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Experimental compound, which has received orphan drug and pediatric rare disease designations from the FDA, displays effectiveness in treating…

Posted: December 10, 2021 at 2:13 am

image:Prof. Illana Gozes view more

Credit: Jonathan Blum, Tel Aviv University.

An extensive international study led by Tel Aviv University found that an experimental drug which has been awarded orphan drug designation by the FDA for future treatment of a rare development disorder can also treat a variety of symptoms relating to autism, intellectual disability, and Alzheimers disease.

The experimental drug, NAP, was discovered in the lab of Prof. Illana Gozes of the Tel Aviv University Sackler Medical Schools Department of Human Molecular Genetics and Biochemistry. In recent years, the FDA has granted the experimental drug with orphan drug designation and pediatric rare disease designation for treatment of a rare developmental disorder called ADNP syndrome, which can cause a variety of symptoms, among them, hallmark features are intellectual disability and autism spectrum disorder.

In the current study, a team of researchers led by Prof. Gozes developed an innovative lab model and found that NAP can be effective in treating a broad spectrum of symptoms of ADNP syndrome, which is caused by mutations in the ADNP gene which is essential to cerebral development and protecting cerebral brain cells. Previous studies showed that ADNP syndrome is related to Alzheimers disease and certain types of mental disabilities, developmental delays, and autism.

Ramot, Tel Aviv University's technology commerce company filed a number of patent applications to protect the technology and its implementation and, in collaboration with Prof. Gozes, is raising funds to finance further clinical research. Similarly, Ramot is in discussions regarding commercial collaboration with pharmaceutical companies. "Were excited by this new discovery and believe that this is groundbreaking technology that will remedy a variety of symptoms and disabilities in a broad spectrum of orphan diseases," said Prof. Keren Primor Cohen, CEO of Ramot.

The study, which is the culmination of the MD/PhD student Dr. Gideon Carmons doctoral research, was joined by a team of researchers from Prof. Gozess lab: Dr. Shlomo Sergovich, Gal Hacohen-Kleiman, Inbar Ben-Horin-Hazak, Dr. Oxana Kapitansky, Alexandra Lubincheva, and Dr. Eliezer Giladi. The team was further joined by Dr. Moran Rubinstein, Prof. Noam Shomron, and Guy Shapira of TAUs Sackler Faculty of Medicine, and Dr. Metsada Pasmanik Chor of Tel Aviv Universitys George S. Wise Faculty of Life Sciences. Researchers from the Czech Republic, Greece, Germany, and Canada also participated. The article was published in the prestigious journal Biological Psychiatry.

Prof. Gozes explained that: NAP, in fact, comprises a short segment of the normal ADNP protein. We previously found that treatment using NAP corrects the function of human nerve cells afflicted with ADNP syndrome in a laboratory test-tube. In this study, we sought to examine the efficacy of NAP in treating various aspects of the syndrome using a model with the most harmful mutation, which allowed us to view brain development and facilitate remedying of behavioral problems.

The study, which examined a model using mice with ADNP syndrome, used objective methods to analyze behavior, electrical activity, and to further identify select protein contents in the brain. The researchers found that the mice suffering from ADNP syndrome demonstrated a broad spectrum of pathological outcomes, including increased rates of neonatal death immediately after birth, slowed development and aberrant gait, primarily among females, as well as poor voice communication.

Cerebral examinations demonstrated additional findings: A relatively small number of synapses the points of contact tween nerve cells, impaired electrophysiological activity demonstrating a low potential for normal cerebral arousal, as well as precipitates (aggregates) of the Tau protein in young mice, similar to those in the brains of elderly Alzheimer's disease patients.

For most of these symptoms, the researchers examined the effect of the future medicinal substance NAP made of a short and normal segment of the ADNP protein, the same protein that is impaired because of the mutation. Prof. Gozes: "In the past, we have found that NAP corrects impaired functioning of ADNP that has mutated in the nerve cell model in the culture. We now examined its effect in vivo in animals modeling the syndrome (ADNP mutation). To our amazement and joy, we discovered that treatment using NAP normalizes the functioning of these mice for most of the symptoms indicated above!"

Researchers further sought to identify in the blood of the mice, a clear biological indicator of ADNP syndrome that will enable diagnosis of this severe disease and effective monitoring of treatment using a simple blood test. With the help of genetic sequencing technologies, they identified an anomaly in a manner characteristic only of females as well as a method for repair using NAP on five proteins (at the messenger RNA level). These findings matched the changes discovered in white blood cells of children suffering from ADNP syndrome. One of the indicators discovered is FOXO3 a protein with an important role in generating cerebral synapses and healthy aging.

Prof. Gozes summarized: "In this study, we examined the effect of the ADNP genes most prevalent mutation in a broad spectrum of aspects and found extensive impairment in physical and cerebral functioning parallel to the symptoms of autism, developmental delay, mental disability, and Alzheimer's disease in humans. Similarly, we examined the potential use of the NAP drug for treating these diseases, and discovered that it is effective against most of these symptoms in lab models. This study is an important milestone on the way to developing a drug, or drugs, that will help children with autism stemming from genetic mutations, as well as Alzheimer's patients."

Link to the article:

https://doi.org/10.1016/j.biopsych.2021.09.018

Biological Psychiatry

Novel ADNP Syndrome Mice Reveal Dramatic Sex-Specific Peripheral Gene Expression With Brain Synaptic and Tau Pathologies

28-Sep-2021

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BIS Research Study Highlights the Global CRISPR Gene Editing Market to Reach $18.85 Billion by 2031 – PRNewswire

Posted: December 10, 2021 at 2:13 am

FREMONT, Calif., Dec. 9, 2021 /PRNewswire/ -- The global CRISPR gene editing marketis projected to reach $18.85 billion by 2031, reveals the premium market intelligence study by BIS Research. The study also highlights that the market is set to witness a CAGR of 29.60% during the forecast period 2021-2031.

The development of genome engineering with potential applications proved to reflect a remarkable impact on the future of the healthcare and life science industry. The high efficiency of the CRISPR-Cas9 system has been demonstrated in various studies for genome editing, which resulted in significant investments within the field of genome engineering. However, with so many advantages, limitations also exist, which need consideration before clinical applications.

The detailed study is a compilation of 09 Market Data Tables and 238 Figures spread through 296 Pages and in-depth TOC on "Global CRISPR Gene Editing Market Analysis and Forecast, 2021-2031"

USP of the report

Analyst's Take on the Market:

To emphasize the dominance of the kits and enzymes segment of CRISPR products market segment over other segments under the product category of CRISPR gene editing market in 2021 and 2031, Nitish Kumar, Lead Analyst BIS Research, states, "The reason for market growth and the dominance of CRISPR products market segment can be attributed to increasing global geriatric population, prevalence of genetic disorders, chronic conditions, rising focus on research and development (R&D) of novel gene therapies, increased government and private funding for improved safety and outcomes provided by the CRISPR gene editing products."

View the Reportfrom BIS Research at Global CRISPR Gene Editing Market

Key Companies Operating in the Market and Competition Landscape

Key insights are drawn from in-depth interviews with the key opinion leaders of more than 20 leading companies, market participants, and vendors. The key players profiled in the report include Abcam plc., Agilent Technologies, Inc., Applied StemCell, Inc., Cellecta, Inc., CRISPR Therapeutics, OriGene Technologies, Inc., GeneCopoeia, Inc., Genscript Biotech Corporation, Horizon Discovery Group Plc, Integrated DNA Technologies (IDT), Inc., Merck KGaA, New England Biolabs, Inc., Rockland Immunochemicals, Inc., Synthego Corporation, System Biosciences, LLC, Thermo Fisher Scientific Inc., ToolGen, Inc., Intellia Therapeutics, Inc., Editas Medicine, Takara Bio, Inc., and Caribou Biosciences, Inc.

The study also offers strategic recommendations that can help organizations track various products, trends, and technologies that are changing the market dynamics. The recommendations by BIS Research also offer bespoke research services to help organizations meet their objectives.

Who should buy this report?

Request for a Sample: https://bisresearch.com/requestsample?id=1210&type=download

Key Questions Answered in the Report:

BIS Research Related Market Studies:

Global Cell and Gene Therapy Market - Analysis and Forecast, 2019-2025

About BIS Research:

BIS Researchis a global B2B market intelligence and advisory firm focusing on deep technology and related emerging trends which can disrupt the market dynamics in the near future. We publish more than 200 market intelligence studies annually that focus on several deep technology verticals.

Our strategic market analysis emphasizes on market estimations, technology analysis, emerging high-growth applications, deeply segmented granular country-level market data, and other important market parameters useful in the strategic decision-making for senior management.

BIS Research offers syndicate as well as custom studies and expert consultations to firms, providing them specific and actionable insights on novel technology markets, business models, and competitive landscape.

BIS Healthcare vertical offers intelligence in the healthcare technology market for Medical Devices, Digital Health, Life Sciences, Robotics and Imaging, Information Technology, Precision Medicine, and other emerging healthcare technologies, covering the entire industry spectrum. In the past 5 years, BIS Healthcare has published more than 50 reports under the precision medicine banner. Additionally, BIS Research has been nominating Top 25 Voices in Precision Medicine on its Insight Monk platform for the past two years successfully.

Contact:Bhavya BangaEmail: [emailprotected] BIS Research Inc.39111 PASEO PADRE PKWY STE 313,FREMONT CA 94538-1686Visit our Blog @ https://blog.bisresearch.com/ Connect with us on LinkedIn @ https://www.linkedin.com/company/bis-researchConnect with us on [emailprotected] https://twitter.com/BISResearch

SOURCE BIS Research

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Laminitis insights show promise for the future | Penn Today – Penn Today

Posted: December 10, 2021 at 2:13 am

Horse owners usually dread hearing the diagnosis of laminitis. The disease plagues horses of many backgrounds, ages, and disciplines. Now, using genetics, scientists from Penns School of Veterinary Medicine and the University of Florida (UF) have made new insights in the disease. Their findings appear in the journal Veterinary Immunology and Immunopathology.

A horses hoof has a tough job. It must support a heavy animal which can move faster than 40 miles per hour. Laminitis occurs when inflammation and damage of the tissue arises between the hoof and coffin bone. The condition causes lameness and a diminished quality of life, and it often results in euthanasia.

Laminitis is a tough problem for the horse and its owner, says Samantha Brooks, UF associate professor of equine physiology and the corresponding author on the work. We have very few tools in our arsenal to manage the disease itself. We treat symptoms, pain, and mechanical instability but do not have anything to target the cause just yet.

By tapping into my labs database and incorporating Dr. Brooks unparalleled expertise in equine genetics and transcriptome analysis, we have identified new and promising pathways in cell stress and inflammatory response that significantly enhance our understanding of supporting limb laminitis and its disease processes, says Hannah Galantino-Homer, senior investigator in laminitis research at Penn Vets New Bolton Center and a coauthor on the paper.

Laminitis studies have previously been hindered by the scarcity of genetic information specific to hoof tissues. Scientists tapped into the New Bolton Center Laminitis Discovery Database, an archive of data and sample sets from naturally occurring laminitis cases collected since 2008. Using that database, researchers examined 36 archived tissues of 20 Thoroughbred horses treated for laminitis.

There are three types of laminitis, and all impair the structure and function of the horses foot. This research provided a snapshot of the active pathways and functions of the hoof, with a focus on supporting limb laminitis, the kind to which famous racehorse Barbaro succumbed.

We understand the situations that trigger an episode of laminitis, but we do not have a good understanding of what is happening in the hoof, says Brooks. This study took a very comprehensive view of the processes early in the development of laminitis.

Using gene-expression analysis, researchers catalogued the changes in gene transcription across the 20 horses. Some had healthy feet, some were early in the disease process, and others were more severe.

Researchers identified three key findings about the disease process.

The first related to keratin, an important structural protein that helps maintain the structural integrity of materials like hair, nails, and hooves. This study was one of the first to examine the changes in the keratin protein family through the laminitis disease process. Some of the keratin-related genes and genes involved in regulating of the cells manufacturing process started to diminish as the disease began. This could be compared to when a car gets a flat tire; it may still be running, but it loses appropriate function and slows down.

Another component of cell machinery often studied in laminitis is a class of enzymes called metalloproteinases that help maintain the cytoskeleton. These enzymes must maintain a careful equilibrium. Hooves must be able to grow yet not break down under the weight of the horse, which requires a balance of remodeling and building tissues. When the metalloproteinases become too active, the hoof begins to lose structural strength. One previous strategy for treating this process was to stop these enzymes from becoming too active. But treatment targeting these enzymes might also stop hoof growth, which would likely lead to further problems.

When keratin degrades, inflammation in the hoof leads to laminitis. Scientists found a collection of genes responsible for triggering that inflammation which could pave the way for future therapies. The genes led researchers to believe that some human medications for autoimmune disorders may help horses with laminitis.

Changes in gene expression in diseased tissue are often reflected in changes in the proteins that can be detected in the blood as the disease progresses. For example, specific proteins, or biomarkers, that increase in the blood in humans following traumatic brain injury were also expressed at higher levels in the samples from the horses with laminitis in this study. Medical doctors have used these compounds to understand the severity of these injuries in humans without using imaging or more invasive testing. The team hopes this could be used as a tool to monitor the progression of laminitis in the horse.

We dont always recognize that a horse has severe laminitis until things have gotten quite bad, says Brooks. Early monitoring tools and ways to combat the disease were exciting findings, but we need further research before these new tools will be ready for use in the field.

The researchers hope that this research can lead to a blood test to detect these new laminitis-related biomarkers as well as medications that are economical and effective for horses with the disease.

Ultimately, these new findings point us towards a more targeted approach for future exploration that we hope will help uncover novel solutions for preventing and treating this debilitating disease, says Galantino-Homer.

This is a big step in improving our understanding of laminitis, says Brooks. Something that could be completely untreatable 10 years ago; in another 10 years we may be able to intervene and make a significant difference in the disease early on.

Galantino-Homer and Brooks coauthors were Heather M. Holl from UF and Caitlin Armstrong from Penn Vet.

The study was supported by funding from The Foundation for the Horse.

University of Florida media contact: Tory Moore, 354-273-3566, torymoore@ufl.edu

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A New Stem-Cell Treatment Looks to Have Cured a Man of Type 1 Diabetes – Good News Network

Posted: December 10, 2021 at 2:09 am

Growing new pancreas cells from unprogrammed stem cells has possibly cured a man of type 1 diabetes.

One doctor told the New York Times that this is the biggest development in treatment for the disease since the discovery of exogenous insulin production 100 years ago.

64-year-old Brian Shelton got an infusion of insulin-producing cells in the pancreasthe kinds which cant function properly in diabetes victims, after his wife signed him up for a trial run by Vertex Pharmaceuticals.

Before, his life had been governed by the levels of his blood sugar. Now, his daily insulin requirements are down 91%, accompanied by robust improvements in glucose control.

Its a whole new life, its a miracle, Shelton told the Times. The trial was organized by a Harvard scientist who had two children join the 1.5 million Americans who suffer from type 1 diabetes.

This is the first of five years in which the trial of 17 patients with severe type 1 will be running. The results of the first stage have not been peer reviewed, and so expectedly, the scientists that are excited about the results are also urging caution because its still early days.

MORE: Patch Inspired by Cactus Eliminates Need for Diabetics to Prick Skin for Blood, Collects Sweat Instead

These results from the first patient treated with [the stem cells] are unprecedented, said Bastiano Sanna, Ph.D., Chief of Cell and Genetic Therapies at Vertex in a statement. What makes these results truly remarkable is that they were achieved with treatment at half the target dose.

Funded by the Howard Hughes Medical Institute, Harvards Dr. Doug Milton took 20 years to convert stem cells into islet cells, the insulin-producing pancreatic denizens.

RELATED: People Whove Tried Psychedelics Have Lower Risk of Heart Disease and Diabetes

In 2014 Milton partnered with Dr. Sanna at his previous job to start a company called Semma for the purpose of bringing to market a potential stem cell treatment, and along with other biologists, was able to demonstrate that for the first time, there was a repeatable, scalable method for growing islet cells and that they could cure diabetes in rodents.

Next, Milton and his partners and colleagues closed a $950 million sale of Semma to Vertex Pharma, who put up the money for the trials as they needed to see if injections of the manufactured islet cells could be done at scale, safely, and if the immunosuppressant drugs, typical of anyone receiving any kind of transplant, did not cause long-term adverse health outcomes.

READ: No More Pricks: Scientists Are Rolling Out First-of-its-Kind Blood Sugar Test for Pain-Free Delivery to Diabetics

The night the trial results came in, Mr. Shelton was taken to dinner by Dr. Milton, who revealed that Shelton was at least for the moment, cured of the disease.

The Times touchingly reports that at that moment Shelton checked his blood sugar levels, which were perfect, and then had dinner, after which they were still perfect.

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Cytovia Therapeutics’ Scientific Leadership to Present at Upcoming Conferences – Business Wire

Posted: December 10, 2021 at 2:09 am

AVENTURA, Fla. & NATICK, Mass.--(BUSINESS WIRE)--Cytovia Therapeutics, Inc., a biopharmaceutical company developing allogeneic off-the-shelf gene-edited iNK cells (NK cells derived from induced pluripotent stem cells, or iPSCs), CAR (Chimeric Antigen Receptor)-iNK cells, and Flex-NK cell engager multifunctional antibodies, announced today that its scientific leadership will present at the following upcoming conferences:

iPSC-Derived Cell Therapies Summit (Virtual Event)

Wednesday, December 8th, 2021, 9.30 AMDemonstrating Safety & Efficacy of iPSC-derived Cells to Maximize Clinical Translation(Industry Leaders Fireside Chat: Paving the Way For the Future of Cell Therapy with iPSCs)Featuring Cytovia Therapeutics CMO Stanley Frankel

Wednesday, December 8th, 2021, 3PMAdvancing Clinical Trial Strategy for Successful Clinical Outcomes(Developing iPSC-Derived NK Cell Therapies Against Solid Tumors)Featuring Cytovia Therapeutics CSO Wei Li

Antibody Engineering & Therapeutics Conference (San Diego, California)

December 12th-15th, 2021Dr. Jean Kadouche, Cytovia Therapeutics scientific co-founder and Global Head of Antibody R&D, will present the Design, Manufacturing, and Activity of FLEX Immune Cell Engager Multi-functional Antibodies poster on behalf of the Cytovia team.

We are proud of the scientific thought leadership of our colleagues Drs. Frankel, Li, and Kadouche, who will present Cytovias latest advances, said Dr. Daniel Teper, CEO of Cytovia Therapeutics. Cytovia is committed to translating disruptive innovation in cell therapy to create therapeutics that advance towards a cancer cure. Cytovia is the first biotech company to develop its own best-in-class gene-edited, iPSC-derived NK cells and NK Engager antibodies, with the potential to combine them for optimal clinical outcomes in both hematological and solid tumors.

About Cytovia Therapeutics

Cytovia Therapeutics aims to accelerate patient access to transformational cell therapies and immunotherapies, addressing several of the most challenging unmet medical needs in cancer. Cytovia focuses on harnessing the innate immune system by developing complementary and disruptive NK-cell and NK-engager antibody platforms. It is developing three types of iPSC-derived (or iNK) cells: unedited iNK cells, TALEN gene-edited iNK cells with improved function and persistence, and TALEN gene-edited iNK cells with chimeric antigen receptors (CAR-iNKs) to improve tumor-specific targeting. The second complementary cornerstone technology is a quadrivalent multifunctional antibody platform designed to engage natural killer cells by targeting NKp46 using its proprietary Flex-NK technology.

These two technology platforms are being used to develop treatment of patients with solid tumors such as hepatocellular carcinoma (HCC) and glioblastoma as well as hematological malignancies such as refractory multiple myeloma.

Cytovias research and development laboratories in Natick, MA and GMP cell manufacturing facility in Puerto Rico are augmented by scientific partnerships with Cellectis, CytoImmune, the Hebrew University of Jerusalem, INSERM, the New York Stem Cell Foundation, and the University of California San Francisco (UCSF).

Cytovia Therapeutics has recently formed CytoLynx Therapeutics, a joint-venture entity focused on research and development, manufacturing, and commercialization activities in Greater China and beyond.

Find out more at http://www.cytoviatx.com and follow us on Facebook, Twitter, LinkedIn, and YouTube.

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BrainStorm Cell Therapeutics and Catalent Announce Completion of Technology Transfer for NurOwn Manufacturing – PRNewswire

Posted: December 10, 2021 at 2:09 am

NEW YORK and SOMERSET, N.J., Dec. 7, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, and Catalent (NYSE: CTLT), a global leader in enabling biopharma, cell, gene and consumer health partners to optimize development, launch, and supply of better patient treatments across multiple modalities, today announced that the technology transfer for NurOwn manufacturing at Catalent's facility has been finalized. NurOwn is BrainStorm's autologous cellular therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS), progressive multiple sclerosis (PMS) and other neurodegenerative diseases.

Catalent entered into a partnership with Brainstorm in 2020 to provide CGMP clinical supply of NurOwn, in anticipation of the product candidate's potential regulatory approval. NurOwn will be manufactured at Catalent's world-class 32,000 square-foot cell therapy manufacturing facility in Houston, Texas.

"The successful completion of this technology transfer with Catalent is an important step in establishing manufacturing preparedness for NurOwn," said Chaim Lebovits, Chief Executive Officer, Brainstorm Cell Therapeutics. "The manufacturing of cellular therapies such as NurOwn is complex, and requires careful planning and very specific expertise. We are very pleased with the progress we have made with our partner Catalent, which has industry-leading capabilities in this area."

Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy, said, "Our extensive experience in cell therapy development and scale-up was key to the completion of this technology transfer to our state-of-the-art cell therapy facility in Houston, Texas. We look forward to continuing our partnership with BrainStorm and are committed to enabling the advancement of their autologous stem cell therapy product candidate toward a potential future commercial launch."

About NurOwn

The NurOwn technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About Catalent

Catalent is the global leader in enabling pharma, biotech, and consumer health partners to optimize product development, launch, and full life-cycle supply for patients around the world.

With broad and deep scale and expertise in development sciences, delivery technologies, and multi-modality manufacturing, Catalent is a preferred industry partner for personalized medicines, consumer health brand extensions, and blockbuster drugs. Catalent helps accelerate over 1,000 partner programs and launch over 150 new products every year. Its flexible manufacturing platforms at over 50 global sites supply over 70 billion doses of more than 7,000 products to over 1,000 customers annually.

Catalent's expert workforce exceeds 17,000, including more than 2,500 scientists and technicians. Headquartered in Somerset, New Jersey, the company generated $4 billion in revenue in its 2021 fiscal year. For more information, visitwww.catalent.com.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contacts:

Brainstorm Contacts

Investor Relations:John MullalyLifeSci Advisors, LLCPhone: +1 617-429-3548[emailprotected]

Media:Mariesa Kemble[emailprotected]

Catalent Media Contact:Chris HallingPhone: +44 (0)7580 041073 [emailprotected]

SOURCE Brainstorm Cell Therapeutics Inc

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Affimed to Host Virtual Investor Call Today to Discuss Treatment of CD30-positive Lymphoma … – The Bakersfield Californian

Posted: December 10, 2021 at 2:09 am

For the 13 patients treated at the recommended phase 2 dose (RP2D) the response rate after one cycle of treatment remains at 100% with a 38.5% complete response (CR) rate; one additional patient completed cycle 1 at the RP2D and was assessed with a partial response (PR)Three of 3 patients treated with two cycles in the dose escalation part of the study at the RP2D remain in CR at 6 months after start of treatmentSide effect profile shows only five instances of transient infusion-related reactions (IRR) in more than 100 AFM13 infusions with no episodes of neurotoxicity, CRS or GvHD

HEIDELBERG, Germany, Dec. 09, 2021 (GLOBE NEWSWIRE) -- Affimed N.V. (Nasdaq: AFMD), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer will host today a financial community call to discuss recent findings from the investigator sponsored trial (IST) at The University of Texas MD Anderson Cancer Center investigating the treatment of CD30-positive lymphoma patients with its innate cell engager (ICE) AFM13, pre-complexed with cord blood-derived natural killer (cbNK) cells (AFM13-104).

A treatment cycle consists of lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed two days later by a single infusion of cytokine-preactivated and expanded cbNK cells that are pre-complexed with AFM13, followed by three weekly infusions of AFM13 (200 mg) monotherapy. Responses are assessed on day 28 by FDG-PET and patients can receive up to two cycles. Three patients were treated with 1106, three patients with 1107 and 13 patients with 1108 AFM13-pre-complexed cbNK cells per kg body weight.

Response Assessment

A total of 19 patients with CD30-positive relapsed or refractory Hodgkin and non-Hodgkin lymphomas (17 and 2 patients, respectively) have been treated to date across three dose cohorts. According to investigator assessment, 17 of 19 patients had achieved an objective response (ORR 89.5%) to the treatment, with seven complete responses (CR 36.8%) and ten partial responses (PR 52.6%).

In patients treated at the RP2D level of 1x108 cbNK cells per kg, 12 of 13 had classical Hodgkin lymphoma and 1 patient had CD30-positive NHL. In this cohort, 100% of patients responded after the first cycle of treatment with five CRs (38.5%) and seven PRs (61.5%). All patients treated at the RP2D have now received a second cycle of therapy. Response evaluation after cycle 2 will be reported at a future scientific conference.

Initial Durability of Response Observations

Nine patients treated in the dose escalation phase of the study had follow-up at 6 months. Of note, the three patients treated at the RP2D remain in remission at 6 months after start of treatment, two without additional treatment and one on anti-PD-1 antibody maintenance.

In the four responders out of six treated at the two lower dose levels, one patient, who started treatment in September 2020, remains in remission after consolidation autologous stem cell transplant, and three relapsed at 3.4, 4.8 and 6.3 months after start of therapy.

Safety

Five reported cases of transient infusion related reactions were reported after the monotherapy infusions of AFM13. Of note, there were no instances of serious adverse events such as cytokine release syndrome, immune cell-associated neurotoxicity syndrome or graft-versus-host disease.

Conference Call/Webcast Information

The event today will include a review of Affimeds approach to activating the innate immune system in the fight against cancer, preclinical data supporting the combination of Affimeds ICE molecules with adoptive NK cell transfer, a review of the treatment challenges and clinical opportunities for CD30+ lymphomas, and review of the interim data from AFM13-104 by the studys principal investigator, Yago L. Nieto, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at of The University of Texas MD Anderson Cancer Center.

Affimed will host a conference call and webcast today, December 9th, 2021, at 8:30 a.m. EST. To access the call, please dial +1 (409) 220-9054 for U.S. callers, or +44 (0) 8000 323836 for international callers, and reference passcode 3065475 approximately 15 minutes prior to the call.

A live audio webcast of the conference call will be available in the Webcasts section on the Investors page of the Affimed website at https://www.affimed.com/webcasts/investor-day/ or https://edge.media-server.com/mmc/p/zzwismtq. A replay of the webcast will be accessible at the same link for 30 days following the call.

About the Phase 1-2 Study

The University of Texas MD Anderson Cancer Center is studying AFM13 in an investigator-initiated phase 1-2 trial in combination with cord blood-derived allogeneic NK cells in patients with recurrent or refractory CD30-positive lymphomas. The first phase of this study involves dose escalation of pre-complexed NK cells, with patients receiving lymphodepleting chemotherapy followed by 1106 NK cells/kg in Cohort 1; 1107 NK cells/kg in Cohort 2; and 1108 NK cells/kg in Cohort 3. The trial is designed to explore safety and to determine the recommended phase 2 dose and evaluate its activity. The recommended phase 2 dose was determined as 1108 NK cells/kg. In each cohort, the dose of the pre-complexed NK cells with AFM13 is followed by weekly doses of 200 mg AFM13 monotherapy for three weeks, with each patient evaluated for dose-limiting toxicities and responses on day 28. MD Anderson has an institutional financial conflict of interest with Affimed related to this research and has therefore implemented an Institutional Conflict of Interest Management and Monitoring Plan. Additional information about the study can be found at http://www.clinicaltrials.gov (NCT04074746).

About AFM13

AFM13 is a first-in-class innate cell engager (ICE) that uniquely activates the innate immune system to destroy CD30-positive hematologic tumors. AFM13 induces specific and selective killing of CD30-positive tumor cells, leveraging the power of the innate immune system by engaging and activating natural killer (NK) cells and macrophages. AFM13 is Affimeds most advanced ICE clinical program and is currently being evaluated as a monotherapy in a registration-directed trial in patients with relapsed/refractory peripheral T-cell lymphoma or transformed mycosis fungoides (REDIRECT). The study is actively recruiting. Additional details can be found at http://www.clinicaltrials.gov (NCT04101331).

About Affimed N.V.

Affimed (Nasdaq: AFMD) is a clinical-stage immuno-oncology company committed to give patients back their innate ability to fight cancer by actualizing the untapped potential of the innate immune system. The companys proprietary ROCK platform enables a tumor-targeted approach to recognize and kill a range of hematologic and solid tumors, enabling a broad pipeline of wholly owned and partnered single agent and combination therapy programs. The ROCK platform predictably generates customized innate cell engager (ICE) molecules, which use patients immune cells to destroy tumor cells. This innovative approach enabled Affimed to become the first company with a clinical-stage ICE. Headquartered in Heidelberg, Germany, with offices in New York, NY, Affimed is led by an experienced team of biotechnology and pharmaceutical leaders united by a bold vision to stop cancer from ever derailing patients lives. For more about the companys people, pipeline and partners, please visit: http://www.affimed.com.

Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. Forward-looking statements appear in a number of places throughout this release and include statements regarding our intentions, beliefs, projections, outlook, analyses and current expectations concerning, among other things, the potential of AFM13, AFM24, and our other product candidates, the value of our ROCK platform, our ongoing and planned preclinical development and clinical trials, our collaborations and development of our products in combination with other therapies, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for our product candidates, our intellectual property position, our collaboration activities, our ability to develop commercial functions, clinical trial data, our results of operations, cash needs, financial condition, liquidity, prospects, future transactions, growth and strategies, the industry in which we operate, the trends that may affect the industry or us, impacts of the COVID-19 pandemic, the benefits to Affimed of orphan drug designation and the risks, uncertainties and other factors described under the heading Risk Factors in Affimeds filings with the SEC. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

Investor Relations Contact Alexander Fudukidis Director, Head of Investor Relations E-Mail: a.fudukidis@affimed.com Tel.: +1 (917) 436-8102

Media Relations Contact Mary Beth Sandin Vice President, Marketing and Communications E-Mail: m.sandin@affimed.com Tel.: +1 (484) 888-8195

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No animals harmed in new chip drug tests that are faster and cheaper – ISRAEL21c

Posted: December 10, 2021 at 2:09 am

Billions of dollars, hundreds of mice and decades of years are sacrificed to research and development for the average pharmaceutical with no guarantee of success. Nothing about that equation makes sense anymore.

And thats why some of the most impressive minds in science are behind Israeli startup Quris,which is rolling out the worlds first clinical-prediction AI (artificial intelligence) platform to evaluate the safety and efficacy of new drugs.

This newly launched, highly scalable automated platform can test thousands of novel drug candidates at once, on hundreds of miniaturized patients-on-a-chip.

Using AI to predict which drug candidates will safely work in humans is expected to improve efficacy and cut drug development costs dramatically.

Israeli Nobel laureate Dr. Aaron Ciechanover and Moderna cofounder Robert S. Langer are actively involved in guiding the science, technology and strategy of Quris, dual-headquartered in Boston and Tel Aviv.

We are at the tipping point of the modernization of drug discovery. I think the Quris platform could be of significant value to pharma companies and the health of society at large, said Langer, one of 12 Institute Professors at MIT.

Saving money and time is the biggest advantage. Eliminating or at least minimizing the use of rodents in drug testing is also important, and not only for animal welfare reasons.

We are not mice, so what works in animal-based trials is not a proper indicator of what will work for people, explained Ciechanover,a physician and research professor at the Technion Israel Institute of Technology in Haifa.

Using a breakthrough way to test drug candidates on miniaturized patients on chips, Quris can demonstrate their safety and efficacy, or lack thereof, through preliminary chip-based clinical trials. This has never been done before, said Ciechanover.

Quris CEO Isaac Bentwich tells ISRAEL21c that AI-driven drug discovery has become the leading frontier for pharma innovation.

However, this wildly expensive technology does not address clinical safety and efficacy. Therefore, most novel drugs still fail clinical trials costing pharma companies more than $30 billion annually.

Quris is the first AI platform to predict which drug candidates will safely work in humans, filling a critical gap in clinical prediction, Bentwich says.

Part of the Quri team in Tel Aviv, with CEO Isaac Bentwich at right. Photo courtesy of Quris

While there are organ-on-chip technologies available to test the effect of drug candidates on, say, the liver, they cannot achieve high throughput.

To generate large amounts of data, you need a platform to perform thousands of experiments, and the current organ-on-a-chip technology does not allow that to happen. Its usually performed one organ at a time, Bentwich explains.

Our chip-on-chip solution does multiple such experiments to train the AI and let the AI define if the drug is safe or not.

Hundreds of genetically diverse patients

Joining together several organs-on-a-chip to build a patient-on-a-chip has proven cumbersome and unscalable, he adds, citing one such device built at Harvard that took up a big box filled with tubes and pipes.

And that was just a single patient-on-a-chip. Our device is two-by-three inches and simulates a hundred patients on a chip by using different architecture and innovations in nanosensing. Its much more scalable and inexpensive, says Bentwich.

Patient-on-a-chip technology will lower the cost and time of drug testing dramatically. Photo courtesy of Quris

Testing to see if a drug is toxic on a single patient-on-a-chip is better than testing it on a mouse. But its not perfect because different people react differently to the same drug, he adds.

Heres where Quris exclusive partnership with the New York Stem Cell Foundation (NYSCF) Research Institutecomes in.

We generate stem cells, and from them different organs, so you have an entire clinical trial on a chip before testing on humans, Bentwich explains.

By testing thousands of drugs known to be safe or unsafe on male and female patients-on-a-chip from different genomic makeups, we are able to train the AI to create representative samples of populations based on many properties.

First clinical trial for Fragile-X drug

Pharma companies can use Quris platform to develop safer drugs faster. At the same time, Quris is using its platform to develop its own pharmaceuticals.

And it made sense to focus on rare genetic diseases that cannot be modeled in mice and arent common enough to attract development dollars.

The first Quris drug addresses Fragile X Syndrome, the most common inherited cause of autism and intellectual disabilities worldwide. This drug candidate was developed initially at the Hebrew University of Jerusalem.

Our Fragile X Syndrome drug is moving into clinical testing in the first half of 2022, says Bentwich, noting that most AI pharma companies do not have drugs at the human clinical testing phase.

Five years to market

This will be a test case to demonstrate how our system can bring a drug to market in five years with millions of dollars, not 20 years with billions, says Bentwich.

We are focusing generally on central nervous system disorders. Brain and liver diseases and cancer are the three areas where safety and efficacy failures of drugs are especially high, he says.

In addition to 18 granted and pending patents, Quris has partnerships with the Technion for various aspects of development, with Tel Aviv University in nanotechnology, and with Hebrew Universitys NewStem drug-discovery spinoff.

The company is doing all this with a lean team of about 20.

Some of the Quris team in Tel Aviv. Photo courtesy of Quris

Its very moving to see extremely talented young guys fresh out of army, not very experienced, working alongside experienced biologists, chemists, nano scientists, micromechanics and robotics experts, using bioconvergenceto solve big problems, says Bentwich.

Dr. Kobi Richter, founder and chief technology officer of Medinol, explained why he participated in Quris recent $9 million seed round.

Our drug discovery process is broken, and technology darlings across biotech, artificial intelligence, machine learning and big data have not been able to overcome the colossal clinical trial failure rate, said.

With a remarkable team of scientific pioneers at the helm and its extraordinary clinical prediction platform, Quris will be a gamechanger for the industry and lead the next era of drug discovery, Richter said.

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Use of CAR T-Cell Therapy in LBCL Could Come Earlier After ASH 2021 – AJMC.com Managed Markets Network

Posted: December 10, 2021 at 2:08 am

Some of the most anticipated presentations at the 63rd Annual American Society of Hematology (ASH) Meeting and Exposition involve phase 3 results for chimeric antigen receptor (CAR) T-cell therapy in second-line treatment.

In 2017, FDA approved the first chimeric antigen receptor (CAR) T-cell therapies, tisangenlecleucel (tisa-cel), sold as Kymriah, and axicabtagene cilocleucel (axi-cel), sold as Yescarta, for patients with B-cell lymphomas treated with at least 2 prior therapies.

Not long after the question arose: would patients have better responses if they received CAR T-cell therapy earlier?

Its a question that will get some answers during the 63rd Annual American Society of Hematology Meeting and Exposition, which takes place December 11-14 in Atlanta and online. Some of the most anticipated presentations at ASH involve phase 3 results for CAR T-cell therapy in second-line treatment, and there are also phase 2 results for axi-cel in the first-line setting coming Monday.

Given the cost of CAR T-cell therapy, which lists for $373,000 to $475,000, depending on the indication, the answer is important to payers, who want to know if these expensive processes are more likely to succeed if patients are treated when their immune systems are less depleted. CAR T-cell therapy works by harnessing the patients own immune system to fight cancer cells.

Theres also the issue of avoiding the cost of prior treatments. However, Peter Marks, MD, PhD, director of FDAs Center for Biologics Evaluation and Research,said during a keynote address in October that widespread use of CAR T-cell therapy at earlier stages might only come if the cost of these treatments came down significantly.

Second-line treatment in LBCL. Two presentations will address this, led by the primary analysis of ZUMA-7, the phase 3 randomized trial that examines the use of axi-cel vs second-line standard of care in relapsed/refractory large B-cell lymphoma (LBCL). Gilead Sciences, which sells axi-cel as Yescarta, announced in June that results showed axi-cel offered a 62% event-free survival benefit over chemotherapy and stem-cell transplant. Results will be presented in Sundays plenary session by Frederick Locke, MD, of Moffitt Cancer Center. Axi-cel is currently approved for LBCL or follicular lymphoma when patients have received at least 2 other therapies.

The race is already on for use of axi-cel in the first-line stage, with phase 2 results from ZUMA-12 to be presented by Sattva S. Neelapu, MD, of MD Anderson Cancer Center.

Getting a jump on ZUMA-7 will be a Saturday morning presentation on interim findings from the phase 3 TRANSFORM study involving lisocabtagene maraleucel (liso-cel), which was approved earlier this year for patients with certain types of LBCL who have received at least 2 treatments. Sold as Breyanzi, liso-cel has a different manufacturing process that investigators say results in less toxicity than earlier CAR T-cell therapies. Officials from Bristol Myers Squibb also announced a significant benefit based on early results; the prespecified interim analysis to be presented at ASH shows event-free survival of 10.1 months, while overall survival data are immature.

Results for tisa-cel in the second-line setting will be presented in the late-breaking session Tuesday, but Novartis announced in August that the BELINDA study did not meet its end point.

Biomarkers and CAR T-cell therapy. Payers will be interested in the effort by 12 academic institutions to track molecular features for 121 patients with diffuse LBCL and assess which biomarkers had effects on clinical outcomes after the patients were later treated with tisa-cel or axi-cel. This study promises to identify possible targetable pathways to bring better responses to CAR T-cell therapy and identify which patients will have the best outcomes.

MCL in real-world settings. FDA approved brexucabtagene autoleucel (brexu-cel) in mantle cell lymphoma (MCL) in July 2020, based on results from the ZUMA-2 trial that showed a 90% objective response rate (ORR) and a 67% complete response rate (CR). A study to be presented by the US Lymphoma Consortium leverages the use of real-world data to show that patients with MCL who fell outside the ZUMA-2 criteria have reported high safety and efficacy rates when treated with brexu-cel.

Beyond R-CHOP. For patients with previously untreated LBCL, there will still be choices beyond CAR T-cell therapy. Results from Tuesday mornings late-breaking abstracts will start with the phase 3 POLARIX study, which examines the use of polatuzumab vedotin with rituximab, cyclophosphamide, doxorubicin, and prednisone, or pola-R-CHP, vs the well-known combination of rituxiumab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) in patients with previously untreated diffuse LBCL.

A discussion, Novel Findings in CAR T-Cell Therapies for Hematologic Malignancies, with a live question and answer session, will take place Monday from 4:30 to 5:15 pm. The session chair will be Marcela V. Maus, MD, Massachusetts General Hospital and Harvard Medical School; panelists will be Maus; David Baker, PhD, University of Washington; and Marco Ruella, MD, University of Pennsylvania.

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Mesoblast has long been the one poster child for stem cell therapy. Now Cynata and other ASX stocks have e … – Stockhead

Posted: December 10, 2021 at 2:08 am

Stem cell therapy, sometimes called regenerative medicine, is one of the most exciting areas of the life sciences sector right now.

Since the pandemic, the sector has emerged into the publics spotlight with new developments in mRNA-based vaccines and therapies.

Nasdaq is the obvious breeding ground for world-class stem cell companies with the likes of Moderna and BioNTech, and lesser known names like Anavex and Enochian.

In Australia, Mesoblast (ASX:MSB) has long been the local poster child for the regenerative medicine industry.

Mesoblast has developed a platform of innovative cellular medicines, but the company has struggled since the FDA rejected its drug in October last year.

Now, other ASX companies like Cynata Therapeutics (ASX:CYP)are making rapid progress to take over the mantle from MSB in this hot field.

Cynata is developing a mesenchymal stem cells (or MSC) technology, which it says has huge therapeutic potential for numerous unmet medical needs.

This includes asthma, heart attack, sepsis, and acute respiratory distress syndrome (ARDS), which all add up to a market opportunity worth $46bn, says the company.

According to CEO Dr Ross Macdonald, who spoke to Stockhead today, MSC is the hottest segment of stem cell therapy at the moment, and has gained a lot of attention recently.

There is a huge interest, and theres been more than 1000 clinical trials conducted around the world using MSC, Dr Macdonald told Stockhead.

He explains that the humans immune system controls many of the bodys functions responsible for repairing tissue after injury or disease, and defending against invading germs like viruses or bacteria.

And just like an orchestral conductor, MSC seems to be playing a central role in that coordination within our immune system.

We now have a firm understanding of how those cells coordinate the bodys responses, and can use that knowledge to enhance those processes that they control, Dr Macdonald explained.

In short, MSC therapies work by expressing a variety of chemokines and cytokines that aid in repair of degraded tissue, restoration of normal tissue metabolism and, most importantly, counteracting inflammation.

And because MSCs play that co-ordination role within the immune system, they can be used to treat different diseases.

However theres one big problem with cell-based therapies, and its not to do with the safety and efficacy.

Its how to manufacture these products on a mass scale, that is the greatest challenge right now, says Dr Macdonald.

Unlike aspirin where it can be synthesised in a chemical lab and produced in bulk, manufacturing a living drug like a cell is a whole lot more complicated.

But that big challenge is the exact area of strength and competitive advantage that Cynata has, Dr Macdonald told Stockhead.

He says Cynata has a technology platform which allows it to manufacture essentially limitless quantities of MSCs, consistently and economically.

Dr Macdonald explains there are two approaches to using cell therapy, the autologous and the allogeneic approach.

The autologous approach is where the patient themselves serves as their own donor.

This is obviously bespoke and inefficient, because the drug can only be manufactured for that one patient, and is obviously not an industrialised process, he said.

But by taking an allogeneic approach, Cynata has the ability to start with a one time donation of cells from one single donor.

Well never have to go back to that human donor ever again, so our process of producing cells has become a very much more typical industrialised process.

The company has a patent for this, with two clinical trials underway and two more under preparation.

A Phase 3 clinical trial for osteoarthritis which is funded by a NHMRC grant has progressed the furthest, while a Phase 2 trial in COVID-19 is ongoing.

Meanwhile a Phase 1 study in GvHD, which was published in prestigious journal Nature Medicine, is probably the closest to commercialisation according to Dr Macdonald.

GvHD is a challenging disease which occurs in patients who have had a bone marrow transplant as part of their chemotherapy treatment for cancer.

Chemo is still very much a sledgehammer therapy where you use very toxic drugs that do kill the cancer cells, but they also kill the surrounding healthy cells that grow hair and bone marrow.

Unfortunately for many patients, the bone marrow transplant reacts against their body and starts to attack all of the tissues in the body, and its ultimately fatal.

Its a horrible death, destroying the lungs, liver, intestines and the skin, Macdonald explains.

Cynatas MSC therapy has been shown to reset that reaction, so the patient can recover from the GvHD, and also recover from their underlying cancer.

With all these clinical trials concurrently under way, Macdonald believes there is a clear significant upside potential for Cynata, particularly given its small market cap of $70m compared to other similar plays like Mesoblast ($1 billion market cap).

Osteopore (ASX:OSX) focuses in bones and specialises in the production of 3D printed bioresorbable implants that are used in surgical procedures to assist with the natural stages of bone healing.

The 3D bio-printer makes a scaffold that mimics bone, with a patented micro-architecture which traps the patients own stem cells.

Orthocell (ASX:OCC) develops collagen medical devices and cellular therapies for the repair and regeneration of human tendons, bone, nerve and cartilage defects.

Its flagship product, the CelGro, is a naturally derived collagen medical device for tissue repair.

Aroa Biosurgery (ASX:ARX) develops FDA-approved medical devices for wounds and tissue repair using its extracellular matrix (ECM) technology, mainly in the United States.

Recent study shows 100% success rates from the use of its Myriad product when patients underwent surgical reconstruction of exposed vital structures such as bone and tendon.

Regeneus (ASX:RGS) Progenza is a cellular therapy targeting pain and inflammation which uses Secretome to improve not only the resident tissue, but the MSCs themselves.

It fills a gap in the current treatment market for osteoarthritis, by providing disease modification and pain relief to address patient symptoms.

Anteris Technologies (ASX:AVR) claims that its Adapt Technology is the first and only bio-scaffold technology that completely re-engineers xenograft tissue into a pure collagen scaffold.

A recent study indicated that Adapt-treated tissue has superior anti-calcification attributes compared with tissues used in competitor valves.

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