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Researchers Convert Astrocytes to Neurons In Vivo to Treat… : Neurology Today – LWW Journals

By daniellenierenberg

Article In Brief

A mouse study shows that select transcription factors to the striatum can effectively and safely convert astrocytes to neurons to treat Huntington's disease.

Delivering two transcription factors to the striatum in a mouse model of Huntington's disease can safely convert astrocytes into neurons with high efficiency, according to a new study in the February 27 issue of Nature Communications.

The neurons grow to and wire up with their targets in the globus pallidus and substantia nigra, and remaining astrocytes proliferate to replace those that have been converted. The treatment extends the lifespan and improves the motor behavior of the mice.

What is exciting about this study is that the authors have clearly made cells that do what they are supposed to do, namely replace dying neurons in existing circuits, said Roger Barker, PhD, professor of clinical neuroscience and honorary consultant in neurology at the University of Cambridge and at Addenbrooke's Hospital, who was not involved in the work. I think the challenge of scaling up this strategy to the human Huntington's disease brain is pretty substantial, but nonetheless, this is an important discovery.

The new study, led by Gong Chen, PhD, builds on discoveries beginning in the mid-2000s showing that a small number of exogenously applied transcription factors could transform skin fibroblasts into stem cells, which could then be further converted to become virtually any cell type. That discovery was quickly followed by advances in direct reprogramming, in which one cell type is directly converted into another, skipping the stem cell intermediate.

Most of that work has taken place in vitro, and most attempts to use the strategy therapeutically have depended on transplantation of stem cells or newly converted cells.

We tried stem cell transplants to the mouse brain 10 years ago, but we couldn't find a lot of functional neurons, said Dr. Chen, professor at Guangdong-Hong Kong-Macau Institute of CNS Regeneration of Jinan University in Guangzhou, China.

It was also clear that anything you do in vitro, you eventually have to transplant, and that didn't seem to be a very promising technology, so I said, Let's try this in vivo, and put transcriptions factors directly into the mouse brain.

Dr. Chen initially tried introducing the transcription factor neurogenin 2, but the efficiency of conversion of astrocytes to neurons was very low, so he turned to the transcription factor NeuroD1, which Dr. Chen's group had previously shown could convert astrocytes into excitatory glutamatergic neurons.

In the current study, in order to generate GABAergic neurons, the team combined NeuroD1 with another transcription factor, D1x2, based on previous work showing its importance for generating GABAergic neurons.

The team packed the genes for the transcription factors into a recombinant adeno-associated virus vector (rAAV 2/5) and used an astrocyte-specific promoter to drive the transgene expression so that it preferentially expresses in astrocytes. They first injected the vector into the normal mouse striatum.

Surprisingly, this strategy worked very well at high efficiency, Dr. Chen said. After seven days, all transfected cells expressed astrocyte markers, indicating a high level of specificity in the vector. Of those cells, 81 percent co-expressed the two transcription factors. By 30 days, 73 percent of the cells expressing the transcription factors now expressed neuronal, rather than astrocytic markers, and were primarily GABAergic in character.

Next, Dr. Chen asked whether the remaining astrocytes could repopulate to replace those lost to conversion. Using immunostaining for astrocytes and neurons, as well as other techniques, the team found that the neuron/astrocyte ratio was unchanged, and that some remaining astrocytes could be found at different stages of cell division, suggesting the process facilitated astrocyte proliferation.

Dr. Chen then turned to the R6/2 mouse, the most common mouse model of Huntington's disease. He treated mice at 2 months of age, just as they began to show motor symptoms

As in the wild-type mice, astrocytes were converted to GABAergic neurons at high efficiency without altering the neuron/astrocyte ratio. The researchers observed similar results in a less-severe HD mouse model as well. Treated mice had only about half the degree of striatal atrophy as untreated mice. The converted neurons still contained aggregated huntingtin protein, but less than in native neurons, and similar to the reduced amount found in astrocytes in the mouse brain.

The real test of any cell therapy in neurodegenerative disease is whether the new cells can link into the existing circuits and provide functional benefit, feats that have been hard to achieve with transplanted fetal cells or stem cells.

Examining striatal slices from the treated mice, Dr. Chen found that the converted neurons displayed electrical properties largely identical to those of normal neurons, including resting potential, action potential threshold, firing amplitude, and firing frequency. They integrated into local circuits and behaved similarly to the native neurons around them. By tracking a marker contained in the AAV gene construct, they showed that converted neurons projected axons to the two basal ganglia targets of medium spiny neurons in the striatum, the globus pallidus and the substantia nigra.

Finally, Dr. Chen found that stride length and travel distance were both significantly improved in treated mice, though still falling below those of wild-type mice, and lifespan was significantly extended.

There were no hints of tumors in the mice, Dr. Chen noted. He suggested that in situ conversion is likely intrinsically safer in this regard than using stem cell-derived neurons, since a proliferative astrocyte is being converted into a non-proliferative neuron, with no residual pool of unconverted and potentially tumorigenic stem cells. We are actually reducing the tumor risk, he said.

Why the converted neurons developed appropriate neuronal connections is an important unanswered question, Dr. Chen said. He suggested there were two important factorsfirst, the astrocytes from which they arose are likely developmentally related to neighboring neurons, and thus may express similar position markers that help guide them to the right targets, just like the native neurons. Second, those remaining neurons may also provide guide tracks for the newly growing axons.

This conversion technique is not limited to Huntington's disease, he stressed, noting that his team last year published a paper showing promise in ischemic stroke, and work is underway to test its potential in Alzheimer's disease, Parkinson's disease, spinal cord injury, and ALS. He is also moving on to testing in non-human primates, setting the stage for eventual human trials.

I think eventually we will want to correct the Huntington's mutation as well, Dr. Chen said, for instance by using CRISPR, but he pointed out that while that strategy can repair diseased neurons, it cannot make new ones, like astrocyte-to-neuron conversion can.

This study is really elegantly done, commented Veronica Garcia, PhD, who has studied astrocytes derived from induced pluripotent stem cells from Huntington's disease patients as a postdoctoral scientist working with Clive Svendsen, PhD, in the Regenerative Medicine Institute at Cedars-Sinai Medical Center in Los Angeles.

The conversion efficiency is similar between wild-type and disease models, suggesting that the disease process is not interfering with the conversion, she said.

Astrocyte depletion does not seem to be a problem, at least in the short term, but Dr. Garcia noted there is a limit on the number of divisions astrocytes appear able to undergo, after which they lose the ability to proliferate. That may be a problem for chronic treatment, she suggested. Nonetheless, these results really look promising for therapeutic development.

The concept of trying to reprogram cells in situ to take on the phenotype of the cells that are lost is not new, commented Dr. Barker, but being able to do it with any degree of efficiency, to make enough cells to make a significant difference, has been problematic. For that reason, and because the cells grow to their target sites and make connections, these results are surprising.

A major hurdle for clinical trials, he noted, will be scaling up to the human striatum, which has approximately 100 times the volume of that in the mouse. Delivering the vector to such a large volume will be a significant challenge, he said, along with determining whether this approach will really work in a disease that affects many different brain structures such as in HD.

Dr. Chen is co-founder of NeuExcell Therapeutics Inc, which will develop clinical trials in the future. Drs. Barker and Garcia disclosed no conflicts.

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Temple University: Spinal Cord Injury and Optic Nerve Damage Repaired With Growth-Regulating Molecule – Gilmore Health News

By daniellenierenberg

Researchers at Temple University have made a discovery that raises hope of restored functions for people with spinal cord injuries and other related issues.

In a study published in Molecular Therapy, researchers said they were able to regenerate neurons in mice that had spinal cord injury and optic nerve damage. They succeeded in restoring lost functions in the animals with the aid of a molecule called Lin28.

Several thousands of people suffer permanent losses of motor function and sensation due to spinal cord injury and similar conditions every year. These are results of severe damage or separation of axons.

Humans can regenerate most of the tissues in their bodies when damaged. Axons, however, fall among key body components that they are unable to redevelop when damaged.

Read Also: University of Freiburg Identifies the Neurons Responsible for Rapid Eye Movements During Sleep

The new research by the Temple University scientists interestingly shows that Lin28 helps to mend axons. With the aid of the molecule, mice showed gains in sensation and recovery of motor function.

Our findings show that Lin28 is a major regulator of axon regeneration and a promising therapeutic target for central nervous injuries, said lead researcher Dr. Shuxin Li, MD, PhD.

The regenerative capacity of Lin28 was observed when it was expressed at levels higher than normal.

It was the first time scientists showed the potential of the molecule to help repair spinal cord injuries.

Axons are nerve fibers that project from neurons. They form networks that help to pass signals from the brain to different parts of the body. These long nerve fibers literally serve as communication cables.

The brain, for instance, depends on axons to be able to communicate with muscles. It is this interaction that enables movement in response to stimuli.

When axons are severed as a result of an injury or accident, sensation and motor function losses result. These changes last for the rest of a persons life since the structures do not regenerate.

In the current research, scientists decided to explore the ability of Lin28 to regrow neurons. They developed an interest in this because of how it determines whether or not stem cells differentiate.

The researchers developed a model involving over-expression of the focal molecule in certain tissues in mice. They put the animals in different groups containing those having a spinal cord injury or optic nerve damage after becoming adults.

Also, the team had a different group of mice with normal levels of Lin28 expression as well as spinal cord or optic nerve injuries. To examine tissue repair effects, the animals were injected with a viral vector that increases the expression of the molecule.

Expressions of Lin28 above normal levels promoted regeneration of axons in all the animals.

Lin28 injections led to axons extending up to 3 mm away from the point of severance or damage in animals with spinal cord injury. Axons grew again all along the whole length of the optic nerve tract.

The use of molecule injections proved to be the most effective means of restoring damaged axons.

When the researchers assessed the walking and sensory capabilities of the animals, they found that Lin28 therapy led to great improvements.

At the moment, there is no restorative treatment for people with injuries involving the spinal cord or optic nerve tracts, which link to the retina.

Li said the level of axon regeneration seen in the study could be highly significant in clinical terms. The professor of anatomy and cell biology revealed that one of his immediate plans was finding a means of making Lin28 helpful to human patients.

Read Also: UC Berkeley Researchers Restore Vision in Mice Through Gene Insertion

He and colleagues need to create a carrier (or vector) for the molecule that would improve its expression when injected. They need to find a means of precisely targeting damaged axons with the treatment.

The researchers also planned to learn more about the Lin28 signaling pathway. Li expressed a suspicion that the molecule possibly uses more than one pathway to promote repair.

To support growth, the molecule seems to work with several others that could potentially be combined with it for more effective therapy.

References

https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(20)30191-X

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Spinal Cord Trauma Treatment Market to Register CAGR 3.7% Growth in Revenue During the Forecast Period 2025 – Jewish Life News

By daniellenierenberg

Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

What the report encloses for the readers:

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Company Profiles

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The global market for spinal cord trauma treatment is is estimated to be valued at US$ 2,276.3 Mn in terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at a CAGR of 3.7% over the forecast period to reach a value of US$ 3,036.2 Mn by 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for 53.2% value share in 2017 and is projected to account for 52.5% share by 2025 end.

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Global Spinal Cord Trauma Treatment Market: Forecast by Injury Type

On the basis of injury type, the global spinal cord trauma treatment market is segmented into complete spinal cord injuries and partial spinal cord injuries.

Partial spinal cord trauma treatment segment is expected to show better growth than the completed spinal cord treatment segment due to higher growth in the incidence rate of partial spinal cord trauma than the complete spinal cord trauma. With US$ 1,870.3 Mn market value in 2025, this segment is likely to expand at CAGR 3.8% throughout the projected period.

Global Spinal Cord Trauma Treatment Market: Forecast by Treatment Type

On the basis of treatment type, the global spinal cord trauma treatment market is segmented into corticosteroid, surgery, and spinal traction segments.

Surgery segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period. Surgery segment is the most attractive segment, with attractiveness index of 2.6 over the forecast period.

Global Spinal Cord Trauma Treatment Market: Forecast by Region

This market is segmented into five regions such as North America, Latin America, Europe, APAC and MEA. Asia-Pacific account for the largest market share in the global spinal cord trauma treatment market.

Large patient population due to the high rate of road accidents and crime is making the Asia Pacific region most attractive market for spinal cord trauma treatment. On the other hand, MEA and Latin America is expected to be the least attractive market for spinal cord trauma treatment, with attractiveness index of 0.3 and 0.5 respectively over the forecast period.

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Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy | DNA RNA and Cells | News Channels -…

By daniellenierenberg

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 06 May 2020 18:08Hits: 268

CARLSBAD, CA, USA I May 06, 2020 ILineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, today announced that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), were published online via the ARVOLearn platform as part of the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting. The presentation entitled, Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results (Abstract # 3363764), was presented by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. Dr. Riemanns presentation is available on the Media page of the Lineage website. Lineage will also host a live call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results of treatment with OpRegen. Interested parties can access the call on the Events and Presentations section of Lineages website.

This update is significant as it builds on our earlier reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry AMD, with meaningful improvements in the progression of geographic atrophy, visual acuity, and reading speed observed in our first Cohort 4 patient and first Orbit SDS with thaw-and-inject formulation dosed patient, stated Brian M. Culley, Lineage CEO. As dry AMD is a slow and progressive disease, it takes many months to observe changes to retinal anatomy or visual acuity. With the benefit of longer follow-up, we now can report that some OpRegen treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression. Notably, we also are reporting additional evidence that OpRegen cells remain present for at least 4 years and hope that longer follow-up periods will reinforce a growing body of evidence that OpRegen is well-tolerated and can provide sustained and clinically meaningful benefits with a single dose of RPE cells. Our near-term objective is to treat and monitor the final four patients in Cohort 4 of the current study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.

As a principal investigator on the OpRegen clinical study, I am excited to present this most recent update, where all Cohort 4 patients treated with OpRegen had improved Best Corrected Visual Acuity up to one year or at their last visit, demonstrating a substantial treatment response, stated Christopher D. Riemann, M.D. The pooled Cohort 4 data demonstrate a significant, greater than 10-letter sustained visual acuity improvement over the entire followup period. Reading center assessments of GA also suggest a reduction in GA progression in the OpRegen treated eye when compared to fellow eye in Cohort 4. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to dosing patients in this study at CEI.

KOL Call Information and Webcast

Lineage will host a conference call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results following treatment with OpRegen. A live webcast of the conference call will be available online in the Events and Presentations section of Lineages website. Interested parties may also access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 19, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 6597936.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC2, a cancer immunotherapy of antigen-presenting dendritic cells in Phase 1 development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

SOURCE: Lineage Cell Therapeutics

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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…

By daniellenierenberg

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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FDA Approves AstraZeneca’s Farxiga for Heart Failure in Adults with Reduced Ejection Fraction – BioSpace

By daniellenierenberg

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

By daniellenierenberg

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

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50 Aussie research projects to beat coronavirus – Tweed Daily News

By daniellenierenberg

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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Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -…

By daniellenierenberg

A recent market study on the global Hematopoietic Stem Cell Transplantation (HSCT) market reveals that the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to reach a value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% during the forecast period (2019-2029). The impact of the COVID-19 pandemic on the global Hematopoietic Stem Cell Transplantation (HSCT) market is discussed in the presented study.

The Hematopoietic Stem Cell Transplantation (HSCT) market study encloses a thorough analysis of the overall competitive landscape and the company profiles of leading market players involved in the global Hematopoietic Stem Cell Transplantation (HSCT) market. Further, the presented study offers accurate insights pertaining to the different segments of the global Hematopoietic Stem Cell Transplantation (HSCT) market such as the market share, value, revenue, and more.

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The following doubts are addressed in the market report:

Key Highlights of the Hematopoietic Stem Cell Transplantation (HSCT) Market Report

The presented report segregates the Hematopoietic Stem Cell Transplantation (HSCT) market into different segments to ensure the readers gain a complete understanding of the different aspects of the Hematopoietic Stem Cell Transplantation (HSCT) market.

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Segmentation of the Hematopoietic Stem Cell Transplantation (HSCT) market

Competitive Outlook

This section of the report throws light on the recent mergers, collaborations, partnerships, and research and development activities within the Hematopoietic Stem Cell Transplantation (HSCT) market on a global scale. Further, a detailed assessment of the pricing, marketing, and product development strategies adopted by leading market players is included in the Hematopoietic Stem Cell Transplantation (HSCT) market report.

Companies Mentioned in the Report

The report profiles key manufacturers in the hematopoietic stem cell transplantation (HSCT) Market based on various attributes such as company details, SWOT analysis, strategic overview, financials, and business overview. Major players profiled in this report include Regen Biopharma, Inc., Escape Therapeutics, Inc., Lonza Group Ltd., and Pluristem Therapeutics Inc.

The global hematopoietic stem cell transplantation (HSCT) Market has been segmented as follows:

By Transplant Type

By Disease Indication

By Application

By Region

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Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -...

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough – MEAWW

By daniellenierenberg

As the world continues to battle the coronavirus pandemic, scientists are looking towards gene therapy to find ways to develop vaccines for the Covid-19 virus. Gene therapy itself was developed based on how viruses work.

When a virus attacks a host, it introduces its genetic material into the host cell as part of its replication cycle. The genetic material serves as an instruction manual on how to produce more copies of the virus, hijacking the host body's normal production machinery to serve the needs of the virus. The host cells then produce additional copies of the virus, leading to more host cells being infected.

Like animals, humans have found a way to domesticate viruses as well, i.e., direct the virus's function to achieve favorable results, which is prominent in gene therapy. Such viruses which physically insert their genes into the host's genome could instead be used to carry "good" genes into a human cell. Scientists would first remove the genes in the virus that cause diseases, and replace those genes with genes encoding the desired effect.

All of this sounds quite sci-fi but it has been done numerous times in the past. Peter Kolchinsky, a virologist and a biotechnology investor, compiled how different viruses have been used for gene therapy in the past.

Kolchinsky tweeted, "SARS2 is a scary menace, but did you know that we've domesticated viruses? Like wolves vs dogs, we've tamed them, including some deadly ones, to perform many useful functions (and may help us stop SARS2)."

The human immunodeficiency virus (HIV) has killed millions of people. It works by disabling the host body's immune system until it can't defend the person against common, normally mild pathogens. Kolchinsky explained that HIV's special trick is to integrate its genome into that of the host body's cells.

This feature of HIV is used for gene therapy, as explained before, by replacing a chunk of the virus's genome with the hemoglobin gene to insert it into bone marrow stem cells of patients with sickle cell anemia, whose hemoglobin genes are malfunctioning.

Kolchinsky also tweeted, "Adenoviruses typically cause mild infections, including common colds. These, too, we are trying to use for gene therapies, particularly when we just want to temporarily make a protein in cells. One company is developing such an adenovirus gene therapy for heart disease to induce growth of new blood vessels when old ones are clogged. Another is using this virus to make oral vaccines that would otherwise require injection (eg flu vaccine pill). When we use a virus to deliver code for making something in cells, we call that a virus vector."

There is now a wealth of clinical experience with numerous vector types that include primarily vaccinia, measles, vesicular stomatitis virus (VSV), polio, reovirus, adenovirus, lentivirus, -retrovirus, adeno-associated virus (AAV) and herpes simplex virus (HSV).

However, as with all other procedures, viral vector-gene therapy has associated risks. Viruses can usually infect more than one type of cell, so, when viralvectorsare used to carrygenesinto the body, they might infect healthy cells as well as cancer cells.

Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. Moreover, when viruses are used to deliver DNA to cells inside the patient's body, there is a slight chance that this DNA could unintentionally be introduced into the patients reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.

One study to help find a vaccine for Covid-19 aims to use the principles behind gene therapy to get the vaccine ready. The researchers' method uses a harmless virus as a vector to bring DNA into the patient's cells. The DNA should then instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.

While a mass-produced vaccine may still take a while, this study is one of at least 90 vaccine projects around the world trying to find a cure for Covid-19. However, some experts are worried that a vaccine may never be available. According to our previous report, Dr David Nabarro, a professor of global health at Imperial College London, who also serves as a special envoy to the WHO on Covid-19, said, "There are some viruses that we still do not have vaccines against."

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough - MEAWW

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What is the Value of iPSC Technology in Cardiac… – The Doctor Weighs In

By daniellenierenberg

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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Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t – The Sun

By daniellenierenberg

MIKE TYSON revealed how he has started training again having not thrown a punch for a staggering 15 years - and is being aided by stem-cell research therapy.

The Baddest Man on the Planet, who hung up his gloves in 2005 following defeat by Kevin McBride, wouldn't elaborate on why he was having the treatment but added that it was 'really wild what scientists can do'.

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Tyson was having an Instagram chat with basketball legend Shaquille O'Neal when he revealed that he had been training for the previous three days after 15 years away - and his new health regime.

Iron Mike said: "You know what I had done? I had stem-cell research therapy.

"I feel like a different person but I can't comprehend why I feel this way. It's really wild what scientists can do."

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.

He did not reveal what the exact condition was that was being treated.

Despite letting the gloves gather dust in the corner, it didn't take long for Iron Mike to show off that lethal punching power.

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The 53-year-old delighted fans in lockdown by uploading a viral video of him laying into a punchbag with his trademark speed and power.

Tyson's weight ballooned after retiring following battles with drug addiction and depression.

But he has since partnered with a new trainer, MMA coach Rafael Cordeiro, to kick-start his training after sensationally revealing his 15-year break.

During their chat, basketball legend O'Neal revealed how he ached for three days after playing with his sons.

Tyson responded: "That's just because you haven't done it for a while.

WHAT IS STEM CELL TREATMENT USED FOR?

Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.

It can also be used to replace damaged blood cells as the result of intensive cancer treatment.

Here are conditions that stem cell transplants can be used to treat:

"If you continue to do it consistently you'll be back to normal.

"It's just like me, I haven't boxed or hit the bag for 15 years - it has been three days so far and I feel incredible."

Tyson, who has a 50-6 record, is reportedly gearing up for a sensational return amid plans to compete in exhibition bouts for charity later this year.

He told rapper T.I. last month: "I've been hitting the mitts for the last week.

"That's been tough, my body is really jacked up and really sore from hitting the mitts.

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"I've been working out, I've been trying to get in the ring, I think I'm going to box some exhibitions and get in shape.

"I want to go to the gym and get in shape to be able to box three or four-round exhibitions for some charities and stuff.

"Some charity exhibitions, make some money, help some homeless and drug-affected motherf****er like me."

4

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Orchard Therapeutics: Updating The Investment Thesis – Seeking Alpha

By daniellenierenberg

Most doctors are prisoners of their education and shackled by their profession.

- Richard Diaz

A month ago we offered up the analysis below on this 'watch item' biotech stock from across the pond exclusively to Biotech Forum members. The stock is up over 40% since then but the concern is still intriguing. Today, we update our original thesis to account for all company-specific news and analyst ratings on this name since our original research was posted.

Orchard Therapeutics (ORTX) is a London, United Kingdom-based biopharmaceutical company that IPOd in 2018. The companys mission is to help patients with rare conditions by leveraging its understanding of gene therapy. The companys approach is to take a patients own blood stem cells and insert into them a working copy of the missing or malfunctioning gene. The companys approach circumvents the need for a bone marrow transplant by taking advantage of blood stem cells' intrinsic capacity to self-renew in a patients bone marrow and produce new blood cells of all types. The ultimate goal is to permanently correct genetic disorders through the use of a single treatment. The companys pipeline is broken down into three overarching programs: neurometabolic disorders, primary immune deficiencies, and blood disorders. The companys lead product candidates are OTL-200, OTL-101 and OTL-103. The companys only commercial product is Strimvelis, which has been approved by the EMA but not by the FDA. Orchard Therapeutics has a market capitalization of just over $1 billion and trades for around $11 a share.

Pipeline:

Source: Company Presentation

OTL-200:

OTL-200 is an ex vivo autologous gene therapy in development to treat Metachromatic leukodystrophy. The drug uses a modified virus to insert an operational copy of the ARSA gene into a patients cells. OTL-200 has received rare pediatric disease designation from the FDA. MLD is a rare and deadly inherited disease. The disease is characterized by the accumulation of fats called sulfatides, which causes a breakdown in the protective fatty layer surrounding nerves in the central and peripheral nerve systems. It is estimated that 1 in 40,000 to 1 in 160,000 people have the disease worldwide. OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Source: Company Presentation

On December 2nd, 2019, under accelerated assessment status, The EMA accepted the review for a marketing application of OTL-200 for MLD. Under accelerated assessment, the review period is 150 days, compared to the normal 210 days. The action date should occur very shortly.

Source: Company Presentation

Looking ahead, the company expects to obtain approval and launch the drug in Europe in the second half of 2020. The company is currently preparing for a commercial launch. Furthermore, the company expects a U.S. regulatory filing, a BLA, in late 2020 to early 2021.

Source: Company Presentation

OTL-101:

OTL-101 is an ex vivo autologous gene therapy in development to treat adenosine deaminase severe combined immunodeficiency. The drug uses a modified virus to insert an operational copy of the ADA gene into a patients cells. OTL-101 has received both Breakthrough Therapy designation and Rare Pediatric Disease designation from the FDA. ADA-SCID is a rare, inherited, pediatric disorder that is commonly fatal when undiagnosed or left untreated. The disorder is the result of a deficiency in adenosine deaminase. The disease culminates in a complete lack of/minimal immune system development. It is estimated that the diseases annual incidence is between 1 in 200,000 and 1 in 1 million live births.

Looking ahead, Orchard Therapeutics will initiate a rolling BLA filing in the U.S. for OTL-101 in ADA-SCID in the first half of 2020 with an anticipated completion of the filing within 12 months.

Source: Company Presentation

OTL-103:

0TL-103 is an ex vivo autologous gene therapy in development to treat Wiskott Aldrich syndrome. The drug uses a modified virus to insert a working copy of the WAS gene into a patients cells. WAS is a rare, X-linked, recessive, inherited immune disorder, which is characterized by reoccurring severe infections, autoimmunity, eczema and severe bleeding episodes. The company has received Rare Pediatric Disease designation and Regenerative Medicine Advanced Therapy designation from the FDA. OTL-103 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy.

Looking ahead, the company is preparing to file a BLA in the U.S. and an MAA in the EU for OTL-103 in WAS in 2021.

Source: Company Presentation

As of December 31st, 2019, the company had cash and cash equivalents of over $300 million including marketable securities. Research and development expenses for the fourth quarter were $30.8 million, compared to $17.4 million in Q4 of 2018. Selling, general and administrative expenses were $18.5 million in the quarter, compared to $11.9 million in the same quarter of 2018. Overall, the company had a net loss of $45.4 million in the quarter, compared to a net loss of $25 million in the same period of 2018. The company believes that its current balance sheet will allow it to fund all anticipated operations and capital expenditures into the second half of 2021.

Despite a sizable market cap, Orchard gets little in the way of attention from analysts. That is probably because it is headquartered overseas. The current median analyst price target on Wall Street is just over $25.00 a share.

Oppenheimer reissued its Buy rating and $28 price target on April 1st. In mid-March, Cowen & Co. did the same with an identical price target in Mid-March. This follows a similar call at Cowen in December with strongly bullish commentary:

Orchard Therapeutics has a differentiated and promising lentiviral, ex vivo pipeline with gene therapies for ADA-SCID, WAS and MLD likely to get approved in 2021-22. With a deep early stage pipeline focused on validated targets, we anticipate strong stock appreciation as drugs are approved and early stage programs yield positive clinical results and advance into pivotal studies in 2020/21."

On September 17th, 2019, Guggenheim initiated coverage with a buy rating and a $31 a share price target. The analyst at Guggenheim views the company's ex vivo, lentiviral gene therapy platform as differentiated and de-risked. Furthermore, three drug approvals within the next 24 months bode well for share appreciation. Finally, in early September of last year, Barclays (NYSE:BCS) initiated coverage with an overweight rating and a $21 price target. The analyst at Barclays sees a company that strategically targets genetic diseases that have validated approaches with hematopoietic stem cell transplant, a fairly de-risked pipeline and multiple catalysts over the next 24 months.

Orchard has multiple shots on goal and several definable milestones in the coming quarters. Given some of the recent turmoil in the credit markets, anything that will need to raise capital in the foreseeable future is taken out and shot when markets have deep down days.

Given that, I still have Orchard as a solid 'watch item' holding and added several hundred shares in my personal account in late March. The shares appear to have an attractive risk/reward profile on a longer term basis, but I would accumulate on dips given the shares run up in April. Once volatility ebbs and we have more confidence in the economy and markets, this is a name we will revisit and maybe upgrade to a larger recommended holding.

The securest place is a prison cell, but there is no liberty

- Ben Franklin

Bret Jensen is the Founder of and authors articles for the Biotech Forum, Busted IPO Forum, and Insiders Forum

Live Chat on The Biotech Forum continues to be very active with new trade and the lucrative covered call ideas available thanks to the spike in market volatility throughout the trading day. If you join the The Biotech Forum today by clicking HERE you will automatically get access to our model portfolio, Live Chat, investment archives and our next 'option play of the week.'

Disclosure: I am/we are long ORTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL – Oncology Nurse Advisor

By daniellenierenberg

The first-in-human, universal chimeric receptor antigen (CAR) T-cell (CAR-T) therapy GC027 was tolerable and resulted in antileukemic responses among patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.1

Theuniversal CAR T cells target CD7, which, according to Xinxin Wang, PhD, ofGracell Biotechnologies Co, Ltd, in China, and lead author and presenter of thestudy, is a good target for T-ALL because it is expressed by more than 95% ofT-ALL patients.

GC027 isallogeneic, which may prevent the development of graft-versus-host disease. Theproduct is introduced using lentivirus for rapid elimination of T-ALL cells. Preclinicalstudies showed efficacy in a T-ALL xenograft model, and this prospective studyevaluated the safety and efficacy in humans.

Thesingle-arm, open-label study treated 5 adult patients with relapsed/refractoryCD7-positive T-ALL with a single infusion of 1 of 3 different dose levels ofG027: 0.6 x 107/kg, 3 x 107/kg, and 1.5 x 107/kg.Lymphodepletion therapy was administered prior to the G027 infusion. Theprimary endpoint was safety and the secondary endpoints included objectiveresponse rate (ORR) within 3 months after G027 infusion.

Patientswith extramedullary or central nervous system disease were excluded. Atbaseline, the median age was 24 (range, 19-38). Patients were heavilypretreated, with 5 median number of prior therapies (range, 1-9). Two patientshad high-risk disease and the median bone marrow tumor burden was a median of38.2% of blasts. None of the patients had undergone a prior allogeneic hematopoieticstem cell transplant.

Allpatients developed cytokine release syndrome (CRS), 4 of which were grade 3 and1 was grade 4. All cases were manageable and resolved with treatment andsupportive care. None of the patients developed neurotoxicity.

The completeremission (CR)/CR with incomplete hematologic recovery was 100%. By day 28, 4patients achieved a CR with negative for minimal residual disease (MRD) and 3of these patients remained MRD negative up to day 161. One patient achieved CRbut was MRD positive, and relapsed by day 29.

Peak CART-cell expansion in peripheral blood occurred between week 1 and 2.

As the first-in-human, universal CAR T-cell therapy for adult relapsed/refractory T-ALL, Dr Wang said, GC027 has demonstrated superior clinical efficacy and induced deep response in patients with acceptable safety profile. She added that trial enrollment is ongoing.

Reference

Wang X, Li S, Gao L, et al. Clinical safety and efficacy study of TruUCAR GC027: The first-in-human, universal CAR-T therapy for adult relapsed/refractory T-cell acute lymphoblastic leukemia (r/r T-ALL). Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I; April 27-28, 2020. Abstract CT052.

This article originally appeared on Cancer Therapy Advisor

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What Is Leukemia? Here’s all you need to know – India Today

By daniellenierenberg

Veteran Bollywood actor Rishi Kapoor, who was battling leukemia for the last two years, died at Mumbais Sir HN Reliance Foundation Hospital on Thursday (April 30) morning. He was 67. Rishi Kapoor got treated for leukemia in the US for a year and had returned to India in September 2019.

Leukemia is a blood cancer caused by a rise in the number of white blood cells in your body. Those white blood cells crowd out the red blood cells and platelets that your body needs to be healthy. The extra white blood cells dont work right. A cancer of blood-forming tissues, hindering the body's ability to fight infection.

Dr Rahul Bhargava, Director, Haematology, Haemato Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram says:

Leukemia, often fatal, is a blood cancer. As with cancer, it is associated with an exponential rise in the number of white blood cells. Symptoms begin to manifest slowly and can often be mistaken for something else sweating uncontrollably, shaking, fever, vomiting. It is caused by huge was is reduced focus on ones own health.

Intake of processed food, soda, artificial and synthetic sweets and preservatives, pesticide use in farms, lack of physical exercise, excessive tobacco and drug consumption, excessive alcohol intake, caffeine consumption are the main culprits causing cancer. Family history also plays an important role in a leukemia diagnosis. Only through screening can it be identified and if caught early managed. One needs to be mindful and ensure that they are employing good lifestyle habits.

This means eating green leafy vegetables and fruits, going for a 30 minutes walk every day or engaging in any other form of exercise for 30 minutes a day, drinking adequate amount of water to keep flushing out the toxins, controlling or completely eliminating our use of tobacco and other unnatural substances. Processed food and sugars should be consumed in control, they are the single largest cause of mutation to cells.

Leukemia symptoms vary, depending on the type of leukemia. Common leukemia signs and symptoms include:

Leukemia can also cause symptoms in organs that have been infiltrated or affected by the cancer cells. For example, if the cancer spreads to the central nervous system, it can cause headaches, nausea and vomiting, confusion, loss of muscle control, and seizures.

Leukemia can also spread to other parts of your body, including:

The major types of leukemia are:

Blood has three types of cells: white blood cells that fight infection, red blood cells that carry oxygen, and platelets that help blood clot. Every day, your bone marrow makes billions of new blood cells, and most of them are red cells. When you have leukemia, your body makes more white cells than it needs. These leukemia cells cant fight infection the way normal white blood cells do. And because there are so many of them, they start to affect the way your organs work. Over time, you may not have enough red blood cells to supply oxygen, enough platelets to clot your blood, or enough normal white blood cells to fight infection.

Leukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer. The treatment depends on the type and stage of the cancer. Some forms of leukemia grow slowly and don't need immediate treatment. However, treatment for leukemia usually involves one or more of the following:

Source: Information has been taken from various health website

Read more| National Honesty Day 2020: Date, history, significance and quotes on the value of honesty

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

By daniellenierenberg

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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Stem Cell Treatment for COVID-19; Doctors Divided on Its Scope – The Quint

By daniellenierenberg

A team of doctors and researchers at the Abu Dhabi Stem Cell Center (ADSCC) administered the treatment in the UAE to 73 COVID-19 patients, who were all successfully treated and cured, without any immediate side effects, according to a statement by the United Arab Emirates (UAE) health ministry from 1 May.

The process involved a minimally invasive method where the patients stem cells are extracted, activated and turned into fine mist to be inhaled into the lungs. This was done in addition to the conventional treatment and is expected to work by supporting the established protocol of management of symptoms.

The ministry said in the statement, It is hypothesised to have its therapeutic effect by regenerating lung cells and modulating the immune response to keep it from overreacting to the COVID-19 infection and causing further damage to healthy cells.

The treatment has successfully undergone the initial phase of clinical trials, demonstrating its safety, and further trials for its efficiency are ongoing; expected to be completed in a couple of weeks.

Dr Fatima al-Kaabi, head of haematology and oncology at the Sheikh Khalifa Medical City in the UAE, told CNBC, Its very early to say at this stage. If all went well, this could reach the market in three months, she added.

Going further back, a pilot study in China on seven COVID-19 patients found that intravenous infusions of donor mesenchymal stem cells (MSC) - multipotent stem cells - improved patient outcomes and helped all of them recover. An Israeli pharmaceutical company, Pluristem Therapeutics, also tested stem cells in seven critical hospitalized patients and found positive results.

Additionally, the US Food and Drug Administration (FDA) approved MSC use in extremely sick COVID-19 patients under expanded access compassionate use on 5 April according to a report in The Scientist, even though the experts seemed divided on the logic on which the investigative treatment may have worked.

A hospital in New York tried the therapy as an experiment on 12 patients, 10 of whom were able to come off ventilators, reports CBS news. The Australian regenerative medicine company Mesoblast has also announced a 300-person trial for its stem cell therapy remestemcel-L (which was used in the New York trial) to determine whether it will work on patients suffering with severe lung inflammation.

Currently, there are over 20 active stem cell trials for COVID-19, most focusing on the use of MSCs.

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Terminally-ill teen with agonizingly-sensitive skin defies doctors to live to 19 – Metro.co.uk

By daniellenierenberg

Marky Jaquez, 19, has recessive dystrophic epidermolysis bullosa, a condition found in fewer than one in one million newborns. (Picture: Melissa Jaquez/Metro.co.uk)

A teenager whose rare skin disorder means that even the slightest touch leaves him in extreme pain has defied doctors predictions to live to adulthood. Marky Jaquez, from Wichita, Kansas, cannot hug his parents, wear clothes or even walk because any friction causes layers of skin to peel away from his body.

Wheelchair-bound, Marky, 19, has never been able to walk because the ground would shred his feet like a cheese grater and must be covered head-to-toe in bandages to protect his fragile skin from tearing. He has recessive dystrophic epidermolysis bullosa, a condition found in an estimated one in one million newborns that often leads to life-threatening skin infections and even cancer.

The illness is caused by a faulty gene, passed onto children by parents. Tragically, Markys brother Carlos was born with it too, and died aged just 14. Doctors warned Markys parents he was unlikely to live beyond the age of 11, but he defied that grim prediction and will celebrate his 20th birthday later this year.

His mom, Melissa Jaquez, 40, said: Marky only has one layer of skin and because of his genetic makeup, the skin has no way of anchoring itself the body. Any form of touch or friction means his skins just tears away.

Its world shattering to think I will lose both of my boys to this and I dont want to think about what life will be like afterwards. I feel like my purpose in life is to give Mary the best life I can for the time that I have him.

I am not able to hug my son because any friction just causes his skin to come away and tear right off. It causes him excruciating pain and I feel so much guilt, but theres nothing I can do to help him.

But despite all of this he is a very happy and joyful boy and he amazes me every single day. Hes an inspiration and proves to everyone that you can keep going with whatever youre going through, no matter how bad it is.

Marky is the youngest of three brothers born to healthcare worker, Melissa. Eldest son Michael is 24, with Carlos dying of heart failure caused by the skin condition in 2015. Marky and Carlos, who would be 21 this year, were both born with the same condition after inheriting a faulty gene from their parents.

Melissa said doctors suspect both she and the boys father carry the faulty gene that causes the genetic mutation. Although both are unaffected themselves, they have an estimated 25% chance of having a child born with recessive dystrophic epidermolysis bullosa. Melissa has since split up with Marky and Carlos father neither of whom knew they carried the faulty gene until Carlos was born, she said.

Melissa said she became aware of the risks involved with having a child after Carlos was born, but claims she was told by a clinician that the chance of her having a second child with the same condition would be less than 1%.

Melissa said: I lost Carlos to the condition because his heart just gave up. His body was in a constant battle to repair his wounds and that took its toll eventually.

His condition was much more progressive than Markys. He got real bad, real quick and by the end could not even breathe on his own.

My pregnancy with Marky was unplanned, but when I got pregnant I was told the chance of me having another baby with the same condition is 1%. They said having two children with it is unheard of.

Marky was born with the most severe type of epidermolysis bullosa meaning he was delivered without skin on his hands, feet and chest because because of the friction of childbirth.

Ever since Marky was little, Melissa, who is now married to Marcos, 30, has had to spend up to three hours a day covering his skin in protective bandages and cleaning open wounds to prevent infection.

She has said every day of motherhood so far has felt like a battle and admits she has struggled to have a normal mother and son relationship with Marky, because of his condition.

Every day is such a massive challenge for us. I struggle with the grief of knowing that when I walk into my sons room in a morning, we cant have fun. I dont get to do the normal things that a mom and a son does.

Instead when I go into his room I know I have to cause him pain and spend two hours doing his bandages. That does give me a lot of anxiety and guilt.

Taking his bandages off does cause excruciating pain. We have to soak his skin every day to soothe his wounds and give him pain medication. Its incredibly upsetting for me.

Whilst there is no cure for epidermolysis bullosa and Marky is highly likely to sadly one day lose his life to the condition, Melissa is hopefully he will survive for several more years.

He has been selected to take part in experimental treatment to create genetically modified skin stem cells that can slow down the effects of the progressive disease. The family plan to travel to Stamford Hospital, Connecticut, later this year to undergo the treatment.

Melissa and Marcos work opposite shifts and have a carer who comes to help look after Marky for three hours each day. Melissa has paid tribute to her amazing son, who she believes can go on to live for many happy years, despite the predictions of doctors.

She said: The treatment at Stamford could add several years to his life. It will reduce the pain and make it easier for his body to cope.

We have tried for that treatment for years, so were thrilled.

Marky has always been an incredibly happy boy and he has just got on with it, without ever moaning or saying why me. Ive had messages from people all over the world to say Marky has inspired them.

Hes an amazing human being and he makes me so proud.

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Terminally-ill teen with agonizingly-sensitive skin defies doctors to live to 19 - Metro.co.uk

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Make-up out, skincare in when working from home. Just ask the stars – AsiaOne

By daniellenierenberg

Too lazy to wear make-up while working from home? You're not alone - according to Bloomberg, a recent survey found about 90 per cent of women working remotely during the coronavirus pandemic are going barefaced.

Even celebrities who rarely show themselves without a face full of make-up have joined the fray.

A-listers including Katy Perry, Julia Roberts, Julianne Moore, Salma Hayek, Halle Berry, Helen Mirren and Jessica Alba have all posted make-up-free selfies while stuck at home.

This does not bode well for beauty companies - data from market research firm NPD Group show total beauty sales in the United States declined 58 per cent year on year in the week ending March 28. It can be beneficial, though, if women begin to put their skincare needs first.

"Now that I am home more, I have no excuse to not be good to my skin. New habits include home facials and face rolling. I have also been using my Foreo face massager weekly and I've been thinking about adding in an LED face mask," says Los Angeles-based entrepreneur Tracy Fong.

Fong is not the only one spending more time on her skin. Beauty retailers such as Joyce Beauty may have noted a decline in lipstick sales, but customers are now stocking up on skincare items such as beauty tools and sheet masks.

"I've added steps to my usual, more basic routine and am trying new things," says fashion executive Jane Tong. "I'm enjoying the little bit of 'me time' that I didn't have pre-quarantine. My skin has definitely improved, but I think it can be attributed to giving it more attention, plus more sleep and a slower-paced lifestyle overall."

While staying at home has given women more time to pamper themselves, working from home can bring new skincare concerns. Being stuck indoors all day means that the skin is exposed to a range of new pollutants.

"We all know how damaging the sun's rays can be - but there are many other light wavelengths that we need to be careful of, including blue light. Wherever you live in the world, indoor pollution can be as bad as outdoor pollution. Additional factors such as mould, heating, air conditioning or poor ventilation can stress out the skin," says Nigma Talib, a Los Angeles-based naturopathic doctor and trained medical aesthetician who counts celebrities such as Gwyneth Paltrow and Penelope Cruz among her clients.

For this reason, Talib advocates that women simplify their routines and focus on high-quality products that help counteract environmental aggressors.

At the top of her list is a resurfacing cleanser, which contains exfoliating agents such as glycolic acid and salicylic acid, to help remove dead skin cells which can build up daily.

A nourishing moisturiser packed with ingredients such as collagen and hyaluronic acid can protect against moisture loss and oxidative stress, which occurs when there is an imbalance between free radicals (unstable molecules with the potential to damage cells) and antioxidants in your body.

Don't forget to apply sunscreen, either. "Layer it on top of a serum loaded with plant stem cells. Sunscreen alone won't prevent light damage to the skin, and the addition of plant stem cells makes your sunscreen 10 times more effective," she says.

Forgoing make-up entirely is not necessarily a good thing. Instead, Talib suggests women pare down their routine and choose products that are multipurpose. Invest in "clean" brands free of toxic chemicals and ingredients, and stick to brands that your skin is already familiar with.

"If it's good quality, make-up can sometimes act as a protective barrier to the skin. I like to suggest a no-make-up look that is simple and free from fussy techniques such as contouring." she says.

"Start with a BB cream or tinted moisturiser with SPF that isn't heavy. Go for a natural colour on your lips - either a simple liner or lip gloss - and a coat of mascara to accentuate your eyes.

"Let your natural beauty shine. You might even come out of quarantine more confident in your skin."

For the latest updates on the coronavirus, visithere.

This article was first published in South China Morning Post.

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Plant Stem Cell Market 2026 Growth Trends by Manufacturers, Regions, Type and Application, Forecast – Latest Herald

By daniellenierenberg

The global Plant Stem Cell Market research report thoroughly explains each and every aspect related to the Plant Stem Cell Market, which facilitates the reports reader to study and evaluate the upcoming market trend and execute the analytical data to promote the business. The growth trend forecasted on account of thorough examination offers in-depth information regarding the global Plant Stem Cell Market. A pathway of development is offered by the market to the several connected networks of businesses under it, which include different firms, industries, organizations, vendors, distributors, and local manufacturers too. All the key Plant Stem Cell Market players compete with each other by offering better products and services at a reasonable price in order to grab significant share at the regional and global level market.

Plant stem cells are undifferentiated cells within the meristems of plants.Plantstem cellsserve as the origin of plant vitality as they maintain themselves and provide a steady supply ofprecursor cellsto form various tissues and organs in plants. Plant stem cells are characterized by two distinctive properties namely its ability to create differentiated celltypes and to self-renew such that the number of stem cells remain stable. The plant stem cell market focuses on this ability of plant cells and thus, processed extracts from the buds, roots and shoots are in high demand and are widely used for topical uses.ription

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The report incorporates an estimated impact of strict standards and regulations set by the government over the market in the upcoming years. The market report also comprises exhaustive research done using several analytical tools such as SWOT analysis to identify the market growth pattern.

Top Manufacturers in GlobalPlant Stem CellMarket Includes:Oriflame Holding AG, MyChelle Dermaceuticals, LLC, Natura Therapeutics Inc, Aidan Products LLC, Mibelle Biochemistry, Phyto Science SDN BHD, and Renature Skin Care Inc.

Regions & Countries Mentioned In The Plant Stem Cell Market Report:

North America ( United States)

Europe ( Germany, France, UK)

Asia-Pacific ( China, Japan, India)

Latin America ( Brazil)

The Middle East & Africa

Key Highlights of the Table of Contents:

Plant Stem Cell Market Study Coverage: It includes key manufacturers covered, key market segments, the scope of products offered in the global market, years considered, and study objectives. Furthermore, it tuches the segmentation study provided in the report on the basis of the type of product and applications.

Plant Stem Cell Market Executive Summary: This section emphasizes on the key studies, market growth rate,Competitive landscape, market drivers, trends, and issues.

Plant Stem Cell Market Production by Region: The report provides information related to import and export, production, revenue, and key players of all regional markets studied are covered in this section.

Plant Stem Cell Market Profile of Manufacturers: Analysis of each market player profiled is detailed in this section. This also provides SWOT analysis, products, production, value, capacity, and other vital factors of the individual player.

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Table of Contents

Report Overview:It includes the Plant Stem Cell market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary:This section of the report gives information about Plant Stem Cell market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players:Here, key players of the Plant Stem Cell market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study:All of the regions and countries analyzed in the Plant Stem Cell market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

An Overview of the Impact of COVID-19 on this Market:

The pandemic of COVID-19 continues to expand and impact over 175 countries and territories. Although the outbreak appears to have slowed in China, COVID-19 has impacted globally. The pandemic could affect three main aspects of the global economy: production, supply chain, and firms and financial markets. National governments have announced largely uncoordinated, country-specific responses to the virus. As authorities encourage social distancing and consumers stay indoors, several businesses are hit. However, coherent, coordinated, and credible policy responses are expected to offer the best chance at limiting the economic fallout.

National governments and international bodies are focused on adopting collaborative efforts to encourage financial institutions to meet the financial needs of customers and members affected by the coronavirus. However, there are some sectors that have remained unscathed from the impact of the pandemic and there are some that are hit the hardest.

We, at Coherent Market Insights, understand the economic impact on various sectors and markets. Using our holistic market research methodology, we are focused on aiding your business sustain and grow during COVID-19 pandemics. With deep expertise across various industries-no matter how large or small- and with a team of highly experienced and dedicated analysts, Coherent Market Insights will offer you an impact analysis of coronavirus outbreak across industries to help you prepare for the future.

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