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InMed Pharmaceuticals Announces Publication of Peer-Reviewed Article on the Use of CBN as a Potential Treatment for Glaucoma – GlobeNewswire

Posted: December 24, 2021 at 2:08 am

VANCOUVER, British Columbia, Dec. 20, 2021 (GLOBE NEWSWIRE) -- InMed Pharmaceuticals Inc. (InMed or the Company) (Nasdaq: INM), a leader in the development, manufacturing and commercialization of rare cannabinoids, today announced that a peer-reviewed scientific article entitled Cannabinol Modulates Neuroprotection and Intraocular Pressure: A Potential Multi-Target Therapeutic Intervention for Glaucoma, has been published in Biochimica et Biophysical Acta (BBA - Molecular Basis of Disease), a leading international journal focused on biochemistry and molecular genetics of disease processes and models of human disease in the area of aging, cancer, metabolic-, neurological-, and immunological-based diseases.

The peer-reviewed article highlights research evaluating the use of cannabinol, or CBN, as a potential treatment option for glaucoma. Several studies were conducted to evaluate the survival of retinal ganglion cells, modulation of intraocular pressure and its effects on extracellular matrix proteins using in vitro and in vivo glaucoma models.

These studies resulted in two key findings: first, CBN may promote neuroprotection of cells in the retina that are responsible for vision; and second, CBN may normalize intraocular pressure by attenuating changes in the extracellular matrix proteins. The article also reports on the comparison of CBN with other cannabinoids, including cannabidiol (CBD) and tetrahydrocannabinol (THC), with results indicating that CBN has a stronger effect and broader neuroprotective therapeutic range. These observations elucidate the therapeutic potential for CBN in the treatment of glaucoma.

Having this research peer-reviewed and published in such a reputable journal is a significant milestone for InMeds glaucoma development program. This provides important external validation for the scientific community as the program advances towards human studies, said InMeds scientific advisor, Dr. Mauro Maccarrone, Professor and Chair of Biochemistry at the Department of Biotechnological and Applied Clinical Sciences, University of LAquila, Italy. We believe InMeds INM-088 program has significant implications for neuroprotection within the glaucoma space and may lead to a potential new treatment for millions of people suffering from this condition worldwide.

Corresponding author Professor Ujendra Kumar, Faculty of Pharmaceuticals, The University of British Columbia, added; The observations in this study indicate the therapeutic potential for the use of CBN in the treatment of glaucoma. Not only does the data suggest a reduction in ocular pressure but also proactive neuroprotection in the retina, both of which are integral to the treatment of glaucoma. This publication is further evidence to suggest rare cannabinoids have therapeutic potential to treat various diseases.

The journal article can be accessed here: https://doi.org/10.1016/j.bbadis.2021.166325

About Glaucoma: Glaucoma is a group of eye diseases which result in damage to the optic nerve due to high intraocular pressure (IOP) of the eye from fluid build-up. According to the American Academy of Ophthalmology, glaucoma is the leading cause of blindness in adults aged 60 and older. It is estimated that 76 million people worldwide have glaucoma. There is a major unmet medical need for new glaucoma treatments that provide neuroprotection of retinal ganglion cells. Current treatments work to reduce intraocular pressure but do not provide direct neuroprotection in the retina.

About CBN: Cannabinol (CBN) is a non-intoxicating rare cannabinoid. It is one of the more than 140 cannabinoid compounds present in the Cannabis plant. CBN is also the active pharmaceutical ingredient in InMeds lead program, INM-755 cannabinol cream, which is currently undergoing a Phase 2 Clinical Trial in the treatment ofEpidermolysis Bullosa (EB). In several preclinical safety pharmacology and toxicology studies using CBN at high doses, no adverse events were seen on central nervous system (CNS) function in a rigorous evaluation of 108 CNS measurements.

Learn about InMeds INM-088, a CBN topical formulation for the treatment of glaucoma: https://www.inmedpharma.com/pharmaceutical/inm-088-for-glaucoma/

Learn more about cannabinol: https://www.inmedpharma.com/learn/what-is-cannabinol/

About InMed: InMed Pharmaceuticals is a global leader in the development, manufacturing and commercialization of rare cannabinoids. Together with its subsidiary, BayMedica LLC, the Company has unparalleled cannabinoid manufacturing capabilities to serve a spectrum of consumer markets, including pharmaceutical and health and wellness. InMed is a clinical-stage company developing a pipeline of rare cannabinoid therapeutics and dedicated to delivering new treatment alternatives to patients that may benefit from cannabinoid-based pharmaceutical drugs. For more information, visitwww.inmedpharma.com and http://www.baymedica.com.

Investor Contact: Colin ClancySenior Director, Investor RelationsT: +1 604 416 0999E: cclancy@inmedpharma.com

Edison Group:Joe Green/Laine YonkerT: +1.646.653.7030/+1.646.653.7035E: jgreen@edisongroup.com / lyonker@edisongroup.com

Cautionary Note Regarding Forward-Looking Information:

This news release contains "forward-looking information" and "forward-looking statements" (collectively, "forward-looking information") within the meaning of applicable securities laws. Forward-looking information is based on management's current expectations and beliefs and is subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Forward-looking information in this news release includes statements about: a topical eye drop formulation under development for the treatment of glaucoma; CBN promoting neuroprotection of the eye and normalizing intraocular pressure and the potential to treat millions suffering from glaucoma; the potential for rare cannabinoids to treat various diseases; being a global leader in the manufacturing and development of rare cannabinoids; and delivering new treatment alternatives to patients that may benefit from cannabinoid-based pharmaceutical drugs.

With respect to the forward-looking information contained in this news release, InMed has made numerous assumptions. While InMed considers these assumptions to be reasonable, these assumptions are inherently subject to significant business, economic, competitive, market and social uncertainties and contingencies.

Additionally, there are known and unknown risk factors which could cause InMed's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking information contained herein. A complete discussion of the risks and uncertainties facing InMeds stand-alone business is disclosed in InMeds Annual Report on Form 10-K and other filings with the Security and Exchange Commission on http://www.sec.gov.

All forward-looking information herein is qualified in its entirety by this cautionary statement, and InMed disclaims any obligation to revise or update any such forward-looking information or to publicly announce the result of any revisions to any of the forward-looking information contained herein to reflect future results, events or developments, except as required by law.

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InMed Pharmaceuticals Announces Publication of Peer-Reviewed Article on the Use of CBN as a Potential Treatment for Glaucoma - GlobeNewswire

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Healthy gut bacteria can reduce severity of Covid-19, UAE researchers say – The National

Posted: December 24, 2021 at 2:08 am

Scientists in the UAE have found that some bacteria in the gut may reduce the severity of Covid-19 in infected people.

The study carried out by scientists at the University of Sharjah, Khalifa University of Science and Technology in Abu Dhabi, and other institutions, said the make-up of the gut microbiome may influence the severity of the disease and the body's immune response.

The work is among the latest of many studies of the relationship between the gut microbiome and Covid-19, some of which have analysed how diet influences a persons ability to fend off the coronavirus.

You dont have to go vegan, but getting more diverse plants on your plate is a great way to boost the health of your gut microbiome, improve your immunity and overall health, and potentially reduce your risk from Covid-19

Prof Tim Spector, King's College London

The study, published in Frontiers in Microbiology, looked at 86 infected people and another 57 without the disease.

It found that in the infected group, there was a greater abundance of some types of disease-causing bacteria in the gut, and higher levels of some that cause inflammation. These, the researchers suggested, may have played a role in the early symptoms of those who were infected.

Since patients gut microbiota were only sampled after they were infected with the virus, we are unsure whether pre-existing gut dysbiosis contributing to more severe Covid-19 symptoms, or whether Covid-19 was the cause of the gut dysbiosis. This is very similar to the chicken and egg question, said the studys senior author, Dr Habiba Alsafar, director of the Centre for Biotechnology and associate professor of molecular biology and genetics at Khalifa University in Abu Dhabi.

But in this group, who had been infected with Covid-19, there was also a greater abundance of certain types of anti-inflammatory bacteria and of several types of bacteria that produce butyrate, a fatty acid that can strengthen the immune response.

These are types of symbiotic bacteria, meaning they have a mutually beneficial relationship with the person in whom they are found.

Altogether, perhaps the gut symbiotic response plays a significant role in counteracting Covid-19 dysregulated immune response, restoring homeostasis [a stable situation], and subsequently reducing Covid-19 pathogenesis and disease manifestations, the researchers said.

While Covid-19 is primarily a respiratory illness, the gut has an immunological function and the microorganisms it contains are important in responding to infection.

The researchers noted that ACE2 receptors, which are abundant on cells in the human respiratory system and are the attachment points for the coronavirus, are also expressed in some cells that line the intestine. As a result, these intestinal cells could be additional sites of infection for SARS-CoV-2, the virus that causes Covid-19.

Dr Habiba Alsafar, director of Khalifa University Centre for Biotechnology, says a healthy diet and exercise is important for a healthy gut. Delores Johnson / The National

The most important connection between gut microbiome and Covid-19 is the involvement of the ACE2 receptor, said Dr Alsafar.

The ACE2 receptors regulate the gut microbiota, and when a viral infection, like SARS-CoV-2, can enter these receptors, it causes a dysregulation of the intestinal system.

Dr Alsafar said specially formulated probiotics might improve the gut microbiome, although a consultation with a doctor would be required for best results.

Healthier diet, including a fruit and vegetable-rich diet, with more exercise, will impact gut microbe communities, she said.

A separate study published early this year in the journal Gut produced some results consistent with the UAE study.

For example, it found that lower levels of a butyrate-producing bacterium, Faecalibacterium, which was also discussed in the UAE study, were associated with more severe infections.

This studys authors, all of whom work at institutions in Hong Kong, also concluded that the gut microbiome may affect a persons immune response to infection with the coronavirus and influence how severe the disease is and the eventual outcome.

They also suggested that ongoing imbalances in the microbiome after infection may cause the persistent symptoms seen in people with long Covid.

The UAE and Hong Kong studies add to the knowledge generated by a major research by Kings College London and Harvard Medical School that involved hundreds of thousands of people contributing data through an app.

It found that people with the healthiest diets were 10 per cent less likely to develop Covid-19 and 40 per cent less likely to become seriously ill from it.

Prof Tim Spector, of genetic epidemiology at Kings College London, said people with diets with low levels of ultra-processed foods had healthier gut microbes that were more likely to fend off infection.

You dont have to go vegan, but getting more diverse plants on your plate is a great way to boost the health of your gut microbiome, improve your immunity and overall health, and potentially reduce your risk from Covid-19, he said at the time of the report's release in September.

Social distancing measures are very much still in place at the farmers' market in Dubai's Business Bay.Reem Mohammed / The National

Updated: December 23rd 2021, 3:15 AM

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People in the News: New Appointments at Myriad Genetics, NeoGenomics, Epigenomics, More – GenomeWeb

Posted: December 24, 2021 at 2:08 am

Myriad Genetics: Nicole Lambert

Myriad Genetics has promoted Nicole Lambert to chief operating officer. Previously, Lambert headed up Myriad's oncology, women's health, and international business divisions and genetic testing laboratory operations. In her new role, Lambert will still report to Myriad CEO Paul Diaz. She will now be in charge of spearheading the company's strategic growth plans, expanding the markets for Myriad's molecular diagnostic products, and implementing operational improvements that allow the firm to better engage with patients and doctors.

NeoGenomics: David Eberhard

NeoGenomics has appointed David Eberhard as chief medical officer of Inivata, NeoGenomics'liquid biopsy-focused subsidiary. Eberhard will be in charge of the clinical and strategic development of the subsidiary's liquid biopsy tests, including the RaDaRtest for minimal residual disease. Hemost recently served as senior medical director of oncology at Illuminaand was previously the senior director of oncology development at Genomic Health, focusing on bringing the firm's OncotypeSEQliquid biopsy test to market.

Epigenomics: Jens Ravens

Epigenomics AG appointed Jens Ravens as chief financial officer, effective Feb. 1, 2022. Ravens will also become a member of the executive board of Epigenomics, overseeing the company's financial and administrative functions.Ravens began his career at Deutsche Bank. He previously held several positions, including VP of controlling and finance and compliance and risk officer at Hermes Group. He also served as CFO and managing director of Pleon Germany and as CFO of Pleon Europe, and as finance director and member of the board of Interseroh CDI.

For additional recent items on executive appointments and promotions in omics and molecular diagnostics, please see the People in the News page on our website.

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People in the News: New Appointments at Myriad Genetics, NeoGenomics, Epigenomics, More - GenomeWeb

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GB HealthWatch raises $3.5 million in pre-series C funding to expand genetic-based preventive health products and services for cardiometabolic…

Posted: December 24, 2021 at 2:08 am

GB HealthWatch raises $3.5 million for genetics-based, preventive health platform to combat cardiometabolic diseases and Alzheimer's

SAN DIEGO (PRWEB) December 21, 2021

GB HealthWatch, a genetic-based preventive health company, announced today it raised $3.5 million in pre-series C funding. The investors participating in the round are Trinity Power LLC, a series B investor, and the founder of GB HealthWatch. The company plans to use the funds to grow its national leadership position in advanced genetic testing and developing precision medicine applications for the prevention and treatment of dyslipidemia, coronary heart disease, type 2 diabetes, and Alzheimers disease, and build the infrastructure of their preventive health ecosystem.

Distinguished from traditional genetics companies that focus primarily on rare monogenic diseases, GB HealthWatch is a pioneer in studying complex, polygenic diseases that can be effectively mitigated with preventive treatment. The pathophysiological processes that underlie cardiometabolic diseases and Alzheimers start decades before the onset of symptoms; genetic testing can identify at-risk individuals before clinical symptoms manifest so that preventive treatment can be initiated before damage is done. Knowing your genetics can help identify the specific root cause of a condition and most effective intervention, said Dr. Mendel Roth, Lead Scientist of Genetics and Molecular Biology at GB HealthWatch.

The value of genetic-based preventive health can be illustrated using familial hypercholesterolemia (FH) as an example. FH is relatively common, occurring in about 1 in every 250 individuals. An individual with a pathogenic FH mutation has a 15-20-fold higher risk of developing premature coronary heart disease. However, if diagnosed early, treatment with lipid-lowering medication and lifestyle modification can effectively halt the progression of the disease. This benefit is even more pronounced if FH is detected at a young age and treatment is started. Research shows that FH patients who initiated lipid-lowering therapies as a child had a 12-fold lower risk of developing coronary heart disease compared to their parents who did not initiate treatment until later in life.

Unfortunately, 90% of people living with FH in the US have not been diagnosed and therefore may never be treated. About 1.3 million people in the US have FH; without treatment, 50% of men and 30% of women living with FH are expected to experience a serious cardiovascular event such as heart attack or stroke before age 50 or 60, respectively. Genetic testing is the gold standard for FH diagnosis and identifying at-risk family members for preventive treatment.

As we are in the genomic health era, the challenges and opportunities converge on same path how to apply genetic data for improving human health. GB is the first company that developed and brought to market comprehensive genetic testing for cardiometabolic diseases including dyslipidemia, atherosclerotic cardiovascular disease, obesity, type 2 diabetes, and Alzheimers which are the leading causes of morbidity and mortality, globally. GBinsight genetic testing developed by GB HealthWatch is used by leading cardiologists, lipidologists, and endocrinologists, and recognized by the National Lipid Association.

With this round of pre-series C funding and subsequent series C funding, GB HealthWatch will further develop and refine a platform that will enable physicians to prescribe genomics-guided, precision therapeutics that will maximize clinical efficacy, limit unnecessary healthcare costs, and empower patients and their at-risk family members to engage in preventive care. We are working to implement a digital preventive health ecosystem that can be applied on a population scale, said Dr. Li Shen, President of GB HealthWatch.

GB HealthWatch Products and Services

GBinsight Genetic Testing and Analysis for:

Digital Preventive Health:

Population Research:

About GB HealthWatch

GB HealthWatch is a genetic-based Preventive Health company. We develop state-of-the-art technologies to facilitate research on the molecular mechanisms, clinical efficacy, and cost-effectiveness of translating genetic insights into personalized prevention and treatment strategies for complex diseases. GB HealthWatch offers the following products and services:

Visit the GB HealthWatch website to learn more.

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The COVID Theory That Got Your Hopes Up Is Actually Bullsh – The Daily Beast

Posted: December 24, 2021 at 2:08 am

A fringe theorythat the novel coronavirus can mutate into a harmless new formseems to have given some people false hope that the pandemic, now entering its third year, might end all on its own, regardless of what government officials, scientists, and everyday people do.

This COVID self-extinction theory isnt new. It got some traction in the media in the spring under slightly different terminology.

The theory was almost certainly wrong then, and its still probably wrong now, experts told The Daily Beast. I think self-extinction is vanishingly unlikely, Jesse Bloom, an investigator at the Fred Hutchinson Cancer Research Center in Washington State, told The Daily Beast.

Its tempting to believe SARS-CoV-2 will just go away. Without expensive, politically fraught efforts to vaccinate everyone. Without unpopular mask mandates and restrictions on schools and businesses. Without anyone sacrificing anything in the interest of public health.

But thats most likely wishful thinking. And potentially dangerous if enough people indulge in it. Beating COVID probably means fighting it with every tool in our kit. Because, lets be honest, its not looking like its going to beat itself.

A smattering of headlines announced the current interest in the possibility of COVID self-extinguishing. Dominant Delta variant may mutate itself into extinction, scientists say, The New York Post teased on Nov. 22. Covid 19: New research suggests Delta strain drove itself to extinction in Japan, the New Zealand Herald crowed the same day.

All the stories cite the same news story promoting the same claim. A Nov. 18 story in The Japan Times in which Ituro Inoue, a professor at Japans National Institute of Genetics, described some preliminary research he and his team had done on the SARS-CoV-2 virus genetic structure and changes over time.

The upshot, according to Inoues theory, is that SARS-CoV-2 basically hit an evolutionary brick wall, piling bad mutation on top of bad mutation until it rendered itself impotent.

Inoues teams research isnt published in a science journal yet, to say nothing of being peer-reviewed. That didnt stop The Japan Times from repeating Inoues claims without a lot of caveats. Neither Inoue nor The Japan Times responded to requests for comment.

I have never seen one that is widespread and just goes away.

Scientists in Japan and across the world urged skepticism. I know some experts are saying such a self-extinction thing, but I will not bet on it, Tanaka Mikihito, an expert on pandemics at Waseda University in Tokyo who also holds a Ph.D in molecular biology, told The Daily Beast.

To be fair, theres a good reason Inoue grasped for a theorya desperate stretch though it might be. He was trying to explain Japans very odd winter.

Japan, like much of the world, suffered a huge spike in COVID infections this fall as the highly transmissible Delta variantlineage is the scientific termbecame dominant, displacing less dangerous forms of the virus.

But Japans Delta wave ended relatively quickly in mid-September. And after that, cases dwindled to, well, nearly zero. Just a hundred or so new cases a day in a country with 126 million people. This at a time when many other countries, including the United States, were seeing fresh surges fueled in part by the new Omicron lineage.

Inoue wanted to understand what happened. He and his team inspected samples of Delta infections in Japanese residents and discovered something interesting: mutations in the NSP14 protein, which helps a virus sneak past our immune systems.

Scientists have long assumed that deliberately altering NSP14 in a particular viral lineageusing, say, a special drug on a bunch of patientscould make it harder for that lineage to spread.

But Inoues team assumed that the Delta lineage present in Japan somehow evolved a weaker NSP14 all on its own.

We were literally shocked to see the findings, Inoue told The Japan Times. The Delta variant in Japan was highly transmissible and keeping other variants out. But as the mutations piled up, we believe it eventually became a faulty virus and it was unable to make copies of itself. Considering that the cases havent been increasing, we think that at some point during such mutations it headed straight toward its natural extinction.

In other words, if the theory holds, Delta became widespread in Japan at the same time that it became harder to transmit. A surge in Delta cases crowded out less aggressive lineages then abruptly ended when changes to the NSP14 protein blocked further transmission.

I havent seen any indication that SARS-CoV-2 is running out of evolutionary space.

It would be as though a fast-breeding family of rats moved into your attic, scaring off all other pests. Then all the rats abruptly died, leaving you pest-free.

As an explanation for Japans weirdly low rate of new COVID cases this winter, Inoues theory is a tantalizing one. But it falls apart quickly under close scrutiny, especially considering that Japans high vaccination rate plus widespread masking and a little luck might be all the explanation we need for the countrys current reprieve from infections.

Edwin Michael, an epidemiologist at the Center for Global Health Infectious Disease Research at the University of South Florida, took Inoues theory into account when he mulled Japans low-COVID winter. Initially, it was thought that the Delta variant there accumulated too many mutations during the summer resurgence that were deleterious to its own survival and hence thought to be a self-extinction, Michael told The Daily Beast.

But Michael said he couldnt figure out how a lineage could, at roughly the same time, both lose its transmissibility and become dominant. Inoues theory is hard to reconcile with why such a mutant was able to spread widely such that most of the Japanese population caught this mutant leading to large-scale extinction of the virus, Michael said.

More to the point, viruses as a rule almost never evolve in ways that lead to their own extinction. I have never seen one that is widespread and just goes away, Lawrence Gostin, a Georgetown University global health expert, told The Daily Beast. I cant think of a single example of that ranging from novel influenza to smallpox, polio and childhood diseases. Viruses dont just go extinct.

If thats what Delta did in Japan, it would be truly unprecedented.

Inoues theory itself isnt unprecedented, howeveralthough its very likely wrong. A similar idea gained some traction this spring, starting with a claim in the pages of The New Yorker. Jason McLellan, a structural biologist at the University of Texas at Austin, told the magazine that the novel coronaviruss spike protein, which helps the pathogen to grab onto and infect our cells, could mutate only so many times before it just stopped changing.

Theres just not a lot of space for the spike to continue to change in ways that allow it to evade antibodies but still bind to its receptor, McLellan said. Substitutions that allow the virus to resist antibodies will probably also decrease its affinity for [the receptor].

In his theory, SARS-CoV-2 can either dodge our antibodies or stick to our cells. But it cant do both. It has a finite number of options, McLellan said. And that, he claimed, should doom the virus to lose power over time.

Experts quickly challenged McLellans theory. Were not in a position to forecast that evolutionary changes are indicative of the virus being trapped, cornered or otherwise weakened, Anna Fagre, a University of Colorado microbiologist, told The Daily Beast.

I havent seen any indication that SARS-CoV-2 is running out of evolutionary space, Bloom chimed in.

History proved the skeptics right. Since McLellan projected the novel coronavirus genetic dead-end, the pathogen has produced several major new lineages, each as bad as or worse than the previous. Delta. Omicron. Even a sublineage of Omicron thats hard to distinguish using standard tests.

McLellan defended his spring comments as more nuanced than critics gave them credit for. He told The Daily Beast he was just trying to explain that the novel coronaviruss spike protein has only so many amino acids to choose between as it evolves.

As for the current self-extinction notion, thats outside my area of expertise, McLellan told The Daily Beast.

The coming months seem likely to disprove Inoues self-extinction claim. Everything we know about viruses points that way. We must remember that a virus is not a living thingit depends on infecting cells so it can replicate, Stephanie James, the head of a COVID testing lab at Regis University in Colorado, told The Daily Beast. When it is done with one host, it must be able to transmit to another to keep perpetuating in a community. So it is more advantageous for a virus to be transmissible.

But lets give McLellan and Inoue and any other future proponents of COVIDs purported potential for self-sabotage the benefit of the doubt. Say one lineage or another does something unprecedented and blunders into a genetic dead-end.

Even that wouldnt end the pandemic, James said. Not when there are so many other lineages in circulation. Even if one variant were to become less transmissible, my concern is that this would simply open a niche for another variant to take its place.

All that is to say, its probably a bad idea to sit around waiting for the pandemic to end itself. The smarter approach is the one weve taken all along: Urge people to wear masks, avoid crowds andmost importantlyget vaccinated and boosted.

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Molecular Biology Enzymes, Kits, and Reagents Market to hit US$ 41104.71 Million, Globally, by 2028 at 17.8% CAGR: The Insight Partners – Digital…

Posted: December 24, 2021 at 2:08 am

The molecular biology enzymes, kits, and reagents market was valued at US$ 10,987.17 million in 2020 and it is projected to reach US$ 41,104.71 million by 2028; it is expected to grow at a CAGR of 17.8% from 2021 to 2028.

According The Insight Partners study on Molecular Biology Enzymes, Kits, and Reagents Market Forecast to 2028 COVID-19 Impact and Global Analysis by Product, Application, End User, The growth of the market is attributed to a few key driving factors such as healthy funding for genomics, declining cost of sequencing procedures, and increasing prevalence of chronic diseases. However, the dearth of skilled professionals hinders the market growth.

Biological enzymes play a prominent role in encouraging chemical reactions in biological systems. Molecular biology enzymes are recommended for the preparation of laboratory samples. These reagents are also utilized during various processes such as polymerase chain reaction (PCR), DNA sequencing, and cell analysis.

Get Sample PDF Copy of Molecular Biology Enzymes, Kits, and Reagents Market at: https://www.theinsightpartners.com/sample/TIPHE100001119/

Market Insights

Healthy Funding for Genomics Drives Market Growth

Genome sequencing was restricted to the research facilities, a few years back. However, it has now became one of the common techniques in clinical practices. Over the next few years, the healthcare sector is expected to generate genomic data of over 60 million patients. The growing implementation of genomic sequencing into healthcare systems is supported by substantial government investments, totaling over US$ 4 billion in at least 14 countries. The UK has announced the worlds largest genome project as part of 200 million publicprivate collaboration between charitable organizations and pharmaceutical companies. The country has already developed the worlds largest genome database through the 100,000 Genomes Project. Led by Innovate UK, a part of UK Research and Innovation, the project was initiated to support researchers and industries through funds to combine data and real-world evidence obtained from UK health services as well as to create new products and services for more efficient and early disease diagnosis.

In November 2018, Stilla Technologies, France, announced the completion of a Series A financing round, gathering US$ 18.3 million (EUR 16 million) in funds; the round was led by Illumina Ventures. The company plans to use these funds to commercialization of its Naica digital PCR system and development of clinical applications. Further, in June 2020, Base Genomics, an epigenetics company based in Oxford, England, closed a seed funding round of US$ 11 million (GBP 9 million). In August 2018, Nebula Genomics, a company based in Boston, Massachusetts, along with 10 leading venture capital firms, raised US$ 4.3 million in seed funding. as a part of partnership with Veritas Genetics, a genome sequencing company. The company aims to use these funds to create a trustworthy, secure, and decentralized marketplace for genomic data. In March 2018, SynbiCITE (UK) received grants worth ~US$ 17.6 million (GBP 13 million) and private investments worth US$ 318.5 million (GBP 234 million). The company is made for increasing synbio applications in the UK. The SynbiCITE has supported the synbio at national level for the increase of gene synthesis applications.

In April 2020, MedGenome, a genetic diagnostics, research, and data company headquartered in Bengaluru, India, raised US$ 55 million (~INR 419 crore) in a new round of funding, led by LeapFrog Investments, a global impact investment firm. MedGenome also claims to have built the largest database of South Asian genetic variants in genetic diagnostics in India and research partnerships. It has conducted over 200,000 genomic tests to date by obtaining samples from more than 550 hospitals and 6,000 clinicians across India.

The Molecular Biology Enzymes, Kits, and Reagents Market, by region, is segmented into North America, Europe, Asia Pacific (APAC), Middle East and Africa (MEA), and South and Central America (SAM).

COVID-19 first began in Wuhan (China) during December 2019 and since then it has spread at a fast pace across the globe. The US, India, Brazil, Russia, France, the UK, Turkey, Italy, and Spain are some of the worst affected countries in terms confirmed cases and reported deaths. The COVID-19 has been affecting economies and industries in various countries due to lockdowns, travel bans, and business shutdowns.

Download the Latest COVID-19 Analysis on Molecular Biology Enzymes, Kits, and Reagents Market Growth Research Report at: https://www.theinsightpartners.com/covid-analysis-sample/TIPHE100001119

Based on product, the molecular biology enzymes, kits, and reagents market is bifurcated into enzymes and kits & reagents. The market, by enzymes, is further segmented into polymerases, ligases, reverse transcriptases, phosphatases, proteases and proteinases, restriction endonuclease, and other. The kits & reagent segment held a larger market share in 2020, and the same segment is estimated to register a higher CAGR during the forecast period.

The molecular biology enzymes, kits, and reagents market, based on application, is segmented into epigenetics, sequencing, synthetic biology, polymerase chain reaction, and other. In 2020, the sequencing segment held the largest market share. However, the polymerase chain reaction segment is estimated to register the highest CAGR during the forecast period.

In terms of end user, the molecular biology enzymes, kits, and reagents market is segmented into biotechnological and pharmaceutical companies, hospitals and diagnostic centers, and academic and research institutes. The biotechnological and pharmaceutical companies segment held the largest share of the market in 2020, and the same segment is estimated to register the highest CAGR during the forecast period.

Molecular Biology Enzymes, Kits, and Reagents Market : Competitive Landscape and Key Developments

THERMO FISHER SCIENTIFIC INC,Illumina, Inc.,Qiagen,Agilent Technologies, Inc.,F. HOFFMANN-LA ROCHE LTD,Merck KGAA, Jena Bioscience GmbH,Bioline Technologies,Promega Corporation, Rockland Immunochemicals

Order a Copy of Molecular Biology Enzymes, Kits, and Reagents Market Shares, Strategies and Forecasts 2021-2028 Research Report athttps://www.theinsightpartners.com/buy/TIPHE100001119/

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Molecular Biology Enzymes, Kits, and Reagents Market to hit US$ 41104.71 Million, Globally, by 2028 at 17.8% CAGR: The Insight Partners - Digital...

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How AI and big data are changing healthcare in the Middle East – CIO

Posted: December 24, 2021 at 2:08 am

AI and big data analytics are allowing healthcare providers in the Middle East to make faster, more cost-effective diagnostics, according to a broad cross-section of healthcare professionals. Along with the increasing use of AI and big data, though, security concerns about data privacy are also growing.

AI is one of the fastest growing segments of the global healthcare market today. According to Frost & Sullivan forecasts, it will reach US$6.6 billion by the end of this year. Such growth rates are possible thanks to the huge amounts of data generated by a wide variety of devices, which can be analysed and acted on.

In the Middle East, healthcare professionals attest to the effectiveness and increasing usage of AI.

We started the digital transformation journey last year with the focus on technology being the core to the foundation of the American Hospital. Weve teamed up with some big names like Oracle and Microsoft to build a new level of intelligence when it comes to models, said Ahmad Yahya, CIO of American Hospital Dubai.

The hospitals IT team created a new COVID diagnostics application built on its clinical database and modelling from Cerner. It was customised and validated by the clinical staff at the hospital intensive care unit, and helped identify risk factors for patients, as well as in deciding who would go to the ICU.

Two other AI-based diagnostic applications are aimed at identifying asthma patients and predicting whether emergency patients will go to an in-patient ward.

We are currently working on all these AI models with one of them, the COVID one, having been already validated, while the other two are close to being validated. We are also working on going live early next year with real-time monitoring of patients sentiments, satisfaction, and (hospital) capacity, which can serve to help allocate resources, Yahyah disclosed earlier this year, at the Arab Health 2021 event in Dubai.

With a wealth of historical medical records available for analysis, AI can be helpful in making a diagnosis and choosing an appropriate treatment, providing the doctor with a third opinion, healthcare professionals say. AI applications are able to analyse all available medical information about a specific disease, and find out which treatments and drugs have been the most effective in the entire history of medical practice.

Medicine is a data-rich field in which accuracy is perhaps the most critical factor. The more data that algorithms process, the more accurately and correctly they will be able to formulate conclusions based on them. Meanwhile, different types of technology in use today are generating an increasing volume of health data, according to Massimo Cannizzo, CEO of Gellify, a venture capital company that in October launched a US$50 million fund with management group Azimut to invest in companies providing healthcare and emerging technology in the Middle East.

There are, for example, various wearable devices that are gaining popularity and generating health information, including portable heart rate and blood pressure monitors devices that can continuously monitor your heart rate or blood sugar.

As their cost decreases and the functionality of the already popular fitness bracelets expands, AI-based diagnostic systems will receive even more data on the health of each individual patient, giving the doctor the opportunity to more accurately and efficiently prescribe a treatment plan.

The rise of so-called augmented healthcare is demonstrated by the growth figures of wearables market in the MENA (Middle East and North Africa) region, where 25% of the adult population is expected to be using a wearable device by 2022, according to Cannizzo.

Algorithms and AI models programmes or sets of algorithms that use a set of data to recognise patterns and perform tasks are constantly improving, and this progress is already finding expression in specific applications in the medical field.

AI will not only help make clinical assessments, but will also streamline operations and workflow, according to Kentaro Suzuki, general manager at medical equipment maker Canon Medical. For example, it can shorten the scan time in MRI, Suzuki said.

We believe AI is a tool for the medical profession in order to improve the current capability of the imaging equipment, Suzuki said.

AI applications can analyse medical images and find early signs of a disease that a doctor may not notice. It will never replace humans or directly diagnose patients, Suzuki said. The third opinion that AI can offer, though, is especially relevant for oncological diseases, in which an early start of treatment can significantly improve the prognosis for a speedy recovery.

AI can also change the way we think about genetics, and push the boundaries of genomics research. Recent research shows that an artificial neural network is capable of identifying and detecting patterns in large amounts of genetic data, thus revealing groups and sequences of genes associated with specific diseases.

Since diseases are encoded in the genetic sequence of a person, the ability to understand genetic information at the most detailed level is currently the key to determining how to treat them.

Advances in genomics have been difficult to make due to the complexity of genetic data. Thanks to the ability of AI to classify and analyse a wide range of data in a short period of time, though, practitioners now expect breakthroughs in genomic study.

I think we are all shifting towards more automated work flows and smart technologies, including in genomics by default because we generate large amount of data that is impossible to manually assess and to make sense of, said Prof. Walid Mohammad Abuhammour, clinical molecular geneticist, director of the genomics centre at Al Jalila Childrens Specialty Hospital, and associate professor of genetics at Mohammed bin Rashid University.

AI can, in particular, make headway in helping to cure rare diseases. Some 80% of the so-called rare diseases are genomic, with 50% of them found in children, noted Joshua Symons, director of data strategy at Genomics England.

We want to embed the combination of AI and genomics into routine medical care to approve outcomes and therapies for patients to discover new drugs and improve lives on a national scale, Symons said.

These types of approaches, embedded into routine clinical medicine, will enable decision support systems to determine what the best therapies are for patients, who, for example, now can perform diabetes blood tests at home, Symons said. Technology is evolving that will allow cancer patients to take their own blood sample and have the ctDNA(circulating tumor DNA) analysed to determine whether they are responding to treatment, he said.

As an increasing number of companies apply emerging technologies such as AI and robotics to healthcare, however, concerns about security and patient privacy are on the rise.

We have a lot of companies in digital health and many of them use AI. We also have companies that use surgical robots or manufacture robotics. The number of those companies is increasing, said Marwan Janahi, managing director of Dubai Science Park (DSP), created to position the UAE as a major destination for research and development.

There are more than 400 companies at DSP, with over 4,000 people working there and the healthcare sector is constantly evolving, Janahi said. The role of AI in healthcare is increasingly important, but at the same time healthcare professionals should be very careful about the information concerning patients and how their privacy is secured, he said.

There are a variety of ways to approach security for patient data. For instance, the UAE ICT law that was issued in January 2019 requires patient data to be stored in the country, where there are very strict data protection laws, Janahi said.

The data should be owned by the patient, but the same time there is a need for flexibility because sometimes there is a need to share information with other parts of the world to advance healthcare knowledge, and to get second opinions, Janahi pointed out.

Healthcare officials should take a variety of approaches toward data security, said American Hospitals Yahyah. We use a typical approach when it comes to cybersecurity, like having firewalls, etc. But the weakest link is your people and what is really important is [security] awareness, which is critical to us, he said.

As a preventive measure the hospital does some mock demo attacks to raise awareness among its people within the enterprise, he said.

While healthcare has evolved to be one of humanitys great success stories due to advances in medicine and technology, at the same time it is in a crisis because of challenges it is facing, said Khalid Ghaloua Adine, director of solutions marketing for digital healthcare at Etisalat Digital.

While life expectancy is higher today than ever before, there is a huge shortage of healthcare professionals to cover the demand. There is also a concern on how to manage and control costs. Thats the reason why healthcare professionals seek new technologies that can optimise costs and support human caregivers, he said.

To be prepared for the challenges ahead, there is a need for more data scientists, data governance engineers, and other types of roles. In addition to CIOs, why not a chief AI officer? Adine asked.

Healthcare organisations should look ahead at skill sets they need to have in the future, he suggested.

For the last 20 years we have been generating data that will need a huge number of people to process it. If we dont have people with the right set of skills to interpret and model that information then we will not be able to overcome those challenges, Adine said.

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How AI and big data are changing healthcare in the Middle East - CIO

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Stem-cell therapy – Wikipedia

Posted: December 24, 2021 at 2:06 am

This article is about the medical therapy. For the cell type, see Stem cell.

Use of stem cells to treat or prevent a disease or condition

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition.[1] As of 2016[update], the only established therapy using stem cells is hematopoietic stem cell transplantation.[2] This usually takes the form of a bone-marrow transplantation, but the cells can also be derived from umbilical cord blood. Research is underway to develop various sources for stem cells as well as to apply stem-cell treatments for neurodegenerative diseases[3] and conditions such as diabetes and heart disease.

Stem-cell therapy has become controversial following developments such as the ability of scientists to isolate and culture embryonic stem cells, to create stem cells using somatic cell nuclear transfer and their use of techniques to create induced pluripotent stem cells. This controversy is often related to abortion politics and to human cloning. Additionally, efforts to market treatments based on transplant of stored umbilical cord blood have been controversial.

For over 30 years, hematopoietic stem cell transplantation (HSCT) has been used to treat people with conditions such as leukaemia and lymphoma; this is the only widely practiced form of stem-cell therapy.[4][5][6] During chemotherapy, most growing cells are killed by the cytotoxic agents. These agents, however, cannot discriminate between the leukaemia or neoplastic cells, and the hematopoietic stem cells within the bone marrow. This is the side effect of conventional chemotherapy strategies that the stem-cell transplant attempts to reverse; a donor's healthy bone marrow reintroduces functional stem cells to replace the cells lost in the host's body during treatment. The transplanted cells also generate an immune response that helps to kill off the cancer cells; this process can go too far, however, leading to graft vs host disease, the most serious side effect of this treatment.[7]

Another stem-cell therapy, called Prochymal, was conditionally approved in Canada in 2012 for the management of acute graft-vs-host disease in children who are unresponsive to steroids.[8] It is an allogenic stem therapy based on mesenchymal stem cells (MSCs) derived from the bone marrow of adult donors. MSCs are purified from the marrow, cultured and packaged, with up to 10,000 doses derived from a single donor. The doses are stored frozen until needed.[9]

The FDA has approved five hematopoietic stem-cell products derived from umbilical-cord blood, for the treatment of blood and immunological diseases.[10]

In 2014, the European Medicines Agency recommended approval of limbal stem cells for people with severe limbal stem cell deficiency due to burns in the eye.[11]

Stem cells are being studied for a number of reasons. The molecules and exosomes released from stem cells are also being studied in an effort to make medications.[12] In addition to the functions of the cells themselves, paracrine soluble factors produced by stem cells, known as the stem cell secretome, have been found to be another mechanism by which stem cell-based therapies mediate their effects in degenerative, autoimmune, and inflammatory diseases.[13]

To be used for research or treatment applications, large numbers of high-quality stem cells are needed. Thus, it is necessary to develop culture systems which produce pure populations of tissue-specific stem-cells in vitro without the loss of stem-cell potential. Two main approaches are taken for this purpose: two-dimensional and three-dimensional cell culture.[14]

Cell culture in two dimensions has been routinely performed in thousands of laboratories worldwide for the past four decades. In two-dimensional platforms, cells are typically exposed to a solid, rigid flat surface on the basal side and to liquid at the apicalsurface. Inhabiting such a two-dimensional rigid substrate requires a dramatic adaption for the surviving cells because they lack the extracellular matrix that is unique to each cell type and which may alter cell metabolism and reduce its functionality.[14]

Three-dimensional cell culture systems may create a biomimicking microenvironment for stem cells, resembling their native three-dimensional extracellular matrix (ECM). Advanced biomaterials have significantly contributed to three-dimensional cell culture systems in recent decades, and more unique and complex biomaterials have been proposed for improving stem-cell proliferation and controlled differentiation. Among them, nanostructured biomaterials are of particular interest because they have the advantage of a high surface-to-volume ratio, and they mimic the physical and biological features of natural ECM at the nanoscale.[14]

Research has been conducted on the effects of stem cells on animal models of brain degeneration, such as in Parkinson's disease, Amyotrophic lateral sclerosis, and Alzheimer's disease.[15][16][17] Preliminary studies related to multiple sclerosis have been conducted,[18][19][20] and a 2020 phase 2 trial found significantly improved outcomes for mesenchymal stem cell treated patients compared to those receiving a sham treatment.[21] In January 2021 the FDA approved the first clinical trial for an investigational stem cell therapy to restore lost brain cells in people with advanced Parkinsons disease.[22]

Healthy adult brains contain neural stem cells, which divide to maintain general stem-cell numbers, or become progenitor cells. In healthy adult laboratory animals, progenitor cells migrate within the brain and function primarily to maintain neuron populations for olfaction (the sense of smell). Pharmacological activation of endogenous neural stem cells has been reported to induce neuroprotection and behavioral recovery in adult rat models of neurological disorder.[23][24][25]

Stroke and traumatic brain injury lead to cell death, characterized by a loss of neurons and oligodendrocytes within the brain. Clinical and animal studies have been conducted into the use of stem cells in cases of spinal cord injury.[26][27][28][20]

A small-scale study on individuals 60 year or older with aging frailty showed, after intravenous treatment with MSCs from healthy young donors, showed significant improvements in physical performance measures.[29]

Stem cells are studied in people with severe heart disease.[30] The work by Bodo-Eckehard Strauer[31] was discredited by identifying hundreds of factual contradictions.[32] Among several clinical trials reporting that adult stem cell therapy is safe and effective, actual evidence of benefit has been reported from only a few studies.[33] Some preliminary clinical trials achieved only modest improvements in heart function following use of bone marrow stem cell therapy.[34][35]

Stem-cell therapy for treatment of myocardial infarction usually makes use of autologous bone-marrow stem cells, but other types of adult stem cells may be used, such as adipose-derived stem cells.[36]

Possible mechanisms of recovery include:[15]

In 2013, studies of autologous bone-marrow stem cells on ventricular function were found to contain "hundreds" of discrepancies.[37] Critics report that of 48 reports, just five underlying trials seemed to be used, and that in many cases whether they were randomized or merely observational accepter-versus-rejecter, was contradictory between reports of the same trial. One pair of reports of identical baseline characteristics and final results, was presented in two publications as, respectively, a 578-patient randomized trial and as a 391-subject observational study. Other reports required (impossible) negative standard deviations in subsets of people, or contained fractional subjects, negative NYHA classes. Overall, many more people were reported as having receiving stem cells in trials, than the number of stem cells processed in the hospital's laboratory during that time. A university investigation, closed in 2012 without reporting, was reopened in July 2013.[38]

In 2014, a meta-analysis on stem cell therapy using bone-marrow stem cells for heart disease revealed discrepancies in published clinical trial reports, whereby studies with a higher number of discrepancies showed an increase in effect sizes.[39] Another meta-analysis based on the intra-subject data of 12 randomized trials was unable to find any significant benefits of stem cell therapy on primary endpoints, such as major adverse events or increase in heart function measures, concluding there was no benefit.[40]

The TIME trial, which used a randomized, double-blind, placebo-controlled trial design, concluded that "bone marrow mononuclear cells administration did not improve recovery of LV function over 2 years" in people who had a myocardial infarction.[41] Accordingly, the BOOST-2 trial conducted in 10 medical centers in Germany and Norway reported that the trial result "does not support the use of nucleated BMCs in patients with STEMI and moderately reduced LVEF".[42] Furthermore, the trial also did not meet any other secondary MRI endpoints,[43] leading to a conclusion that intracoronary bone marrow stem cell therapy does not offer a functional or clinical benefit.[44]

The specificity of the human immune-cell repertoire is what allows the human body to defend itself from rapidly adapting antigens. However, the immune system is vulnerable to degradation upon the pathogenesis of disease, and because of the critical role that it plays in overall defense, its degradation is often fatal to the organism as a whole. Diseases of hematopoietic cells are diagnosed and classified via a subspecialty of pathology known as hematopathology. The specificity of the immune cells is what allows recognition of foreign antigens, causing further challenges in the treatment of immune disease. Identical matches between donor and recipient must be made for successful transplantation treatments, but matches are uncommon, even between first-degree relatives. Research using both hematopoietic adult stem cells and embryonic stem cells has provided insight into the possible mechanisms and methods of treatment for many of these ailments.[45]

Fully mature human red blood cells may be generated ex vivo by hematopoietic stem cells (HSCs), which are precursors of red blood cells. In this process, HSCs are grown together with stromal cells, creating an environment that mimics the conditions of bone marrow, the natural site of red-blood-cell growth. Erythropoietin, a growth factor, is added, coaxing the stem cells to complete terminal differentiation into red blood cells.[46] Further research into this technique should have potential benefits to gene therapy, blood transfusion, and topical medicine.

In 2004, scientists at King's College London discovered a way to cultivate a complete tooth in mice[47] and were able to grow bioengineered teeth stand-alone in the laboratory. Researchers are confident that the tooth regeneration technology can be used to grow live teeth in people.

In theory, stem cells taken from the patient could be coaxed in the lab turning into a tooth bud which, when implanted in the gums, will give rise to a new tooth, and would be expected to be grown in a time over three weeks.[48] It will fuse with the jawbone and release chemicals that encourage nerves and blood vessels to connect with it. The process is similar to what happens when humans grow their original adult teeth. Many challenges remain, however, before stem cells could be a choice for the replacement of missing teeth in the future.[49][50]

Heller has reported success in re-growing cochlea hair cells with the use of embryonic stem cells.[51]

In a 2019 review that looked at hearing regeneration and regenerative medicine, stem cell-derived otic progenitors have the potential to greatly improve hearing.[52]

Since 2003, researchers have successfully transplanted corneal stem cells into damaged eyes to restore vision. "Sheets of retinal cells used by the team are harvested from aborted fetuses, which some people find objectionable." When these sheets are transplanted over the damaged cornea, the stem cells stimulate renewed repair, eventually restore vision.[53] The latest such development was in June 2005, when researchers at the Queen Victoria Hospital of Sussex, England were able to restore the sight of forty people using the same technique. The group, led by Sheraz Daya, was able to successfully use adult stem cells obtained from the patient, a relative, or even a cadaver. Further rounds of trials are ongoing.[54]

People with Type 1 diabetes lose the function of insulin-producing beta cells within the pancreas.[55] In recent experiments, scientists have been able to coax embryonic stem cell to turn into beta cells in the lab. In theory if the beta cell is transplanted successfully, they will be able to replace malfunctioning ones in a diabetic patient.[56]

Use of mesenchymal stem cells (MSCs) derived from adult stem cells is under preliminary research for potential orthopedic applications in bone and muscle trauma, cartilage repair, osteoarthritis, intervertebral disc surgery, rotator cuff surgery, and musculoskeletal disorders, among others.[57] Other areas of orthopedic research for uses of MSCs include tissue engineering and regenerative medicine.[57]

Stem cells can also be used to stimulate the growth of human tissues. In an adult, wounded tissue is most often replaced by scar tissue, which is characterized in the skin by disorganized collagen structure, loss of hair follicles and irregular vascular structure. In the case of wounded fetal tissue, however, wounded tissue is replaced with normal tissue through the activity of stem cells.[58] A possible method for tissue regeneration in adults is to place adult stem cell "seeds" inside a tissue bed "soil" in a wound bed and allow the stem cells to stimulate differentiation in the tissue bed cells. This method elicits a regenerative response more similar to fetal wound-healing than adult scar tissue formation.[58] Researchers are still investigating different aspects of the "soil" tissue that are conducive to regeneration.[58] Because of the general healing capabilities of stem cells, they have gained interest for the treatment of cutaneous wounds, such as in skin cancer.[59]

Destruction of the immune system by the HIV is driven by the loss of CD4+ T cells in the peripheral blood and lymphoid tissues. Viral entry into CD4+ cells is mediated by the interaction with a cellular chemokine receptor, the most common of which are CCR5 and CXCR4. Because subsequent viral replication requires cellular gene expression processes, activated CD4+ cells are the primary targets of productive HIV infection.[60] Recently scientists have been investigating an alternative approach to treating HIV-1/AIDS, based on the creation of a disease-resistant immune system through transplantation of autologous, gene-modified (HIV-1-resistant) hematopoietic stem and progenitor cells (GM-HSPC).[61]

Stem cells are thought to mediate repair via five primary mechanisms: 1) providing an anti-inflammatory effect, 2) homing to damaged tissues and recruiting other cells, such as endothelial progenitor cells, that are necessary for tissue growth, 3) supporting tissue remodeling over scar formation, 4) inhibiting apoptosis, and 5) differentiating into bone, cartilage, tendon, and ligament tissue.[62][63]

To further enrich blood supply to the damaged areas, and consequently promote tissue regeneration, platelet-rich plasma could be used in conjunction with stem cell transplantation.[64][65] The efficacy of some stem cell populations may also be affected by the method of delivery; for instance, to regenerate bone, stem cells are often introduced in a scaffold where they produce the minerals necessary for generation of functional bone.[64][65][66][67]

Stem cells have also been shown to have a low immunogenicity due to the relatively low number of MHC molecules found on their surface. In addition, they have been found to secrete chemokines that alter the immune response and promote tolerance of the new tissue. This allows for allogeneic treatments to be performed without a high rejection risk.[68]

The ability to grow up functional adult tissues indefinitely in culture through Directed differentiation creates new opportunities for drug research. Researchers are able to grow up differentiated cell lines and then test new drugs on each cell type to examine possible interactions in vitro before performing in vivo studies. This is critical in the development of drugs for use in veterinary research because of the possibilities of species-specific interactions. The hope is that having these cell lines available for research use will reduce the need for research animals used because effects on human tissue in vitro will provide insight not normally known before the animal testing phase.[69]

Stem cells are being explored for use in conservation efforts. Spermatogonial stem cells have been harvested from a rat and placed into a mouse host and fully mature sperm were produced with the ability to produce viable offspring. Currently research is underway to find suitable hosts for the introduction of donor spermatogonial stem cells. If this becomes a viable option for conservationists, sperm can be produced from high genetic quality individuals who die before reaching sexual maturity, preserving a line that would otherwise be lost.[70]

Most stem cells intended for regenerative therapy are generally isolated either from the patient's bone marrow or from adipose tissue.[65][67] Mesenchymal stem cells can differentiate into the cells that make up bone, cartilage, tendons, and ligaments, as well as muscle, neural and other progenitor tissues. They have been the main type of stem cells studied in the treatment of diseases affecting these tissues.[71][72] The number of stem cells transplanted into damaged tissue may alter the efficacy of treatment. Accordingly, stem cells derived from bone marrow aspirates, for instance, are cultured in specialized laboratories for expansion to millions of cells.[65][67] Although adipose-derived tissue also requires processing prior to use, the culturing methodology for adipose-derived stem cells is not as extensive as that for bone marrow-derived cells.[73] While it is thought that bone-marrow-derived stem cells are preferred for bone, cartilage, ligament, and tendon repair, others believe that the less challenging collection techniques and the multi-cellular microenvironment already present in adipose-derived stem cell fractions make the latter the preferred source for autologous transplantation.[64]

New sources of mesenchymal stem cells are being researched, including stem cells present in the skin and dermis which are of interest because of the ease at which they can be harvested with minimal risk to the animal.[74] Hematopoietic stem cells have also been discovered to be travelling in the blood stream and possess equal differentiating ability as other mesenchymal stem cells, again with a very non-invasive harvesting technique.[75]

There has been more recent interest in the use of extra embryonic mesenchymal stem cells. Research is underway to examine the differentiating capabilities of stem cells found in the umbilical cord, yolk sac and placenta of different animals. These stem cells are thought to have more differentiating ability than their adult counterparts, including the ability to more readily form tissues of endodermal and ectodermal origin.[68]

There is widespread controversy over the use of human embryonic stem cells. This controversy primarily targets the techniques used to derive new embryonic stem cell lines, which often requires the destruction of the blastocyst. Opposition to the use of human embryonic stem cells in research is often based on philosophical, moral, or religious objections.[76] There is other stem cell research that does not involve the destruction of a human embryo, and such research involves adult stem cells, amniotic stem cells, and induced pluripotent stem cells.

On 23 January 2009, the US Food and Drug Administration gave clearance to Geron Corporation for the initiation of the first clinical trial of an embryonic stem-cell-based therapy on humans. The trial aimed to evaluate the drug GRNOPC1, embryonic stem cell-derived oligodendrocyte progenitor cells, on people with acute spinal cord injury. The trial was discontinued in November 2011 so that the company could focus on therapies in the "current environment of capital scarcity and uncertain economic conditions".[77] In 2013 biotechnology and regenerative medicine company BioTime (AMEX:BTX) acquired Geron's stem cell assets in a stock transaction, with the aim of restarting the clinical trial.[78]

Scientists have reported that MSCs when transfused immediately within few hours post thawing may show reduced function or show decreased efficacy in treating diseases as compared to those MSCs which are in log phase of cell growth (fresh), so cryopreserved MSCs should be brought back into log phase of cell growth in invitro culture before administration. Re-culturing of MSCs will help in recovering from the shock the cells get during freezing and thawing. Various MSC clinical trials which used cryopreserved product immediately post thaw have failed as compared to those clinical trials which used fresh MSCs.[79]

Research has been conducted on horses, dogs, and cats can benefit the development of stem cell treatments in veterinary medicine and can target a wide range of injuries and diseases such as myocardial infarction, stroke, tendon and ligament damage, osteoarthritis, osteochondrosis and muscular dystrophy both in large animals, as well as humans.[80][81][82][83] While investigation of cell-based therapeutics generally reflects human medical needs, the high degree of frequency and severity of certain injuries in racehorses has put veterinary medicine at the forefront of this novel regenerative approach.[84] Companion animals can serve as clinically relevant models that closely mimic human disease.[85][86]

Veterinary applications of stem cell therapy as a means of tissue regeneration have been largely shaped by research that began with the use of adult-derived mesenchymal stem cells to treat animals with injuries or defects affecting bone, cartilage, ligaments and/or tendons.[87][71][88] There are two main categories of stem cells used for treatments: allogeneic stem cells derived from a genetically different donor within the same species[67][89] and autologous mesenchymal stem cells, derived from the patient prior to use in various treatments.[64] A third category, xenogenic stem cells, or stem cells derived from different species, are used primarily for research purposes, especially for human treatments.[69]

Bone has a unique and well documented natural healing process that normally is sufficient to repair fractures and other common injuries. Misaligned breaks due to severe trauma, as well as treatments like tumor resections of bone cancer, are prone to improper healing if left to the natural process alone. Scaffolds composed of natural and artificial components are seeded with mesenchymal stem cells and placed in the defect. Within four weeks of placing the scaffold, newly formed bone begins to integrate with the old bone and within 32 weeks, full union is achieved.[90] Further studies are necessary to fully characterize the use of cell-based therapeutics for treatment of bone fractures.

Stem cells have been used to treat degenerative bone diseases. The normally recommended treatment for dogs that have LeggCalvePerthes disease is to remove the head of the femur after the degeneration has progressed. Recently, mesenchymal stem cells have been injected directly in to the head of the femur, with success not only in bone regeneration, but also in pain reduction.[90]

Autologous stem cell-based treatments for ligament injury, tendon injury, osteoarthritis, osteochondrosis, and sub-chondral bone cysts have been commercially available to practicing veterinarians to treat horses since 2003 in the United States and since 2006 in the United Kingdom. Autologous stem cell based treatments for tendon injury, ligament injury, and osteoarthritis in dogs have been available to veterinarians in the United States since 2005. Over 3000 privately owned horses and dogs have been treated with autologous adipose-derived stem cells. The efficacy of these treatments has been shown in double-blind clinical trials for dogs with osteoarthritis of the hip and elbow and horses with tendon damage.[91][92]

Race horses are especially prone to injuries of the tendon and ligaments. Conventional therapies are very unsuccessful in returning the horse to full functioning potential. Natural healing, guided by the conventional treatments, leads to the formation of fibrous scar tissue that reduces flexibility and full joint movement. Traditional treatments prevented a large number of horses from returning to full activity and also have a high incidence of re-injury due to the stiff nature of the scarred tendon. Introduction of both bone marrow and adipose derived stem cells, along with natural mechanical stimulus promoted the regeneration of tendon tissue. The natural movement promoted the alignment of the new fibers and tendocytes with the natural alignment found in uninjured tendons. Stem cell treatment not only allowed more horses to return to full duty and also greatly reduced the re-injury rate over a three-year period.[68]

The use of embryonic stem cells has also been applied to tendon repair. The embryonic stem cells were shown to have a better survival rate in the tendon as well as better migrating capabilities to reach all areas of damaged tendon. The overall repair quality was also higher, with better tendon architecture and collagen formed. There was also no tumor formation seen during the three-month experimental period. Long-term studies need to be carried out to examine the long-term efficacy and risks associated with the use of embryonic stem cells.[68] Similar results have been found in small animals.[68]

Osteoarthritis is the main cause of joint pain both in animals and humans. Horses and dogs are most frequently affected by arthritis. Natural cartilage regeneration is very limited. Different types of mesenchymal stem cells and other additives are still being researched to find the best type of cell and method for long-term treatment.[68]

Adipose-derived mesenchymal cells are currently the most often used for stem cell treatment of osteoarthritis because of the non-invasive harvesting. This is a recently developed, non-invasive technique developed for easier clinical use. Dogs receiving this treatment showed greater flexibility in their joints and less pain.[93]

Stem cells have successfully been used to ameliorate healing in the heart after myocardial infarction in dogs. Adipose and bone marrow derived stem cells were removed and induced to a cardiac cell fate before being injected into the heart. The heart was found to have improved contractility and a reduction in the damaged area four weeks after the stem cells were applied.[94]

A different trial is underway for a patch made of a porous substance onto which the stem cells are "seeded" in order to induce tissue regeneration in heart defects. Tissue was regenerated and the patch was well incorporated into the heart tissue. This is thought to be due, in part, to improved angiogenesis and reduction of inflammation. Although cardiomyocytes were produced from the mesenchymal stem cells, they did not appear to be contractile. Other treatments that induced a cardiac fate in the cells before transplanting had greater success at creating contractile heart tissue.[95]

Recent research, such as the European nTRACK research project, aims to demonstrate that multimodal nanoparticles can structurally and functionally track stem cell in muscle regeneration therapy. The idea is to label stem cells with gold nano-particles that are fully characterised for uptake, functionality, and safety. The labelled stem cells will be injected into an injured muscle and tracked using imaging systems.[96] However, the system still needs to be demonstrated at lab scale.

Spinal cord injuries are one of the most common traumas brought into veterinary hospitals.[90] Spinal injuries occur in two ways after the trauma: the primary mechanical damage, and in secondary processes, like inflammation and scar formation, in the days following the trauma. These cells involved in the secondary damage response secrete factors that promote scar formation and inhibit cellular regeneration. Mesenchymal stem cells that are induced to a neural cell fate are loaded onto a porous scaffold and are then implanted at the site of injury. The cells and scaffold secrete factors that counteract those secreted by scar forming cells and promote neural regeneration. Eight weeks later, dogs treated with stem cells showed immense improvement over those treated with conventional therapies. Dogs treated with stem cells were able to occasionally support their own weight, which has not been seen in dogs undergoing conventional therapies.[97][98][99]

In a study to evaluate the treatment of experimentally induced MS in dogs using laser activated non-expanded adipose derived stem cells. The results showed amelioration of the clinical signs over time confirmed by the resolution of the previous lesions on MRI. Positive migration of the injected cells to the site of lesion, increased remyelination detected by Myelin Basic Proteins, positive differentiation into Olig2 positive oligodendrocytes, prevented the glial scar formation and restored axonal architecture.[20]

Treatments are also in clinical trials to repair and regenerate peripheral nerves. Peripheral nerves are more likely to be damaged, but the effects of the damage are not as widespread as seen in injuries to the spinal cord. Treatments are currently in clinical trials to repair severed nerves, with early success. Stem cells induced to a neural fate injected in to a severed nerve. Within four weeks, regeneration of previously damaged stem cells and completely formed nerve bundles were observed.[74]

Stem cells are also in clinical phases for treatment in ophthalmology. Hematopoietic stem cells have been used to treat corneal ulcers of different origin of several horses. These ulcers were resistant to conventional treatments available, but quickly responded positively to the stem cell treatment. Stem cells were also able to restore sight in one eye of a horse with retinal detachment, allowing the horse to return to daily activities.[75]

In the late 1990s and early 2000s, there was an initial wave of companies and clinics offering stem cell therapy, while not substantiating health claims or having regulatory approval.[100] By 2012, a second wave of companies and clinics had emerged, usually located in developing countries where medicine is less regulated and offering stem cell therapies on a medical tourism model.[101][102] Like the first wave companies and clinics, they made similar strong, but unsubstantiated, claims, mainly by clinics in the United States, Mexico, Thailand, India, and South Africa.[101][102] By 2016, research indicated that there were more than 550 stem cell clinics in the US alone selling generally unproven therapies for a wide array of medical conditions in almost every state in the country,[103] altering the dynamic of stem cell tourism. In 2018, the FDA sent a warning letter to StemGenex Biologic Laboratories in San Diego, which marketed a service in which it took body fat from people, processed it into mixtures it said contained various forms of stem cells, and administered it back to the person by inhalation, intravenously, or infusion into their spinal cords; the company said the treatment was useful for many chronic and life-threatening conditions.[104]

Costs of stem cell therapies range widely by clinic, condition, and cell type, but most commonly range between $10,000-$20,000.[105] Insurance does not cover stem cell injections at clinics so patients often use on-line fundraising.[106] In 2018, the US Federal Trade Commission found health centers and an individual physician making unsubstantiated claims for stem cell therapies, and forced refunds of some $500,000.[107] The FDA filed suit against two stem cell clinic firms around the same time, seeking permanent injunctions against their marketing and use of unapproved adipose stem cell products.[108]

Although according to the NIH no stem cell treatments have been approved for COVID-19 and the agency recommends against the use of MSCs for the disease,[109] some stem cell clinics began marketing both unproven and non-FDA-approved stem cells and exosomes for COVID-19 in 2020.[110] The FDA took prompt action by sending letters to the firms in question.[111][112] The FTC also warned a stem cell firm for COVID-19-related marketing.[113][114]

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Omicron is here, and many prisons and jails are not ready – The Marshall Project

Posted: December 24, 2021 at 2:03 am

In the Philadelphia jail, the number of COVID-19 cases has tripled in the last two months. In Chicagos lockup, infections have increased 11-fold in the same period. And in New York, city jails are struggling with a mushrooming 13-fold increase in less than a month.

From local lockups in California to prisons in Wisconsin to jails in Pennsylvania, COVID-19 is once again surging behind bars, posing a renewed threat to a high-risk population with spotty access to healthcare and little ability to distance.

At this point its unclear whether the surge in infections is due to the highly contagious omicron variant. Still, as caseloads across the country skyrocket and omicron becomes the dominant variant, experts worry the coronavirus is once again poised to sweep through jails and prisons. As in the world outside prison bars, many incarcerated people are struggling with pandemic fatigue. Theyre also facing uncertain access to booster shots, widespread vaccine hesitancy and pandemic-driven staffing shortfalls that have created even harsher conditions.

As with previous iterations of the virus, everything about prisons and jails makes them a setup to magnify the harms of omicron. The overcrowding. The poor sanitary conditions. The lack of access to health care, said Monik Jimenez, an epidemiologist at Harvards School of Public Health. Masking is only going to do so much when you have people on top of you.

Though scientists warn that the new variant is far more contagious than previous ones, a half dozen prisoners who spoke with The Marshall Project for this story said they hadnt noticed any widespread concern about it at this point and that prison officials had given them little information.

They're not telling us anything about omicron or anything else for that matter, wrote Rachel Padgett, a federal prisoner in Florida. Many pandemic-weary prisoners said they were less concerned about catching the virus than about being locked down because of it, once again facing months confined to their cells and bunks with no way to call home, see their families or go outside.

Thats the only part people are worried about these days taking away our rec, said a man incarcerated in a federal prison in Mississippi who asked not to be named for fear of retaliation from prison officials.

For some, its hard to get worried again considering how bad things got in some prisons before. John J. Lennon recalled the end of last year when the virus seemed particularly relentless, making the people incarcerated with him in upstate New York fearful and anxious. All thats changed. There is no sense of urgency about it, he said.

I havent seen watery eyes coming off the phones anymore. There arent ambulances coming in and out, said Lennon, a journalist who is a contributing writer to the Marshall Project and Esquire. There was a time when thats what I saw every day.

Though some early reports suggested that omicron may cause less severe disease, there are also indications that the new variant is better able to evade vaccines which means that access to booster shots is even more important. But there is little good public information about how widely boosters are available to incarcerated people or how widespread booster uptake is among correctional staff, many of whom resisted vaccination in the first place.

Though officials in more than half a dozen prison systems including New York, Texas and Arkansas said theyd offered booster shots to prisoners, not all were able to specify how many received them so far. In Nebraska, inspector general Doug Koebernick said prisoners have only been offered access to the Johnson & Johnson booster shot, which early research suggests is the least effective against the new variant.

The CDC recently said the 2-dose Pfizer and Moderna vaccines are preferred over the Johnson & Johnson. Homer Venters, a former New York City jail medical director and correctional healthcare consultant, said that makes the coming months a lot trickier for the people who are trying to engage with people behind bars and promote vaccination, which is crucial to prevent omicron.

Aside from the lack of data about booster availability, many states that routinely released real-time data about infections and vaccinations in the first year of the pandemic are now releasing information monthly or not at all, said Lauren Brinkley-Rubinstein, an associate professor and co-founder of the COVID Prison Project at the University of North Carolina School of Medicine. We basically know nothing, and its really disheartening in that we have less and less data every day, she said.

Even so, the ripple effects of the mutating virus could soon become clear.

If the slope of the curve with omicron is as steep as we fear it is, we may have some really devastating staff shortages, said Venters, who has been performing court-ordered COVID-19 prison inspections since the beginning of the pandemic. With large numbers of staff unavailable to work very quickly, you cant actually do any of the jobs of running the facilities. Venters said to expect large scale lockdowns or very serious security consequences.

For several prison systems, the dwindling number of guards has already been a problem. In Texas, longstanding staff shortages exacerbated by the pandemic have created unsafe conditions for prisoners and workers alike. For the first time in recent memory, the Texas agency that runs state prisons is down more than 7,200 officers, leaving several facilities at less than half-staff and relying on overtime. Jeff Ormsby, executive director for the states biggest union representing corrections employees, said officers are more likely to die from a car wreck going home from a 24-hour shift than to die from omicron.

Meanwhile, populations of people in prison and jail have crept back toward pre-pandemic levels in many places, after early efforts to keep as many people out of jail as possible and to release people from prisons en masse as a tactic to stem the spread of coronavirus. By the middle of last year, the number of people in jails nationwide was down by roughly one-quarter its lowest point in more than two decades. And, from 2019 to 2020, the number of new prisoners admitted to state and federal prisons went down by 40%.

Now, courthouses are slogging through the backlog of cases that accumulated while they were closed, leading to many new people entering the system. Systems set up to process plea bargains have returned to business as usual. And new spikes in certain violent crimes and public fears of violence have put pressure on city leaders across the country to call for additional policing.

So with omicron poised to sweep through the nations lockups, they are increasingly crowded a dangerous setup for the viruss spread, said Jaimie Meyer, an epidemiologist and infectious disease doctor at Yale medical school. Were looking at another potential tinderbox scenario.

Staff writers Weihua Li and Katie Park contributed to this story.

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A look at prospects for the US gene therapy industry – BioPharma-Reporter.com

Posted: December 24, 2021 at 2:01 am

Today, you could estimate that one family per day is being treated with and impacted by gene therapy. We want to see that increase to 10s, 100s, 1,000s per day, and reaching that goal comes from investing in research, clinical trials and manufacturing, said Ken Mills, CEO of clinical stage US biotechnology company, Regenxbio.

Regenxbio, said its CEO, has played a pivotal role in the gene therapy industry since its founding in 2008, as a result of research from the lab of gene therapy pioneer, Jim Wilson, University of Pennsylvania.

The company's NAV technology platform consists of over 100 novel adeno-associated virus (AAV) vectors, one of which was used in the US Food and Drug Administration (FDA) approved gene therapy, ZOLGENSMA, for spinal muscular atrophy in children under two years old.

The developer has also licensed out its technology to a growing list of partners and licensees that includes Novartis, Eli Lily and Pfizer and has a robust in-house pipeline of candidates for retinal and rare diseases.

Looking at the US gene therapy regulatory landscape, what are the current roadblocks?

AAV-mediated gene therapies offer the possibility of a one-time administration that could address the underlying disease and change the way critical medicine is delivered to patients, but the regulatory landscape has not evolved as quickly as the innovations of AAV gene therapy.

Weve seen that the FDA is open to working with industry and gene therapy stakeholders to determine the appropriate path forward, to streamline clinical development and get medicines to patients faster. Leveraging accelerated approvals and surrogate endpoints in clinical trials, such as a biomarker, may play a large role, Mills told BioPharma-Reporter.

The Pathway Development Consortium (PDC) launched in 2021 by Regenxbio and Solid Biosciences is working to advance opportunities to leverage the FDAs accelerated approval pathway for gene therapy candidates.

Our mission to bring together patients, industry, regulators, academia, payers and other stakeholders for meaningful scientific and policy discussion, said the CEO.

There is a significant strain on manufacturing capabilities in the gene therapy sector both capacity and reproducibility, but more importantly, talent, commented Mills.

As the sector has grown rapidly and expanded broadly, we have seen these rate-limiting factors continue to persist. In addition, significant strain on supply chains is likely to continue into 2022 and will impact pharma and biotech. Consistent, reliable manufacturing is critical to gene therapy trial development, product approval, and commercialization, so it is crucial to overcome these capabilities challenges.

Contract development and manufacturing organizations (CDMOs) are critical partners, said the executive, and he anticipates continued investment in manufacturing capabilities through in-house facilities and CDMOs next year.

And as the field continues to advance, we will start to see more and more efficiencies that companies like Regenxbio can capitalize upon to allow for rapid manufacturing and formulation development.

Regenxbio has invested in the establishment of a robust suspension cell culture-based manufacturing process and new manufacturing facility at its headquarters in Rockville, Maryland.

We have also invested to ensure the hiring of the right people to make this possible. Five to 10 years ago, you did not see a lot of process development teams, and now they are crucial to drive the scalability of capabilities across clinical and commercial strategy.

Through the expansion of its expert manufacturing team and facility build out, he said the companys researchers and process development team have been able to work side by side to mitigate potential issues early in the development process.

The goal is always to get therapies approved and to patients as quickly as possible, and a reliable, scalable chemistry, manufacturing and controls (CMC) process is crucial in accomplishing this, said Mills.

Our philosophy initially was to develop the best process platform that could be utilized across multiple programs with a highly similar process that could be easily transferred to a CDMO. We also have a platform downstream process developed that works across our programs, giving consistent downstream yields that are appropriate for the current phase of development.

We have developed proprietary formulations that are indication-specific. The formulations are stable at the intended storage conditions over several years and we have ongoing monitoring of product quality during that period to ensure consistent performance.

In terms of the highlights for the biotech this year, Mills said it was a fast paced, high-achieving 12 months for the company.

In September, we announced a partnership with AbbVie to develop and commercialize RGX-314, our gene therapy for the treatment of wet AMD, diabetic retinopathy, and other chronic retinal diseases. Under the terms of the agreement, AbbVie will provide Regenxbio a US$370m upfront payment with the potential for the company to receive up to US$1.38bn in additional development, regulatory and commercial milestones.

We are currently running a pivotal program of RGX-314 for the treatment of wet AMD, and we expect to file a BLA in 2024. We are also conducting additional trials evaluating RGX-314 delivered directly to the suprachoroidal space of the eye for the treatment of wet AMD and diabetic retinopathy. In 2021, we reported initial data from both of those trials.

Regenxbio also announced, early in 2021, a new pipeline candidate for treating Duchenne muscular dystrophy (Duchenne) - RGX-202. That is designed, said Mills, to deliver an optimized microdystrophin transgene with a unique C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne.

We received Orphan Drug Designation from the FDA in November and shared that we expect to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021.

Commercial-scale cGMP material has already been produced at 1,000 liter capacity using our suspension cell culture manufacturing process, and the company's internal cGMP facility is expected to allow for production up to 2,000 liters for the clinical development of RGX-202.

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