Exosome Therapeutic Marketanalyses the current market size, status, enterprise competition pattern, advantages and disadvantages of enterprise products, development trends regional industrial layout characteristics and macroeconomic policies and industrial policy. By focusing on all the necessities and requirements of the businesses for achieving a successful business growth, the Exosome Therapeutic Market Report are created. The CAGR values estimate the fluctuation about the rise or fall of demand for the specific forecasted period with respect to investment. The Exosome Therapeutic Market report also recognizes and analyses the expanding trends along with major drivers, restraints, challenges and opportunities in the market.

Exosome Therapeutic Marketis expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018.

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Synopsis of Global Exosome Therapeutic Market:-Exosomes is used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Some Of The Major Competitors Currently Working In Global Exosome Therapeutic Market Are:Bayer AG, Iso-Tex Diagnostics, Inc., Bracco Diagnostic Inc., Novalek Pharmaceuticals Pvt. Ltd., iMAX, Taejoon Pharm, Unijules Medicals Ltd, General Electric, Guerbet LLC, J.B.Chemicals & Pharmaceuticals Ltd among others players domestic and global. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

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North America Dominates The Exosome Therapeutic Market as the U.S. Is leaderin exosome therapeutic manufacturing as well as research activities required for exosome therapeutics. At present time Stem Cells Group holding shares around 60.00%. In addition global exosomes therapeutics manufacturers like EXOCOBIO, evox THERAPEUTICS and others are intensifying their efforts in China. The Europe region is expected to grow with the highest growth rate in the forecast period of 2019 to 2026 because of increasing research activities in exosome therapeutic by population.

Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

Browse in-depth TOC on Exosome Therapeutic Market

50 Tables

250 No of Figures

150 Pages

This Exosome Therapeutic Market report contains all aspects that are directly or indirectly related to the multiple areas of the global market. Our experts have carefully collated the global Exosome Therapeutic Market data and estimated the change in the forecast period. This information in the report helps customers make accurate decisions about market activity Exosome Therapeutic Market based on forecasting trends. This report also discusses current or future policy research or regulations that must be initiated by management and market strategies.

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Global Exosome Therapeutic Market Scope and Market Size

Global Exosome Therapeutic Market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Based on therapy, the market is segmented into immunotherapy, gene therapy and chemotherapy. Chemotherapy is dominating in the market because chemotherapy is basically used in treatment of cancer which is major public health issues. The multidrug resistance (MDR) proteins and various tumors associated exosomes such as miRNA and IncRNA are include in in chemotherapy associated resistance.

Based on transporting capacity, the market is segmented into bio macromolecules and small molecules. Bio macromolecules are dominating in the market because bio macromolecules transmit particular biomolecular information and are basically investigated for their delicate properties such as biomarker source and delivery system

Based on application, the market is segmented into oncology, neurology, metabolic disorders, cardiac disorders, blood disorders, inflammatory disorders, gynecology disorders, organ transplantation and others. Oncology segment is dominating in the market due to rising incidence of various cancers such as lung cancer, breast cancer, leukemia, skin cancer, lymphoma. As per the National Cancer Institute, in 2018 around 1,735,350 new cases of cancer was diagnosed in the U.S. As per the American Cancer Society Inc in 2019 approximately 268,600 new cases of breast cancer diagnosed in the U.S.To be continued..Detailed Segmentation ofExosome Therapeutic Market

The Countries Covered In The Exosome Therapeutic Market Report Are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific in the Asia-Pacific, South Africa, Rest of Middle East and Africa as a part of Middle East and Africa, Brazil and Rest of South America as part of South America.

Along with the elaborated information about the key contenders, the globalExosome Therapeutic Marketreport efficiently provides information by segmenting the market on the basis of the type services and products offerings, form of the product, applications of the final products, technology on which the product is based, and others. The report is also bifurcated the market on the basis of regions to analyze the growth pattern of the market in different geographical areas.

The Exosome Therapeutic Market report includes the leading advancements and technological up-gradation that engages the user to inhabit with fine business selections, define their future-based priority growth plans, and to implement the necessary actions. The global Exosome Therapeutic Market report also offers a detailed summary of key players and their manufacturing procedure with statistical data and profound analysis of the products, contribution, and revenue.

Global Exosome Therapeutic Market Report includes Detailed TOC points:

1 Introduction

2Market Segmentation

3 Market Overview

3.3 Opportunities

4 Executive Summaries

5 Premium Insights

6 Regulatory Procedure

7 Global Exosome Therapeutic Market, By Type

8 Global Exosome Therapeutic Market, by disease type

9 Global Exosome Therapeutic Market, By Deployment

10 Global Exosome Therapeutic Market, By End User

11 Global Exosome Therapeutic Market, By Distribution Channel

12 Global Exosome Therapeutic Market, By Geography

13 Global Exosome Therapeutic Market, Company Landscape

14 Company Profile

Continued!!!

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Exosome Therapeutic Market Size, Share, Trends, Global Research, Technology Implementation and Geographical Overview Till 2027 - Cole of Duty

Cardiac Stem Cells Comments Off on Exosome Therapeutic Market Size, Share, Trends, Global Research, Technology Implementation and Geographical Overview Till 2027 – Cole of Duty | May 24th, 2020

Indias slum population is ill-prepared to face the novel coronavirus disease (COVID-19) pandemic, with livelihoods threatened as a consequence of the lockdown

The novel coronavirus disease (COVID-19) pandemic infected over four million people worldwide as of May 7, 2020 and killed over 200,000 people. After receiving its first cases on January 28, India witnessed a spike in late March, that led to a nationwide lockdown on March 24 in order to stem the infection.

The number of cases, however, only increased in the past couple of months. As of May 10, 63,975 cases were reported, mostly in densely populated and highly urbanised areas, resulting in almost 2,109 deaths.

One of the measures adopted by the Union and state governments following World Health Organization guidelines was to tell people to stay home and follow the practice of social distancing where emphasis is laid on the maintenance of distance of at least six feet between individuals.

Access to safe drinking water, proper sanitation and health conditions to protect from the infection is necessary to stop human-to-human transmission along with ensuring good consistency in hand washing and waste management practices.

A question, however, emerges over the efficacy of such measures, given that India has the second-most dense population in the world, with a less than adequate healthcare system and a high migration rate, compounded by the fact that 21.9 per cent of the countrys total population lives below the poverty line, according to 2018 data from the National Sample Survey Office.

Is social distancing a viable strategy for this largely informal society? Can a largely poor population, consisting primarily of marginalised communities and migrant daily wage workers adopt such strict guidelines?

We focus on this question by analysing the socio-economic conditions of people living in informal urban settlements and slums within the country.

What is clear is that the slum population is ill-prepared to fight the pandemic, with livelihoods threatened as a consequence of the lockdown. It also sounds impractical to assume slum households have hygienic access to water, toilets, sewers and drainage in informal settlements.

In overcrowded slums, measures like physical distancing and self-quarantine remain far from implementation.

Dharavi Asias biggest slum located in Mumbai with an approximate one million population shows this reality. As on April 29, Dharavi reported a total of 590 cases, with 20 deaths. These deaths include youth and children, according to a media report.

Researchers from the Centre for Sustainability estimate the reproductive ratio (R naught) for the SARS-CoV-2 virus that causes COVID-19 on a global scale. They suggest this number in India's slums to be 20 per cent on an average, higher because of dense living conditions, according to a report in news daily Hindustan Times. This vulnerability is borne out in statistics as well.

We analysed data from the fourth round of the National Family Health Survey (NFHS-4) and the 2011 census, focusing on more urbanised states like Maharashtra, Madhya Pradesh, Uttar Pradesh, Delhi, Tamil Nadu and Telangana.

Within these states, we further classified urban households residing in slums in Mumbai, suburban Mumbai, Nagpur, Indore, Meerut, Hyderabad, Chennai and Delhi.

Distribution of SARS-CoV-2

To begin with, we described the basic characteristics of confirmed cases of the virus in Indian states, with basic calculations from COVID-19 India. We analysed details such as growth rate, fatality rate, recovery rate, test per million and share of active cases.

Basic method to understand the novel coronavirus pandemic select states in India

Source: NFHS-4, Census-2011,COVID19

Based on the reported rise in the pace of infection in various states, we attempted its association with share of migrants and slum population. The statistics indicate a clear pattern of intensity among major cities.

The share in total active COVID-19 cases is higher in Maharashtra (36.4 per cent per million), followed by Gujarat (12.5 per cent), Tamil Nadu (12 per cent) and Delhi (10.9 per cent).

With respect to fatality rates, West Bengal, Gujarat and Madhya Pradesh lead the table. Recovery rate too, follows the same trend with the notable exceptions of Kerala and Telangana.

When one reads the pace of increase in infection, it is evident that the most urbanised and in-migration states like Maharashtra, Delhi, Gujarat and Telangana according to the 2011 census show higher incidences of infection.

At the same time, there is a reasonable rise in COVID-19 cases in Bihar, Jharkhand, West Bengal and Uttar Pradesh because of reverse migration following the lockdown.

On the other hand, its association with the degree of urbanisation may very well be due to under-served and under-privileged residents with limitation of housing and basic amenities that inhibits adhering to the required prevention protocol. The most vulnerable therefore become the slum residents with poor sanitation practices.

Percentage of the novel coronavirus cases and risk in select urban districts

Source: NFHS-4, Census-2011, COVID-19.org

Approximately 65 million people, or 22 per cent, of Indias total population lives in urban slums, according to a report. Most of the temporary and semi-temporary migrants, however, live in slums across major cities.

A few districts of Maharashtra, including Mumbai, suburban Mumbai and Nagpur are home to 1.39 million temporary migrants and 5.74 million semi-permanent migrants.

These migrant labourers form the backbone of Indias economy, work on lower daily wages and do not have the privilege of working from home with stable wages.

They either risk infection to work in the current lockdown or face unemployment and starvation. It is these workers who are prone to a higher risk of comorbidity and consequently at a greater risk to get infected with the virus.

Risks with migrants, slum households

When we look at migrants, they mainly live in metropolitan hubs, including Mumbai, Delhi and Chennai and are vulnerable due to the informal nature of their livelihoods in these slums.

While associating the share of migration and the quantum of infection, we find a positive correlation indicating the migration related vulnerability of this infection. This association is further strengthened when one considers the share of the most vulnerable population living in the urban slums of Mumbai, Central Delhi, Chennai and Hyderabad with a positive correlation.

SARS-CoV-2infections correlation with migrants within slum households

Assessing social distancing, sanitation

Slum lanes are so narrow that when we cross each other, we cannot cross without our shoulders rubbing against the other person, said a slum dweller, quoted by international news outlet The Guardian. This is, unfortunately, the case in most slum pockets in India.

The World Economic Forum, for example, said Dharavi has a high population density of over 270,000 people living per square kilometre.

This undoubtedly makes social distancing norms impractical. Most of the migrant families or migrant workers who live in single rooms depend oncommunity toilets and water taps.

Findings from NFHS-4 show that among slum households, 56.94 per cent use unimproved toilets, 63.76 per cent live in one room tenements and 76.25 per cent have limited access to water for hand wash.

Unsurprisingly, the evidence shows a positive correlation among virus infections and residences with unimproved toilet facilities, one room households and unavailability of water.

For almost all slum households, using hand sanitiser is very expensive and moreover, access to water for hand-washing is inadequate to begin with. Maintaining social distancing is also not possible given the high density of the population within these slums.

The current crisis should send alarm bells ringing for governments and urban planners in the country over the sustainability of our cities for its citizens. Ensuring good and consistently applied water sanitation and hygiene and waste management practices in communities is a pre-requisite for containing the spread of the epidemic.

Given the evidence on living reality of slums residents and urban migrants in Indian cities, norms like social distancing and hygiene practices seem far from reality.

SARS-CoV-2correlation with hygiene and sanitation conditions in slum households

Policy implications

The analysis highlights the inadequacies of human living and its vulnerability in the face of a pandemic. Overcrowded slums in major cities house an important but extremely vulnerable section of our society.

This population was severely affected due to both the typical infectiveness of the virus as well as the lockdown, leading to substantial losses in lives and livelihoods of these people.

A Stranded Workers Action Network survey recently conducted across various states showed out of 11,159 migrant workers, 96 per cent did not get ration and 90 per cent of them did not get wages.

In case of such an outbreak and the associated measures for its containment, basic deficiencies of the urban poor are overlooked, not just in terms of their compromised living, but also limited access to medical care. It is evident that measures adopted originate from a middle-class mindset that assumes a lot more and is remote from prevailing realities.

This pandemic is a reminder for the need of a slum emergency planning map for every urban settlement and developed solid waste management strategy.

Most importantly, urban planners need to urgently rethink about the sustainability of mega cities in the wake of the outbreak. We have seen vulnerable populations disproportionately affected all the worlds major cities from Wuhan in China, to New York in the United States and Mumbai and Delhi in India.

While the more privileged sections of society have the luxury of sequestering themselves and maintain social-distancing, large swathes of people who do not have the luxury to do so often suffer.

This episode should is an eye-opener to reveal that if we are to recover from crises in the future, we need to strengthen and equip the basic health and public health infrastructure of the urban cities to cater to all its residents to guarantee sustainable urban living.

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COVID-19: How do India's urban informal settlements fight the pandemic - Down To Earth Magazine

Cardiac Stem Cells Comments Off on COVID-19: How do India’s urban informal settlements fight the pandemic – Down To Earth Magazine | May 22nd, 2020

Latest Patent Further Strengthens Intellectual Property Portfolio Covering Novel Platform for Precisely Controlling Core Cell Migration Mechanisms

Decelerator Technology Has Key Potential Applications in Treatment of Cancer and Fibrosis and Serves as Key Complement to Companys Cell Motility Accelerator Platform for Enhanced Tissue Repair

NEW YORK, May 20, 2020 (GLOBE NEWSWIRE) -- MicroCures, a biopharmaceutical company developing novel therapeutics that harness the bodys innate regenerative mechanisms to accelerate tissue repair, today announced the issuance of a new European patent providing broad protection for the companys first-of-its-kind cell movement decelerator technology, which has potential therapeutic applications in combating cancer metastases and fibrosis. The companys decelerator technology is being developed alongside MicroCures accelerator technology, which is designed to enhance repair of tissue, nerves, and organs following trauma. With the newly issued patent in the European Union (#3052117), the companys global patent estate now includes eight issued and 12 pending patents covering its underlying technology, as well as the therapeutic programs that have emerged from the platform.

Our proprietary platform technology represents a fundamentally new way of thinking about how to harness the bodys natural cell movement processes to drive therapeutic outcomes in response to a range of medical challenges. Whether it is removing the brakes from cells to accelerate their migration and drive tissue, nerve and organ repair, or putting the brakes on cell movement to combat tumor metastases and fibrosis, we are pioneering an entirely new treatment paradigm, said Derek Proudian, co-founder and chief executive officer of MicroCures. While we clearly recognize the importance of the development work we are undertaking in support of this platform, it is equally important to build a strong, wide-reaching intellectual property portfolio to protect it. This latest patent issuance provides us yet another key piece of intellectual property, bringing our total number of issued and pending patents to 20.

MicroCures technology is based on foundational scientific research at Albert Einstein College of Medicine regarding the fundamental role that cell movement plays as a driver of the bodys innate capacity to repair tissue, nerves, and organs. The company has shown that complex and dynamic networks of microtubules within cells crucially control cell migration, and that this cell movement can be reliably modulated to achieve a range of therapeutic benefits. Based on these findings, the company has established a first-of-its-kind proprietary platform to create siRNA-based therapeutics capable of precisely controlling the speed and direction of cell movement by selectively silencing microtubule regulatory proteins.

The company has developed a broad pipeline of therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. Unlike regenerative medicine approaches that rely upon engineered materials or systemic growth factor/stem cell therapeutics, MicroCures technology directs and enhances the bodys inherent healing processes through local, temporary modulation of cell motility. Additionally, the company is developing a decelerator technology based on the same foundational science. Instead of accelerating cell movement for therapeutic repair and regeneration, this technology is designed to slow or halt the movement of cells, potentially offering a unique, natural approach to preventing cancer metastases and fibrosis.

About MicroCures

MicroCures develops biopharmaceuticals that harness innate cellular mechanisms within the body to accelerate and improve recovery after traumatic injury. MicroCures has developed a first-of-its-kind therapeutic platform that precisely controls the rate and direction of cell migration, offering the potential to deliver powerful therapeutic benefits for a variety of large and underserved medical applications.

MicroCures has developed a broad pipeline of novel therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. The companys lead therapeutic candidate, siFi2, targets excisional wound healing, a multi-billion dollar market inadequately served by current treatments. Additional applications for the companys cell migration accelerator technology include dermal burn repair, corneal burn repair, cavernous nerve repair/regeneration, spinal cord repair/regeneration, and cardiac tissue repair. Cell migration decelerator applications include combatting cancer metastases and fibrosis. The company protects its unique platform and proprietary therapeutic programs with a robust intellectual property portfolio including eight issued patents, as well as 12 pending patent applications.

Story continues

For more information please visit: http://www.microcures.com

Contact:

MicroCuresinfo@microcures.com

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MicroCures Announces Issuance of New European Patent Providing Broad Protection for First-of-its-Kind Cell Movement Decelerator Technology - Yahoo...

Cardiac Stem Cells Comments Off on MicroCures Announces Issuance of New European Patent Providing Broad Protection for First-of-its-Kind Cell Movement Decelerator Technology – Yahoo… | May 21st, 2020

Aggressive public health measures tostem the tidal wave of coronavirus infections have left people isolated,unemployed and wondering when it will all end. Life probably wont gocompletely back to normal until vaccines against the virus are available,experts warn.

Researchers are working hard on thatfront. At least six vaccines are currently being tested in people, says EstherKrofah, chief executive of the FasterCures center at the Milken Institute in Washington,D.C. We expect about two dozen more toenter clinical trials by this summer and early fall. That is a huge number,Krofah said at an April 17 briefing. Dozens more are in earlier stages oftesting.

In unpublished, preliminary results of a test of one vaccine, inoculated people made as many antibodies against the coronavirus as people who have recovered from COVID-19 (SN: 5/18/20). The mRNA-based vaccine induces human cells to make one of the viruss proteins, which the immune system then builds antibodies to attack. That study was small, only eight people, but a second phase of safety testing has begun.

But vaccinestake time to test thoroughly (SN: 2/21/20). Even with acceleratedtimelines and talk of emergency use of promising vaccines for health care workersand others at high risk of catching the virus, the general public will likelywait a year or more to be vaccinated.

In the meantime, new treatments may helpsave lives or lessen the severity of disease in people who become ill.Researchers around the world are experimenting with more than 130 drugs to findout if any can help COVID-19 patients, according to atracker maintained by the Milken Institute.

Some of those drugs are aimed atstopping the virus, while others may help calm overactive immune responses thatdamage lungs and other organs. Although researchers are testing a battery ofrepurposed drugs and devising new ones, there is still a great deal ofuncertainty over whether the drugs help, or maybe even hurt.

The wait is frustrating, but theres still much doctors and scientists dont know about how this new coronavirus affects the body. Getting answers will take time, and finding measures to counter the virus that are both safe and effective will take even more. Early results suggest that the antiviral drug remdesivir can modestly speed recovery from COVID-19 (SN: 5/13/20). It is not a cure, but the drug may become the new standard of care as researchers continue to test other therapies.

Scientists and journalists share a core belief in questioning, observing and verifying to reach the truth. Science News reports on crucial research and discovery across science disciplines. We need your financial support to make it happen every contribution makes a difference.

Antiviral drugs interfere with a viruss ability to replicate itself, though such drugs are difficult to create. Remdesivir is being tested in half a dozen clinical trials worldwide. The drug mimics a building block of RNA, the genetic material of the coronavirus (SN: 3/10/20). When the virus copies its RNA, remdesivir replaces some of the building blocks, preventing new virus copies from being produced, laboratory studies have shown.

Early results in COVID-19 patients given the drug outside of a clinical trial showed that 68 percent needed less oxygen support after treatment, as reported online April 10 in the New England Journal of Medicine (SN: 4/29/20). The drug went to very sick patients, including those who needed oxygen from a ventilator or through tubes in the nose. Other researchers have disputed those results, questioning the study methods and statistical analyses, which may have given an exaggerated impression of good outcomes. The studys authors say they have reanalyzed the data and still conclude that remdesivir has benefits.

Soon after, the U.S. National Instituteof Allergy and Infectious Diseases announced that hospitalized patients withCOVID-19 who got intravenous remdesivir recoveredmore quickly than those on a placebo: in 11 days versus 15. Those findingshad not been reviewed by other scientists at the time of the announcement. Thedug provides researchers with a baseline for comparing other treatments. Wethink its really opening the door to the fact that we now have the capabilityof treating, Anthony Fauci, director of the NIAID said April 29 in a newsbriefing at the White House.

Antiviral medications used against HIV are also being tested against COVID-19. The combination of lopinavir and ritonavir stops an HIV enzyme called the M protease from cutting viral proteins so that the virus can replicate itself. The SARS-CoV-2 virus produces a similar enzyme. But early results from a small study in China showed that the combination didnt stop viral replication or improve symptoms (SN: 3/19/20), and there were side effects.

For now, the Society of Critical CareMedicine recommendsagainst using the drugs, and the Infectious Diseases Society of Americasays patients should get the drugs onlyas part of a clinical trial. Several large trials may report results soon.

The HIV drugs may not work well against SARS-CoV-2, even though the viruses have similar M proteases: The coronaviruss enzyme lacks a pocket where the drugs fit in the HIV version of the enzyme.

This illustrates why antiviral drugs areso difficult to develop. Designing a drug requires knowing the 3-D structure ofthe viruss proteins, which can take months to years. But researchers arealready getting some close-up views of the new coronavirus. A team in Chinaexamined the structure of the coronaviruss M protease and designed smallmolecules that could block a part of the protein necessary to do its job. Theteam describedtwo such molecules, dubbed 11a and 11b, April 22 in Science.

In test tubes, both molecules stopped the virus from replicating in monkey cells. In mice, 11a stuck around longer in the blood than 11b, so the researchers tested 11a further and found it seemed safe in rats and beagles. More animal tests will probably be needed to show whether it stops the virus, then multiple stages of human tests will have to follow. The drug development and testing process often takes on average 10 years or more, and can fail at any point along the way.

Meanwhile, hundreds of thousands of people worldwide have already recovered from COVID-19, and many are donating blood that might contain virus-fighting antibodies. Clinical trials are under way to test whether antibodies from recovered patients blood plasma can help people fight off the virus (SN: 4/25/20, p. 6). More such trials are planned.

Stopping the virus is only half the problem. In some people seriously ill with COVID-19, their immune system becomes the enemy, unleashing storms of immune chemicals called cytokines. Those cytokines trigger immune cells to join the fight against the virus, but sometimes the cells go too far, causing damaging inflammation.

Some of the drugs used to calm cytokines in cancer patients (SN: 6/27/18, p. 22) may also help people with COVID-19 ride out the storm, says cancer researcher Lee Greenberger, chief scientific officer of Leukemia and Lymphoma Society. Several of those drugs are being tested against the coronavirus now.

Hydroxychloroquine, a drug approved totreat autoimmune disorders such as lupus and rheumatoid arthritis, became ahousehold word after President Trump touted it as a possible COVID-19treatment.

The drug is being tested in numerouslarge clinical trials around the world to see if it might help calm cytokinestorms in COVID-19 patients as well. But so far, there is no solid evidence thatit works either to prevent infection in people or to treat people who alreadyhave the disease.

And in some studies the drug has caused serious side effects, including causing irregular heartbeats, says Raymond Woosley, a pharmacologist at the University of Arizona College of Medicine in Phoenix. People with heart problems, low potassium or low oxygen levels in their blood are at higher risk of these side effects, he says. And those are exactly the kinds of patients who are most vulnerable to COVID-19. So, the very sickest COVID patients are those at most risk for these life-threatening arrhythmias and cardiac effects.

Results of some rigorous clinical trialsof hydroxychloroquine are expected this summer. Meanwhile, the U.S. Food andDrug Administration allows the drug to be used when no other treatment isavailable and patients cant join a clinical trial.

Todays enthusiasm for any drug thatseems promising feels familiar, says Woosley. He remembers the excitement overAZT, the first drug used to fight HIV in the 1980s. It wasnt the best drug tocombat the AIDS epidemic, and better ones came later. Likewise, the firsttreatments for COVID-19 might be better than nothing, but not the best we willultimately get.

Meanwhile, we wait.

With hundreds of clinical trials going on around the world, some answers may come soon. But for now, keeping the coronavirus contained will probably require aggressive testing, tracing and isolating contacts of people who have the virus and continued social distancing.

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As we wait for a vaccine, heres a snapshot of potential COVID-19 treatments - Science News

Cardiac Stem Cells Comments Off on As we wait for a vaccine, heres a snapshot of potential COVID-19 treatments – Science News | May 21st, 2020

Despite many breakthroughs in cardiovascular treatment, heart attacks and heart failure still pose a very large threat to the American population. The largest challenge in patients who have had a cardiac complication is the restoration of function to the damaged heart. Since damaged heart tissue is very difficult for the body to replace, physicians are continually looking for new methods of treating the heart.

Regenerative treatment through the use of stem cells is showing a large amount of potential at not only helping reverse the resulting damage of a cardiac attack, but in actually re-growing the damaged tissue in order to restore function.

To understand what stem cells can potentially do for the heart, it is important to first understand the different heart cells that can be damaged in a cardiac event. Destruction of the heart muscle cells, called cardiomyocytes, is the primary cause behind loss of function in a damaged heart. These cells are the muscle behind heart contraction, which sends blood to the rest of the body.

Secondly, vascular endothelial cells (inner lining of blood vessels) and smooth muscle cells (outer lining of blood vessels) each play an important role in the formation of new arteries. These serve to draw nutrients and oxygen to the remaining cardiomyocytes following heart damage, directly influencing the capabilities of a damaged heart.

Numerous studies are being conducted into the purposing of stem cells this manner, with one study providing evidence that bone marrow stem cells were able to develop into the required myocardial cells in mice. The ability to develop human hematopoietic stem cells for heart muscle is already documented technique, with the method of application into humans and the results of implantation still under study.

The research currently being performed on stem cells for cardiac treatment is focused on developing known stem cell traits into a working cure for cardiac complications. The current hurdles researchers face include how to best expand stem cells injected into the heart, how to best deliver the cells, and how to discover new niches (groupings) of stem cells in the body.

Delivery: Current methods of delivery include generic IV injection, which is minimally invasive with varying degrees of success. The most dependable method is to have direct injection into the heart, which requires surgery for visualization. Complications may include potentially clogging the arteries with the introduced stem cells and the invasiveness of the surgery.

Expanding Stem Cells: The majority of transplanted stem cells fail to reach the area of damage. It is crucial for a physician to be able to accurately deliver a large amount of stem cells to offer the patient the best treatment. Methods of better stem cell isolation, identification, and expansion into tissue are currently being developed.

Physician First Choice offers stem cell therapy for cardiac disease. The clinic has multiple stem cell doctors with extensive experience in stem cell therapy for numerous conditions. The stem cell clinic sees patients from all over California and the country, providing both IV stem cell therapy and injections into arthritic joints and areas with tendonitis and ligament injury.

Call (888) 988-0515 for more information and scheduling today!

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Cardiac Stem Cells Comments Off on cardiac disease and stem cells | Stem Cell Treatment in … | May 18th, 2020

The report on the global Progenitor Cell Product market is comprehensively prepared with main focus on the competitive landscape, geographical growth, segmentation, and market dynamics, including drivers, restraints, and opportunities. It sheds light on key production, revenue, and consumption trends so that players could improve their sales and growth in the GlobalProgenitor Cell Product Market.It brings to light key factors affecting the growth of different segments and regions in the global Progenitor Cell Product market. It also offers SWOT, Porters Five Forces, and PESTLE analysis to thoroughly examine the global Progenitor Cell Product market.It offers a detailed analysis of the competition and leading companies of the global Progenitor Cell Product market. Here, it concentrates on the recent developments, sales, market value, production, gross margin, and other important factors of the business of top players operating in the global Progenitor Cell Product market.

Key companies operating in the global Progenitor Cell Product market include:NeuroNova AB, StemCells, ReNeuron Limited, Asterias Biotherapeutics, Thermo Fisher Scientific, STEMCELL Technologies, Axol Bio, R&D Systems, Lonza, ATCC, Irvine Scientific, CDI

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With deep quantitative and qualitative analysis, the report provides encyclopedic and accurate research study on important aspects of the global Progenitor Cell Product market. It gives a detailed study on manufacturing cost, upstream and downstream buyers, distributors, marketing strategy, and marketing channel development trends of the global Progenitor Cell Product market. Furthermore, it provides strategic bits of advice and recommendations for players to ensure success in the global Progenitor Cell Product market.

Segmental Analysis

The report has classified the global Progenitor Cell Product industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the Progenitor Cell Product manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall Progenitor Cell Product industry.

Global Progenitor Cell Product Market Segment By Type:

, Pancreatic progenitor cells, Cardiac Progenitor Cells, Intermediate progenitor cells, Neural progenitor cells (NPCs), Endothelial progenitor cells (EPC), Others

Global Progenitor Cell Product Market Segment By Application:

Progenitor Cell Product

Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Progenitor Cell Product industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key companies operating in the global Progenitor Cell Product market includeNeuroNova AB, StemCells, ReNeuron Limited, Asterias Biotherapeutics, Thermo Fisher Scientific, STEMCELL Technologies, Axol Bio, R&D Systems, Lonza, ATCC, Irvine Scientific, CDI

Regions and Countries

The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Key Questions Answered

What is the size and CAGR of the global Progenitor Cell Product market?

Which are the leading segments of the global Progenitor Cell Product market?

What are the key driving factors of the most profitable regional market?

What is the nature of competition in the global Progenitor Cell Product market?

How will the global Progenitor Cell Product market advance in the coming years?

What are the main strategies adopted in the global Progenitor Cell Product market?

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Table of Contents

Table of Contents 1 Progenitor Cell Product Market Overview1.1 Progenitor Cell Product Product Overview1.2 Progenitor Cell Product Market Segment by Type1.2.1 Pancreatic progenitor cells1.2.2 Cardiac Progenitor Cells1.2.3 Intermediate progenitor cells1.2.4 Neural progenitor cells (NPCs)1.2.5 Endothelial progenitor cells (EPC)1.2.6 Others1.3 Global Progenitor Cell Product Market Size by Type1.3.1 Global Progenitor Cell Product Sales and Growth by Type1.3.2 Global Progenitor Cell Product Sales and Market Share by Type1.3.3 Global Progenitor Cell Product Revenue and Market Share by Type1.3.4 Global Progenitor Cell Product Price by Type1.4 North America Progenitor Cell Product by Type1.5 Europe Progenitor Cell Product by Type1.6 South America Progenitor Cell Product by Type1.7 Middle East and Africa Progenitor Cell Product by Type 2 Global Progenitor Cell Product Market Competition by Company2.1 Global Progenitor Cell Product Sales and Market Share by Company (2014-2019)2.2 Global Progenitor Cell Product Revenue and Share by Company (2014-2019)2.3 Global Progenitor Cell Product Price by Company (2014-2019)2.4 Global Top Players Progenitor Cell Product Manufacturing Base Distribution, Sales Area, Product Types2.5 Progenitor Cell Product Market Competitive Situation and Trends2.5.1 Progenitor Cell Product Market Concentration Rate2.5.2 Global Progenitor Cell Product Market Share of Top 5 and Top 10 Players2.5.3 Mergers & Acquisitions, Expansion 3 Progenitor Cell Product Company Profiles and Sales Data3.1 NeuroNova AB3.1.1 Company Basic Information, Manufacturing Base and Competitors3.1.2 Progenitor Cell Product Product Category, Application and Specification3.1.3 NeuroNova AB Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.1.4 Main Business Overview3.2 StemCells3.2.1 Company Basic Information, Manufacturing Base and Competitors3.2.2 Progenitor Cell Product Product Category, Application and Specification3.2.3 StemCells Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.2.4 Main Business Overview3.3 ReNeuron Limited3.3.1 Company Basic Information, Manufacturing Base and Competitors3.3.2 Progenitor Cell Product Product Category, Application and Specification3.3.3 ReNeuron Limited Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.3.4 Main Business Overview3.4 Asterias Biotherapeutics3.4.1 Company Basic Information, Manufacturing Base and Competitors3.4.2 Progenitor Cell Product Product Category, Application and Specification3.4.3 Asterias Biotherapeutics Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.4.4 Main Business Overview3.5 Thermo Fisher Scientific3.5.1 Company Basic Information, Manufacturing Base and Competitors3.5.2 Progenitor Cell Product Product Category, Application and Specification3.5.3 Thermo Fisher Scientific Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.5.4 Main Business Overview3.6 STEMCELL Technologies3.6.1 Company Basic Information, Manufacturing Base and Competitors3.6.2 Progenitor Cell Product Product Category, Application and Specification3.6.3 STEMCELL Technologies Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.6.4 Main Business Overview3.7 Axol Bio3.7.1 Company Basic Information, Manufacturing Base and Competitors3.7.2 Progenitor Cell Product Product Category, Application and Specification3.7.3 Axol Bio Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.7.4 Main Business Overview3.8 R&D Systems3.8.1 Company Basic Information, Manufacturing Base and Competitors3.8.2 Progenitor Cell Product Product Category, Application and Specification3.8.3 R&D Systems Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.8.4 Main Business Overview3.9 Lonza3.9.1 Company Basic Information, Manufacturing Base and Competitors3.9.2 Progenitor Cell Product Product Category, Application and Specification3.9.3 Lonza Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.9.4 Main Business Overview3.10 ATCC3.10.1 Company Basic Information, Manufacturing Base and Competitors3.10.2 Progenitor Cell Product Product Category, Application and Specification3.10.3 ATCC Progenitor Cell Product Sales, Revenue, Price and Gross Margin(2014-2019)3.10.4 Main Business Overview3.11 Irvine Scientific3.12 CDI 4 Progenitor Cell Product Market Status and Outlook by Regions4.1 Global Progenitor Cell Product Market Status and Outlook by Regions4.1.1 Global Progenitor Cell Product Market Size and CAGR by Regions4.1.2 North America4.1.3 Europe4.1.4 Asia-Pacific4.1.5 South America4.1.6 Middle East and Africa4.2 Global Progenitor Cell Product Sales and Revenue by Regions4.2.1 Global Progenitor Cell Product Sales Market Share by Regions (2014-2019)4.2.2 Global Progenitor Cell Product Revenue Market Share by Regions (2014-2019)4.2.3 Global Progenitor Cell Product Sales, Revenue, Price and Gross Margin (2014-2019)4.3 North America Progenitor Cell Product Sales, Revenue, Price and Gross Margin4.3.1 North America Progenitor Cell Product Sales by Countries4.3.2 United States4.3.3 Canada4.3.4 Mexico4.4 Europe Progenitor Cell Product Sales, Revenue, Price and Gross Margin4.4.1 Europe Progenitor Cell Product Sales by Countries4.4.2 Germany4.4.3 France4.4.4 UK4.4.5 Italy4.4.6 Russia4.5 Asia-Pacific Progenitor Cell Product Sales, Revenue, Price and Gross Margin4.5.1 Asia-Pacific Progenitor Cell Product Sales by Regions4.5.2 China4.5.3 Japan4.5.4 South Korea4.5.5 India4.5.6 Australia4.5.7 Indonesia4.5.8 Thailand4.5.9 Malaysia4.5.10 Philippines4.5.11 Vietnam4.6 South America Progenitor Cell Product Sales, Revenue, Price and Gross Margin4.6.1 South America Progenitor Cell Product Sales by Countries4.6.2 Brazil4.7 Middle East and Africa Progenitor Cell Product Sales, Revenue, Price and Gross Margin4.7.1 Middle East and Africa Progenitor Cell Product Sales by Countries4.7.2 Turkey4.7.3 GCC Countries4.7.4 Egypt4.7.5 South Africa 5 Progenitor Cell Product Application5.1 Progenitor Cell Product Segment by Application5.1.1 Medical care5.1.2 Hospital5.1.3 Laboratory5.2 Global Progenitor Cell Product Product Segment by Application5.2.1 Global Progenitor Cell Product Sales by Application5.2.2 Global Progenitor Cell Product Sales and Market Share by Application (2014-2019)5.3 North America Progenitor Cell Product by Application5.4 Europe Progenitor Cell Product by Application5.5 Asia-Pacific Progenitor Cell Product by Application5.6 South America Progenitor Cell Product by Application5.7 Middle East and Africa Progenitor Cell Product by Application 6 Global Progenitor Cell Product Market Forecast6.1 Global Progenitor Cell Product Sales, Revenue Forecast (2019-2025)6.1.1 Global Progenitor Cell Product Sales and Growth Rate Forecast (2019-2025)6.1.2 Global Progenitor Cell Product Revenue and Growth Rate Forecast (2019-2025)6.2 Global Progenitor Cell Product Forecast by Regions6.2.1 North America Progenitor Cell Product Sales and Revenue Forecast (2019-2025)6.2.2 Europe Progenitor Cell Product Sales and Revenue Forecast (2019-2025)6.2.3 Asia-Pacific Progenitor Cell Product Sales and Revenue Forecast (2019-2025)6.2.4 South America Progenitor Cell Product Sales and Revenue Forecast (2019-2025)6.2.5 Middle East and Africa Progenitor Cell Product Sales and Revenue Forecast (2019-2025)6.3 Progenitor Cell Product Forecast by Type6.3.1 Global Progenitor Cell Product Sales and Revenue Forecast by Type (2019-2025)6.3.2 Pancreatic progenitor cells Growth Forecast6.3.3 Cardiac Progenitor Cells Growth Forecast6.4 Progenitor Cell Product Forecast by Application6.4.1 Global Progenitor Cell Product Sales Forecast by Application (2019-2025)6.4.2 Global Progenitor Cell Product Forecast in Medical care6.4.3 Global Progenitor Cell Product Forecast in Hospital 7 Progenitor Cell Product Upstream Raw Materials7.1 Progenitor Cell Product Key Raw Materials7.1.1 Key Raw Materials7.1.2 Key Raw Materials Price7.1.3 Raw Materials Key Suppliers7.2 Manufacturing Cost Structure7.2.1 Raw Materials7.2.2 Labor Cost7.2.3 Manufacturing Expenses7.3 Progenitor Cell Product Industrial Chain Analysis 8 Marketing Strategy Analysis, Distributors8.1 Sales Channel8.2 Distributors8.3 Downstream Customers 9 Research Findings and Conclusion 10 Appendix10.1 Methodology/Research Approach10.1.1 Research Programs/Design10.1.2 Market Size Estimation10.1.3 Market Breakdown and Data Triangulation10.2 Data Source10.2.1 Secondary Sources10.2.2 Primary Sources10.3 Author List10.4 Disclaimer

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Progenitor Cell Product Market 2020| Worldwide Industry Share, Size, Gross Margin, Trend, Future Demand, Analysis by Top Leading Player and Forecast...

Cardiac Stem Cells Comments Off on Progenitor Cell Product Market 2020| Worldwide Industry Share, Size, Gross Margin, Trend, Future Demand, Analysis by Top Leading Player and Forecast… | May 16th, 2020

Over the past six months, we have been able to deliver on our promise of bringing important new medicines to certain patients with HER2-positive metastatic breast cancer and metastatic urothelial cancer through two U.S. FDA approvals, said Clay Siegall, Ph.D., Chief Executive Officer at Seattle Genetics. We look forward to sharing data in the ASCO virtual scientific program that reinforce our ability to rapidly advance novel targeted agents across multiple tumor types.

An Expanding Portfolio of Marketed Therapies

Key data presentations will showcase progress for certain patients with HER2-positive metastatic breast cancer and metastatic urothelial cancer as well as for patients with classical Hodgkin lymphoma (HL). Highlights include:

TUKYSA Update in Patients with Brain Metastases

Results for TUKYSA in combination with trastuzumab and capecitabine in patients with brain metastases from the HER2CLIMB pivotal trial of previously treated patients with HER2-positive metastatic breast cancer will be featured in an oral session (Abstract #1005). Data will be presented from these exploratory analyses on findings from the TUKYSA arm of the study on reduction in the risk of death (OS), reduction in the risk of intracranial progression or death (CNS-PFS) and improvement of the intracranial confirmed objective response rate (ORR-IC) compared to trastuzumab and capecitabine. Data will be presented by Nancy U. Lin, Director of the Metastatic Breast Cancer Program in the Susan F. Smith Center for Womens Cancers at Dana-Farber in Boston, MA, during an oral presentation available on demand at 8:00 a.m. ET on May 29, 2020. A separate analysis of adverse events (AE) from the same trial will be presented (Abstract #1043; poster presentation).

PADCEV (enfortumab vedotin-ejfv) in Combination and in Other Solid Tumors

Additional results and durability data from the phase 1b EV-103 trial of PADCEV plus pembrolizumab in first-line metastatic urothelial cancer will be presented (Abstract #5044), and a separate Trials-in-Progress poster will provide details about a new randomized cohort added to the EV-103 study, Cohort K, which is evaluating PADCEV as monotherapy or in combination with pembrolizumab (#TPS5092). Both presentations will be featured in the Genitourinary CancerKidney and Bladder session. Data from the Cohort K, along with other data from the EV-103 trial evaluating PADCEV combined with pembrolizumab as first-line therapy for cisplatin-ineligible patients, could potentially support registration under accelerated approval regulations in the United States.

Additionally, information about the phase 2 EV-202 trial, which is studying PADCEV in six different types of locally advanced and metastatic solid tumors (HR-positive/HER2-negative and triple-negative breast cancers, squamous and non-squamous non-small cell lung cancers, head and neck cancer and gastroesophageal cancers), will be discussed in a Trials-in-Progress poster during the Developmental Therapeutics Molecularly Targeted Agents and Tumor Biology Poster Session (Abstracts #TPS3647).

ADCETRIS (brentuximab vedotin) Continues to Advance

Data to be presented on ADCETRIS will demonstrate the companys progress in efforts to continue expanding clinical research on combination regimens and monotherapy in a variety of HL and peripheral T-cell lymphoma (PTCL) patient populations, including in both older and younger disease settings. A poster presentation will highlight the potential of ADCETRIS in combination with nivolumab or dacarbazine and as a monotherapy for previously untreated older HL patients who typically have poorer outcomes than younger patients due to comorbidities and toxicities related to standard first-line chemotherapy (Abstract #8032). The primary analysis from an ongoing clinical trial evaluating ADCETRIS plus nivolumab in children, adolescents and young adults with standard-risk relapsed or refractory classical HL will also be presented (Abstract #8013; poster discussion). Lastly, two Trials-in-Progress poster presentations will highlight ongoing clinical trials evaluating ADCETRIS as a monotherapy in frontline older HL or CD30-expressing PTCL patients and in a combination regimen in frontline advanced-stage HL patients (Abstracts #TPS8069 and #TPS8068).

A Strong, Diverse Pipeline of Investigational Therapies

An additional four Trials-in-Progress posters for investigational therapies will showcase the companys continued clinical development of pipeline candidates in first-line cervical cancer (Abstract #TPS6095), metastatic breast cancer (Abstract #TPS1104), metastatic pancreatic ductal adenocarcinoma (PDAC) (Abstract #TPS4671) and other solid tumors (Abstract #TPS3652).

The abstracts published in advance of the ASCO meeting were made available today on the ASCO website. All data presentations will be available on-demand on May 29, 2020.

Details of Key Seattle Genetics Presentations at ASCO20 Virtual:

Abstract Title

Abstract #

Presentation Type

Presenter

ADCETRIS (brentuximab vedotin)

Nivolumab and brentuximab vedotin (BV)-based, responseadapted treatment in children, adolescents, and young adults (CAYA) with standard-risk relapsed/refractory classical Hodgkin lymphoma (R/R cHL): Primary analysis

8013

Poster discussion

P. Cole

Frontline Brentuximab Vedotin as Monotherapy or in Combination for Older Hodgkin Lymphoma Patients

8032

Poster presentation

C. Yasenchak

PADCEV (enfortumab vedotin-ejfv)

Study EV-103: Durability results of enfortumab vedotin plus pembrolizumab for locally advanced or metastatic urothelial carcinoma

5044

Poster presentation

J. Rosenberg

TUKYSA (tucatinib)

Tucatinib vs Placebo Added to Trastuzumab and Capecitabine for Patients with Previously Treated HER2+ Metastatic Breast Cancer with Brain Metastases (HER2CLIMB)

1005

Oral presentation

N. Lin

Management of adverse events in patients with HER2+ metastatic breast cancer treated with tucatinib, trastuzumab, and capecitabine (HER2CLIMB)

1043

Poster presentation

A. Okines

Trials-in-Progress

ADCETRIS (brentuximab vedotin)

Frontline brentuximab vedotin in Hodgkin lymphoma and CD30-expressing peripheral T-cell lymphoma for older patients and those with comorbidities

TPS8069

Poster presentation

C. Yasenchak

Brentuximab Vedotin in Combination with Nivolumab, Doxorubucin, and Dacarbazine in Newly Diagnosed Patients with Advanced Stage Hodgkin Lymphoma

TPS8068

Poster presentation

J. Friedman

PADCEV (enfortumab vedotin-ejfv)

Study EV-103: New randomized cohort testing enfortumab vedotin as monotherapy or in combination with pembrolizumab for locally advanced or metastatic urothelial carcinoma

TPS5092

Poster presentation

N. Mar

EV-202: A Phase 2 Study of Enfortumab Vedotin in Patients With Select Previously Treated Locally Advanced or Metastatic Solid Tumors

TPS3647

Poster presentation

J. Bruce

Investigational Therapies

Phase 1b/2 trial of tisotumab vedotin (TV) bevacizumab (BEV), pembrolizumab (PEM), or carboplatin (CBP) in recurrent or metastatic cervical cancer (innovaTV 205/ENGOT-cx8/GOG-3024)

TPS6095

Poster presentation

I. Vergote

SGNLVA-001: A phase 1 open-label dose escalation and expansion study of SGN-LIV1A administered weekly in breast cancer

TPS1104

Poster presentation

H. Beckwith

SGN228-001: A phase 1 open-label dose escalation and expansion study of SGN-CD228A in select advanced solid tumors

TPS3652

Poster presentation

A. Patnik

Phase 1 study of SEA-CD40, gemcitabine, nab-paclitaxel, and pembrolizumab in patients (pts) with metastatic pancreatic ductal adenocarcinoma (PDAC)

TPS4671

Poster presentation

A. Coveler

About ADCETRIS (brentuximab vedotin)

ADCETRIS is an antibody-drug conjugate (ADC) comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells. Seattle Genetics and Takeda are jointly developing ADCETRIS.

About PADCEV (enfortumab vedotin-ejfv)

PADCEV is an antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer. Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis). PADCEV is co-developed by Seattle Genetics and Astellas.

About TUKYSA (tucatinib)

TUKYSA is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. In vitro (in lab studies), TUKYSA inhibited phosphorylation of HER2 and HER3, resulting in inhibition of downstream MAPK and AKT signaling and cell growth (proliferation), and showed anti-tumor activity in HER2-expressing tumor cells. In vivo (in living organisms), TUKYSA inhibited the growth of HER2-expressing tumors. The combination of TUKYSA and the anti-HER2 antibody trastuzumab showed increased anti-tumor activity in vitro and in vivo compared to either medicine alone.

ADCETRIS (brentuximab vedotin) U.S. Important Safety Information

BOXED WARNING

PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY (PML): JC virus infection resulting in PML and death can occur in ADCETRIS-treated patients.

Contraindication

ADCETRIS concomitant with bleomycin due to pulmonary toxicity (e.g., interstitial infiltration and/or inflammation).

Warnings and Precautions

Administer G-CSF primary prophylaxis beginning with Cycle 1 for patients who receive ADCETRIS in combination with chemotherapy for previously untreated Stage III/IV cHL or previously untreated PTCL.

Monitor complete blood counts prior to each ADCETRIS dose. Monitor more frequently for patients with Grade 3 or 4 neutropenia. Monitor patients for fever. If Grade 3 or 4 neutropenia develops, consider dose delays, reductions, discontinuation, or G-CSF prophylaxis with subsequent doses.

Most Common (20% in any study) Adverse Reactions

Peripheral neuropathy, fatigue, nausea, diarrhea, neutropenia, upper respiratory tract infection, pyrexia, constipation, vomiting, alopecia, decreased weight, abdominal pain, anemia, stomatitis, lymphopenia, and mucositis.

Drug Interactions

Concomitant use of strong CYP3A4 inhibitors or inducers has the potential to affect the exposure to monomethyl auristatin E (MMAE).

Use in Specific Populations

Moderate or severe hepatic impairment or severe renal impairment: MMAE exposure and adverse reactions are increased. Avoid use.

Advise males with female sexual partners of reproductive potential to use effective contraception during ADCETRIS treatment and for at least 6 months after the final dose of ADCETRIS.

Advise patients to report pregnancy immediately and avoid breastfeeding while receiving ADCETRIS.

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Seattle Genetics Highlights Data from Expanding Oncology Portfolio During Virtual Scientific Program of the 2020 ASCO Annual Meeting - BioSpace

Cardiac Stem Cells Comments Off on Seattle Genetics Highlights Data from Expanding Oncology Portfolio During Virtual Scientific Program of the 2020 ASCO Annual Meeting – BioSpace | May 15th, 2020

The novel coronavirus cant be killed or stopped with the current drugs that we have, the WHO said earlier this week. Dr. Anthony Fauci said separately that its virtually impossible to eradicate the virus. But there are plenty of therapies that can be used to reduce the severity of COVID-19 and shorten the recovery period.

The WHO is studying four or five of the best drugs for the new illness, but there are plenty of new lines of therapy that are discovered on a regular basis. The latest one consists of a treatment thats usually given to Duchenne muscular dystrophy patients.

Cedars-Sinai doctors have given six patients an experimental treatment consisting of cells grown from human heart tissues, according to ABC7. This therapy improved the overall condition of all patients, each of whom were critically ill before the Hail Mary treatment was administered. Four of them have come off ventilators and were discharged, while the other two are still in the hospital, but theyre alive.

Dr. Eduardo Marban and his colleagues were using the treatment for muscular dystrophy patients with heart failure before considering it for COVID-19. The novel coronavirus can do severe damage to the heart, and that may have been the reason why the doctors attempted this novel therapy.

This can only be considered anecdotal evidence at best, but the doctors are hoping that the FDA can approve a more extensive study that can evaluate the benefits of the therapy. The doctors have additional doses available in the freezer for the research.

Cells grown from human heart tissues sound a lot like stem cells, although the report doesnt refer to them as such. This wouldnt be the first time that stem cell use would prove to be helpful in COVID-19 cases. A few weeks ago, doctors from Mount Sinai reported theyve treated 12 patients using stem cells derived from bone marrow, and the therapy allowed 10 of them to come off ventilators. Those physicians also noted that further study is required.

Marban and his colleagues detailed the benefits of injections of cardiac progenitor cells (cardiosphere-derived cells or CDCs) for patients with muscular dystrophy in February 2018. Cardiosphere-derived cells are stem cells derived from cardiac tissue.

We unexpectedly found that treating the heart made the whole body better, Marban said at the time. These basic findings, which have already been translated to clinical trials, rationalize why treating the heart may also benefit skeletal muscle function in boys and young men with Duchenne.

The study showed the stem cells acted not just on the heart tissue, but also on skeletal muscle, and that the benefits persisted. We found that within a few weeks, the injected cells were undetectable, Marban said, but the benefits persisted for at least three months, which led us to discover that exosomes secreted by CDCs are responsible.

The same type of therapy was likely used to treat COVID-19 patients.

Image Source: John Minchillo/AP/Shutterstock

Chris Smith started writing about gadgets as a hobby, and before he knew it he was sharing his views on tech stuff with readers around the world. Whenever he's not writing about gadgets he miserably fails to stay away from them, although he desperately tries. But that's not necessarily a bad thing.

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Doctors just discovered another promising coronavirus therapy - BGR

Cardiac Stem Cells Comments Off on Doctors just discovered another promising coronavirus therapy – BGR | May 14th, 2020

Four of six patients in case series were weaned off respiratory support

An investigational allogeneic cell therapy using cardiosphere-derived cells (CDC) showed an acceptable safety profile with early evidence of efficacy in the treatment of very severe Covid-19 in a case series involving six patients treated at Cedars-Sinai Medical Center in Los Angeles.

All six patients treated with the intravenous allogeneic CDC formulation CAP-1002 (Capricor Therapeutics) as a compassionate therapy required respiratory support prior to treatment, with five on mechanical ventilation.

No adverse events related to the treatment were reported, and four of the six patients were successfully weaned from respiratory support and were discharged from the hospital as of late April.

The other two patients are still alive, but remain intubated, Cedars-Sinai cardiologist Raj Makkar, MD, confirmed to BreakingMED Wednesday, May 13.

While we are encouraged by these findings, it is important to point out that the only way that we can assess the efficacy of this treatment in a definitive way is with a randomized clinical trial, and that is what we intend to do, Makkar said.

He added that the clinical trial, which is in the planning stages, is likely to include Covid-19 patients who are not as critically ill as the six in the case series.

All of these patients required respiratory support and they were all on a downward trajectory when treated, he said. They were getting worse and we had nothing else to offer them.

Cardiosphere-derived cells are stromal/progenitor cells from heart tissue with a distinctive antigenic profile (CD105+, CD45-, CD90low).

In their case series, published in the journal Basic Research in Cardiology, Makkar and colleagues noted that the cells are entirely distinct from the controversial c-kit+ putative cardiac progenitors, which have been the subject of various retracted studies.

Since CDCs were first isolated in 2007, the cells have been tested in more than 200 patients in clinical trials for a variety of conditions with a good safety profile, including in young boys with Duchenne muscular dystrophy.

Makkar said the anti-inflammatory and antifibrotic properties of CDCs in animal models make them a possible target therapy for Covid-19.

The prior testing gave us reasonable confidence that this treatment was safe, he said, adding that there is also evidence of a favorable effect on the same type of proinflammatory cytokines that are up-regulated in Covid-19.

Comparisons to mesenchymal stem cells (MSCs) in pre-clinical models suggest that CDCs may also be more effective for paracrine factor secretion and myocardial remodeling.

Given the safety record of CDCs in humans, and the substantial body of evidence confirming relevant disease-modifying bioactivity, applicability to Covid-19 seemed compelling, particularly in the hyperinflammatory stage of the illness, the researchers wrote.

All six patients treated with the intravenous CDC formulation had severe, confirmed Covid-19 with respiratory failure and they were not receiving any other experimental agent, with the exception of hydroxychloroquine and tocilizumab.

Lack of clinical improvement or deterioration despite standard care was the primary reason for considering patients for treatment with CAP-1002. Exclusion criteria included known hypersensitivity to DMSO, which is a component of CAP-1002; prior stem cell therapy; pre-existing terminal illness; and need for mechanical circulatory support and dialysis.

In general, patients with multi-organ failure who were deemed to be too sick for any intervention were excluded from the study, Makkar and colleagues wrote.

All patients had acute respiratory distress syndrome (ARDS) prior to infusion, with decreased PaO2/FiO2 ratios (range 69-198; median 142), diffuse bilateral pulmonary infiltrates on chest imaging and evidence of preserved cardiac function on transthoracic echocardiography (LVEF range, 50-75%). SOFA scores ranged from 2 to 8 prior to stem cell treatment.

The six patients (age range, 19-75 years) had IV infusions of CAP-1002 containing 150 million allogeneic CDCs, and two of the six had a second dose of the treatment.

Following treatment, four patients (67%) were weaned from respiratory support and discharged from the hospital.

A contemporaneous control group of critically ill Covid-19 patients (n = 34) at our institution showed 18% overall mortality at a similar stage of hospitalization, the researchers wrote.

Ferritin was elevated in all patients at baseline (range of all patients 605.43-2991.52 ng/ml) and decreased in five of the six patients (range of all patients 252.891029.90 ng/ml).

Absolute lymphocyte counts were low in five of the six patients at baseline (range 0.260.82 103/l) but had increased in 3 of these five at last follow-up (range 0.231.02 103/l).

Administration of CAP-1002 as a compassionate therapy for patients with severe Covid-19 and significant comorbidities was safe, well tolerated without serious adverse events, and associated with clinical improvement, as evidenced by extubation (or prevention of intubation, the researchers wrote.

Stem cell therapy utilizing cardiosphere-derived cells (CDC) showed an acceptable safety profile with early evidence of efficacy in the treatment of very severe Covid-19 in an early case series involving 6 patients treated at Cedars-Sinai Medical Center, Los Angeles.

No adverse events related to the treatment were reported, and four of the six patients were successfully weaned from respiratory support and were discharged from the hospital.

Salynn Boyles, Contributing Writer, BreakingMED

Funding for this story was provided by the Smidt Family Foundation. The cell product, CAP-1002, was provided by manufacturer Capricor Therapeutics.

ResearcherEduardo Marban reported owning founders equity in Cariricor Therapeutics, and researcher Linda Marban reported being an employee and owning equity in the company.

Cat ID: 125

Topic ID: 79,125,254,930,287,728,932,570,574,730,933,125,190,926,192,927,151,928,925,934

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Cardio Stem Cell Therapy Used to Treat Critically Ill Covid-19 Patients - Physician's Weekly

Cardiac Stem Cells Comments Off on Cardio Stem Cell Therapy Used to Treat Critically Ill Covid-19 Patients – Physician’s Weekly | May 14th, 2020

Presentation of new and updated results from ongoing Phase 1/2 HGB-206 study of LentiGlobin for sickle cell disease will include additional patients treated in the study

New and updated data, including analysis of healthy red blood cell production in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) to be shared

CAMBRIDGE, Mass. bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene therapy programs for sickle cell disease (SCD), transfusion-dependent -thalassemia (TDT) and its cell therapy program for relapsed and refractory multiple myeloma (RRMM) will be presented during the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

New data from the companys Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will be presented, including updated data from patients in Group C.

bluebird bio will also present data from its ongoing clinical studies of betibeglogene autotemcel (formerly LentiGlobin gene therapy for -thalassemia), including the Phase 3 Northstar-2 (HGB-207) study in patients who do not have a 0/0 genotype and the Phase 3 Northstar-3 (HGB-212) study in patients who have 0/0, 0/+IVS-I-110, or +IVS-I-110/+IVS-I-110 genotypes.

Data from studies of idecabtagene vicleucel (ide-cel; bb2121), the companys anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy in development with Bristol Myers Squibb, will be presented, including an encore presentation of results from the pivotal Phase 2 KarMMa study.

Sickle Cell Disease Data at EHA25

Oral Presentation: Outcomes in patients treated with LentiGlobin for sickle cell disease (SCD) gene therapy: Updated results from the Phase 1/2 HGB-206 group C study Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala.

Transfusion-Dependent -Thalassemia Data at EHA25

Oral Presentation: Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study Presenting Author: John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospital, London, UK

Poster: Betibeglogene autotemcel (LentiGlobin) in patients with transfusion-dependent -thalassemia and 0/0, +IVS-I-110/+IVS-I-110, or 0/+IVS-I-110 genotypes: Updated results from the HGB-212 study Presenting Author: Evangelia Yannaki, M.D., George Papanicolaou Hospital, Thessaloniki, Greece

Multiple Myeloma Data at EHA25

Oral Presentation:Phase II KarMMa study: Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma Presenting Author: Jesus San-Miguel, M.D., Ph.D., Clinica Universidad de Navarra, Navarra, Spain

Poster: Quality of life in patients with relapsed and refractory multiple myeloma treated with the BCMA-targeted CAR T cell therapy Idecabtagene vicleucel (ide-cel; bb2121): results from the KarMMa Trial Presenting Author: Michel Delforge, M.D., Ph.D., Leuven University College, Brussels, Belgium

Poster: Matching-adjusted indirect comparisons of efficacy outcomes for idecabtagene vicleucel from the KarMMa study vs selinexor PLUS dexamethasone (STORM part 2) and belantamab mafodotin (DREAMM-2) Presenting Author: Paula Rodriguez-Otero, M.D., Clinica Universidad de Navarra, Navarra, Spain

Poster: Baseline and postinfusion pharmcodynamic biomarkers of safety and efficacy in patients treated with idecabtagene vicleucel (ide-cel; bb2121) in the KarMMa study Presenting Author: Justine DellAringa, Bristol Myers Squibb, Seattle, Wash.

Poster: Correlation of tumor BCMA expression with response and acquired resistance to idecabtagene vicleucel in the KarMMa study in relapsed and refractory multiple myeloma Presenting Author: Nathan Martin, Bristol Myers Squibb, Seattle, Wash.

Abstracts outlining bluebird bios accepted data at the EHA25 Virtual Congress have been made available on the EHA25 conference website. On Friday, June 12 at 8:30 AM CEST, the embargo will lift for poster and oral presentations accepted for EHA25.

About betibeglogene autotemcel The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO including three patients with up to five years of follow-up.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during the clinical studies that were attributed to betibeglogene autotemcel were abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted betibeglogene autotemcel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. Betibeglogene autotemcel is not approved in the United States.

Betibeglogene autotemcel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

About LentiGlobin for Sickle Cell Disease LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive crises (VOCs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration (FDA) granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

LentiGlobin for SCD is investigational and has not been approved by the European Medicines Agency (EMA) or FDA.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel and LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About idecabtagene vicleucel (ide-cel; bb2121) Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

In addition to the pivotal KarMMa trial evaluating ide-cel in patients with relapsed and refractory multiple myeloma, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) and PRIority Medicines (PRIME) designation, as well as Accelerated Assessment status, by the European Medicines Agency for relapsed and refractory multiple myeloma.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

Ide-cel is not approved for any indication in any geography.

About KarMMa KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 10P6P CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

About bluebird bio, Inc. bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: regarding the potential for betibeglogene autotemcel to treat transfusion-dependent -thalassemia and the potential for LentiGlobin for sickle cell disease (SCD) to treat SCD; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200514005234/en/

Contacts

Media: Catherine Falcetti, 339-499-9436 cfalcetti@bluebirdbio.com Victoria von Rinteln, 617-914-8774 vvonrinteln@bluebirdbio.com

Investors: Ingrid Goldberg, 410-960-5022 Ingrid.goldberg@bluebirdbio.com Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com

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bluebird bio to Present Data from Its Gene and Cell Therapy Programs During the Virtual Edition of the 25th European Hematology Association Annual...

Cardiac Stem Cells Comments Off on bluebird bio to Present Data from Its Gene and Cell Therapy Programs During the Virtual Edition of the 25th European Hematology Association Annual… | May 14th, 2020

This discovery may have clinical importance in management of heart failure.

This discovery may have clinical importance in management of heart failure.Many heart diseases are linked to oxidative stress, an overabundance of reactive oxygen species. The body reacts to reduce oxidative stress where the redox teeter-totter has gone too far up through production of endogenous antioxidants that reduce the reactive oxygen species. This balancing act is called redox homeostasis.

But what happens if the redox teeter-totter goes too far down, creating antioxidative stress, also known as reductive stress? Rajasekaran Namakkal-Soorappan, Ph.D., associate professor in the University of Alabama at Birmingham Department of Pathology, and colleagues have found that reductive stress, or RS/AS, is also pathological. This discovery, they say, may have clinical importance in management of heart failure.

They report that RS causes pathological heart enlargement and diastolic dysfunction in a mouse model. This study, published in the journal Antioxidants and Redox Signaling, was led by Namakkal-Soorappan and Pei Ping, Ph.D., David Geffen School of Medicine at the University of California-Los Angeles.

Antioxidant-based therapeutic approaches for human heart failure should consider a thorough evaluation of antioxidant levels before the treatment, they said. Our findings demonstrate that chronic RS is intolerable and adequate to induce heart failure.

The study used transgenic mice that had upregulated genes for antioxidants in the heart, which increased the amounts of antioxidant proteins and reduced glutathione, creating RS. One mouse line had low upregulation, and one had high upregulation, creating chronic low RS and chronic high RS, respectively, in the hearts of the mice.

The mice with high RS showed pathological heart changes called hypertrophic cardiomyopathy, and had an abnormally high heart ejection fraction and diastolic dysfunction at 6 months of age. Sixty percent of the high-RS mice died by 18 months of age.

The mice with low RS had normal survival rates, but they developed the heart changes at about 15 months of age, suggesting that even moderate RS can lead to irreversible damage in the heart over time.

Giving high-RS mice a chemical that blocked biosynthesis of glutathione, beginning at about 6 weeks of age, prevented RS and rescued the mice from pathological heart changes.

Gobinath Shanmugam, Ph.D., postdoctoral fellow in the UAB Department of Pathology, and Namakkal-Soorappan point out that a 2019 survey found about 77 percent of Americans are consuming dietary supplements every day, and within this group, about 58 percent are consuming antioxidants as multivitamins. Thus, a chronic consumption of antioxidant drugs by any individual without knowing their redox state might result in RS, which can induce pathology and slowly damage the heart.

In a related study, published in the journal Redox Biology, Namakkal-Soorappan looked at the impact of RS on myosatellite cells, which are also known as muscle stem cells. These cells, located near skeletal muscle fibers, are able to regenerate and differentiate into skeletal muscle after acute or chronic muscle injury. The regulation of myosatellite cells is of interest given the loss of skeletal muscle mass during aging or in chronic conditions like diabetes and AIDS.

Recently, Namakkal-Soorappan reported that tilting the redox teeter-totter to oxidative stress impaired regeneration of skeletal muscle. Now, in the Redox Biology paper, he has shown that tilting the redox to RS also causes significant inhibition of muscle satellite cell differentiation.

Rather than genetic manipulation to induce RS, as was done in the heart study, the researchers used the chemical sulforaphane or direct augmentation of intracellular glutathione to induce RS in cultured mouse myoblast cells. Both treatments inhibited myoblast differentiation. Finally, authors attempted to withdraw antioxidative stress by growing cells in medium without sulforaphane, which removes the RS and accelerates the differentiation. Namakkal-Soorappan and colleagues found that a pro-oxidative milieu, through a mild generation of reactive oxygen species, was required for myoblast differentiation.

The researchers also showed that genetic silencing of a negative regulator of the antioxidant genes also inhibited myoblast differentiation.

Co-authors with Namakkal-Soorappan and Ping, and first-author Shanmugam, in the Antioxidants and Redox Signaling study, Reductive stress causes pathological cardiac remodeling and diastolic dysfunction, are Silvio H. Litovsky and Rajesh Kumar Radhakrishnan, UAB Department of Pathology; Ding Wang, UCLA; Sellamuthu S. Gounder, Kevin Whitehead, Sarah Franklin and John R. Hoidal, University of Utah School of Medicine; Jolyn Fernandes and Dean P. Jones, Emory University, Atlanta, Georgia; Thomas W. Kensler, Fred Hutch Cancer Research Center, Seattle, Washington; Louis DellItalia, UAB Department of Medicine; Victor Darley-Usmar, UAB Department of Pathology; and E. Dale Abel, University of Iowa.

In the Redox Biology study, Reductive stress impairs myogenic differentiation, co-authors with Namakkal-Soorappan are Sandeep Balu Shelar, UAB Department of Pathology; Dean P. Jones, Emory University; and John R. Hoidal, University of Utah School of Medicine.

Support for both studies came from National Institutes of Health grants HL118067 and AG042860, American Heart Association grant BGIA 0865015F, the University of Utah, and UAB.

In the two studies, Namakkal-Soorappans name is listed as Namakkal S. Rajasekaran.

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Surplus antioxidants are pathogenic for hearts and skeletal muscle - The Mix

Cardiac Stem Cells Comments Off on Surplus antioxidants are pathogenic for hearts and skeletal muscle – The Mix | May 14th, 2020

Repeated setbacks aside, little Capricor has suggested it has generated some long-term data to support its pursuit to garner approval for its stem cell therapy for Duchenne muscular dystrophy, although some of the data appeared to underwhelmed investors.

The data from the small, placebo-controlled mid-stage study, HOPE-2, tracked the effects of the companys stem cell therapy CAP-1002, which is designed to temper the inflammation associated with DMD, in 8 boys and young men who are in advanced stages of DMD. The remaining 12 enrolled patients received the placebo.

The main goal of the study was a measure that evaluates shoulder, arm and hand strength in patients who are generally non-ambulant (performance of the upper limb (PUL) 2.0), as suggested by the FDA, Capricor said. It is one of several ways Capricor quantified skeletal muscle improvement in the trial.

The intravenous infusion of CAP-1002, given every 3 months, induced a statistically meaningful improvement of 2.4 points (p=0.05) versus the placebo group, in which patient declines were consistent with natural history data. However, on another measure of upper limb function, the trend was in favor of the Capricor drug, but did not hit statistical significance.

The companys shares $CAPR were down nearly 13% to $6.89 in morning trading.

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Meanwhile, there were also some encouraging data on cardiac function the genetic condition is characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles.

As reflected above, CAP-1002 elicited an improvement across different measures of cardiac function, although the effect was not always statistically significant. In particular, the drug also caused a reduction in the levels of the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage.

Armed with these data and an RMAT and orphan drug designation from the FDA, Capricor is now hoping to eke out a plan with the FDA for marketing approval.

LA-based Capricor initially set out to test the potential of technology that Eduardo Marbn, CEO Linda Marbns husband, developed at Johns Hopkins. But repeated setbacks clobbered the company, which in 2014 traded north of $14 a share. In 2017, J&J walked away from a collaboration on a stem cell therapy for damaged hearts after it flopped in the clinic.

In late 2018, the company voluntarily halted a DMD clinical trial, following a severe allergic reaction that occurred during infusion. In February 2019, the company said it is exploring strategic alternatives for one or more of its products and cutting 21 jobs to keep financially afloat, but had resumed dosing in its DMD trial.

The first batch of positive data on CAP-1002, which consists of progenitor cells derived from donor hearts and is designed to exude exosomes that initiate muscle repair by suppressing inflammation and driving immunomodulation, came last July when the company announced the drug had generated a positive effect at the interim analysis juncture of HOPE-2. Capricor is now working on to flexing its therapeutic muscle with CAP-1002 to fight the Covid-19 pandemic.

DMD is a rare muscle-wasting disease caused by the absence of dystrophin, a protein that helps keep muscle cells intact. It disproportionately affects boys and affects roughly 6,000 in the United States.

Patients are essentially treated with steroids. Sarepta Therapeutics now has two exon-skipping drugs designed to treat certain subsets of the disease, although the magnitude of their effect is controversial given that approvals were not based on placebo-controlled data. Meanwhile, Sarepta and others are also pursuing one-time cures in the form of gene therapies to replace the missing dystrophin gene in patients.

Social: Linda Marbn, Capricor CEO (Twitter)

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One year on, Capricor's stem cell therapy appears to help DMD patients in small study, but investors balk at the data - Endpoints News

Cardiac Stem Cells Comments Off on One year on, Capricor’s stem cell therapy appears to help DMD patients in small study, but investors balk at the data – Endpoints News | May 13th, 2020

You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes that are included elsewhere in this QuarterlyReport on Form 10-Q and our Annual Report on Form 10-K for the fiscal year endedDecember 31, 2019 filed with the U.S. Securities and Exchange Commission, or theSEC, on March 6, 2020, or our 2019 Form 10-K. This discussion containsforward-looking statements based upon current plans, expectations and beliefsthat involve risks and uncertainties. Our actual results may differ materiallyfrom those anticipated in these forward-looking statements as a result ofvarious factors, including, but not limited to, those discussed in the sectionentitled "Risk Factors" and elsewhere in this Quarterly Report on Form 10-Q. Inpreparing this MD&A, we presume that readers have access to and have read theMD&A in our 2019 Form 10-K, pursuant to Instruction 2 to paragraph (b) of Item303 of Regulation S-K. Unless stated otherwise, references in this QuarterlyReport on Form 10-Q to "us," "we," "our," or our "Company" and similar termsrefer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on thedevelopment of first, only and best-in-class gene therapies, with directon-target mechanism of action and clear clinical endpoints, for rare anddevastating diseases. We currently have three clinical-stage ex vivo lentiviralvector ("LVV") programs currently enrolling patients in the US and EU forFanconi Anemia ("FA"), a genetic defect in the bone marrow that reducesproduction of blood cells or promotes the production of faulty blood cells,Leukocyte Adhesion Deficiency-I ("LAD-I"), a genetic disorder that causes theimmune system to malfunction and Pyruvate Kinase Deficiency ("PKD"), a rare redblood cell autosomal recessive disorder that results in chronic non-spherocytichemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-Iprogram are in registration-enabling studies in the US and EU. In addition, inthe US we have a clinical stage in vivo adeno-associated virus ("AAV") programfor Danon disease, a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Finally, we have a pre-clinical stage LVV programfor Infantile Malignant Osteopetrosis ("IMO"), a genetic disorder characterizedby increased bone density and bone mass secondary to impaired bone resorption -this program is anticipated to enter the clinic in 2020. We have globalcommercialization and development rights to all of these product candidatesunder royalty-bearing license agreements. Additional work in the discovery stagefor an FA CRISPR/CAS9 program as well as a gene therapy program for the lesscommon FA subtypes C and G is ongoing.

Recent Developments

On February 20, 2020, we entered into separate, privately negotiated exchangeagreements (the "Exchange Agreements") with certain holders of our outstanding5.75% Convertible Senior Notes due 2021 (the "2021 Convertible Notes") to extendthe maturity date by one year. Pursuant to the Exchange Agreements, we exchangedapproximately $39.35 million aggregate principal amount of the 2021 ConvertibleNotes (which represents approximately 76% of the aggregate outstanding principalamount of the 2021 Convertible Notes) for (a) approximately $39.35 millionaggregate principal amount of 6.25% Convertible Senior Notes due August 2022(the "2022 Convertible Notes") (an exchange ratio equal to 1.00 2022 ConvertibleNote per exchanged 2021 Convertible Note) and (b) $119,416 in cash to pay theaccrued and unpaid interest on the exchanged 2021 Convertible Notes from, andincluding, February 1, 2020 to February 20, 2020. The 2022 Convertible Noteswere issued in private placements exempt from registration in reliance onSection 4(a) (2) of the Securities Act of 1933, as amended (the "SecuritiesAct"). Upon completion of the exchange transactions, approximately $12.65million aggregate principal amount of 2021 Convertible Notes remainedoutstanding.

Gene Therapy Overview

Genes are composed of sequences of deoxyribonucleic acid ("DNA"), which code forproteins that perform a broad range of physiologic functions in all livingorganisms. Although genes are passed on from generation to generation, geneticchanges, also known as mutations, can occur in this process. These changes canresult in the lack of production of proteins or the production of alteredproteins with reduced or abnormal function, which can in turn result in disease.

Gene therapy is a therapeutic approach in which an isolated gene sequence orsegment of DNA is administered to a patient, most commonly for the purpose oftreating a genetic disease that is caused by genetic mutations. Currentlyavailable therapies for many genetic diseases focus on administration of largeproteins or enzymes and typically address only the symptoms of the disease. Genetherapy aims to address the disease-causing effects of absent or dysfunctionalgenes by delivering functional copies of the gene sequence directly into thepatient's cells, offering the potential for curing the genetic disease, ratherthan simply addressing symptoms.

We are using modified non-pathogenic viruses for the development of our genetherapy treatments. Viruses are particularly well suited as delivery vehiclesbecause they are adept at penetrating cells and delivering genetic materialinside a cell. In creating our viral delivery vehicles, the viral (pathogenic)genes are removed and are replaced with a functional form of the missing ormutant gene that is the cause of the patient's genetic disease. The functionalform of a missing or mutant gene is called a therapeutic gene, or the"transgene." The process of inserting the transgene is called "transduction."Once a virus is modified by replacement of the viral genes with a transgene, themodified virus is called a "viral vector." The viral vector delivers thetransgene into the targeted tissue or organ (such as the cells inside apatient's bone marrow). We have two types of viral vectors in development, LVVand AAV. We believe that our LVV and AAV-based programs have the potential tooffer a long-lasting and significant therapeutic benefit to patients.

Gene therapies can be delivered either (1) ex vivo (outside the body), in whichcase the patient's cells are extracted and the vector is delivered to thesecells in a controlled, safe laboratory setting, with the modified cells thenbeing reinserted into the patient, or (2) in vivo (inside the body), in whichcase the vector is injected directly into the patient, either intravenously("IV") or directly into a specific tissue at a targeted site, with the aim ofthe vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greaterregulatory acceptance of gene therapy have created a promising environment toadvance gene therapy products as these products are being designed to restorecell function and improve clinical outcomes, which in many cases includeprevention of death at an early age.

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The chart below shows the current phases of development of Rocket's programs andproduct candidates:

LVV Programs. Rocket's LVV-based programs utilize third-generation,self-inactivating lentiviral vectors to target selected rare diseases.Currently, Rocket is developing LVV programs to treat FA, LAD-I, PKD, and IMO.

Fanconi Anemia Complementation Group A (FANCA):

FA, a rare and life-threatening DNA-repair disorder, generally arises from amutation in a single FA gene. An estimated 60 to 70% of cases arise frommutations in the Fanconi-A ("FANCA") gene, which is the focus of our program. FAresults in bone marrow failure, developmental abnormalities, myeloid leukemiaand other malignancies, often during the early years and decades of life. Bonemarrow aplasia, which is bone marrow that no longer produces any or very few redand white blood cells and platelets leading to infections and bleeding, is themost frequent cause of early morbidity and mortality in FA, with a median onsetbefore 10 years of age. Leukemia is the next most common cause of mortality,ultimately occurring in about 20% of patients later in life. Solid organmalignancies, such as head and neck cancers, can also occur, although at lowerrates during the first two to three decades of life.

Although improvements in allogeneic (donor-mediated) hematopoietic stem celltransplant ("HSCT"), currently the most frequently utilized therapy for FA, haveresulted in more frequent hematologic correction of the disorder, HSCT isassociated with both acute and long-term risks, including transplant-relatedmortality, graft versus host disease ("GVHD"), a sometimes fatal side effect ofallogeneic transplant characterized by painful ulcers in the GI tract, livertoxicity and skin rashes, as well as increased risk of subsequent cancers. Ourgene therapy program in FA is designed to enable a minimally toxic hematologiccorrection using a patient's own stem cells during the early years of life. Webelieve that the development of a broadly applicable autologous gene therapy canbe transformative for these patients.

Each of our LVV-based programs utilize third-generation, self-inactivatinglentiviral vectors to correct defects in patients' HSCs, which are the cellsfound in bone marrow that are capable of generating blood cells over a patient'slifetime. Defects in the genetic coding of HSCs can result in severe, andpotentially life-threatening anemia, which is when a patient's blood lacksenough properly functioning red blood cells to carry oxygen throughout the body.Stem cell defects can also result in severe and potentially life-threateningdecreases in white blood cells resulting in susceptibility to infections, and inplatelets responsible for blood clotting, which may result in severe andpotentially life-threatening bleeding episodes. Patients with FA have a geneticdefect that prevents the normal repair of genes and chromosomes within bloodcells in the bone marrow, which frequently results in the development of acutemyeloid leukemia ("AML"), a type of blood cancer, as well as bone marrow failureand congenital defects. The average lifespan of an FA patient is estimated to be30 to 40 years. The prevalence of FA in the US and EU is estimated to be about4,000, and given the efficacy seen in non-conditioned patients, the addressableannual market opportunity is now thought to be in the 400 to 500 range.

We currently have one LVV-based program targeting FA, RP-L102. RP-L102 is ourlead lentiviral vector based program that we in-licensed from Centro deInvestigaciones Energticas, Medioambientales y Tecnolgicas ("CIEMAT"), whichis a leading research institute in Madrid, Spain. RP-L102 is currently beingstudied in our sponsored Phase 2 registrational enabling clinical trialstreating FA patients initially at the Center for Definitive and CurativeMedicine at Stanford University School of Medicine ("Stanford") and HospitalInfantil de Nino Jesus ("HNJ") in Spain. The Phase 2 portion of the trial isexpected to enroll ten patients total from the U.S. and EU. Patients willreceive a single IV infusion of RP-L102 that utilizes fresh cells and "ProcessB" which incorporates a modified stem cell enrichment process, transductionenhancers, as well as commercial-grade vector and final drug product.

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Table of ContentsIn October 2019, at the European Society of Cell and Gene Therapy ("ESGCT") 2019Annual Congress, long-term Phase 1/2 clinical data of RP-L102, from the clinicaltrial sponsored by CIEMAT, for FA "Process A", without the use of myeloablativeconditioning was presented demonstrating evidence of increasing and durableengraftment leading to bone marrow restoration exceeding the 10% thresholdagreed to by the FDA and EMA for the ongoing registration-enabling Phase 2trial. In patient 02002, who received what we consider adequate drug product,hemoglobin levels are now similar to those in the first year after birth,suggesting hematologic correction over the long term.

During the third quarter of 2019, we received alignment from the FDA on thetrial design and the primary endpoint. This alignment was similar to thatpreviously received from the European Medicines Agency ("EMA"). Resistance tomitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum timepoint of one year to serve as the primary endpoint for our Phase II study. InDecember 2019, we announced that the first patient of the global Phase 2 studyfor RP-L102 "Process B" for FA received investigational therapy. There will betotal of 10 patients enrolled in the global Phase 2 studies.

In December 2019, we also announced preliminary results from two pediatricpatients treated with "Process B" RP-L102 prior to development of severe bonemarrow failure in our Phase 1 trial of RP-L102 for FA. To evaluate transductionefficiency, an analysis of the proportion of the MMC-resistant colony formingcells was conducted and both patients have thus far exhibited early signs ofengraftment, including increases in blood cell lineages in one patient. Nodrug-related safety or tolerability issues have been reported.

Leukocyte Adhesion Deficiency-I (LAD-I):

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion andmigration, resulting from mutations in the ITGB2 gene encoding for the Beta-2Integrin component, CD18. Deficiencies in CD18 result in an impaired ability forneutrophils (a subset of infection-fighting white blood cells) to leave bloodvessels and enter into tissues where these cells are needed to combatinfections. As is the case with many rare diseases, true estimates of incidenceare difficult; however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease. SevereLAD-I is notable for recurrent, life-threatening infections and substantialinfant mortality in patients who do not receive an allogeneic HSCT. Mortalityfor severe LAD-I has been reported as 60 to 75% by age two in the absence ofallogeneic HCST.

We currently have one program targeting LAD-I, RP-L201. RP-L201 is a clinicalprogram that we in-licensed from CIEMAT. We have partnered with UCLA to leadU.S. clinical development efforts for the LAD-I program. UCLA and its Eli andEdythe Broad Center of Regenerative Medicine and Stem Cell Research is servingas the lead U.S. clinical research center for the registrational clinical trialfor LAD-I, and HNJ is serving as the lead clinical site in Spain.

The ongoing open-label, single-arm, Phase 1/2 registration enabling clinicaltrial of RP-L201 has dosed one severe LAD-I patient in the U.S. to assess thesafety and tolerability of RP-L201. The first patient was treated with RP-L201in third quarter 2019. This study has received $6.5 million CLIN2 grant awardfrom the California Institute for Regenerative Medicine ("CIRM") to support theclinical development of gene therapy for LAD-I.

In December 2019, we announced initial results from the first pediatric patienttreated with RP-L201, demonstrating early evidence of safety. Analyses ofperipheral vector copy number ("VCN"), and CD18-expressing neutrophils wereperformed through three months after infusion of RP-L201 to evaluate engraftmentand phenotypic correction. The patient exhibited early signs of engraftment withVCN myeloid levels at 1.5 at three months and CD-18 expression of 45%. No safetyor tolerability issues related to RP-L201 administration (or investigationalproduct) had been identified as of that date. The study is expected to enrollnine patients globally.

Pyruvate Kinase Deficiency (PKD):

Red blood cell PKD is a rare autosomal recessive disorder resulting frommutations in the pyruvate kinase L/R ("PKLR") gene encoding for a component ofthe red blood cell ("RBC") glycolytic pathway. PKD is characterized by chronicnon-spherocytic hemolytic anemia, a disorder in which RBCs do not assume anormal spherical shape and are broken down, leading to decreased ability tocarry oxygen to cells, with anemia severity that can range from mild(asymptomatic) to severe forms that may result in childhood mortality or arequirement for frequent, lifelong RBC transfusions. The pediatric population isthe most commonly and severely affected subgroup of patients with PKD, and PKDoften results in splenomegaly (abnormal enlargement of the spleen), jaundice andchronic iron overload which is likely the result of both chronic hemolysis andthe RBC transfusions used to treat the disease. The variability in anemiaseverity is believed to arise in part from the large number of diverse mutationsthat may affect the PKLR gene. Estimates of disease incidence have rangedbetween 3.2 and 51 cases per million in the white U.S. and EU population.Industry estimates suggest at least 2,500 cases in the U.S. and EU have alreadybeen diagnosed despite the lack of FDA-approved molecularly targeted therapies.Enrollment is currently ongoing and we anticipate treating the first patient inthe third quarter of 2020.

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Table of ContentsWe currently have one LVV-based program targeting PKD, RP-L301. RP-L301 is aclinical stage program that we in-licensed from CIEMAT. The IND for RP-L301 toinitiate a global Phase 1 study was cleared by the FDA in October 2019. Thisprogram has been granted EMA orphan drug disease designation and FDA orphan drugdisease designation ("ODD").

This global Phase 1 open-label, single-arm, clinical trial is expected to enrollsix adult and pediatric transfusion-dependent PKD patients in the U.S. andEurope. Lucile Packard Children's Hospital Stanford will serve as the lead sitein the U.S. for adult and pediatric patients, and Hospital InfantilUniversitario Nio Jess will serve as the lead site in Europe for pediatricsand Hospital Universitario Fundacin Jimnez Daz will serve as the lead site inEurope for adult patients.

Infantile Malignant Osteopetrosis (IMO):

IMO is a genetic disorder characterized by increased bone density and bone masssecondary to impaired bone resorption. Normally, small areas of bone areconstantly being broken down by special cells called osteoclasts, then madeagain by cells called osteoblasts. In IMO, the cells that break down bone(osteoclasts) do not work properly, which leads to the bones becoming thickerand not as healthy. Untreated IMO patients may suffer from a compression of thebone-marrow space, which results in bone marrow failure, anemia and increasedinfection risk due to the lack of production of white blood cells. Untreated IMOpatients may also suffer from a compression of cranial nerves, which transmitsignals between vital organs and the brain, resulting in blindness, hearing lossand other neurologic deficits.

We currently have one LVV-based program targeting IMO, RP-L401. RP-L401 is apreclinical program that we in-licensed from Lund University, Sweden. Thisprogram has been granted ODD and Rare Pediatric Disease designation from theFDA. The FDA defines a "rare pediatric disease" as a serious andlife-threatening disease that affects less than 200,000 people in the U.S. thatare aged between birth to 18 years. The Rare Pediatric Disease designationprogram allows for a sponsor who receives an approval for a product topotentially qualify for a voucher that can be redeemed to receive a priorityreview of a subsequent marketing application for a different product. We havepartnered with UCLA to lead U.S. clinical development efforts for the IMOprogram and anticipate that UCLA will serve as the lead U.S. clinical site forIMO. We intend to file an IND for IMO and commence our clinical trial in thefourth quarter of 2020.

Danon disease is a multi-organ lysosomal-associated disorder leading to earlydeath due to heart failure. Danon disease is caused by mutations in the geneencoding lysosome-associated membrane protein 2 ("LAMP-2"), a mediator ofautophagy. This mutation results in the accumulation of autophagic vacuoles,predominantly in cardiac and skeletal muscle. Male patients often require hearttransplantation and typically die in their teens or twenties from progressiveheart failure. Along with severe cardiomyopathy, other Danon disease symptomscan include skeletal muscle weakness, liver disease, and intellectualimpairment. There are no specific therapies available for the treatment of Danondisease. RP-A501 is in clinical trials as an in vivo therapy for Danon disease,which is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S.and the EU, however new market research is being performed and the prevalence ofpatients may be updated in the future.

In January 2019, we announced the clearance of our IND application by the FDAfor RP-A501, and in February 2019, we were notified by the FDA that we weregranted Fast Track designation for RP-A501. University of California San DiegoHealth is the initial and lead center for our Phase 1 clinical trial.

On May 2, 2019, we presented additional preclinical data at the ASCGT annualmeeting, indicating that high VCN, in Danon disease-relevant organs in both miceand non-human primates ("NHN's"), with high concentrations in heart and livertissue (for NHP, cardiac VCN was approximately 10 times higher on average thanin skeletal muscle and central nervous system), which is consistent withreported results in several studies of heart tissue across different species.There were no treatment-related adverse events or safety issues up to thehighest dose. We have dosed three patients in the RP-A501 phase 1 clinicaltrial. We will continue further enrollment with clinical data read-outs in thefourth quarter of 2020.

As of March 2020, we have dosed three patients in the RP-A501 phase 1 clinicaltrial. This completes the first low dose cohort of the Phase 1 study. Based onthe preliminary safety and efficacy data review of this completed cohort, boththe FDA and IDMC has provided clearance to advance to a higher dose cohort inPhase 1 Trial of RP-A501 for Danon Disease. We will continue further enrollmentwith clinical data read-outs in the second half of 2020.

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In addition to its LVV and AAV programs, we also have a program evaluatingCRISPR/Cas9-based gene editing for FA. This program is currently in thediscovery phase. CRISPR/Cas9-based gene editing is a different method ofcorrecting the defective genes in a patient, where the editing is very specificand targeted to a particular gene sequence. "CRISPR/Cas9" stands for Clustered,Regularly Interspaced Short Palindromic Repeats ("CRISPR") Associated protein-9.The CRISPR/Cas9 technology can be used to make "cuts" in DNA at specific sitesof targeted genes, making it potentially more precise in delivering genetherapies than traditional vector-based delivery approaches. CRISPR/Cas9 canalso be adapted to regulate the activity of an existing gene without modifyingthe actual DNA sequence, which is referred to as gene regulation.

Strategy

We seek to bring hope and relief to patients with devastating, undertreated,rare pediatric diseases through the development and commercialization ofpotentially curative first-in-class gene therapies. To achieve these objectives,we intend to develop into a fully-integrated biotechnology company. In the near-and medium-term, we intend to develop our first-in-class product candidates,which are targeting devastating diseases with substantial unmet need, developproprietary in-house analytics and manufacturing capabilities and continue tocommence registration trials for our currently planned programs. In the mediumand long-term, we expect to submit our first biologics license applications("BLAs"), and establish our gene therapy platform and expand our pipeline totarget additional indications that we believe to be potentially compatible withour gene therapy technologies. In addition, during that time, we believe thatour currently planned programs will become eligible for priority review vouchersfrom the FDA that provide for expedited review. We have assembled a leadershipand research team with expertise in cell and gene therapy, rare disease drugdevelopment and commercialization.

We believe that our competitive advantage lies in our disease-based selectionapproach, a rigorous process with defined criteria to identify target diseases.We believe that this approach to asset development differentiates us as a genetherapy company and potentially provides us with a first-mover advantage.

Financial Overview

Since our inception, we have devoted substantially all of our resources toorganizing and staffing the Company, business planning, raising capital,acquiring or discovering product candidates and securing related intellectualproperty rights, conducting discovery, research and development activities forthe programs and planning for potential commercialization. We do not have anyproducts approved for sale and have not generated revenue from product sales.From inception through March 31, 2020, we raised net cash proceeds ofapproximately $373.1 million from investors through both equity and convertibledebt financing to fund operating activities. As of March 31, 2020, we had cash,cash equivalents and investments of $275.9 million.

Since inception, we have incurred significant operating losses. Our ability togenerate product revenue sufficient to achieve profitability will depend heavilyon the successful development and eventual commercialization of one or more ofthe current or future product candidates and programs. We had net losses of$24.7 million for the three months ended March 31, 2020 and $77.3 million forthe year ended December 31, 2019. As of March 31, 2020, we had an accumulateddeficit of $207.8 million. We expect to continue to incur significant expensesand higher operating losses for the foreseeable future as we advance our currentproduct candidates from discovery through preclinical development and clinicaltrials and seek regulatory approval of our product candidates. In addition, ifwe obtain marketing approval for any of their product candidates, we expect toincur significant commercialization expenses related to product manufacturing,marketing, sales and distribution. Furthermore, we expect to incur additionalcosts as a public company. Accordingly, we will need additional financing tosupport continuing operations and potential acquisitions of licensing or otherrights for product candidates.

Until such a time as we can generate significant revenue from product sales, ifever, we will seek to fund our operations through public or private equity ordebt financings or other sources, which may include collaborations with thirdparties and government programs or grants. Adequate additional financing may notbe available to us on acceptable terms, or at all. We can make no assurancesthat we will be able to raise the cash needed to fund our operations and, if wefail to raise capital when needed, we may have to significantly delay, scaleback or discontinue the development and commercialization of one or more productcandidates or delay pursuit of potential in-licenses or acquisitions.

Because of the numerous risks and uncertainties associated with productdevelopment, we are unable to predict the timing or amount of increased expensesor when or if we will be able to achieve or maintain profitability. Even if weare able to generate product sales, we may not become profitable. If we fail tobecome profitable or are unable to sustain profitability on a continuing basis,then we may be unable to continue our operations at planned levels and be forcedto reduce or terminate our operations.

Revenue

To date, we have not generated any revenue from any sources, including fromproduct sales, and we do not expect to generate any revenue from the sale ofproducts in the near future. If our development efforts for product candidatesare successful and result in regulatory approval or license agreements withthird parties, we may generate revenue in the future from product sales.

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Research and Development Expenses

Our research and development program ("R&D") expenses consist primarily ofexternal costs incurred for the development of our product candidates. Theseexpenses include:

expenses incurred under agreements with research institutions that conduct

research and development activities including, process development,

preclinical, and clinical activities on Rocket's behalf;

costs related to process development, production of preclinical and clinical

materials, including fees paid to contract manufacturers and manufacturing

input costs for use in internal manufacturing processes;

consultants supporting process development and regulatory activities; and

costs related to in-licensing of rights to develop and commercialize our

product candidate portfolio.

We recognize external development costs based on contractual payment schedulesaligned with program activities, invoices for work incurred, and milestoneswhich correspond with costs incurred by the third parties. Nonrefundable advancepayments for goods or services to be received in the future for use in researchand development activities are recorded as prepaid expenses.

Our direct research and development expenses are tracked on a program-by-programbasis for product candidates and consist primarily of external costs, such asresearch collaborations and third party manufacturing agreements associated withour preclinical research, process development, manufacturing, and clinicaldevelopment activities. Our direct research and development expenses by programalso include fees incurred under license agreements. Our personnel, non-programand unallocated program expenses include costs associated with activitiesperformed by our internal research and development organization and generallybenefit multiple programs. These costs are not separately allocated by productcandidate and consist primarily of:

Our research and development activities are central to our business model.Product candidates in later stages of clinical development generally have higherdevelopment costs than those in earlier stages of clinical development. As aresult, we expect that research and development expenses will increasesubstantially over the next several years as we increase personnel costs,including stock-based compensation, support ongoing clinical studies, seek toachieve proof-of-concept in one or more product candidates, advance preclinicalprograms to clinical programs, and prepare regulatory filings for productcandidates.

We cannot determine with certainty the duration and costs to complete current orfuture clinical studies of product candidates or if, when, or to what extent wewill generate revenues from the commercialization and sale of any of our productcandidates that obtain regulatory approval. We may never succeed in achievingregulatory approval for any of our product candidates. The duration, costs, andtiming of clinical studies and development of product candidates will depend ona variety of factors, including:

the scope, rate of progress, and expense of ongoing as well as any future

clinical studies and other research and development activities that we

undertake;

future clinical trial results;

uncertainties in clinical trial enrollment rates;

changing standards for regulatory approval; and

the timing and receipt of any regulatory approvals.

We expect research and development expenses to increase for the foreseeablefuture as we continue to invest in research and development activities relatedto developing product candidates, including investments in manufacturing, as ourprograms advance into later stages of development and as we conduct additionalclinical trials. The process of conducting the necessary clinical research toobtain regulatory approval is costly and time-consuming, and the successfuldevelopment of product candidates is highly uncertain. As a result, we areunable to determine the duration and completion costs of research anddevelopment projects or when and to what extent we will generate revenue fromthe commercialization and sale of any of our product candidates.

Our future research and development expenses will depend on the clinical successof our product candidates, as well as ongoing assessments of the commercialpotential of such product candidates. In addition, we cannot forecast with anydegree of certainty which product candidates may be subject to futurecollaborations, when such arrangements will be secured, if at all, and to whatdegree such arrangements would affect our development plans and capitalrequirements. We expect our research and development expenses to increase infuture periods for the foreseeable future as we seek to complete development ofour product candidates.

The successful development and commercialization of our product candidates ishighly uncertain. This is due to the numerous risks and uncertainties associatedwith product development and commercialization, including the uncertainty of:

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Table of Contents

the scope, progress, outcome and costs of our clinical trials and other

research and development activities;

the efficacy and potential advantages of our product candidates compared to

alternative treatments, including any standard of care;

the market acceptance of our product candidates;

obtaining, maintaining, defending and enforcing patent claims and other

intellectual property rights;

significant and changing government regulation; and

the timing, receipt and terms of any marketing approvals.

A change in the outcome of any of these variables with respect to thedevelopment of our product candidates that we may develop could mean asignificant change in the costs and timing associated with the development ofour product candidates. For example, if the FDA or another regulatory authoritywere to require us to conduct clinical trials or other testing beyond those thatwe currently contemplate for the completion of clinical development of any ofour product candidates that we may develop or if we experience significantdelays in enrollment in any of our clinical trials, we could be required toexpend significant additional financial resources and time on the completion ofclinical development of that product candidate.

General and Administrative Expenses

General and administrative ("G&A") expenses consist primarily of salaries andrelated benefit costs for personnel, including stock-based compensation andtravel expenses for our employees in executive, operational, finance, legal,business development, and human resource functions. In addition, othersignificant general and administrative expenses include professional fees forlegal, patents, consulting, investor and public relations, auditing and taxservices as well as other expenses for rent and maintenance of facilities,insurance and other supplies used in general and administrative activities. Weexpect general and administrative expenses to increase for the foreseeablefuture due to anticipated increases in headcount to support the continuedadvancement of our product candidates. We also anticipate that we will incurincreased accounting, audit, legal, regulatory, compliance and director andofficer insurance costs as well as investor and public relations expenses.

Interest Expense

Interest expense is related to the 2021 Convertible Notes, which mature inAugust 2021, and the 2022 Convertible Notes, which mature in August 2022.

Interest Income

Interest income is related to interest earned from investments.

Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generallyaccepted accounting principles in the U.S. The preparation of our financialstatements and related disclosures requires us to make estimates and judgmentsthat affect the reported amounts of assets, liabilities, costs and expenses, andthe disclosure of contingent assets and liabilities in our financial statements.We base our estimates on historical experience, known trends and events andvarious other factors that we believe are reasonable under the circumstances,the results of which form the basis for making judgments about the carryingvalues of assets and liabilities that are not readily apparent from othersources. We evaluate estimates and assumptions on an ongoing basis. Actualresults may differ from these estimates under different assumptions orconditions.

Our significant accounting policies are described in more detail in our 2019Form 10-K, except as otherwise described below.

Results of Operations

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(MENAFN - iCrowdNewsWire) May 8, 2020

According to the new market research report " Cell Therapy Technologies Market by Product (Consumables, Equipment, Software), Cell Type (Human Stem & Differentiated, Animal), Process Stages (Cell Processing, Distribution, Handling, QC), End User, and Region - Global Forecast to 2023, , published by MarketsandMarkets, The global cell therapy technologies market is projected to reach USD 19.9 billion by 2023 from USD 10.2 billion in 2018, at a CAGR of 14.4% during the forecast period.

Browse in-depth TOC on 'Cell Therapy Technologies Market" 75 - Table30 Figures116 Pages

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Rising government investments for cell-based research, the increasing number of GMP-certified production facilities, and the large number of oncology-oriented cell-based therapy clinical trials are the key factors driving the growth of this market. China, India, Japan, Korea, and Brazil are emerging markets for cell therapy instruments. These markets boast comparatively lenient standards and government regulations as opposed to developed markets in North America and the EU, and thus offer significant growth potential for providers. However, the high cost of cell-based research and the low success rate is expected to restrain market growth to some extent during the forecast period.

Consumables are expected to account for the largest cell therapy technologies market share in 2018 : By product, the cell therapy technologies market is segmented into consumables, equipment, and systems & software. The consumables segment is expected to account for the largest share of the market in 2018. Factors such as increasing investments by companies to develop advanced products as well as government initiatives for enhancing cell-based research are contributing to the growth of the cell therapy consumables market.

Cell processing segment to witness the highest growth during the forecast period :

Based on process, the cell therapy technologies market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control. The cell processing segment is expected to account for the largest market share in 2018 and is projected to witness the highest CAGR during the forecasted period.

Human cells segment accounts for the large share of the cell therapy instruments market, by cell type :

Based on cell type, the market is segmented into human cells and animal cells. In 2018, the human cells segment is expected to account for the largest share of the cell therapy technologies market. The rising adoption of human cells over animal cells for cell therapeutics research, technological advancements, and the rising incidence of diseases such as cancer and cardiac abnormalities are the key factors driving the growth of this segment.

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North America to dominate the cell therapy technologies market during the forecast period : The market is segmented into four major regions, namely, North America, Europe, Asia Pacific, and the Rest of the World (RoW). North America is expected to dominate the market in 2018 owing to the high burden of chronic diseases and increasing R & D activities in the pharmaceutical and biotechnology industries. The Asia Pacific region is expected to register the highest CAGR during the forecast period.

The major players in the western blotting market are Beckman Coulter (US), Becton, Dickinson and Company (US), GE Healthcare (US), Lonza (Switzerland), Merck KGaA (Germany), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Terumo BCT (US), and Thermo Fisher Scientific (US).

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LYON, France & HALLE, Belgium--(BUSINESS WIRE)--Mrieux Equity Partners and Korys announce the launch of a new investment platform in Venture Capital to support innovative companies in the healthcare and nutrition sectors, in Europe and North America.

OMX Europe Venture Fund FPCI (OMX Europe) was launched with a significant financial commitment representing more than two thirds of the target size of the fund (EUR 90 million) with the support of Korys and Mrieux Dveloppement as sponsors and the contribution of new third party subscribers. The fund will benefit from the solid expertise and network of its sponsors and will be operated by a dedicated team covering direct investments in Venture Capital.

OMX [-miks] refers to a field of study in biology ending in -omics, such as genomics, proteomics, metabolomics or microbiomics. The promise of precision medicine and a biology-led industrial revolution hinge on the ability to reduce the complex interconnections of large, multi-omic data sets into useful products, services and information to enable a more personalized healthcare and nutrition.

The crisis caused by COVID-19 has raised significant public and government awareness around the importance of biology, for a better understanding of infectious diseases but also for providing adequate solutions in the field of prevention, diagnostic and therapeutic intervention. The OMX Europe investment fund focuses on entrepreneurs and life science companies driving breakthroughs in this field at international level, ultimately contributing to a better and cost-effective healthcare while also addressing global challenges.

A number of investments have already been completed by the fund:

OMX Europe will be managed by Mrieux Equity Partners in Europe, with the operational support of Korys Life Science team as a key advisor to the fund. Mrieux Equity Partners currently employs four FTEs dedicated to venture investment and plans to expand its team over the coming months. To add additional geographic and sector expertise a strategic partnership was recently established with a team of senior business executives who have co-invested on several venture deals in the United States with Mrieux Equity Partners over the last ten years. The US-based OMX Ventures investment team, composed of Craig Asher, Nick Haft and Dan Fero, with the support of Paul Conley, operating as Senior Advisor to the fund, will bring an outstanding track record and deep experience in the life science sector.

The American OMX Ventures team have established OMX Ventures Fund I (OMX US) in the US to support innovative companies, with a similar investment strategy to that of the OMX Europe fund and a privileged right of co-investment with OMX Europe. Targeting at least USD 100 million, OMX US has recently completed an initial closing and secured over two thirds of the funding for OMX Ventures Fund I.

We are honored to welcome Korys as a sponsor and key advisor to the fund. Together with Mrieux Dveloppements sponsorship, privileged access to the experience and industrial network of Korys will bring real additional value to our portfolio of fast growing companies, said Valrie Calenda, Partner at Mrieux Equity Partners.

Our collaboration with Mrieux Equity Partners is based on an entrepreneurial history spanning several generations, common values and the shared ambition to dedicate significant, long-term resources within the healthcare and nutrition sectors. We look forward to a successful partnership, said Christoph Waer from Korys.

Thanks to significant support from Mrieux Dveloppement, Korys and the contribution of our business partners in North America, we are increasing our investment capacity in the life science sector, at a time when understanding and mastering biology is more important than ever, added Franois Valencony, President of Mrieux Equity Partners.

About Mrieux Equity Partners - http://www.merieux-partners.com

Mrieux Equity Partners is a management company registered with the Autorit des Marchs Financiers (AMF) since June 2018 that is dedicated to growth equity and venture capital investments. Mrieux Equity Partners currently operates with an international team of 20 employees and regional partners based in Europe and North America. With over EUR 650 million under management, Mrieux Equity Partners actively supports entrepreneurs and industrial companies whose products and services bring differentiated and innovative solutions in the healthcare and nutrition sectors by providing privileged access to its expertise and the industrial, scientific and commercial network of Institut Mrieux, in compliance with the current regulations.

About Korys - http://www.korys.be

Korys is the investment company of the Colruyt family. Today, it has more than EUR 4.5 billion of assets under management. Besides holding a significant participation in the Colruyt Group, a leading retail company in Belgium and France, it actively manages participations in privately held companies and in private equity funds. Korys has also set up proprietary funds to manage its portfolio of listed investments. Across its activities, Korys investment decisions are taken with a long-term perspective and on basis of strict economic (Profit), social (People) and ecological (Planet) criteria. Korys aims to create sustainable value in three major ecosystems: Life Sciences, Energy Transition and Conscious Consumer. To do this, Korys can count on a motivated team of 30 professionals based in Belgium and Luxembourg.

This press release is not a marketing communication in the European Union member states or non-member states. This press release is not an offer of securities for sale in the United States. Securities may not be offered or sold in the United States absent registration or an exemption from registration. Any public offering of securities made in the United States will be made by means of a prospectus that may be obtained from the issuer and will contain detailed information about the company and management, as well as financial statements.

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Mrieux Equity Partners and Korys Announce the Launch of OMX Europe Venture Fund, Dedicated to Venture Investment Within the Healthcare and Nutrition...

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The U.S. Food and Drug Administration (FDA) announced on Tuesday that it has approved dapagliflozin, also known under the brand name Farxiga, for the treatment of heart failure in adults with reduced ejection fraction. The drug can potentially reduce the risk of cardiovascular death and hospitalization for heart failure.

AstraZenecas Farxiga is now the first in its drug class of sodium-glucose co-transporter 2 (SGLT2) inhibitors to be approved to treat adults with the New York Heart Associations functional class II-IV heart failure with reduced ejection fraction. AstraZeneca was granted with the approval of Farxiga related to heart failure by the FDA.

In a clinical trial, Farxiga appeared to improve survival and reduce the need for hospitalization in adults with heart failure and reduced ejection fraction.

To determine the efficacy of the drug, researchers looked at the number of instances of cardiovascular death, hospitalization for heart failure and urgent heart failure visits. Some trial participants were given a once-daily dose of 10mg of Farxiga, while others were given a placebo. After approximately 18 months, those who were given Farxiga had fewer cardiovascular deaths, hospitalizations for heart failure and urgent heart failure visits compared to their counterparts.

Heart failure is a serious health condition that contributes to one in eight deaths in the U.S. and impacts nearly 6.5 million Americans, said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDAs Center for Drug Evaluation and Research. This approval provides patients with heart failure with reduced ejection fraction an additional treatment option that can improve survival and reduce the need for hospitalization.

Farxiga can cause side effects including dehydration, urinary tract infections and genetical yeast infections. It can also potentially result in serious cases of necrotizing fasciitis of the perineum in people with diabetes and low blood sugar when combined with insulin.

On Tuesday, BioCardia, Inc. also announced positive preclinical data supporting its new drug application for anti-inflammatory cell therapy for heart failure. BioCardias allogenic neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) therapy appeared to improve heart function in a study. NK1R+ MSC is being marketed under the name CardiALLO.

Researchers looked at 26 animals treated with both low dose and high dose CardiALLO in their study. Echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow all notably improved in the animals.

In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter, said BioCardia Chief Scientific Officer Ian McNiece, PhD. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

BioCardia also intends to submit an IND for the use of NK1R+ MSC delivered via intravenous infusion for the treatment of Acute Respiratory Distress Syndrome caused by COVID-19.

Approximately 6.5 million adults in the U.S. are living with heart failure, according to the Centers for Disease Control and Protection. In 2017, it was a contributing cause of death in one out of eight people.

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FDA Approves AstraZeneca's Farxiga for Heart Failure in Adults with Reduced Ejection Fraction - BioSpace

Cardiac Stem Cells Comments Off on FDA Approves AstraZeneca’s Farxiga for Heart Failure in Adults with Reduced Ejection Fraction – BioSpace | May 7th, 2020

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that new data from its oncology program will be presented at the 2020 American Society of Clinical Oncology (ASCO20) Virtual Scientific Program from May 29-31. More than 80 abstracts in nearly 20 types of solid tumors and blood cancers have been accepted across Mercks broad cancer portfolio and investigational pipeline, including KEYTRUDA, Mercks anti-PD-1 therapy; LENVIMA (in collaboration with Eisai); LYNPARZA (in collaboration with AstraZeneca); and MK-6482 (formerly PT2977), an investigational, oral hypoxia-inducible factor-2 alpha (HIF-2) inhibitor.

Despite the challenges we all face due to the COVID-19 pandemic, Merck remains fully committed to supporting the cancer community and to advancing important scientific research from our clinical program, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. The data to be presented at this years ASCO demonstrate how our deep and diverse oncology portfolio continues to show meaningful outcomes for patients in new tumor types and stages of disease, while long-term survival data for KEYTRUDA in non-small cell lung cancer, renal cell carcinoma and melanoma further support its important role in these types of cancer.

Key abstracts including late-breakers, oral sessions, and select poster discussions and posters to be presented at ASCO include:

Merck Investor Event

Merck will hold a virtual investor event in conjunction with the ASCO20 Virtual Scientific Program on Tuesday, June 2 at 2 p.m. ET. Details will be provided at a date closer to the event at http://investors.merck.com/home/default.aspx.

Details on Abstracts Listed Above, Additional Presentations and Key Abstracts with Mercks Collaboration Partners

KEYTRUDA

Breast Cancer

Bladder Cancer

Classical Hodgkin Lymphoma

Colorectal Cancer

Lung Cancer

Renal Cell Carcinoma

Prostate Cancer

Melanoma

Ovarian Cancer

Head and Neck Cancer

KEYTRUDA plus LENVIMA (in collaboration with Eisai)

Hepatocellular Carcinoma

Renal Cell Carcinoma

Endometrial Cancer

LYNPARZA (in collaboration with AstraZeneca)

Ovarian Cancer

MK-6482

Renal Cell Carcinoma

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

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Merck to Present New Data from its Broad Oncology Portfolio and Pipeline at the ASCO20 Virtual Scientific Program - Business Wire

Cardiac Stem Cells Comments Off on Merck to Present New Data from its Broad Oncology Portfolio and Pipeline at the ASCO20 Virtual Scientific Program – Business Wire | May 7th, 2020

Australian researchers are at the forefront of world efforts to find vaccines, treatments and new tests for coronavirus with more than 50 projects already under way.

The Australian Association of Medical Research Institutes has compiled a list of major COVID-19 research projects in every state.

Apart from the University of Queensland's project to develop a vaccine, there are trials under way that repurpose existing drugs to treat COVID-19 patients, teams of scientists are developing new antiviral drugs, others are working on monoclonal antibody treatments that harness the body's immune system to fight the virus.

Burnet Institute in Victoria is screening ACE 2 inhibitor drugs for blood pressure to see if they can prevent or treat COVID-19 infection.

They plan to turn the best performing drugs into formulations that can be inhaled to deliver the drug directly to where the virus is in the lungs.

It is also developing monoclonal antibodies essential for profiling the immune response in humans infected with COVID-19 to help develop point of care tests.

Hudson Institute in Victoria, in collaboration with Biotech company Noxopharm, has found a cancer drug Veyonda could block pathways that causes a deadly inflammatory reaction in COVID-19 patients called a cytokine storm.

Noxopharm is seeking approval from the US FDA for a clinical trial in COVID-19 patients of Veyonda.

The Walter and Eliza Hall Institute in Melbourne is developing 'biologics' medicines for coronavirus infections.

The race for a COVID-19 cure is on and Australia is at the forefront. Picture: Shutterstock

These mimic antibodies to fight infection and are already in clinical use for diseases such as cancer and auto-immune conditions.

The Institute is also leading COVID-19 SHIELD, Australia's first clinical trial to assess whether the drug hydroxychloroquine is effective in preventing COVID-19 in frontline healthcare workers.

Doherty Institute Melbourne was the first group outside China to grow the COVID-19 virus, it is conducting the testing for COVID-19 in patients and validating commercial tests for the virus.

The institute's experts have been conducting pandemic modelling for the government on the spread of COVID-19.

It is helping Melbourne University run the ASCOT trial that will initially test two treatments for hospitalised COVID-19 patients, using drugs that are currently used to treat HIV (lopinavir/ritonavir) and malaria (hydroxychloroquine).

It is also developing a vaccine for COVID-19.

CSIRO is running animal trials of two potential COVID-19 vaccines, it has helped test the University Queensland vaccine on mice.

It is conducting research into genetic changes in the virus and doing computer modelling to understand how the virus behaves.

It is tracing where the virus came from and how it jumped from animals to humans and is working on how it spread so quickly.

The Monash Biomedicine Discovery Institute (BDI) and the Doherty Institute in Victoria have shown that head lice treatment ivermectin kills the COVID-19 in a test tube.

It is now checking whether it works at doses that are safe to use in humans and will then trial it on animals.

Garvan Institute in Sydney in collaboration with UNSW Sydney's Kirby Institute, is developing antibodies designed to target proteins the virus needs to infect human cells.

The potential antiviral therapy could help the elderly and chronically ill and be administered as a preventative therapy to health workers on the frontline.

Victor Chang Cardiac Research Institute Sydney in collaboration with St Vincent's Hospital Sydney and two hospitals in Victoria are planning a clinical trial of a stem cell to dampen down the hyperactivity of the immune system that causes severe heart and lung problems in patients with COVID-19.

The Kirby Institute at UNSW Sydney is researching hyperimmune globulin-based therapy based on the blood plasma of people who have recovered from the virus, it is engineering monoclonal antibodies for COVID-19 protection and therapy and is working on a treatment that could be delivered direct to the lungs via an inhaler or puffer.

There are more than 50 vaccines underway in Australia alone. Picture: Marieke De Lorijn/Supplied

QMIR Berghofer is conducting a randomised controlled trial of anti-inflammatory drug tocilizumab on critically ill patients with COVID-19 in the hope it can prevent the cytokine storm that is killing some COVID-19 patients.

It is also testing existing, widely used, and safe drugs to reduce COVID-19's ability to infect cells and help the immune system fight the disease and it is adapting its patented liquid biopsy assay system that has been successfully used in cancer patients to predict disease progression in patients with COVID-19.

It is developing antiviral gene-based drugs, looking factors that make some people more susceptible to severe COVID-19 symptoms.

It is using its 'human-heart-muscle-in-a-dish' to examine how COVID-19 causes cardiotoxicity and screen for drugs to limit heart injury in COVID-19 patients.

It is collecting information on COVID-19 from 80,000 Australians for whom researchers already have detailed genetic data will enable them to rapidly and cheaply identify genetic risk factors that might fast-track targets for drug development.

It is researching the way people with blood cancers respond to COVID-19.

Blood cancer treatments target immune cells that make antibodies to fight viruses.

It is developing a test to detect who has immunity to the virus.

The Institute for Glycomics (QLD), building on many years of vaccine development in streptococcus and malaria, is trying to identify critical target points on the coronavirus that may be susceptible to immune attack and to use that information to develop a highly focused vaccine.

Griffith Institute for Drug Discovery's rapid response technologies that allow fast design and manufacture of vaccines to combat pandemic threats has found five COVID-19 vaccine candidates that have been designed and manufactured and are currently being evaluated in animal trials.

The South Australian Health and Medical Research Institute is studying the protein-making pathway that are activated by coronavirus in an attempt to slow the growth of the virus.

They already know how to inhibit this pathway using a drug that is already in phase 2 clinical trials and cleared for use in humans.

Originally published as 50 Aussie research projects to beat coronavirus

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50 Aussie research projects to beat coronavirus - Tweed Daily News

Cardiac Stem Cells Comments Off on 50 Aussie research projects to beat coronavirus – Tweed Daily News | May 7th, 2020

According to the World Health Organization (WHO), cardiovascular disease, specifically ischemic heart disease, is one of the leading causes of death worldwide. Cardiovascular diseases result in an estimated 17.9 million deaths each year. This is about 31% of all deaths worldwide (1). Medical researchers are continually working on ways to reduce those numbers, including the development of new technologies to combat premature deaths from cardiovascular diseases. This article will focus, in particular, on the value of induced pluripotent stem cells (iPSCs) in cardiac research.

iPSCs are a type of pluripotent stem cell. These are master cells that can differentiate into any cell or tissue the body needs. They are generated directly from somatic cells through ectopic expression of various transcription factors, such as

Theyve become key tools to model biological processes, particularly in cell types that are difficult to access from living donors. Many research laboratories are working to enhance reprogramming efficiency by testing different cocktails of transcription factors.

iPSCs have become essential in a number of different research fields, including cardiac research.

They are a valuable and advantageous technologic development for two main reasons:

Most people have heard of embryonic stem cells, which are one variation of pluripotent cells. Like iPSCs, they can be used to replace or restore tissues that have been damaged.

The problem is that embryonic stem cells are only found in preimplantation stage embryos (3). Whereas iPSCs are adult cells that have been genetically modified to work like embryonic stem cells. Thus, the term, inducedpluripotent stem cells.

The development of iPSCs was helpful because embryos are not needed. This reduces the controversy surrounding the creation and use of stem cells. Further, iPSCs from human donors are also more compatible with patients than animal iPSCs, making them even closer to their embryonic cousins.

The Japanese inventor of iPSCs, Professor Shinya Yamanaka earned a Nobel Prize in 2012 for the discovery that mature cells can be reprogrammed to become pluripotent. (4) The Prize was awarded to Dr. Yamanaka because of the significant medical and research implications this technology holds.

iPSCs hold a lot of promise for transplantation medicine. Further, they are highly useful in drug development and modeling of diseases.

iPSCs may become important in transplantation medicine because the tissues developed from them are a nearly identical match to the cell donors. This can potentially reduce the chances of rejection by the immune system (5).

In the future, and with enough research, it is highly possible that researchers may be able to perfect the iPSC technology so that it can efficiently reprogram cells and repair damaged tissues throughout the body.

iPSCs forgo the need for embryos and can be made to match specific patients. This makes them extremely useful in both research and medicine.

Every individual with damaged or diseased tissues could have their own pluripotent stem cells created to replace or repair them. Of course, more research is needed before that becomes a reality. To date, the use of iPSCs in therapeutic transplants has been very limited.

One of the most significant areas where iPSCs are currently being used is in cardiac research. With appropriate nutrients and inducers, iPSC can be programmed to differentiate into any cell type of the body, including cardiomyocyte. This heart-specific cell can then serve as a great model for therapeutic drug screening or assay development.

Another notable application of iPSCs in cardiac research is optical mapping technology. Optical mapping technology employs high-speed cameras and fluorescence microscopy to examines the etiology and therapy of cardiac arrhythmias in a patient-like environment. This is typically done by looking into electrical properties of multicellular cardiac preparations., e.g. action potential or calcium transient, at high spatiotemporal resolution (6).

Optical mapping technology can correctly record or acquire data from iPSCs. iPSCs are also useful in mimicking a patients cardiomyocytes with their specific behaviors, resulting in more reliable and quality data of cardiac diseases.

iPSCs are vital tools in cardiac research for the following reasons:

iPSCs are patient-specific because they are 100% genetically identical with their donors. This genomic make-up allows researchers to study patients pathology further and develop therapeutic agents for treating their cardiac diseases.

Induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), help researchers predict the cardiotoxicity of drugs like with widely used chemotherapy reagents (10). Predictions like this were close to impossible before iPSC technology entered the research game.

iPSCs really come into play with their ability to model diseases. Because iPSCs are genetic matches to their living donors, they are uniquely useful for the study of genetic cardiac diseases like monogenic disorders. iPSCs help researchers understand how disease genotypes at the genetic level manifest as phenotypes at the cellular level (5).

Long QT syndrome, a condition that affects the repolarization of a patients heart after a heartbeat, is a notable example of iPSC-based disease modeling (7). This syndrome has been successfully modeled using iPSCs and is an excellent model for other promising target diseases (7).

Long QT syndrome is not the only disease that has been modeled by iPSCs. Other cardiac diseases like Barth syndrome-associated cardiomyopathy and drug-induced kidney glomerular injuries have been modeled as well (8).

The advent of iPSC technology has created a wealth of new opportunities and applications in cardiovascular research and treatments. In the near future, researchers hope that iPSC-derived therapies will be an option for thousands, if not millions of patients worldwide.

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What is the Value of iPSC Technology in Cardiac... - The Doctor Weighs In

Cardiac Stem Cells Comments Off on What is the Value of iPSC Technology in Cardiac… – The Doctor Weighs In | May 6th, 2020

SAN CARLOS, Calif., May 05, 2020 (GLOBE NEWSWIRE) -- BioCardia, Inc.[Nasdaq: BCDA], a leader in the development of comprehensive solutions for cardiovascular regenerative therapies, today announced data from a recent animal study performed by the Company that demonstrate meaningful improvements in heart function for subjects treated with its allogenic (from another donor, or off the shelf) neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) program for heart failure, known as CardiALLO. In addition, the Company is planning further exploration and discussion with the U.S. Food and Drug Administration (FDA) on the use of its allogenic cells for COVID-19 related Acute Respiratory Distress Syndrome (ARDS).

In the 26 animals treated with both low dose and high dose NK1R+ MSC, echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow were meaningfully improved, with all three measures being statistically significant for both dosage levels over control animals.

The CardiALLO cell therapy is being developed initially to treat heart failure patients whose cells do not qualify for its lead autologous cell therapy, CardiAMP (BCDA-01).

BioCardia Chief Scientific Officer Ian McNiece, PhD, said, In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

COVID-19 Induced Acute Respiratory Distress Syndrome (ARDS) Exploration

The Company also intends to submit an IND for the use of its NK1R+ MSC delivered via intravenous (IV) infusion for Acute Respiratory Distress Syndrome (ARDS) caused by COVID-19.

Based on preliminary clinical reports on COVID-19, respiratory failure complicated by ARDs is the leading cause of death for COVID-19 patients.1 ARDS is a type of respiratory failure characterized by the rapid onset of widespread inflammation in the lungs.

The anti-inflammatory effects of MSC have been well-documented and MSC have been shown to reduce inflammation and injury in models of lung disease.2 The specific MSCs used in BioCardias allogenic cell therapy are expanded from cells selected for the presence of the NK1 receptor, which is known to bind to substance P, an important neuropeptide associated with inflammation throughout the body and a primary mediator of inflammation in the airways.3,4

Our NK1R+ allogenic MSC may have more potential than other MSC approaches being advanced today due to their interaction with Substance P, said BioCardia CEO Peter Altman, PhD. This COVID-19 related work will be the Companys first clinical investigation outside of the cardiac space and our first exploring therapy for the lung. A recent patent publication (US 2020/0101113 A1) shows that BioCardia has long intended for these remarkable reparative cells to be targeted for respiratory disorders, in addition to cardiovascular disease. Addressing inflammation in the lungs is an important contribution we may be able to make using our NK1R+ allogenic MSC therapy.

The Companys allogenic cells are expected to be manufactured at BioCardias clinical stage cell manufacturing facility in San Carlos, California.

About BioCardiaBioCardia, Inc., headquartered in San Carlos, California, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the Companys biotherapeutic product candidates in clinical development. The Company's approved products include the Helix transendocardial delivery system and its steerable guide and sheath catheter portfolio. BioCardia also partners with other biotherapeutic companies to provide its Helix System and clinical support to their programs studying therapies for the treatment of heart failure, chronic myocardial ischemia and acute myocardial infarction.

Forward Looking Statements This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, references to the development of NK1R+ cells for the treatment of heart failure and ARDS secondary to COVID-19, potential FDA IND acceptances, and potential FDA filings, statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations. Such risks and uncertainties include, among others, the inherent uncertainties associated with developing new products or technologies. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

We may use terms such as believes, estimates, anticipates, expects, plans, intends, may, could, might, will, should, approximately or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardias Form 10-K filed with the Securities and Exchange Commission on April 9, 2020, under the caption titled Risk Factors. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

Media Contact: Michelle McAdam, Chronic Communications, Inc.michelle@chronic-comm.com(310) 902-1274

Investor Contact: David McClung, Chief Financial Officerinvestors@BioCardia.com(650) 226-0120

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BioCardia Announces Positive Preclinical Results Supporting Investigational New Drug Application for Anti-Inflammatory Cell Therapy in Heart Failure -...

Cardiac Stem Cells Comments Off on BioCardia Announces Positive Preclinical Results Supporting Investigational New Drug Application for Anti-Inflammatory Cell Therapy in Heart Failure -… | May 5th, 2020