Top liver disease conference showcases positive clinical data from Yaqrit’s technologies

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Top liver disease conference showcases positive clinical data from Yaqrit’s technologies

NANTES, France, June 24, 2021 (GLOBE NEWSWIRE) -- OSE Immunotherapeutics (ISIN: FR0012127173; Mnemo: OSE) today announced that all the resolutions submitted to a vote at the Combined General Shareholders’ Meeting were approved as proposed by OSE Immunotherapeutics’ Board of Directors.

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OSE Immunotherapeutics Reports on its 2021 Virtual Combined General Shareholder’s Meeting

SAN DIEGO, June 24, 2021 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") today announced that following its receipt of EUA approval from COFEPRIS (Comisión Federal para la Protección contra Riesgos Sanitarios) under registration number “OFICIO: CAS/10720/2021”, it is establishing a local “Sorrento Mexico Ltd” subsidiary to manage commercial operations in Mexico.

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Sorrento Receives EUA Approval and Import License From Mexico Authority COFEPRIS For COVI-STIX™ (COVID-19 Virus Rapid Antigen Detection Test) and...

Company invites individual and institutional investors, as well as advisors and analysts, to attend real-time, interactive presentations on VirtualInvestorConferences.com Company invites individual and institutional investors, as well as advisors and analysts, to attend real-time, interactive presentations on VirtualInvestorConferences.com

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Lowell Farms to Webcast Live at 2021 VirtualInvestorConferences.com on June 29

-- Christopher J. DeRespino appointed acting CFO ---- Peter Soparkar appointed COO --

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Adverum Announces Changes to Management Team

FOSTER CITY, Calif., June 24, 2021 (GLOBE NEWSWIRE) -- Terns Pharmaceuticals, Inc. (“Terns” or the “Company”) (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule single-agent and combination therapy candidates for the treatment of non-alcoholic steatohepatitis (NASH) and other chronic liver diseases, today announced the initiation of patient dosing in the AVIATION Trial, a Phase 1b clinical trial evaluating TERN-201, an orally-administered, highly selective inhibitor of vascular adhesion protein-1 (VAP-1) in patients with NASH.

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Terns Announces Initiation of Patient Dosing in AVIATION Phase 1b NASH Clinical Trial of VAP-1 Inhibitor TERN-201

Findings Published in Nature Chemical Biology Support Clinical Development of RMC-5552, the Company’s First-in-Class, Phase 1/1b Bi-steric mTORC1-Selective Inhibitor Findings Published in Nature Chemical Biology Support Clinical Development of RMC-5552, the Company’s First-in-Class, Phase 1/1b Bi-steric mTORC1-Selective Inhibitor

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Revolution Medicines Announces Publication of Scientific Paper Describing Novel Class of Anti-Tumor Compounds Targeting mTORC1

Ultrasonic Technology to Heal Diabetic Foot Ulcers Ultrasonic Technology to Heal Diabetic Foot Ulcers

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Bioventus Invests in Vaporox

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Avicanna Announces Results of Annual General Meeting and Provides Corporate Update

Libtayo® (cemiplimab) approved by the European Commission for first-line treatment of patients with advanced non-small cell lung cancer with ?50% PD-L1 expression

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Sanofi: Libtayo® (cemiplimab) approved by the European Commission for first-line treatment of patients with advanced non-small cell lung cancer with...

Timeline Guidance for Both Programs Confirmed

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Oxurion NV to Focus Resources on Clinical Assets THR-687 and THR-149

Libtayo® (cemiplimab) approved by the European Commission as the first immunotherapy indicated for patients with advanced basal cell carcinoma

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Sanofi: Libtayo® (cemiplimab) approved by the European Commission as the first immunotherapy indicated for patients with advanced basal cell...

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Nicox’s Licensee Bausch + Lomb Receives Approval for VYZULTA® (latanoprostene bunod ophthalmic solution), 0.024% in the United Arab Emirates

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Sanofi: Availability of the Q2 2021 Memorandum for modelling purposes

Cases of mucormycosis or "black fungus," a rare but serious fungal infection, are climbing in India among some coronavirus patients, per news reports.

Infections have risen to over 30,000 in three weeks, with at least 2,100 deaths, the New York Times reported, compounding the need to protect the country's population, a large percentage of which remain unvaccinated against COVID-19 ahead of a feared third wave this fall.

The infection is caused by a group of molds called mucormycetes which live throughout the environment and typically do not agitate otherwise healthy people, according to the Centers for Disease Control and Prevention (CDC).

However, in those who have health problems, or take medications that lower the bodys ability to fight off germs and sickness, it could infect the sinuses or lungs when inhaled through the air or on injured skin. Diabetes, cancer, organ transplants, stem cell transplants, low white blood cells, long-term corticosteroid use, injection drug use, too much iron, skin injury and premature or low birth weight are all considered to be risk factors.

INDIA'S CORONAVIRUS DOCTORS REPORT BLACK FUNGUS INFECTIONS AMONG SOME PATIENTS

Doctors in India suspect the countrys overwhelmed hospitals and shortage of medical oxygen left patients vulnerable to the fungal infection. Dr. Bela Prajapati, in charge of treatment for hundreds of mucormycosis patients, told the Times that doctors excessive use of steroids to fight inflammation in coronavirus patients, to help them breathe easier, in turn spiked blood sugar levels and left diabetes patients at risk of infection.

Researcher and microbiologist Dr. Arunaloke Chakrabarti noted many doctors didnt have time to question patients over underlying conditions before turning to the steroids.

"Doctors hardly had any time to do patient management," Chakrabarti told the newspaper. "They were all looking at how to take care of the respiratory tract."

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'Black fungus' infections on the rise in India: report - Yahoo News

Continued, heavy thunderstorms along the Gulf Coast will further flash flooding concerns left by the tropical storm. Accuweather

Planning a hike or a nature walk? You probably know to avoid poison ivy by its distinguishable three leaves on a single stem or stinging nettle by its tiny hair-like projections. But there may be one seemingly innocuous plant not on your radar, as one Facebook post claims.

"(It's) about that time of the year again and we are terrified of the kidscoming into contact with Wild Parsnip. Please, please be aware of how dangerous this plant really is!" claims the May 31, 2019, post,since shared over 142,000 times on the social media platform.

The poster says the yellow flowering plant, resembling an upturned umbrella in accompanying pictures, produces a sapthat reacts violently with skin after exposure to sunlight, causing blisters, burns and potentially blindness.

Large, fluid-filled blisters on the chest, hands and arms of an unidentified child, purportedly the poster's son, are included, emphasizing the need for awareness and caution.

Fact check: Brood X cicadas are infected with a sexually transmitted fungus

"I have heard about Wild Parsnip but never knew it was this bad. Poor little guy," commented one Facebook user.

"Oh my gosh, (I) had this in the field behind my house last year, wonder if it comes back every year," said another.

Wild parsnip, an invasive plant species from Europe and Asia likely brought by European settlers to North America for its edible root, is a widespread problem in Canada and in states like Ohio, New Yorkand Minnesota. USA TODAY cannot verify the images shared in the post, but its word of warning about the plant is indeed scientifically true.

Standing at almost 5-feet tall with a single, deeply ridged stem about 2- to 5-centimetersthick, wild parsnip is found throughout southern Canada and the northern U.S. All parts of the plant from the stems, leaves and flowers contain phytochemicals called psoralens, which kill skin cells by inserting themselves into our DNA.

Normally, our skin shields us from a type of radiation emitted by the sun called long-wave ultraviolet radiation, or LWUVR. But with psoralens essentially hijacking our genetic code, they boost the amount of LWUVR our skin absorbsand stop cellular growth.

This may sound bad, and does result in a condition called phytophotodermatitis characterized by angry-looking blisters and burn-like symptoms, but psoralens in combination with ultraviolet light therapy havebeen usedto safely treat skin diseases like psoriasis and vitiligo since the 1970s.

Fact check: Image of human-elephant hybrid is art, doesn't show real baby

Other conditions resulting from coming into contact with wild parsnipcan include blindness if the sap gets into your eyes.

The plant can also inflict injury in animals; livestock that eat it tend to lose weight and may have fertility issues.

Wild parsnip grows in a whole range of environments butis commonly foundalong roadsides, pastures, abandoned fields and any place where soil has been disturbed and native vegetation has yet to be established, according tothe New York Invasive Species Information Clearinghouse.

It's advised if you do come into contact with the yellow-flowered planttoget out of the sun as soon as possible or immediately cover the affected areas before washing with warm water and soap. While photosensitivity and discoloration typically last up to eight hours, these symptoms can linger for up to two years.

Based on our research, we rate TRUE the claim wild parsnip sap can cause skin blisters. A phytochemical secreted by wild parsnip, called psoralens, reacts when exposed to sunlight, affecting cell growth, which results in blisters, other burn-like symptoms and even blindness if the sap gets in the eyes.

Thank you for supporting our journalism. You can subscribe to our print edition, ad-free app or electronic newspaper replica here.

Our fact check work is supported in part by a grant from Facebook.

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Fact check: Contact with wild parsnip harmful to humans and animals - USA TODAY

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Both internal and external factors cause the skin to age. As we age, collagen and other supportive tissues in the deeper layers of the skin decrease, causing the skin to lose its elasticity. Excessive sun exposure, air pollution, stress and chronic ill-health can hasten the process. Aesthetic and surgical treatments for men are now coming into prominence. Here are some:

Laser treatments: New technology is giving laser treatments an impetus, which are good to treat pigmentation and other skin problems. Carbon dioxide and erbium lasers are good options. Botulinum toxin and dermal filler injections: Fat is a very good, popular and natural filler. Botox is a protein that paralyses muscles and prevents them from contracting, thus smoothening fine lines and wrinkles.

Platelet Rich Plasma (PRP) therapy: This is a simple, quick and effective therapy that restores radiance. A small amount of blood is drawn from the patient, centrifuged (spun at fast speed) to separate the platelet-rich plasma and is then injected into the facial skin using a fine needle.

Autologous fat grafting: Fat is harvested from one part of the body, purified and injected into the facial area. With new techniques, the fat can be treated to extract fat stem cells, which have better rejuvenating and repairing potential. This technique is called Nano fat grafting and can be combined with micro-needling.

Thermage: Also known as the lunchtime facelift, it is a quick, non-invasive facial rejuvenation technique. A handpiece delivers radiofrequency heat energy into the collagen-containing layers of the skin, stimulating the collagen to renew and repair itself, and thus adding volume and improving the texture of the facial skin.

HIFU skin tightening: It is a new cosmetic treatment that is a non-invasive and painless replacement for facelifts. Surgical facelift: This is useful when the degree of sagging and wrinkles is unlikely to be corrected by non-surgical techniques. It is done under general anaesthesia.

The author is a SeniorConsultant and Cosmetic Surgeon, Apollo Hospital, New Delhi

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Mens Guide to Cosmetic Beauty - The New Indian Express

ABU DHABI, 22nd June, 2021 (WAM) -- The Ministry of Health and Prevention (MoHAP) and the Emirates Health Services (EHS) recently revealed innovative immunotherapy for cancer and viral infections in cooperation with Japans Kyoto University.

This came during the participation of the ministry and the EHS at the Arab Health 2021 which began in Dubai on 21st June and concludes on 24th June.

The treatment is based on the clinical application of the therapy using T cell preparation after it was discovered that such cells can fight cancer and viral infections. The T cell medicine will be produced using the iPS cell technology.

T Cell makes up a group of lymphocytes present in the blood and plays a major role in cellular immunity. It is possible to produce T cells in large numbers and store them in appropriate conditions to be administered to patients when needed.

Thus, by the success of this project, patients with cancer or viral infection may have great merit in which they can make very easy access to T cell therapy.

Strategic partnerships Dr. Youssef Mohamed Al Serkal, Director-General of the Emirates Health Services, spoke about the commitment of the ministry and the EHS to having strategic partnerships with the most prestigious medical research centres while keeping an eye on the sustainable investment in future healthcare services.

"Although the prevalence of cancer in the UAE is considered lower than in other parts of the world, we work hard to make a qualitative shift in cancer and viral infection healthcare," Al Serkal stated, adding, "This is part of our strategy to provide healthcare services in innovative and sustainable ways and implement the national strategy to reduce cancer mortality rates."

Al Serkal pointed out that the ministry and EHS support the National Cancer Control Programme and prepare a road map to achieve the target indicator. They also analyse the current status of cancer diseases and their diagnostic and therapeutic pathways, support research and studies on the control of cancer diseases and viral infections, and back workshops and educational and training activities. awareness campaigns, and innovative initiatives.

Dr. Kalthum Al Balushi, Director of Hospitals Department, said, "The ground-breaking treatment technology for cancer and viral infections, in cooperation with the Kyoto University, represents a paradigm shift in health services provided by the Ministry and the EHS."

The treatment is based on stimulating immune cells to fight cancer cells using pluripotent stem cells, which is a recent global trend that has begun to open great prospects for improving the quality of life of patients, Al Balushi added.

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MoHAP, EHS reveal immunotherapy for cancer, viral infections at Arab Health 2021 - WAM EN

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced that the results of a Phase 3 clinical study of omidubicel have been published in Blood, the official journal of the American Society of Hematology. Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant solution for patients with hematologic malignancies.

The results demonstrate that transplantation with omidubicel leads to faster neutrophil and platelet recovery compared to a standard umbilical cord blood graft, and results in fewer early bacterial and viral infections and less time in the hospital.

We are pleased that the data from this well-conducted international Phase 3 trial have been published in Blood, the highly respected, peer-reviewed journal of the American Society of Hematology, said Ronit Simantov, M.D., chief medical officer of Gamida Cell. The robust results of this clinical trial have demonstrated that omidubicel could provide an important new option for patients with hematologic malignancies in need of a bone marrow transplant.

Data from this study were previously presented at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research, and most recently during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation. The pivotal study was an international, multi-center, randomized Phase 3 trial designed to compare the safety and efficacy of omidubicel to standard umbilical cord blood transplant in patients with high-risk hematologic malignancies undergoing a bone marrow transplant.

Previous studies have shown that engraftment with omidubicel is durable, with some patients in the Phase 1/2 study now a decade past their transplant. The Phase 3 data reinforce omidubicels potential to be a new standard of care for patients who are in need of stem cell transplantation but do not have access to an appropriate matched donor, said Mitchell Horwitz, M.D., lead author of the paper and a professor of medicine at the Duke Cancer Institute.

The full Blood manuscript is available here: https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.

Details of Phase 3 Efficacy and Safety Results Shared in Blood

The intent-to-treat analysis included 125 patients aged 1365 years with a median age of 41. Forty-four percent of the patients treated on study were non-Caucasian, a population known to be underrepresented in adult bone marrow donor registries. Patient demographics and baseline characteristics were well-balanced across the two study groups. Patients with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma were enrolled at more than 30 clinical centers in the United States, Europe, Asia, and Latin America.

Gamida Cell previously reported in May 2020 that the study achieved its primary endpoint, showing that omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment, a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells and a key milestone in a patients recovery from a bone marrow transplant. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001).

All three secondary endpoints, details of which were first reported in December 2020, demonstrated a statistically significant improvement among patients who were randomized to omidubicel compared to patients randomized to standard cord blood graft. Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 61 days, compared to 48 days for the comparator (p=0.005). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p=0.027). Additional data reported in the manuscript included a comparison of infection density, or the number of infections during the first year following transplantation, which showed that the risk for grade 2 and grade 3 infections was significantly lower among recipients of omidubicel compared to control (risk ratio 0.5, p<0.001).

Data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the studys primary and secondary endpoints. There was no statistically significant difference between the two patient groups in incidence of grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).

These clinical data results form the basis of a Biologics License Application (BLA) that Gamida Cell plans to submit to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2021.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplants for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About Gamida Cell

Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the potential for omidubicel to become a new standard of care and the anticipated submission of a BLA for omidubicel, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to Gamida Cells ability to prepare regulatory filings and the review process therefor; complications in Gamida Cells plans to manufacture its products for commercial distribution; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on March 9, 2021, as amended on March 22, 2021, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

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Gamida Cell Announces Publication in Blood, the Journal of the American Society of Hematology, of the First Pivotal Trial to Evaluate a Cell Therapy...

Imagine being able to reverse blindness, cure multiple sclerosis (MS), or rebuild your heart muscles after a heart attack. For the past few decades, research into stem cells, the building blocks of tissues and organs, has raised the prospect of medical advances of this kind yet it has produced relatively few approved treatments. But that could be about to change, says Robin Ali, professor of human molecular genetics of Kings College London. Just as gene therapy went from being a fantasy with little practical value to becoming a major area of treatment, stem cells are within a few years of reaching the medical mainstream. Whats more, developments in synthetic biology, the process of engineering and re-engineering cells, could make stem cells even more effective.

Stem cells are essentially the bodys raw material: basic cells from which all other cells with particular functions are generated. They are found in various organs and tissues, including the brain, blood, bone marrow and skin. The primary promise of adult stem cells lies in regenerative medicine, says Professor Ali.

Stem cells go through several rounds of division in order to produce specialist cells; a blood stem cell can be used to produce blood cells and skin stem cells can be used to produce skin cells. So in theory you can take adult stem cells from one person and transplant them into another person in order to promote the growth of new cells and tissue.

In practice, however, things have proved more complicated, since the number of stem cells in a persons body is relatively limited and they are hard to access. Scientists were also previously restricted by the fact that adult stem cells could only produce one specific type of cell (so blood stem cells couldnt produce skin cells, for instance).

In their quest for a universal stem cell, some scientists initially focused on stem cells from human embryos, but that remains a controversial method, not only because harvesting stem cells involves destroying the embryo, but also because there is a much higher risk of rejection of embryonic stem cells by the recipients immune system.

The good news is that in 2006 Japanese scientist Shinya Yamanaka of Kyoto University and his team discovered a technique for creating what they call induced pluripotent stem cells (iPSC). The research, for which they won a Nobel Prize in 2012, showed that you can rewind adult stem cells development process so that they became embryo-like stem cells. These cells can then be repurposed into any type of stem cells. So you could turn skin stem cells into iPSCs, which could in turn be turned into blood stem cells.

This major breakthrough has two main benefits. Firstly, because iPSCs are derived from adults, they dont come with the ethical problems associated with embryonic stem cells. Whats more, the risk of the body rejecting the cells is much lower as they come from another adult or are produced by the patient. In recent years scientists have refined this technique to the extent that we now have a recipe for making all types of cells, as well as a growing ability to multiply the number of stem cells, says Professor Ali.

Having the blueprint for manufacturing stem cells isnt quite enough on its own and several barriers remain, admits Professor Ali. For example, we still need to be able to manufacture large numbers of stem cells at a reasonable cost. Ensuring that the stem cells, once they are in the recipient, carry out their function of making new cells and tissue remains a work in progress. Finally, regulators are currently taking a hard line towards the technology, insisting on exhaustive testing and slowing research down.

The good news, Professor Ali believes, is that all these problems are not insurmountable as scientists get better at re-engineering adult cells (a process known as synthetic biology). The costs of manufacturing large numbers of stem cells are falling and this can only speed up as more companies invest in the area. There are also a finite number of different human antigens (the parts of the immune system that lead a body to reject a cell), so it should be possible to produce a bank of iPSC cells for the most popular antigen types.

While the attitude of regulators is harder to predict, Professor Ali is confident that it needs only one major breakthrough for the entire sector to secure a large amount of research from the top drug and biotech firms. Indeed, he believes that effective applications are likely in the next few years in areas where there are already established transplant procedures, such as blood transfusion, cartilage and corneas. The breakthrough may come in ophthalmology (the treatment of eye disorders) as you only need to stimulate the development of a relatively small number of cells to restore someones eyesight.

In addition to helping the body repair its own tissues and organs by creating new cells, adult stem cells can also indirectly aid regeneration by delivering other molecules and proteins to parts of the body where they are needed, says Ralph Kern, president and chief medical officer of biotechnology company BrainStorm Cell Therapeutics.

For example, BrainStorm has developed NurOwn, a cellular technology using peoples own cells to deliver neurotrophic factors (NTFs), proteins that can promote the repair of tissue in the nervous system. NurOwn works by modifying so-called Mesenchymal stem cells (MSCs) from a persons bone marrow. The re-transplanted mesenchymal stem cells can then deliver higher quantities of NTFs and other repair molecules.

At present BrainStorm is using its stem-cell therapy to focus on diseases of the brain and nervous system, such as amyotrophic lateral sclerosis (ALS, also known as Lou Gehrigs disease), MS and Huntingtons disease. The data from a recent final-stage trial suggests that the treatment may be able to halt the progression of ALS in those who have the early stage of the disease. Phase-two trial (the second of three stages of clinical trials) of the technique in MS patients also showed that those who underwent the treatment experienced an improvement in the functioning of their body.

Kern notes that MSCs are a particularly promising area of research. They are considered relatively safe, with few side effects, and can be frozen, which improves efficiency and drastically cuts down the amount of bone marrow that needs to be extracted from each patient.

Because the manufacture of MSC cells has become so efficient, NurOwn can be used to get years of therapy in one blood draw. Whats more, the cells can be reintroduced into patients bodies via a simple lumbar puncture into the spine, which can be done as an outpatient procedure, with no need for an overnight stay in hospital.

Kern emphasises that the rapid progress in our ability to modify cells is opening up new opportunities for using stem cells as a molecular delivery platform. Through taking advantage of the latest advances in the science of cellular therapies, BrainStorm is developing a technique to vary the molecules that its stem cells deliver so they can be more closely targeted to the particular condition being treated. BrainStorm is also trying to use smaller fragments of the modified cells, known as exosomes, in the hope that these can be more easily delivered and absorbed by the body and further improve its ability to avoid immune-system reactions to unrelated donors. One of BrainStorms most interesting projects is to use exosomes to repair the long-term lung damage from Covid-19, a particular problem for those with long Covid-19. Early preclinical trials show that modified exosomes delivered into the lungs of animals led to remarkable improvements in their condition. This included increasing the lungs oxygen capacity, reducing inflammation, and decreasing clotting.

Overall, while Kern admits that you cant say that stem cells are a cure for every condition, there is a lot of evidence that in many specific cases they have the potential to be the best option, with fewer side effects. With Americas Food and Drug Administration recently deciding to approve Biogens Alzheimers drug, Kern thinks that they have become much more open to approving products in diseases that are currently considered untreatable. As a result, he thinks that a significant number of adult stem-cell treatments will be approved within the next five to ten years.

Adult stem cells and synthetic biology arent just useful in treatments, says Dr Mark Kotter, CEO and founder of Bit Bio, a company spun out of Cambridge University. They are also set to revolutionise drug discovery. At present, companies start out by testing large numbers of different drug combinations in animals, before finding one that seems to be most effective. They then start a process of clinical trials with humans to test whether the drug is safe, followed by an analysis to see whether it has any effects.

Not only is this process extremely lengthy, but it is also inefficient, because human and animal biology, while similar in many respects, can differ greatly for many conditions. Many drugs that seem promising in animals end up being rejected when they are used on humans. This leads to a high failure rate. Indeed, when you take the failures into account, it has been estimated that it may cost as much to around $2bn to develop the typical drug.

As a result, pharma companies are now realising that you have to insert the human element at a pre-clinical stage by at least using human tissues, says Kotter. The problem is that until recently such tissues were scarce, since they were only available from biopsies or surgery. However, by using synthetic biology to transform adult stem cells from the skin or other parts of the body into other types of stem cells, researchers can potentially grow their own cells, or even whole tissues, in the laboratory, allowing them to integrate the human element at a much earlier stage.

Kotter has direct experience of this himself. He originally spent several decades studying the brain. However, because he had to rely on animal tissue for much of his research he became frustrated that he was turning into a rat doctor.

And when it came to the brain, the differences between human and rat biology were particularly stark. In fact, some human conditions, such as Alzheimers, dont even naturally appear in rodents, so researchers typically use mice and rats engineered to develop something that looks like Alzheimers. But even this isnt a completely accurate representation of what happens in humans.

As a result of his frustration, Kotter sought a way to create human tissues. It initially took six months. However, his company, Bit Bio, managed to cut costs and greatly accelerate the process. The companys technology now allows it to grow tissues in the laboratory in a matter of days, on an industrial scale. Whats more, the tissues can also be designed not just for particular conditions, such as dementia and Huntingdons disease, but also for particular sub-types of diseases.

Kotter and Bit Bio are currently working with Charles River Laboratories, a global company that has been involved in around 80% of drugs approved by the US Food and Drug Administration over the last three years, to commercialise this product. They have already attracted interest from some of the ten largest drug companies in the world, who believe that it will not only reduce the chances of failure, but also speed up development. Early estimates suggest that the process could double the chance of a successful trial, effectively cutting the cost of each approved drug by around 50% from $2bn to just $1bn. This in turn could increase the number of successful drugs on the market.

Two years ago my colleague Dr Mike Tubbs tipped Fate Therapeutics (Nasdaq: FATE). Since then, the share price has soared by 280%, thanks to growing interest from other drug companies (such as Janssen Biotech and ONO Pharmaceutical) in its cancer treatments involving genetically modified iPSCs.

Fate has no fewer than seven iPSC-derived treatments undergoing trials, with several more in the pre-clinical stage. While it is still losing money, it has over $790m cash on hand, which should be more than enough to support it while it develops its drugs.

As mentioned in the main story, the American-Israeli biotechnology company BrainStorm Cell Therapeutics (Nasdaq: BCLI) is developing treatments that aim to use stem cells as a delivery mechanism for proteins. While the phase-three trial (the final stage of clinical trials) of its proprietary NurOwn system for treatment of Amyotrophic lateral sclerosis (ALS, or Lou Gehrigs disease) did not fully succeed, promising results for those in the early stages of the disease mean that the company is thinking about running a new trial aimed at those patients. It also has an ongoing phase-two trial for those with MS, a phase-one trial in Alzheimers patients, as well as various preclinical programmes aimed at Parkinsons, Huntingtons, autistic spectrum disorder and peripheral nerve injury. Like Fate Therapeutics, BrainStorm is currently unprofitable.

Australian biotechnology company Mesoblast (Nasdaq: MESO) takes mesenchymal stem cells from the patient and modifies them so that they can absorb proteins that promote tissue repair and regeneration. At present Mesoblast is working with larger drug and biotech companies, including Novartis, to develop this technique for conditions ranging from heart disease to Covid-19. Several of these projects are close to being completed.

While the US Food and Drug Administration (FDA) controversially rejected Mesoblasts treatment remestemcel-L for use in children who have suffered from reactions to bone-marrow transplants against the advice of the Food and Drug Administrations own advisory committee the firm is confident that the FDA will eventually change its mind.

One stem-cell company that has already reached profitability is Vericel (Nasdaq: VCEL). Vericels flagship MACI products use adult stem cells taken from the patient to grow replacement cartilage, which can then be re-transplanted into the patient, speeding up their recovery from knee injuries. It has also developed a skin replacement based on skin stem cells.

While earnings remain relatively small, Vericel expects profitability to soar fivefold over the next year alone as the company starts to benefit from economies of scale and runs further trials to expand the range of patients who can benefit.

British micro-cap biotech ReNeuron (Aim: RENE) is developing adult stem-cell treatments for several conditions. It is currently carrying out clinical trials for patients with retinal degeneration and those recovering from the effects of having a stroke. ReNeuron has also developed its own induced pluripotent stem cell (iPSC) platform for research purposes and is seeking collaborations with other drug and biotech companies.

Like other small biotech firms in this area, it is not making any money, so it is an extremely risky investment although the rewards could be huge if any of its treatments show positive results from their clinical trials.

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Investing in stem cells, the building blocks of the body - MoneyWeek