Grady Smith, 10, and Southfield resident Jessica Carroll were able to meet for the first time via Zoom in September at the DKMS Gala. Carroll was the bone marrow donor that helped save the boys life.

Photo provided by the Smith family

SOUTHFIELD If you were to take one look at 10-year-old Grady Smith, youd see a young boy who enjoys sports and school.

But the young Salem, New Hampshire, boy has been through more in 10 years than some people have in 50.

Grady was diagnosed with adrenoleukodystrophy, or ALD, back in 2018.

According to Boston Childrens Hospital, ALD is a rare genetic condition that causes the buildup of very long chain fatty acids in the brain. When the fatty acids accumulate, they destroy the protective myelin sheath around nerve cells, responsible for brain function. Without the myelin sheath, the nerves can no longer relay information to and from the brain.

Every single thing I read said, terminal, slow deterioration to death, one to five years, horrible, horrible death, Jillian Smith said. We just died. I havent been the same person since that day. It just changes you for the rest of your life.

With a diagnosis, Grady and his parents looked for options on how to help. Grady had a lesion with a Loes score which is a way of rating severity of 10. Scores range from 0-34.

His parents werent sure Grady would qualify for a bone marrow transplant because they usually only perform transplants for boys with scores of 9 and under. Grady was in luck, however, as Boston Childrens Hospital decided to move forward anyway.

The next move was to find a match for the boy, but that process could take weeks, months or even years. In Gradys case, it took just a few weeks.

Southfield resident Jessica Carroll registered as a potential bone marrow/blood stem cell donor with DKMS, a German bone marrow donor file, in 2014, but she didnt think much would come of it.

Four years later she got a call from the nonprofit organization letting her know that she was a match for a young boy. After some research, Carroll was totally on board with donating.

It was great knowing during that donation that this little bit that I went through was potentially saving somebodys life, Carroll said. Thats all I really cared about, was that I was helping somebody.

Grady was able to get his transplant in 2018.

According to his mother, Grady hasnt had any progression and has even made some recovery. Hes back in line with his academics and is playing sports again.

Grady has auditory processing issues, which make it hard for him to comprehend language and sound. His mother said he relies on reading lips to communicate.

Theres still a lot to it. It stopped the monster thats how we look at it but its not just so cut and dry, Jillian said. Hes a very rare outcome with his Loes score and with just how well hes doing. Hes just a really, really good boy. He works really hard to help bring awareness.

Carroll and the Smiths have talked via text, and they were able to meet virtually for the first time in September at the DKMS Gala.

For the Smiths and Carroll, the meeting was emotional. Grady was finally able to put a face to his donor, and vice versa for Carroll.

It was of course emotional, Carroll said. Being able to hear everything they went through, though, definitely made me so happy that I had chosen to register.

The Smiths and Carroll still talk periodically throughout the year, and Sept. 20 was the two-year anniversary of the transplant.

They are hoping to be able to meet in person soon, and the DKMS team wants to bring them to next years gala to help make that happen. However, they hope it will be sooner.

Throughout this journey with Grady, the Smiths have advocated, learned and spoken more about ALD.

Prior to Gradys birth, Massachusetts wasnt testing for ALD in newborns, but it has since started. New Hampshire wasnt either, but the Smiths got the state to add ALD to the newborn screening panel.

The next goal is to get more states to add the ALD screening. The family has also spoken at conferences to share Gradys story and have become big proponents of what DKMS has been able to do for not only their family, but families around the world.

I think a big thing, too, that we really want to get out there is bone marrow transplant or stem cell transplant, how easy it is, Jillian said. All people need to do is go on DKMSs website, and they can get a packet sent out to them. They just swab their cheeks, send it in and they could be saving anyones life, someone just like Grady.

According to the DKMS website, the organization is dedicated to the fight against blood cancer and blood disorders by creating awareness, recruiting bone marrow donors to provide a second chance at life, raising funds to match donor registration costs and supporting the improvement of blood cancer therapies by our own research.

Those looking for more information or wanting to register can visit dkms.org/en or call (212) 209-6700.

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Southfield woman meets boy she saved with bone marrow donation - C&G Newspapers

Emmanuelle Charpentier and Jennifer Doudna have scooped the 2020 Nobel prize in chemistry for the development of a method for genome editing. Specifically, theyve been awarded the prize for their discovery of the CrisprCas9 genome editing technique that allows scientists to make precise alterations to the genetic code of living organisms. CrisprCas9 is a powerful tool that could revolutionise many aspects of our lives, from medical treatments to the way we produce food. Its also seen its fair share of controversy in recent years. Here, we take a deeper look at these genetic scissors and why theyve won the Nobel prize.

Since Charpentier and Doudna began investigating the CrisprCas9 system in 2011, the field has exploded. Due to the relative simplicity and affordability of Crispr systems, researchers around the world have been able to apply the tools to all manner of different problems. Today there are entire journals, conferences and companies dedicated to the technique.

The ability to cut any DNA molecule at a chosen site has huge potential from treating genetic illnesses to creating disease-resistant crops. Trials have even shown how Crispr-delivered genetic modifications can spread through populations of mosquitoes and stop malaria infections such gene-drives offer a way to eliminate the disease altogether. And in the face of the Covid-19 pandemic, researchers have found ways to use Crispr in rapid coronavirus diagnostic tests and have also proposed using it to attack the viruss genome.

As Claes Gustafsson, chair of the Nobel committee for chemistry, said at the award announcement, There is enormous power in this genetic tool, which affects us all.

Crispr technology has even been used to make more delicious beer.

The whole Crispr gene editing tool has been adapted from the immune system of bacteria. The term Crispr comes from clustered regularly interspaced short palindromic repeats, which refers to distinct genetic sequences found in the genomes of bacteria. Each Crispr sequence is transcribed into RNA sequences that will target the DNA of a virus. These sequences also include cas (Crispr-associated) genes that code for DNA-cutting Cas enzymes. Together, the guide RNA and Cas enzyme form a complex that hunts out viral DNA and chops it up.

In Crispr gene editing, scientists repurpose this system by designing a guide RNA sequence of around 20 nucleobases that matches up to a DNA sequence they wish to target in a cells genome. This RNA sequence is paired with the Cas9 enzyme that will cut the DNA strand at the targeted site. The whole DNA sequence coding for both these components of the Crispr-Cas9 tool can be delivered to the target cell via a plasmid.

The tool can therefore be used to edit a cells genome with incredible precision for example, it can cut out a dysfunctional gene associated with a hereditary illness. And if the healthy version of the gene is also delivered to the cell, the cells own repair system will then incorporate the healthy strands at the site where it has been cleaved.

In 2011, when investigating the bacteria Streptococcus pyogenes, Charpentier discovered a molecule called tracrRNA that forms a key part of the CrisprCas system in bacteria.

Meanwhile, Doudna had been investigated the function of the cas genes, and learned that the Cas proteins they code for are involved in cutting up DNA as part of the bacterial immune system against pathogenic viruses.

That year Charpentier teamed up with Doudna to investigate the system further. Together they revealed how the Cas9 protein, CrisprRNA and tracrRNA worked together to snip DNA strands into two parts. They then simplified the system by combining the CrisprRNA and tracrRNA into a single molecule guide RNA making it easier to use, and showed how this could be used to cut any DNA strand at a site of their choosing, opening the door to using the tool in all manner of genome editing experiments.

While previous tools for genetic editing existed before Crispr-Cas9, the new tools are much simpler and cheaper. This has led to the huge expansion of the field by making gene editing accessible for scientists all around the globe.

For years Crispr has been at the centre of a long-running patent dispute. Shortly after Doudna and Charpentiers discovery, Feng Zhangs team the Broad Institute in Cambridge, US, patented a way to use the technique in eukaryotic cells. There have been protracted court battles between Doudnas group at the University of California in Berkeley, US, and the Broad team over who holds the key piece of intellectual property. In the meantime, numerous groups and companies have been granted patents for many new Crispr-related technologies, meaning that as time goes on, the original patents at the centre of the dispute are becoming less relevant.

Another area of controversy surrounds the potential consequences of using genome editing tools at all. As the genome is so complex, we cant always know what will happen when we edit genes. Some genes have multiple and often unknown functions editing them to correct for one problem could end up creating new unforeseen ones. This is particularly important when it comes to editing germline cells (those that can be passed on to an organisms children), because the modified genes can be inherited by future generations.

As a relatively new technique, we also know that Crispr itself isnt perfect. Some studies have shown off-target cuts, where the tool has snipped DNA strands at additional locations to the desired site. This clearly can have harmful consequences, and so many researchers are looking into ways to improve the technique and make it more suitable for medical uses.

With these concerns in mind, scientists worldwide including Doudna and Charpentier have called for a moratorium on editing human germline cells, until we can know more about the consequences. Such calls intensified after the rogue Chinese scientist He Jiankui edited human embryos that were then brought to term in 2018. He is now serving a three year prison sentence for conducting the study.

Several clinical trials have already begun on Crispr-based therapies, with promising reports emerging this year. In February, the first study to look at a cancer treatment using Crispr-edited immune cells reported that the modified cells were safe, with no serious side-effects in the three patients studied. While the efficacy of the treatment on the cancers was minimal, it may help to inform future Crispr-based T-cell treatments.

One month later, a patient with hereditary blindness became the first person ever to have a CrisprCas9 therapy directly administered into their body. And in June, the Swiss gene-editing company Crispr Therapeutics announced that two patients with beta thalassaemia and one with sickle cell disease would no longer require blood transfusions after their bone marrow stem cells were edited using Crispr techniques.

Earlier this week, Doudna launched a new company, Scribe Therapeutics, to begin work on treatments for amyotrophic lateral sclerosis.

Other Crispr-based technologies are coming closer to commercial reality. For example, the US genome engineering company eGenesis is developing ways to use the technique to edit pigs genes so that their organs might be transplanted safely into humans. In the agricultural sector, many companies are working on ways to use Crispr to speed up the selection process for crops with desirable traits such as disease-resistance or improved flavour.

At the fundamental level, researchers are working on ways to improve the system itself. By using alternative Cas proteins, some groups hope to make the tool more effective and easier to use in certain settings. Doudnas group recently reported on a CasX protein that is smaller than Cas9 and potentially easier to introduce into target cells.

Delivering DNA into cells and tissues is an important part of gene therapy, even more so for Crispr-Cas9 approaches because plasmids carrying this system are very large. This research paper describes a non-viral vector for delivering plasmid DNA carrying Crispr-Cas9 into tumour spheroids, which are good in vitro models for tissues but also challenging transfecting targets.

1 S J Zamolo, T Darbre and J-L Reymond, Transfecting tissue models with CRISPR/Cas9 plasmid DNA using peptide dendrimers, Chem. Commun., 2020, DOI: 10.1039/d0cc04750c

Regulating the function of Crispr-Cas9 is on the agenda for many researchers because the ability to restrict it in a spatial and temporal manner opens the door to precisely manipulating genomes and minimising any side effects. By introducing photolabile groups into the system, these researchers have shown how they can regulate Cas9 activity with light

2 Y Wang et al, Photocontrol of CRISPR/Cas9 function by site-specific chemical modification of guide RNA,Chem. Sci., 2020, DOI: 10.1039/d0sc04343e

It seems that Crispr-Cas systems arent just handy for gene editing. This paper describes how the Crispr-Cas system was used to assemble a multi-enzyme cascade containing five distinct enzymes. The team behind the work hope it could be the beginnings of a general method for building complex scaffolded biocatalytic pathways

3 S Lim et al, CRISPR/Cas-directed programmable assembly of multi-enzyme complexes, Chem. Commun., 2020, 56, 4950 (DOI: 10.1039/d0cc01174f)

And to round things off, here are some reviews on how Crispr-Cas9 works, the delivery processes for therapeutic nanoparticles and the physiological obstacles for those process

4 Y Xu, R Liu and Z Dai, Key considerations in designing CRISPR/Cas9-carrying nanoparticles for therapeutic genome editing, Nanoscale, 2020, DOI: 10.1039/d0nr05452f

5 Y Gong et al,Lipid and polymer mediated CRISPR/Cas9 gene editing, J. Mater. Chem. B, 2020,8, 4369 (DOI: 10.1039/d0tb00207k)

Excerpt from:
Explainer: What is Crispr and why did it win the Nobel prize? - Chemistry World

Autologous Stem Cell Based Therapies Market Report Delivering Growth Analysis with Key Trends of Top Companies (2020-2026)

A comprehensive research study on the Autologous Stem Cell Based Therapies Marketwas recently published by Market Report Expert. This is an up-to-date report, covering the current COVID-19 impact on the market. The Coronavirus (COVID-19) has affected every aspect of life globally and thus altering the global market scenario. The changes in the market conditions are drastic. The swiftly changing market scenario and initial and future assessment of the impact on Autologous Stem Cell Based Therapies market is covered in the report.The Autologous Stem Cell Based Therapies Market report is a precise and deep-dive study on the current state that aims at the major drivers, market strategies, and imposing growth of the key players. Worldwide Autologous Stem Cell Based Therapies Industry also offers a granular study of the dynamics, segmentation, revenue, share forecasts, and allows you to make superior business decisions. The report serves imperative statistics on the market stature of the prominent manufacturers and is an important source of guidance and advice for companies and individuals involved in the Autologous Stem Cell Based Therapies industry.

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Regeneus, Mesoblast, Pluristem Therapeutics Inc, U.S. STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe

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Majortype, primarily split into

Embryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Major applications/end users, including

Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

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Information related to the growth rate, revenue, sales, production, consumption, during the forecast period is included in the report. The Autologous Stem Cell Based Therapies Market report claims that the industry is projected to generate significant revenue and sales during the forecast period. The report consists of information related to the market dynamics such as challenges involved in this vertical, growth opportunities, and driving factors affecting the market.

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Autologous Stem Cell Based Therapies Market Size, Business Revenue Forecast, Leading Competitors And Growth Trends 2026| Regeneus, Mesoblast,...

CRANFORD, N.J., Oct. 7, 2020 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company developing and commercializing critical care drug products, announced that it has signed an exclusive agreement with Novellus Therapeutics Limited ("Novellus") to license iPSC-derived mesenchymal stem cells (iMSCs), and has created a new subsidiary, NoveCite, that will be focused on developing cellular therapies.

NoveCite has a worldwide exclusive license from Novellus, an engineered cellular medicines company, to develop and commercialize NoveCite mesenchymal stem cells ("NC-iMSCs") to treat acute respiratory conditions with a near term focus on Acute Respiratory Distress Syndrome ("ARDS") associated with COVID-19. Several cell therapy companies using donor-derived MSC therapies in treating ARDS have demonstrated that MSCs reduce inflammation, enhance clearance of pathogens and stimulate tissue repair in the lungs. Almost all these positive results are from early clinical trials or under the emergency authorization program.

NC-iMSCs are the next generation mesenchymal stem cell therapy. They are believed to be differentiated and superior to donor-derived MSCs. Human donor-derived MSCs are sourced from human bone marrow, adipose tissue, placenta, umbilical tissue, etc. and have significant challenges (e.g., variable donor and tissue sources, limited supply, low potency, inefficient and expensive manufacturing). iMSCs overcome these challenges because they:

Globally, there are 3 million cases of ARDS every year out of which approximately 200,000 cases are in the United States. The COVID-19 pandemic has added significantly to the number of ARDS cases. Once the COVID patients advance to ARDS, they are put on mechanical ventilators. Death rate among patients on ventilators can be as high as 50% depending on associated co-morbidities. There are no approved treatments for ARDS, and the current standard of care only attempts to provide symptomatic relief.

"NoveCite iMSCs have the potential to be a breakthrough in the field of cellular therapy for acute respiratory conditions because of the high potency seen in Novellus' pre-clinical studies, and because iMSCs are iPSC-derived, and therefore overcome the manufacturing challenges associated with donor derived cells," said Myron Holubiak, Chief Executive Officer of Citius.

"We are excited to be part of this effort because of the promise to save lives and reduce long term sequelae in patients with devastating respiratory diseases such as ARDS caused by COVID-19," said Dr. Matthew Angel, Chief Science Officer of Novellus. "Our iMSC technology has multimodal immunomodulatory mechanisms of action that make it potentially promising therapy to treat acute respiratory diseases."

About Citius Pharmaceuticals, Inc.

Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Therapeutics, Limited

Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its patented non-immunogenic mRNA high specificity gene editing, mutation-free & footprint-free cell reprogramming and serum insensitive mRNA lipid delivery technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

About NoveCite iMSC (NC-iMSC)

NoveCite's mesenchymal stem cell therapy product is derived from a human induced pluripotent stem cell (iPSC) line generated using a proprietary mRNA-based (non-viral) reprogramming process. The NC-iMSCs produced from this clonal technique are differentiated from human donor-derived MSCs (bone marrow, placenta, umbilical cord, adipose tissue, or dental pulp) by providing genetic homogeneity. In in-vitro studies, NC-iMSCs exhibit superior potency and high cell viability. NC-iMSCs secrete immunomodulatory proteins that may reduce or prevent pulmonary symptoms associated with acute respiratory distress syndrome (ARDS) in patients with COVID-19. NC-iMSC is an allogeneic (unrelated donor) mesenchymal stem-cell product manufactured by expanding material from a master cell bank.

First generation (human donor-derived) MSCs are isolated from donated tissue followed by "culture expansion". Since only a relatively small number of cells are isolated from each donation, first generation MSCs are increased by growing the cells in culture. Unfortunately, these type of MSCs start to lose potency, and ultimately become senescent. Each donation produces a limited number of MSCs, so a continuous supply of new donors is needed to produce commercial scale. The number and quality of MSCs that can be isolated from different donors can vary substantially.

About Acute Respiratory Distress Syndrome (ARDS)

ARDS is an inflammatory process leading to build-up of fluid in the lungs and respiratory failure. It can occur due to infection, trauma and inhalation of noxious substances. ARDS accounts for approximately 10% of all ICU admissions and almost 25% of patients requiring mechanical ventilation. Survivors of ARDS are often left with severe long-term illness and disability. ARDS is a frequent complication of patients with COVID-19. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

Safe Harbor

This press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risks associated with developing the NoveCite technology as a treatment for ARDS; risks associated with developing any of our product candidates, including any licensed from Novellus, Inc., including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; our need for substantial additional funds; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; the early stage of products under development, including the NoveCite technology; our ability to obtain, perform under and maintain licensing, financing and strategic agreements and relationships; our ability to attract, integrate, and retain key personnel; risks related to our growth strategy; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677 x105(M) 646-522-8410ascott@citiuspharma.com

View original content:http://www.prnewswire.com/news-releases/citius-pharmaceuticals-signs-an-exclusive-worldwide-licensing-agreement-with-novellus-therapeutics-for-unique-imsc-therapy-for-acute-inflammatory-respiratory-conditions-including-covid-19-related-acute-respiratory-distress-syndrom-301147409.html

SOURCE Citius Pharmaceuticals, Inc.

Company Codes: NASDAQ-SMALL:CTXR

Original post:
Citius Pharmaceuticals Signs an Exclusive Worldwide Licensing Agreement with Novellus Therapeutics for Unique iMSC-Therapy for Acute Inflammatory...

Despite the challenges brought about by Covid-19, leading cosmetic manufacturers, including Mibelle Biochemistry, Cargill Beauty, Givaudan Active Beauty and Lucas Meyer, put forward their most creative innovations for consideration by the in-cosmetics Global Awards jury.

Judged across three categories - Best Active Ingredient, Best Functional Ingredient and Best Green Ingredient - by some of the industrys foremost experts, the awards recognise the cosmetic ingredient manufacturers who are helping to push the boundaries of innovation, while responding to evolving consumer demands.

The winners were announced yesterday during a virtual ceremony from a list of finalists dominated by skin care ingredients.

Scooping both the Gold and Silver awards in the Best Active Ingredient category, Vytrus Biotech was recognised for its Kannabia Sense and Deobiome Noni ingredients.

Kannabia Sense took the top accolade. This probiotic treatment stimulates the skin microbiota to produce an in-situ postbiotic cocktail that promotes the synthesis of cutaneous oxytocin. Recommended for use in sensitive skin treatments, anti-ageing cosmetic products and delicate facial treatments, it is made from plant stem cells of Cannabis sativa.

Deobiome Noni is a prebiotic deodorant treatment that reduces the generation of body odour while allowing the skin to breathe and respecting the skin microbiota.

Closing the category, Mibelle Biochemistry and Clariant Active were announced as this years joint Bronze Award winners.

Alpine Rose Active, Mibelle Biochemistrys novel senolytics concept, was chosen for its ability to eliminate senescent skin cells and protect skin proteins from oxidative stress.

Clariant Active was applauded for Prenylium. Thanks to Clariants Plant Milking technology, Prenylium is extracted from the root of the mulberry tree (Morus Alba) without causing any damage. This innovative and sustainable approach to sourcing helps to stimulate root growth and results in a concentration of prenylated flavonoids 2000% higher than what is typically found in mulberry root extracts.

German cosmetic manufacturer Symrise took the top award in the Best Functional Ingredient category for its Symrise SymEffect Sun. Based entirely on renewable raw materials from responsible sources, the new ingredient combines the advantages of conventional sunscreen products with the increasing sustainability requirements of consumers.

Also demonstrating a sustainable approach to sun protection, Dow Chemical was awarded the Silver award for its SunSpheres BIO SPF Booster, a bio-based and readily biodegradable SPF boost that enables greater SPF efficiency in suncare and daily skincare products.

The category also honoured two bronze award winners: Clariant and Gattefoss. The former was awarded for Velsan Flex which, thanks to its high water solubility and Renewable Carbon Index (RCI) of 93%, offers preservation boosting powers together with broad formulation flexibility. Gattefosss Emulium Dolcea MB a natural origin O/W emulsifier - has the ability to create a wider range of textures, from fluid serum to thick butter in a wide range of applications, such as skincare, suncare, make up and haircare.

As sustainability continues to drive cosmetic manufacturers to assess the ingredients used in their formulations, this years Green Ingredient Award - in partnership with Ecovia Intelligence - recognises the ingredients championing sustainability in the personal care and beauty market.

Henry Lamotte Oils Paradise Nut Oil/Magdalena River Nut Oil scooped the Gold Award for its ability to support the regeneration of the skins lipid film, while improving the water retention capability of the skin, reducing trans-epidermal water loss.

Unveiled as the Silver Award winner, Cargill Beautys FiberDesign Sensation was praised for its approach to sustainability and upcycling. Derived from 100% natural origins, the texturiser and emulsion stabiliser, designed specifically for skincare, is based on citrus peel fibres from the pectin production side stream.

Joining the line-up of award winners, Minasolve SAS scooped the Bronze Award for its A-Leen Aroma-3, a nature-derived perfuming agent that also offers a broad-spectrum antimicrobial effect. It is a 100% natural version of phenylpropanol, it is produced from cassia essential oil.

Cosmetic Ingredients - October 2020

For more information on the latest cosmetic ingredients launches, also read our special issue:

How far will clean beauty go?

Interest in upcycled ingredients expected to rise in the cosmetics industry

Driven by new consumer preferences, demand for cupuau butter is on the rise

in-cosmetics Awards 2020

Ingredient news: Akott, BASF, Berkem, Clariant, Codif, Covestro, DSM, Firmenich, Givaudaun, Grolman, Imerys, Inter Actifs, Lubrizol, Mibelle, Sederma, Seppic, Silab, Symrise

Read online for free or download the pdf version here.

Read more here:
Who are the winners of the in-cosmetics Global Awards 2020? - Premium beauty

Essentially, graying hair is just natural, whether you have a full head of silver or patches of gray throughout. Loss of pigmentation in hair is something out of our control, agrees celebrity colorist and Redken brand ambassadorMatt Rez. Its largely due to genetics and aging in general, and unfortunately there arent any existing treatments to reverse those grays to their natural vigor.

Specifically, your pigment-producing cells (called melanocytes) start to deteriorate, leading to sprouted grays. The melanocytes are pigment producing cells located near the bulb of the hair and their stem cells, explains hair transplant surgeon James S. Calder, M.D., medical director of Ziering Medical. When your hair ages, those melanocytes dont function as well or start to migrate away from the hair bulb, which causes a sprinkling of grays. And according to Calder, no supplement, nor diet, can successfully prevent the process forever.

Read more:
Can You Prevent Gray Hair? The Answer Isn't Black & White, Experts Say - mindbodygreen.com

MINNEAPOLIS, Oct. 6, 2020 /PRNewswire/ --The National Marrow Donor Program (NMDP)/Be The Match today announced a collaboration with the Minnesota health system M Health Fairview and Marcus Center for Cellular Cures (MC3)/Carolinas Cord Blood Bank at Duke University (Duke) to offer cryopreservation services to transplant centers through the Be The Match BioBank. The collaboration brings together industry-leading expertise in cryopreservation and storage of patient-directed donor blood stem cell products to improve donor availability, collection quality, and ultimately, to provide a more reliable path to transplant for patients.

Through the Be The Match BioBank, blood stem cell donors will be able to donate bone marrow or peripheral blood stem cells (PBSC) for an intended patient on a timeline that is convenient for the donor. The cells are then cryopreserved and stored for the transplant center at no cost to them and shipped to coincide with initiation of the patient's conditioning regimen and optimal treatment timeline.

"We're excited to expand our partnership with Duke University by adding the expertise of physicians and researchers at M Health Fairview University of Minnesota Medical Center to continue to overcome logistical barriers to blood and marrow transplantation that might otherwise disrupt optimal patient care. Through the flexibility offered by the Be The Match BioBank, we believe we can provide transplant centers with a well-matched, available donor more often, and allow the transplant to occur at the best time for the patient," explained Steven Devine, MD, Chief Medical Officer, NMDP/Be The Match, and Associate Scientific Director, CIBMTR (Center for International Blood and Marrow Transplant Research). "The team at the Duke University lab was instrumental in the development of the Be The Match BioBank, as well as supporting donor product cryopreservation during the COVID-19 pandemic to ensure patients can continue to receive the transplants they need."

"We are proud to extend our partnership with the NMDP/Be The Match in a new way. Be The Match BioBank is an innovative way to remove barriers that otherwise may stand in the way of a patient's transplant," said Joanne Kurtzberg, MD, who leads the Marcus Center for Cellular Cures (MC3)/Carolinas Cord Blood Bank at Duke University.

"We are thrilled to be working with the NMDP/Be The Match to offer Be The Match BioBank. Through this partnership, transplant physicians can have confidence a high-quality bone marrow or PBSC product will be available from the donor they requested in the timeframe that works best for their patient," said David McKenna, MD, who leads the Molecular and Cellular Therapeutics program at M Health Fairview.

Be The Match BioBank can be used by any transplant center in the NMDP/Be The Match Network of more than 180 transplant centers worldwide. Blood stem cell donors are informed that the transplant center is requesting cryopreservation and provide consent prior to collection. Donors can also consent to having their donated cells made available to other searching patients in the unlikely event the intended patient is unable to proceed to transplant as planned.

To learn more about Be The Match BioBank, visit Network.BeTheMatchClinical.org/BioBank.

About the National Marrow Donor Program/Be The Match The National Marrow Donor Program/Be The Match is the global leader in providing a cure to patients with life-threatening blood and marrow cancers like leukemia and lymphoma, as well as other diseases. The organization manages the world's largest registry of potential blood stem cell donors and cord blood units. The NMDP/Be The Match partners with a global network to connect patients to their donor match for a transplant, and provides education and support for patients. Through Be The Match BioTherapies, the NMDP/Be The Match partners with cell and gene therapy companies to support the development and delivery of new therapies. The organization conducts research through its research program, CIBMTR (Center for International Blood and Marrow Transplant Research), in collaboration with Medical College of Wisconsin.

About M Health Fairview M Health Fairview is the newly expanded collaboration betweenthe University of Minnesota, University of Minnesota Physicians,and Fairview Health Services. The healthcare system combines the best of academic and community medicine expanding access to world-class, breakthrough care through its 10 hospitals and 60 clinics.

SOURCE Be The Match

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NMDP/Be The Match partners with M Health Fairview and Duke University cryopreservation labs to launch Be The Match BioBank - PRNewswire

By Dylan Adams News Editor

Timothy Ray Brown, the first known person to be cured of HIV, died on Sept. 29 at age 54 after battling cancer.

Timothy Ray Brown, a figurehead in the AIDS and HIV community, passed away surrounded by friends after a five-month battle with leukemia, stated Tim Hoeffgen, Browns partner.

Brown received a positive HIV diagnosis in 1995 while studying in Berlin.

In 2006, Brown was diagnosed with acute myeloid leukemia, which is a cancer that builds in the bone marrow and blood interfering with blood cell production. After bouts of infections from several rough rounds of chemotherapy, Browns leukemia came out of remission.

Due to leukemia in his bones, Brown required a stem cell transplant, a process that allows healthy stem cells to be introduced into a host to stimulate the immune system and healthy bone marrow growth. At the time, the survival rates for stem cell transplant were around fifty percent.

Doctors found a match to Browns genetic type, a donor with the CCR5 Delta 32 mutation, a protein that acts as a doorway to stop the HIV from infecting new cells. Three months after Brown stopped taking his HIV medication, doctors found he no longer had HIV in his blood.

After another round of stem cell treatment in February of 2008, Brown went through several near-death complications, almost going blind and becoming paralyzed but slowly recovering. His body was still successfully fighting off HIV.

In July 2012, the Timothy Ray Brown Foundation was created during the World AIDS Conference in Washington, DC. This foundation was built for Brown to show his support and work with medical institutions and scientists to develop a unifying cure and vaccination against HIV.

Brown would often donate large amounts of blood and tissue samples to researchers in the hope of progressing closer towards an HIV cure. According to his partner, Hoeffgen, Tims lifework was to tell his story about his HIV cure and become an ambassador of hope to those in need.

Doctors have since used Brown as a blueprint to work on a potential cure and vaccine for HIV. Most notably for the second person to ever be cured of HIV the London Patient, Adam Castillejo who went through similar stem cell transplants in 2019 before coming forward to the public.

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Gosselies, Belgium, 5 October 2020, 7am CEST BONE THERAPEUTICS(Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, Link Health Pharma Co., Ltd (Link Health) and Shenzhen Pregene Biopharma Company, Ltd (Pregene) today announce the signing of an exclusive license agreement for the manufacturing, clinical development and commercialization of Bone Therapeutics allogeneic, off-the-shelf, bone cell therapy platform ALLOB in China (including Hong Kong and Macau), Taiwan, Singapore, South Korea, and Thailand.

Under the agreement, Bone Therapeutics is eligible to receive up to 55 million in development, regulatory and commercial milestone payments including 10 million in upfront and milestone payments anticipated in the next 24 months. Bone Therapeutics is also entitled to receive tiered double-digit royalties on annual net sales of ALLOB. Bone Therapeutics retains development and commercialization rights to ALLOB in all other geographies outside of those covered by this agreement. As a result, Bone Therapeutics will continue to concentrate on its development and commercialization plans for ALLOB in the US and Europe and novel innovative cell-based products globally.

This collaboration between Bone Therapeutics, Link Health and Pregene expands our geographic reach and demonstrates the global commercial potential of ALLOB,said Miguel Forte, MD, PhD, Chief Executive Officer of Bone Therapeutics. We already have operational experience in Asia with the Phase III clinical trial of our lead product JTA-004 in Hong Kong. We selected Link Health and Pregene to partner with us in Asia as a result of their expertise in advanced therapeutics and cell therapies, their proven track record of development and commercial implementation in Chinese and Asian markets, and Pregenes well established cell therapy manufacturing capacity. Bone Therapeutics will continue to develop the ALLOB cell therapy platform for other markets while exploring additional partnership opportunities in the U.S. and Europe.

The agreement grants Link Health and Pregene exclusive rights to clinically develop and commercialize ALLOB for the treatment of human bone disorders in Greater China, Taiwan, Singapore, South Korea, and Thailand. All rights for China will be transferred to Pregene and Link Health will gain rights for the remaining countries Bone Therapeutics will share its patented proprietary manufacturing expertise for the expansion and differentiation of bone-forming cells and has the option to sell clinical supplies to Link Health and Pregene in preparation for their clinical development of ALLOB.

This collaboration and license agreement for Bone Therapeutics ALLOB provides a strong addition to our pipeline. ALLOB has demonstrated the potential to reduce the recovery time and stimulate bone growth for a variety of bone conditions, and to have a considerable impact on patients lives,said Yan Song, PhD, Chief Executive Officer of Link Health. It is important for Link Health to collaborate with companies that have strong therapeutic product portfolios and entrepreneurial management. This partnership with Bone Therapeutics is a direct result of our shared commitment to appreciate the enormous potential of cell therapy and regenerative medicine.

Pregene now has a flourishing portfolio of CAR-T cell therapy-based cancer treatments. Bone Therapeutics ALLOB provides anallogeneic, off-the-shelf cell therapy that expands our portfolio of cell therapies to include the sizable commercial potential of orthopedics,said Hongjian Li, Co-founder and Chief Executive Officer of Pregene. We expect to be able to leverage our extensive international cell and gene therapy experience to develop Bone Therapeutics ALLOB platform and subsequently launch products in China and Southeast Asian markets.

ALLOB, an allogeneic and off-the-shelf cell therapy product manufactured through a proprietary, scalable production process, consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors. In preclinical studies ALLOB has shown to reduce healing time in a delayed-union fracture model by half, and has demonstrated good tolerability and signs of efficacy in two Phase IIa studies for two separate indications. The Companys randomized, placebo-controlled, double-blind Phase IIb clinical trial in patients with difficult tibial fractures has received approval from regulatory authorities in six of the seven planned European countries to date, and is expected to enroll the first patient later this year.

About Link Health Pharma Co., Ltd

Link Health is a leading Chinese pharmaceutical company based in Guangzhou, Southern China, focusing on the development of innovative drugs for unmet medical needs.

Link Health has created a highly professional team with diverse expertise in drug development, medical affairs and regulatory affairs. Leveraging deep understanding of China market, regulatory environment and strong network with global biopharmaceutical companies, Link Health is well positioned to bring innovative drugs to the market efficiently. The company has a drug development pipeline of 5 clinical stage assets and 1 under NDA reviewing in China.

The company has also established a fully owned subsidiary in Amsterdam, the Netherlands. The Dutch office builds and further strengthen collaborations with global pharma/biotech partners and research institutes.

About Pregene Biopharma Co., Ltd

Shenzhen Pregene Biopharma Co. Ltd is a leading enterprise in the cell and gene therapy field with the core technology for industrialization. The companys core team comes from well-known institutions and companies including the Academy of Military Medical Sciences, the University of Toronto, and the US FDA.

Pregene has established the gene editing platform, viral vector and cell production platform, nanobody selection platform and other small to pilot trial manufacturing system, with total investment over 100 million CNY. It has the laboratories and GMP plants for cell and gene therapy of over 10,000 square meter.

The company focuses on the research and development of cell and gene therapy drugs, and participated in the drafting the national standard Considerations for CAR-T Cell Quality Study and Non-clinical Evaluation issued by the National Institutes for Food and Drug Control in June 2018. The CAR-T cell therapy for the treatment of multiple myeloma have obtained NMPA IND clearance as the Class I new drug, which is the first in China and fastest in the world using the humanized single domain antibody in CAR construct, and phase I clinical trials are now in progress. Other pipelines such as CAR-T, TCR-T and mRNA drugs for tumors, autoimmune diseases and other indications are in the development at different stages. The company has broad development prospects with the abundant backup technologies.

Looking forward to the future, the company will build the core capacity in one-stop solution for cell and gene therapy drugs, and fulfill the Express of innovative medicine development from drug discovery to clinical products.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company is ready to start the phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available atwww.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

For US Media and Investor Enquiries:LHA Investor RelationsYvonne BriggsTel: +1 310 691 7100ybriggs@lhai.com

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -...

Hematopoietic Stem Cell Transplantation (HSCT) Market Scenario 2020-2026:

The Global Hematopoietic Stem Cell Transplantation (HSCT) market exhibits comprehensive information that is a valuable source of insightful data for business strategists during the decade 2014-2026. On the basis of historical data, Hematopoietic Stem Cell Transplantation (HSCT) market report provides key segments and their sub-segments, revenue and demand & supply data. Considering technological breakthroughs of the market Hematopoietic Stem Cell Transplantation (HSCT) industry is likely to appear as a commendable platform for emerging Hematopoietic Stem Cell Transplantation (HSCT) market investors.

This Hematopoietic Stem Cell Transplantation (HSCT) Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution, etc., these data help the consumer know about the competitors better.

Get Sample Report: https://grandviewreport.com/sample/63228

The complete value chain and downstream and upstream essentials are scrutinized in this report. Essential trends like globalization, growth progress boost fragmentation regulation & ecological concerns. This Market report covers technical data, manufacturing plants analysis, and raw material sources analysis of Hematopoietic Stem Cell Transplantation (HSCT) Industry as well as explains which product has the highest penetration, their profit margins, and R&D status. The report makes future projections based on the analysis of the subdivision of the market which includes the global market size by product category, end-user application, and various regions.

Topmost Leading Manufacturer Covered in this report:Escape Therapeutics Inc., Cryo-Save AG, Regen Biopharma Inc., CBR Systems Inc., ViaCord Inc., Lonza Group Ltd., Pluristem Therapeutics Inc., China Cord Blood Corp.

Product Segment Analysis: Allogeneic, Autologous

Application Segment Analysis:Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

Get Discount @ https://grandviewreport.com/discount/63228

Regional Analysis For Hematopoietic Stem Cell Transplantation (HSCT)Market

North America(the United States, Canada, and Mexico)Europe(Germany, France, UK, Russia, and Italy)Asia-Pacific(China, Japan, Korea, India, and Southeast Asia)South America(Brazil, Argentina, Colombia, etc.)The Middle East and Africa(Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

Market Synopsis:The market research report consists of extensive primary research, as well as an in-depth analysis of the qualitative and quantitative aspects by various industry specialists and professionals, to gain a deeper insight into the market and the overall landscape.

The objectives of the report are:

To analyze and forecast the market size of Hematopoietic Stem Cell Transplantation (HSCT)Industry in theglobal market. To study the global key players, SWOT analysis, value and global market share for leading players. To determine, explain and forecast the market by type, end use, and region. To analyze the market potential and advantage, opportunity and challenge, restraints and risks of global key regions. To find out significant trends and factors driving or restraining the market growth. To analyze the opportunities in the market for stakeholders by identifying the high growth segments. To critically analyze each submarket in terms of individual growth trend and their contribution to the market. To understand competitive developments such as agreements, expansions, new product launches, and possessions in the market. To strategically outline the key players and comprehensively analyze their growth strategies.

View Full Report @ https://grandviewreport.com/industry-growth/Hematopoietic-Stem-Cell-Transplantation-(HSCT)-Market-63228

At last, the study gives out details about the major challenges that are going to impact market growth. They also report provides comprehensive details about the business opportunities to key stakeholders to grow their business and raise revenues in the precise verticals. The report will aid the companys existing or intend to join in this market to analyze the various aspects of this domain before investing or expanding their business in the Hematopoietic Stem Cell Transplantation (HSCT) markets.

Contact Us:Grand View Report(UK) +44-208-133-9198(APAC) +91-73789-80300Email : [emailprotected]

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Hematopoietic Stem Cell Transplantation (HSCT) Market to eyewitness massive growth by 2026 | Escape Therapeutics Inc., Cryo-Save AG, Regen Biopharma...

LONDON, UK / ACCESSWIRE / October 6, 2020 / Hemogenyx Pharmaceuticals plc (LSE:HEMO), the biopharmaceutical group developing new therapies and treatments for blood diseases, is pleased to announce the following update on its activities.

As previously announced, Hemogenyx Pharmaceuticals' CDX bi-specific antibody has the potential to treat Acute Myeloid Leukemia ("AML") directly as well as to provide a benign conditioning regimen for blood stem cell replacement therapy. The Company has now carried out extensive work developing treatments for AML and has to date obtained encouraging results.

As announced on 20 February 2020, the Company has constructed and successfully tested in vivo Chimeric Antigen Receptor ("CAR") programmed T cells ("HEMO-CAR-T") for the potential treatment of AML. HEMO-CAR was constructed using the Company's proprietary humanized monoclonal antibody against a target on the surface of AML cells.

It was also announced that the Company was engaging in additional engineering of HEMO-CAR-T cells to increase their safety and versatility. The Company has now introduced and successfully in vitro tested a safety switch within the HEMO-CAR. The aim of this safety switch is to modulate the activity of HEMO-CAR-T cells and to turn them into a "controllable drug" - SAFE-HEMO-CAR-T. The purpose of these efforts is to dramatically improve the safety and potential versatility of HEMO-CAR-T cells for the treatment of AML and/or conditioning of bone marrow transplants, as well as a number of additional potential indications.

Following the successful completion of these in vitro tests, in vivo tests of the efficacy of SAFE-HEMO-CAR-T against AML are being conducted using a model of AML established on the background of Advanced peripheral blood Hematopoietic Chimera (ApbHC) - humanized mice developed by Immugenyx, LLC, a subsidiary of Hemogenyx Pharmaceuticals. If these in vivo tests are successful, the Company will discuss its findings with its partners under the Sponsored Research Agreement with the University of Pennsylvania, announced on 11 August 2020, with a view to considering the inclusion of SAFE-HEMO-CAR-T in the program of pre-clinical trials currently underway there.

Dr Vladislav Sandler, Chief Executive Officer, commented, "We are encouraged by this new data which demonstrates our continuing progress in the development of novel treatments for blood cancers such as AML. The development of SAFE-HEMO-CAR-T further expands the Company's pipeline and advances it into a cutting-edge area of cell-based immune therapy. We are excited to have developed another unique product candidate that should, if successful, provide a new and potentially effective treatment for blood cancers for which survival rates are currently very poor."

About AML and CAR-T

AML, the most common type of acute leukemia in adults, has poor survival rates (a five-year survival rate of less than 25% in adults) and is currently treated using chemotherapy, rather than the potentially more benign and effective form of therapy being developed by Hemogenyx Pharmaceuticals. The successful development of the new therapy for AML would have a major impact on treatment and survival rates for the disease.

CAR-T therapy is a treatment in which a patient's own T cells, a type of immune cell, are modified to recognize and kill the patient's cancer cells. The procedure involves: isolating T cells from the patient, modifying the isolated T cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient.

Market Abuse Regulation (MAR) Disclosure

Certain information contained in this announcement would have been deemed inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 until the release of this announcement.

Enquiries:

Hemogenyx Pharmaceuticals plc

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Dr Vladislav Sandler, Chief Executive Officer & Co-Founder

headquarters@hemogenyx.com

Peter Redmond, Director

peter.redmond@hemogenyx.com

SP Angel Corporate Finance LLP

Tel: +44 (0)20 3470 0470

Matthew Johnson, Vadim Alexandre, Soltan Tagiev

Peterhouse Capital Limited

Tel: +44 (0)20 7469 0930

Lucy Williams, Duncan Vasey, Charles Goodfellow

About Hemogenyx Pharmaceuticals plc

Hemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility.

The Company is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development.

For more than 50 years, bone marrow transplantation has been used to save the lives of patients suffering from blood diseases. The risks of toxicity and death that are associated with bone marrow transplantation, however, have meant that the procedure is restricted to use only as a last resort. The Company's technology has the potential to enable many more patients suffering from devastating blood diseases such as leukemia and lymphoma, as well as severe autoimmune diseases such as multiple sclerosis, aplastic anemia and systemic lupus erythematosus (Lupus), to benefit from bone marrow transplantation.

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit http://www.rns.com.

SOURCE: Hemogenyx Pharmaceuticals PLC

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Hemogenyx Pharmaceuticals PLC Announces SAFE-HEMO-CAR-T Effective against AML in vitro - BioSpace

Massachusetts-based AVROBIO announced today that it has entered an exclusive global license agreement, as well as a collaborative research funding agreement, with The University of Manchester. Together, the university and AVROBIO will look into an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome.

The condition, which impacts an estimated one in 100,000 males worldwide, causes complications throughout the body and brain. Children with severe cases typically show symptoms beginning in their toddler years. At the moment, the standard of care is weekly enzyme replacement therapy, but it does not halt progression of the disease or address cognitive issues that may arise.

We believe a lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function. If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures, said Geoff MacKay, AVROBIOs president and CEO. We believe our deep experience with investigational gene therapies for lysosomal disorders will enable us to efficiently move the program through clinical development in collaboration with Prof. Brian Bigger, who has done tremendous work to develop and optimize this investigational gene therapy. Were proud to add this program to our leading lysosomal disorder pipeline and excited about its potential to change the lives of patients and families living with Hunter syndrome.

The investigational gene therapy, titled AVR-RD-05, includes ex vivo transduction of the patients own hematopoietic stem cells with a therapeutic transgene. The transgene is meant to express functional enzymes that the patient needs to maintain cellular health. When reinfused back into the patient, the modified stem cells are designed to engraft in the bone marrow and produce generations of daughter cells, each carrying the transgene.

This is just one company looking toward making an impact in the MPS II realm as of late. REGENXBIO announced at the end of September that it was expanding its RGX-121 program, looking into the treatment of MPS II. RGX-121 is an investigational one-time gene therapy that uses the AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system.

An ongoing Phase I/II study is evaluating a single intracisternal administration of RGX-121 in severe instances of MPS II in patients under the age of five. As of Sept. 16, RGX-121 was reported to be well-tolerated in patients and there were no drug-related serious adverse events.

"MPS II is a serious and debilitating lysosomal disease that affects 1 in 100,000 children, and available treatments are inadequate to treat the neurodegenerative manifestations of the disease, said Terri Klein, President and Chief Executive Officer of the National MPS Society. Initiating a natural history study will increase the understanding of neurocognitive effects and key biomarkers of severe MPS II, and is critical to advancing the development of new treatment options. We are grateful for REGENXBIO's dedication to MPS and commitment to share the learnings from this observational study with the community.

REGENXBIO has also announced that the U.S. Food and Drug Administration cleared an Investigational New Drug application. The company plans on initiating a second Phase I/II multicenter, open-label trial of RGX-121 for the treatment of pediatric patients with severe MPS II between the ages of five and 18.

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AVROBIO and University of Manchester Enter Agreement for MPS II Research - BioSpace

WASHINGTON Heres a look at how Georgias members of Congress voted over the previous week.

Along with its roll call votes this week, the House also passed the Cyber Sense Act (H.R. 360) to require the Secretary of Energy to establish a voluntary Cyber Sense program to test the cybersecurity of products and technologies intended for use in the bulk-power system; the Consumer Product Safety Inspection Enhancement Act (H.R. 8134) to support the Consumer Product Safety Commissions capability to protect consumers from unsafe consumer products; the School-Based Allergies and Asthma Management Program Act (H.R. 2468) to increase the preference given in awarding certain allergies and asthma-related grants to states that require certain public schools to have allergies and asthma management programs; and the Effective Suicide Screening and Assessment in the Emergency Department Act (H.R. 4861) to establish a program to improve the identification, assessment and treatment of patients in the emergency department who are at risk of suicide.

DISCLOSING TIES TO UYGHUR LABOR: The House has passed the Uyghur Forced Labor Disclosure Act (H.R. 6270) sponsored by Rep. Jennifer Wexton, D-Va., to require publicly traded companies to disclose whether they have business ties to Chinas Uyghur Autonomous Region in Xinjiang province. Wexton said the requirement would let investors know of a given companys passive complicity or active exploitation of one of the most pressing and ongoing human rights violations of our lifetime. A bill opponent, Rep. Anthony Gonzalez, R-Ohio, said it wrongly tried to have the Securities and Exchange Commission police human rights violations, a role that would be better handled by the Treasury Department. The vote, on Sept. 30, was 253 yeas to 163 nays.

NAYS: Loudermilk R-GA (11th), Allen R-GA (12th), Scott, Austin R-GA (8th), Collins R-GA (9th), Carter R-GA (1st), Woodall R-GA (7th), Ferguson R-GA (3rd), Hice R-GA (10th)

YEAS: Bishop D-GA (2nd), Scott, David D-GA (13th), McBath D-GA (6th), Johnson D-GA (4th)

NOT VOTING: Graves R-GA (14th)

PRESIDENTIAL ELECTION: The House has passed a resolution (H. Res. 1155) sponsored by Rep. Eric Swalwell, D-Calif., reaffirming the Houses commitment to an orderly and peaceful transfer of presidential power after the November election. Swalwell said: The peaceful transition of power is not only a bedrock principle of Americas founding, it is a living ideal that we must exercise and pass down to our children. An opponent, Rep. Matt Gaetz, R-Fla., called the resolution a way for Democrats to attack the president and disguise the fact that they will refuse to accept the election results unless they win. The vote, on Sept. 29, was 397 yeas to 5 nays.

YEAS: Entire delegation, except Collins R-GA (9th), Graves R-GA (14th), who did not vote

DISEASE THERAPIES: The House has passed the Timely ReAuthorization of Necessary Stem-cell Programs Lends Access to Needed Therapies Act (H.R. 4764) sponsored by Rep. Doris O. Matsui, D-Calif. The bill would reauthorize a program for transplanting umbilical cord blood, stem cells, and bone marrow to adults and children suffering from various diseases. The vote, on Sept. 30, was unanimous with 414 yeas.

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YEAS: Entire delegation, except Graves R-GA (14th), who did not vote

FURTHER COVID-19 SPENDING: The House has approved an amendment to the Americas Conservation Enhancement Act (H.R. 925). The amendment would spend $2.2 trillion on new COVID-19 measures, including testing and treatment efforts and unemployment benefits. A supporter, Rep. James P. McGovern, D-Mass., said the spending was needed for families to pay for necessities like food, utilities, and rent during this pandemic. An opponent, Rep. Tom Cole, R-Okla., said the amendment had been hurriedly brought to the floor without minority input or adequate time for review, and that it would not pass the Senate. The vote, on Oct. 1, was 214 yeas to 207 nays.

NOT VOTING: Loudermilk R-GA (11th), Graves R-GA (14th)

YEAS: Bishop D-GA (2nd), Scott, David D-GA (13th), McBath D-GA (6th), Johnson D-GA (4th)

NAYS: Allen R-GA (12th), Scott, Austin R-GA (8th), Collins R-GA (9th), Carter R-GA (1st), Woodall R-GA (7th), Ferguson R-GA (3rd), Hice R-GA (10th)

CONTINUING APPROPRIATIONS: The Senate has passed the Continuing Appropriations Act and Other Extensions Act (H.R. 8337) sponsored by Rep. Nita M. Lowey, D-N.Y., to extend through Dec. 11 funding for health programs, including Medicare, surface transportation, and many other government programs. The vote, on Sept. 30, was 84 yeas to 10 nays.

AFFORDABLE CARE ACT LITIGATION: The Senate has rejected a cloture motion to end debate on a motion to consider a bill (S. 4653) sponsored by Senate Minority Leader Chuck Schumer, D-N.Y., that would block the Justice Department from making arguments in court for cancelling any provision of the 2010 health care reform law (ACA). The vote to end debate on Oct. 1, was 51 yeas to 43 nays, with a three-fifths majority needed for approval.

Link:
Congress Votes | News | albanyherald.com - The Albany Herald

Alex Peters is Dazed Beautys news writer. She picked up the phrase Its all happening from Almost Famous as a teen and it now exclusively makes up her vocabulary. Although in theory she likes the idea of a queer mullet, she is currently rocking the latest lockdown trend: Kajillionaire hair. Here, she shares her choice for Product of the Week a weekly round-up of the Dazed Beauty team and wider communitys must-have buys.

Brand: Augustinus Bader

Product: The Cream

Price: 205

As head of stem cell research at the University of Leipzig, Professor Augustinus Bader spent 30 years working with burns victims, developing a groundbreaking gel in 2008 that could heal third-degree burns without the need for skin grafts. Its this revolutionary stem cell technology the patented Trigger Factor Complex (TFC8) which lies at the heart of his skincare label and its hero product The Cream.

Powered by this technology, with consistent use (the Cream asks that you remain devoted to it for 27 days, using nothing else), the products activate and orchestrate the body's innate regenerative processes which basically means that when you use them your skin cells repair themselves. This helps with reducing the signs of ageing and environmental damage, reducing scarring and redness, and improving tone and texture.

Thats all very scientific, but the most important question is does it actually work? And for me it does. My skin is on the oily side as well as being sensitive and acne-prone so I use the Cream, rather than the Rich Cream which is better suited to dry skin types. The cream itself has a light texture that absorbs quickly and doesnt feel sticky. Two pumps is enough to cover your face and it goes on very smoothly. It has quite a distinctive scent, so if you are nose-sensitive be prepared. Its hard to describe clinical rather than artificial. It comes in a weighty blue and copper tube that feels fancy when youre using it.

I am not always great at using it consistently, particularly since we test out so many products as part of our job, but I have found that when I do, my skin looks noticeably better. My tone is more even and less red, the texture is better, scars look more faded, and I would tentatively say that it seems like I get fewer spots.

I would like to say, however, that the product is definitely on the higher end of the spectrum price-wise, and if you cannot afford it, please do not worry. There are a lot of great options out there that are more affordable and also very effective. If this is something that you can afford, I would highly recommend.

If I were communicating to an alien only using hand gestures, I would describe it with... Just a solid thumbs up. Or through elaborately miming the process of cell renewal.

It sounds like... A reassuring older German professor telling you efferyzing vill be alright and in the background the sound of a fire crackling.

If it was a meme it would be... Sorry to this man. Totally unrelated but its my favourite meme.

The fictional character who would use it is... Bette Porter from The L Word.

Alex Peters's Product of the Week

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The luxury skincare splurge that will help repair your skin cells - Dazed

Advances in the stem cell therapy sector have been phenomenal over the years. Its assistance in curing humans of various diseases and disorders has generated expansive advancements. These advancements are not just limited to humans. Stem cell therapy has also acquired a prominent place in the veterinary sector.

The influence of animal stem cell therapy for the treatment of various animals for diverse diseases and disorders is growing rapidly. Therefore, this factor may help the global animal stem cell therapy market to generate exponential growth across the forecast period of 2019-2029. Stem cells help in the replacement of neurons affected by stroke, Parkinsons disease, spinal cord injury, Alzheimers disease, and others.

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This animal stem cell therapy market report has extensive information on various aspects associated with bringing growth. Important factors such as emerging trends, mergers and acquisitions, and the regional scenario of the animal stem cell therapy market have been analyzed and included in the report. The stakeholders can derive a treasure of information from this report. This report also includes a scrutinized take on the COVID-19 impact on the animal stem cell therapy market.

Animal Stem Cell Therapy Market: Competitive Prospects

The competitive landscape of the animal stem cell therapy market can be described as mildly fragmented. With a considerable chunk of players, the animal stem cell therapy market is surrounded by substantial competition. Research and development activities form an important part of the growth landscape because they help gain novel insights.

Activities such as mergers, acquisitions, joint ventures, collaborations, and partnerships form the foundation of the growth of the animal stem cell therapy market. These activities help manufacturers to gain influence and eventually help in increasing the growth rate of the animal stem cell therapy market. Prominent participants in the animal stem cell therapy market are Magellan Stem Cells, Medivet Biologics LLC, Kintaro Cells Power, U.S. Stem Cell, Inc., Celavet Inc., VETSTEM BIOPHARMA, and VetCell Therapeutics.

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Animal Stem Cell Therapy Market: Key Trends

Infections are scaling up among animals at a rapid rate. The alarming increase is proving fatal for many animals. Therefore, to avoid such incidences and treat existing diseases and disorders, animal stem cell therapy is being applied seamlessly. Hence, this aspect may bring great growth opportunities for the animal stem cell therapy market.

Developments have been observed across the animal stem cell therapy market for long. Autologous adipose-derived mesenchymal stem cells are gaining traction for successfully resolving a range of issues in animals. These stem cells help in treating ligament and tendon injuries to a certain extent. The strengthening influence of this stem cell type in companion animals is also proving to be a prominent growth prospect for the animal stem cell therapy market.

Recent research has also found that stem cell-derived CC exosomes showed improved recovery from myocardial infarction (MI) among pigs. Such developments assure promising growth for the animal stem cell therapy market.

Animal Stem Cell Therapy Market: Regional Analysis

The animal stem cell therapy market is spread across North America, Latin America, the Middle East and Africa, Europe, and Asia Pacific. The animal stem cell therapy market may derive significant growth from North America. The escalating awareness regarding animal stem cell therapy may attract profound growth. Strengthening research and development activities in the region regarding animal stem cell therapy is further expanding the growth prospects.

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Animal Stem Cell Therapy Market - Great Growth Opportunities for the Market in the Coming Year | TMR Research Study - BioSpace

DetailsCategory: AntibodiesPublished on Saturday, 03 October 2020 15:56Hits: 667

Opdivo + Yervoy is the first new systemic therapy in over 15 years to be approved by the FDA in this setting1,2

Approval is based on CheckMate -743 in which Opdivo + Yervoy demonstrated superior overall survival vs. standard of care chemotherapy1

Approval marks third indication for Opdivo + Yervoy-based treatments in thoracic cancers and seventh indication overall

PRINCETON, NJ, UA I October 2, 2020 IBristol Myers Squibb (NYSE: BMY) today announced that Opdivo (nivolumab) 360 mg every three weeks plus Yervoy (ipilimumab) 1 mg/kg every six weeks (injections for intravenous use) was approved by the U.S. Food and Drug Administration (FDA) for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).1 This approval is based on a pre-specified interim analysis from the Phase 3 CheckMate -743 trial in which Opdivo + Yervoy (n=303) demonstrated superior overall survival (OS) versus the platinum-based standard of care chemotherapy (n=302) (Hazard Ratio [HR]: 0.74 [95% Confidence Interval [CI]: 0.61 to 0.89]; P=0.002), with a median OS (mOS) of 18.1 months (95% CI: 16.8 to 21.5) versus 14.1 months (95% CI: 12.5 to 16.2), respectively.1 These results were observed after 22.1 months of minimum follow-up.3 At two years, 41% of patients treated with Opdivo + Yervoy were alive and 27% with chemotherapy.1,3

Malignant pleural mesothelioma is a rare cancer with limited treatment options. When it is diagnosed in advanced stages, the five-year survival rate is approximately 10 percent, said study investigator Anne S. Tsao, M.D., professor and Section Chief Thoracic Medical Oncology and Director of the Mesothelioma Program at The University of Texas M.D. Anderson Cancer Center.2,4 The survival results from the CheckMate -743 trial show that the combination of nivolumab and ipilimumab could become a new front-line standard of care option. This is exciting news, instilling hope for patients with this devastating disease and for the healthcare providers who care for them.1,3

Opdivo and Yervoy are associated with Warnings and Precautions including immune-mediated: pneumonitis, colitis, hepatitis, endocrinopathies, nephritis and renal dysfunction, skin adverse reactions, encephalitis, other adverse reactions; infusion reactions; complications of stem-cell transplant that uses donor stem cells (allogeneic); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials.1Yervoy is associated with the following Warnings and Precautions: severe and fatal immune-mediated adverse reactions, infusion-related reactions, complications of allogeneic hematopoietic stem cell transplant after Yervoy, embryo-fetal toxicity and risks associated when administered in combination with Opdivo.5 Please see the Important Safety Information section below.

This is the third indication for an Opdivo + Yervoy-based combination in the first-line treatment of a form of thoracic cancer.1Opdivo + Yervoy is approved by the FDA as a first-line treatment for patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L11% as determined by an FDA-approved test, and without EGFR or ALK genomic tumor aberrations.1 It is also approved in combination with limited chemotherapy for the first-line treatment of adult patients with metastatic or recurrent NSCLC with no EGFR or ALK genomic tumor aberrations regardless of PD-L1 expression.1

Thoracic cancers can be complex and difficult to treat, and we are focused on developing immunotherapy options that may have the potential to extend patients lives, said Adam Lenkowsky, general manager and head, U.S., Oncology, Immunology, Cardiovascular, Bristol Myers Squibb.2,6 Just a few months ago, Opdivo + Yervoy-based combinations received two first-line indications for certain patients with non-small cell lung cancer. Now, Opdivo + Yervoy is approved for use in another type of thoracic cancer, previously untreated unresectable MPM. With todays announcement, Opdivo + Yervoy becomes the first new systemic therapy approved in more than 15 years in this setting, and may offer these patients a chance for a longer life. 1

Opdivo + Yervoy is a unique combination of two immune checkpoint inhibitors that features a potentially synergistic mechanism of action, targeting two different checkpoints (PD-1 and CTLA-4) to help destroy tumor cells: Yervoy helps activate and proliferate T cells, while Opdivo helps existing T cells discover the tumor.1,7 Some of the T cells stimulated by Yervoy can become memory T cells, which may allow for a long-term immune response.7,8,9,10,11,12 Targeting of normal cells can also occur and result in immune-mediated adverse reactions, which can be severe and potentially fatal.1 Please see the Important Safety Information section below.

This approval was granted less than six weeks following the submission of a new supplemental Biologics License Application (sBLA), which was reviewed under the FDAs Real-Time Oncology Review (RTOR) pilot program. The RTOR program aims to ensure that safe and effective treatments are available to patients as early as possible.13 The review was also conducted under the FDAs Project Orbis initiative, enabling concurrent review by the health authorities in Australia, Brazil, Canada and Switzerland.

About CheckMate -743

CheckMate -743 is an open-label, multi-center, randomized Phase 3 trial evaluating Opdivo plus Yervoy compared to chemotherapy (pemetrexed and cisplatin or carboplatin) in patients with histologically confirmed unresectable malignant pleural mesothelioma and no prior systemic therapy or palliative radiotherapy within 14 days of initiation of therapy (n=605).1 Patients with interstitial lung disease, active autoimmune disease, medical conditions requiring systemic immunosuppression, or active brain metastasis were excluded from the trial.1In the trial, 303 patients were randomized to receive Opdivo 3 mg/kg every two weeks and Yervoy 1 mg/kg every six weeks; 302 patients were randomized to receive cisplatin 75 mg/m2 or carboplatin AUC 5 plus pemetrexed 500 mg/m2 in 3-week cycles for six cycles.1 Treatment in both arms continued until disease progression or unacceptable toxicity or, in the Opdivo + Yervoy arm, up to 24 months.1 The primary endpoint of the trial was OS in all randomized patients.1 Additional efficacy outcome measures included progression-free survival (PFS), objective response rate (ORR) and duration of response (DOR), as assessed by BICR utilizing modified RECIST criteria.1

Select Safety Profile from CheckMate -743 Study

Treatment was permanently discontinued for adverse reactions in 23% of patients treated with Opdivo + Yervoy, and 52% had at least one dose withheld for an adverse reaction.1 An additional 4.7% of patients permanently discontinued Yervoy alone due to adverse reactions. Serious adverse reactions occurred in 54% of patients receiving Opdivo + Yervoy.1 The most frequent (2%) serious adverse reactions in patients receiving Opdivo + Yervoy were pneumonia, pyrexia, diarrhea, pneumonitis, pleural effusion, dyspnea, acute kidney injury, infusion-related reaction, musculoskeletal pain, and pulmonary embolism.1 Fatal adverse reactions occurred in 4 (1.3%) patients and included pneumonitis, acute heart failure, sepsis, and encephalitis.1 The most common (20%) adverse reactions were fatigue (43%), musculoskeletal pain (38%), rash (34%), diarrhea (32%), dyspnea (27%), nausea (24%), decreased appetite (24%), cough (23%) and pruritus (21%).1 The median number of doses was 12 for Opdivo and 4 for Yervoy.3

About Malignant Pleural Mesothelioma

Mesothelioma is a rare but aggressive form of cancer that often forms in the lining of the lungs.2,14 There are approximately 3,000 cases diagnosed in the United States each year.14 Malignant pleural mesothelioma is the most common type of the disease.2 It is most frequently caused by exposure to asbestosand diagnosis is often delayed, with the majority of patients presenting with advanced disease.2,15 Prognosis is generally poor: in patients with advanced malignant pleural mesothelioma, median survival is approximately one year and the five-year survival rate is approximately 10%.2

INDICATIONS

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC).

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

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

Bristol Myers Squibb: Advancing Cancer Research

At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.

Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early- to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.

About Bristol Myers Squibbs Patient Access Support

Bristol Myers Squibb remains committed to providing assistance so that cancer patients who need our medicines can access them and expedite time to therapy.

BMS Access Support, the Bristol Myers Squibb patient access and reimbursement program, is designed to help appropriate patients initiate and maintain access to BMS medicines during their treatment journey. BMS Access Support offers benefit investigation, prior authorization assistance, as well as co-pay assistance for eligible, commercially insured patients. More information about our access and reimbursement support can be obtained by calling BMS Access Supportat 1-800-861-0048 or by visiting http://www.bmsaccesssupport.com.

About the Bristol Myers Squibb and Ono Pharmaceutical Collaboration

In 2011, through a collaboration agreement with Ono Pharmaceutical Co., Bristol Myers Squibb expanded its territorial rights to develop and commercialize Opdivo globally, except in Japan, South Korea and Taiwan, where Ono had retained all rights to the compound at the time. On July 23, 2014, Ono and Bristol Myers Squibb further expanded the companies strategic collaboration agreement to jointly develop and commercialize multiple immunotherapies as single agents and combination regimens for patients with cancer in Japan, South Korea and Taiwan.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

References

SOURCE: Bristol-Myers Squibb

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US Food and Drug Administration Approves Opdivo (nivolumab) + Yervoy (ipilimumab) as the First and Only Immunotherapy Treatment for Previously...

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

If youre a parent, you know the thoughts, feelings and emotions that come with protecting your child. Youd do anything for them to ensure they live a long, healthy life. But what will you do when there are times when health conditions, such as childhood cancer, stand in the way?

Cancer in children is more common than you may think, as it accounts for the second-leading cause of death behind accidents. While a cancer diagnosis, or even the thought of your child developing cancer one day, is overwhelming, modern medicine has improved survival rates. As with any cancer, early detection is key. To help you familiarize yourself with childhood cancer, we asked Michael Confer, M.D., a radiation oncologist at the INTEGRIS Cancer Institute, about the different types of cancers, which signs to look for and how they can be treated.

Unlike adult cancers, which can result from the environment or exposure to certain things, childhood cancers result from genetic mutations that occur early on in life or before birth.

It all comes down to changes in genes. Your DNA contains information to make different types of cells in your body. In other words, your skin cells contain information to be brain cells, while your kidney cells contain information to be heart muscle cells. As cells mature, they become specialized, turning on and off certain genes to allow them to perform specific duties. Cells need to be able to replicate to replace damaged cells of the same category. They grow with help from genes called proto-oncogenes.

When your DNA changes, it leads to genetic mutations, and cells can become permanently activated. This can lead to cells duplicating uncontrollably, known as cancer.

Tumor suppressor genes slow down cell division. They repair DNA mistakes before cells divide and control cells internal death process (apoptosis or programmed cell death), Dr. Confer says. DNA mutations within tumor suppressor genes can also allow cells to duplicate uncontrollably. Children can be born with mutated proto-oncogenes or tumor suppressor genes in certain cells. These abnormally programmed cells lead to most childhood cancers.

So, what causes DNA changes? Your child can inherit genes from a parent that increases their risk of cancer or they can acquire these genes. Cancers from acquired, sporadic gene mutations are more common than those from inherited gene mutations 5% of all childhood cancers come from inherited mutations.

Breast cancer and ovarian cancer are the most common types of cancer caused by inherited DNA changes from BRCA1 or BRCA2 gene mutations. Even with how well-known these are, only 5 to 10% of breast cancer cases come from BRCA1/BRCA2 inherited mutations. Plus, breast cancer and ovarian cancer are more common in adults than children. Talk to your doctor or visit a genetic counselor if you have specific questions about inherited mutations.

Cancer can impact any part of your body, ranging from your bones and blood cells to your brain, spinal cord and other internal organs. You may be most familiar with leukemia, lymphoma, and brain and spinal tumors, since they are the most common. But, here is a full overview of cancers that commonly affect children, according to the American Cancer Society.

Leukemia: This is the most common type of cancer in children, accounting for 28% of cases. It generally starts in white blood cells and becomes fast growing (acute). Acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML) are the two most common types of leukemia. Three out of every four children with leukemia have ALL. This type of cancer starts in the lymphoid cells, called lymphocytes, whereas AML starts in myeloid cells. Chronic myeloid leukemia (CML) and chronic lymphocytic leukemia (CLL) are two types of rare cancers

Brain and spinal cord tumors: These types of cancers make up 26% of all cases. Brain tumors are more common than spinal cord tumors. The cancer generally starts in the lower part of the brain.

Neuroblastoma: This type of cancer affects infants and young children. Neuroblastoma starts in nerve cells during pregnancy and accounts for 6% of childhood cancers. Abdomen swelling is a common sign of neuroblastoma.

Wilms tumor: This type of cancer starts in the kidneys and impacts children who are 3 or 4 years old. It accounts for 5% of childhood cancers.

Lymphoma: Although lymphoma isnt as common as other types of cancers, youve likely heard of Hodgkin lymphoma and non-Hodgkin lymphoma, the two main types of cancer that show up in the lymphocytes. Non-Hodgkin lymphoma (accounts for 5% of childhood cancers) appears in younger children and is more common than Hodgkin lymphoma (accounts for 3%), which is more common in younger adults. You may notice a swollen lymph node under your childs arm or near their throat.

Rhabdomyosarcoma: This type of cancer develops in areas that your child uses to move their body, such as the head, pelvis, arms or legs. It accounts for 3% of childhood cancer cases.

Retinoblastoma: This cancer develops in the eyes, and your child is most at risk around the age of 2 until the age of 6. It accounts for 2% of childhood cancers.

Bone cancer: Bone cancer is more prominent in teens, but it still accounts for 3% of childhood cancers. There are two types, osteosarcoma and Ewing sarcoma, that show up via swelling around the bones. Ewing sarcoma is a less common type of cancer that is more prominent in older children and younger teens. Osteosarcoma, meanwhile, is more common in teens and in areas where bones grow quickly.

There isn't a one-size-fits-all guide to know if your child has cancer. In general, Dr. Confer says to keep an eye on any changes in your childs behavior, such as walking, eating, playing or sleeping. If theyre older, listen to any complaints they may have. Some cancers may produce a lump or swelling and pain in certain areas. Other symptoms include a loss of energy, weight loss, sudden eye or vision changes, frequent headaches with vomiting or a persistent fever that signifies the body is fighting an infection.

For example, leukemia, the most common type of childhood cancer, affects most children between the ages of 2 and 4. Typical symptoms include fever, bleeding, deep pain in the bones, small red spots on the skin called petechiae, bruises and enlarged lymph nodes.

Notify your childs pediatrician if any of these concerns arise. Aside from that, you should schedule your child for routine checkups and wellness visits.

Routine checkups and wellness visits help monitor normal growth and development. A good pediatrician-patient relationship helps the physician better identify subtle signs of cancer and gives parents a trusted sounding board for the concerns parents or children may have, Dr. Confer says.

Many childhood cancers have become increasingly treatable, leading to longer survival rates. Dr. Confer says acute lymphoblastic leukemia, lymphoma or kidney tumors known as Wilms tumor all have more than a 90% five-year survival rate (the percentage of patients who are alive five years after receiving treatment or a diagnosis).

In fact, the overall five-year survival rate for childhood cancers has improved from 58% in the mid-1970s to 84%, according to the American Cancer Society. But, certain types of aggressive cancer still exist. Diffuse intrinsic pontine glioma (DIPG), a rare brain tumor, is often cited as the childhood cancer with the poorest survival rate (less than 1% for five years).

No matter the diagnosis, continual hope and quality, proven therapies are the most important factors for children and families facing childhood cancers, Dr. Confer says.

Here are some of the most common forms of therapies to treat childhood cancer.

Surgery can help many patients, whether you need an entire tumor removed or a procedure to ease pain caused by a tumor. Your childs surgeon can also debulk a tumor, meaning they remove part of it and treat the rest with another method. Surgery has the highest success rate when its contained to one area, before the cancer has an opportunity to metastasize (spread to other parts of the body).

High doses of radiation help reduce cancer by either killing the cells or damaging their DNA to slow growth. Over time, these cells die and your body removes them. You can either receive internal or external radiation. External radiation comes from a beam that treats a specific body part, whereas internal radiation is in solid or liquid form. More specifically, brachytherapy is the medical term for solid internal radiation. Your doctor will place capsules, seeds or ribbons near the tumor. Systemic therapy is the medical term for liquid internal radiation. With this method, the radiation travels through your blood via a pill, injection or IV to kill cancer cells.

Chemotherapy comes in many methods of application, such as IV, oral, injection, topical or through a catheter, port or pump. Chemotherapy also kills healthy cells, which is one of the downsides. This is why many chemotherapy patients lose their hair and experience other side effects. Depending on the type and progression of the cancer, chemotherapy can help shrink a tumor to increase the success rate of surgery or radiation. Chemotherapy can also fight against any lingering cancer cells following surgery or radiation. Its also used to treat cancer that returns or metastasizes.

The immune system is your bodys way of defending itself against harmful germs, bacteria and viruses. When it comes to cancer, the immune system can have trouble recognizing and fighting off harmful cells because cancer starts in healthy cells. Immunotherapy helps your body pinpoint cancer kills to better defend against them. There are many types of immunotherapy treatments to boost your immune system. One type, chimeric antigen receptor (CAR) T-cell therapy, mixes your own T-cells with a virus that teaches the T-cells how to kill cancer cells.

Targeted therapy is a form of chemotherapy. But, as the name suggests, these drugs zero in on a specific area of the cancer cells. Depending on the drug, targeted therapy can change the protein levels in cancer cells or block chemical signals that help cancer cells grow. Other targeted therapy drugs can limit blood vessel production to cut off the cancer cells or distribute toxins to specifically kill the cancer while sparing healthy cells.

Stem cells, which originate in the bone marrow, make red blood cells, white blood cells and platelets. Leukemia and lymphoma start in the blood cells, causing damage to the cells your body needs to function. A stem cell transplant involves destroying cancer cells via chemotherapy and/or radiation before replacing them with new, healthy cells. This allows doctors to use stronger doses of chemotherapy or radiation knowing new cells, via a transplant, will replace old, damaged cells. Stem cell transplants can come from your own cells or the cells of another person. Donated cells are often more effective since they can help kill off cancer cells.

While you cant do anything to prevent your child from developing cancer, you can be proactive by scheduling regular checkups and looking out for warning signs and symptoms. Contact an INTEGRIS pediatrician if you have any concerns, and they can refer you to an oncologist at the INTEGRIS Cancer Institute.

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Different Types of Childhood Cancer | INTEGRIS - Integris

Heres a look at how Maines members of Congress voted over the previous week.

Along with its roll call votes this week, the House also passed these measures: the Cyber Sense Act (H.R. 360), to require the Secretary of Energy to establish a voluntary Cyber Sense program to test the cybersecurity of products and technologies intended for use in the bulk-power system; the Consumer Product Safety Inspection Enhancement Act (H.R. 8134), to support the Consumer Product Safety Commissions capability to protect consumers from unsafe consumer products; the School-Based Allergies and Asthma Management Program Act (H.R. 2468), to increase the preference given, in awarding certain allergies and asthma-related grants, to states that require certain public schools to have allergies and asthma management programs; and the Effective Suicide Screening and Assessment in the Emergency Department Act (H.R. 4861), to establish a program to improve the identification, assessment, and treatment of patients in the emergency department who are at risk of suicide.

House Vote 1:

PRESIDENTIAL ELECTION: The House has passed a resolution (H. Res. 1155), sponsored by Rep. Eric Swalwell, D-Calif., reaffirming the Houses commitment to an orderly and peaceful transfer of presidential power after the November election. Swalwell said: The peaceful transition of power is not only a bedrock principle of Americas founding; it is a living ideal that we must exercise and pass down to our children. An opponent, Rep. Matt Gaetz, R-Fla., called the resolution a way for Democrats to attack the president and disguise the fact that they will refuse to accept the election results unless they win. The vote, on Sept. 29, was 397 yeas to 5 nays.

YEAS: Pingree D-ME (1st), Golden D-ME (2nd)

House Vote 2:

DISCLOSING TIES TO UYGHUR LABOR: The House has passed the Uyghur Forced Labor Disclosure Act (H.R. 6270), sponsored by Rep. Jennifer Wexton, D-Va., to require publicly traded companies to disclose whether they have business ties to Chinas Uyghur Autonomous Region in Xinjiang province. Wexton said the requirement would let investors know of a given companys passive complicity or active exploitation of one of the most pressing and ongoing human rights violations of our lifetime. A bill opponent, Rep. Anthony Gonzalez, R-Ohio, said it wrongly tried to have the Securities and Exchange Commission police human rights violations, a role that would be better handled by the Treasury Department. The vote, on Sept. 30, was 253 yeas to 163 nays.

YEAS: Pingree D-ME (1st), Golden D-ME (2nd)

House Vote 3:

DISEASE THERAPIES: The House has passed the Timely ReAuthorization of Necessary Stem-cell Programs Lends Access to Needed Therapies Act (H.R. 4764), sponsored by Rep. Doris O. Matsui, D-Calif. The bill would reauthorize a program for transplanting umbilical cord blood, stem cells and bone marrow to adults and children suffering from various diseases. The vote, on Sept. 30, was unanimous with 414 yeas.

YEAS: Pingree D-ME (1st), Golden D-ME (2nd)

House Vote 4:

FURTHER COVID-19 SPENDING: The House has approved an amendment to the Americas Conservation Enhancement Act (H.R. 925). The amendment would spend $2.2 trillion on new COVID-19 measures, including testing and treatment efforts and unemployment benefits. A supporter, Rep. James P. McGovern, D-Mass., said the spending was needed for families to pay for necessities like food, utilities, and rent during this pandemic. An opponent, Rep. Tom Cole, R-Okla., said the amendment had been hurriedly brought to the floor without minority input or adequate time for review, and that it would not pass the Senate. The vote, on Oct. 1, was 214 yeas to 207 nays.

YEAS: Pingree D-ME (1st)

NAYS: Golden D-ME (2nd)

Senate Vote 1:

CONTINUING APPROPRIATIONS: The Senate has passed the Continuing Appropriations Act and Other Extensions Act (H.R. 8337), sponsored by Rep. Nita M. Lowey, D-N.Y., to extend through December 11 funding for health programs, including Medicare, surface transportation, and many other government programs. The vote, on Sept. 30, was 84 yeas to 10 nays.

YEAS: Collins R-ME, King I-ME

Senate Vote 2:

OBAMACARE LITIGATION: The Senate has rejected a cloture motion to end debate on a motion to consider a bill (S. 4653), sponsored by Senate Minority Leader Chuck Schumer, D-N.Y., that would block the Justice Department from making arguments in court for cancelling any provision of the 2010 health care reform law (Obamacare). The vote to end debate, on Oct. 1, was 51 yeas to 43 nays, with a three-fifths majority needed for approval.

YEAS: Collins R-ME, King I-ME

Continued here:
How Maine's members of Congress voted this week - Bangor Daily News

HOUSTON, Oct. 1, 2020 /PRNewswire/ -- FibroGenesis, the leading developer of fibroblast based therapeutic solutions for unmet medical needs has entered into a clinical collaboration agreement with Brazilian R4D Biotech.Holding the world's largest patent portfolio in the field of cell therapies using fibroblasts, FibroGenesis is expanding its ongoing clinical programs internationally. The partnership will pave the way for clinical studies of PneumoBlast in Brazil as a unique treatment of acute respiratory distress syndrome (ARDS) for patients affected by COVID-19, in parallel to clinical studies in the United States upon approval by the FDA.

Administration of PneumoBlast in pre-clinical and animal studies resulted in dramatic improvement of immunological signaling molecules, reducing concentrations of the inflammatory cytokines interleukin-1 beta, interleukin-6, interleukin-8, interleukin-17, interleukin-18, and Tumor Necrosis Factor alpha TNFa. Company scientists have also demonstrated that PneumoBlast has induced statistically significant reduction of lung fibrosis and lung scarring in COVID-19 infected animals, particularly when compared to more conventional treatments using bone marrow derived mesenchymal stem cells (BMSCs). Furthermore, recent data supports the potential benefits of PneumoBlast for preventing COVID-19 blood clotting. Both companies will collaborate on a clinical study design that meets the needs of Brazilian patients.

"As the scientific and medical community is discovering more about the biological and medical consequences of the COVID-19 infection, FibroGenesis is eager to contribute to the therapeutic cure options currently being created to fight this global war against this virus," commented Pete O'Heeron, Chief Executive Officer, FibroGenesis. "The collaboration with R4D Biotech is another strategic milestone that emphasizes our commitment to expand fibroblast research globally."

"The lab results which indicate our cell therapy approach possesses both therapeutic effects on animal models of the acute stage of COVID-19, and also benefits a cure for residual pathology seen in COVID-19 patients, has our research team extremely excited," said Thomas Ichim, Ph.D., Chief Scientific Officer, FibroGenesis.

"Technology transfer is at the core of this partnership," said Paulo Ferraz, BRICS/Emerging Markets Director of international fund Newstar Ventures and an advisor for FibroGenesis on this transaction. "R4D Biotech has access to sophisticated resources comprising research facilities and hospitals, and its talent pool includes scientific advisors who are recognized academics and distinguished members of the Brazilian Academy of Pharmaceutical Sciences. PneumoBlast clinical study will represent the first step in a long-term relationship designed to aid in the discovery of advanced therapeutic solutions for chronic medical needs."

About R4D Biotech:R4D Biotech is a Brazilian emerging company headquartered in the state of So Paulo focused on research and development for biotechnology and healthcare, with the mission of bringing disruptive technology innovation across all steps of clinical development in life sciences.

About FibroGenesis:Based in Houston, Texas, FibroGenesis is a regenerative medicine company developing an innovative solution for chronic disease treatment using human dermal fibroblasts. Currently, FibroGenesis holds 240+ U.S. and international issued patents/patents pending across a variety of clinical pathways, including Disc Degeneration, Multiple Sclerosis, Parkinson's, Chronic Traumatic Encephalopathy, Cancer, Diabetes, Liver Failure, Colitis and Heart Failure. FibroGenesis represents the next generation of medical advancement in cell therapy.Visit http://www.Fibro-Genesis.com.

View original content to download multimedia:http://www.prnewswire.com/news-releases/fibrogenesis-expands-fight-against-covid-19-in-brazil-with-international-collaboration-301144126.html

SOURCE FibroGenesis

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FibroGenesis Expands Fight Against COVID-19 In Brazil with International Collaboration - BioSpace