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Cell and gene therapy services market forecast to see significant growth over the next decade – BioPharma-Reporter.com

April 17th, 2021 1:52 am

Roots Analysis has released its latest overview on theCell and Gene Therapy CROs Market (2nd Edition), 2021-2030, covering key aspects of the industry and identifying areas for potential future growth.

The opportunity is likely to be well distributed across therapeutic areas, scales of operation, types of therapies and key geographical regions, said the analysts.

The advanced therapy medicinal products (ATMPs) sector, including cell and gene therapy (CGTs) developers. is a relatively niche area, with the presence of several start-ups/small-sized developers dominating the development landscape. About 52% of cell and gene therapy developers are small players.

And, over time, said the analysts, outsourcing has become a preferred operating model for product development activity in this domain, with CROs making significant investments towards acquiring advanced technologies and the capacity to accommodate their growing clientele.

The R&D of biologics is complex and time intensive process, requiring sophisticated techniques and dedicated infrastructure, which is readily available with contract service providers. Further, these service providers adhere to the rapidly evolving regulatory environment, acting as a key enabler to the broader industrys evolution in the coming years.

The study noted that, currently, over 100 industry players claim to have the necessary capabilities to offer contract research and clinical trial support for ATMPs.

Close to 38% of the CROs engaged in this domain are large companies, having required capabilities to provide services for both types of therapies. Examples of new entrants those established since 2018 - that claim to offer clinical services include Geistek Pharma, The Discovery Labs, KCT and Longmore 60 Biotech, reported the market specialists.

Around 45% of cell and gene therapy CROs offer research related services across both, preclinical and clinical scales of operations, while some 74% of players offer CRO players offer regulatory and data management services for cell and gene therapies, according to the analysis.

Both established players and new entrants have forged several partnerships in the recent past; most of the deals were focused on cell therapy, said the analysts, citing examples of the companies that have signed multiple international deals: Accelovance, Charles River, Laboratories, and CitoxLab.

In pursuit of a competitive edge and to eventually establish themselves as one-stop shops, stakeholders are also actively consolidating their capabilities related to cell and gene therapies through mergers and acquisitions, commented the authors.

Some are pursuing such activity for geographical consolidation reasons as shown by the acquisition of Synteract by Syneos Health in December 2020, some for portfolio addition, as seen in Comparative Biosciences being bought by Genesis Biotechnology in August 2020. Others have gone down the M&A route to expand their portfolios: the acquisition of Absorption Systems by Pharmaron in December last year is a case in point, while geographic expansion was the rationale behind the acquisition of Veristat by TCTC Group in January 2020, according to the market specialists.

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TeamedOn and AGTC Announce a Licensing Agreement Advancing X-Linked Retinoschisis Gene Therapy Program – BioSpace

April 17th, 2021 1:52 am

AGTC Transfers Clinical Trial Materials and Phase 1/2 Data To TeamedON

ROCKVILLE, Md., April 13, 2021 (GLOBE NEWSWIRE) -- TeamedOn International, Inc., a biotechnology company dedicated to advancing gene therapies for rare diseases, including ophthalmic indications, and Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced a licensing agreement to advance gene therapy to treat X-linked retinoschisis (XLRS), an inherited disease that causes loss of vision due to degeneration of the retina in males. Under the terms of the agreement, AGTC will provide TeamedOn with the clinical trial material, pre-clinical and clinical data generated for the development of AGTCs investigational intravitreal gene therapy candidate, rAAV2tYF-CB-hRS1.

The license between AGTC and TeamedOn re-opens the possibility of a much-needed treatment for individuals with XLRS, said Peter Mu, CEO of TeamedOn. TeamedOn is very pleased to be able to build on AGTCs efforts, carry on their investment in XLRS to bring potential benefit to patients.

Under the agreement, TeamedOn will conduct all activities required to reinitiate clinical development of the program. AGTC will be eligible to receive milestones and royalties based on clinical progress.

There is no cure for XLRS, and patients living with this disease have an urgent need for disease-modifying therapies that have the potential to stabilize and/or improve their long-term vision outcomes, said Dr. Paul Yang, Assistant Professor of Ophthalmology at the Oregon Health and Sciences University, who was a clinical investigator for AGTCs prior XLRS Phase 1/2 clinical trial and recently engaged by TeamedOn. Intravitreal injection of this gene therapy for XLRS previously demonstrated a reasonable safety profile. When administered through subretinal injection as TeamedOn is planning, this investigational gene therapy may have an increased likelihood of producing detectable biological activity.

AGTC discontinued its XLRS clinical program and development of rAAV2tYF-CB-hRS1 in 2018 because defined efficacy endpoints were not met using intravitreal injection.

We are excited at the prospect of TeamedOn taking a license for this program to explore the opportunity for subretinal injection of rAAV2tYF-CB-hRS1 to provide potential clinical benefit to mitigate the devastating impact on the vision of individuals with XLRS for whom there are no effective treatments, said Sue Washer, President and CEO of AGTC.

Forward-Looking StatementsThis release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs, including statements regarding the licensing agreement between AGTC and TeamedOn International, Inc. for AGTC to provide TeamedOn with the clinical trial material, pre-clinical and clinical data generated for the development of AGTCs investigational intravitreal gene therapy candidate, rAAV2tYF-CB-hRS1. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions, and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

About TeamedOnTeamedOn International, Inc. (TeamedOn) (https://www.TeamedOn.com) is advancing gene therapies for rare diseases and currently focused on ophthalmic indications. We are developing innovative medicine to treat inherited retinal disorders (IRDs) and other eye diseases with significant unmet medical need. In addition to our internal R&D programs, we also seek partners for collaboration or co-development. Our goal is to restore sight and prevent vision loss for patients all around the world. TeamedOn is privately funded and located in Rockville, MD.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, an innovator in the emerging field of optogenetics and retinal coding.

TeamedOn PR CONTACTS:Peter Mu, PhD, MBACEOTeamedOn International, Inc.(301) 461-1617Peter_Mu@teamedon.com

AGTC IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

AGTC Corporate Contacts:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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Jaguar Gene Therapy gains $139 million from investors – Daily Herald

April 17th, 2021 1:52 am

LAKE FOREST -- Jaguar Gene Therapy said it has raised $139 million through its most recent round of investments, led by Eli Lilly and Co. and Deerfield Management.

Also participating in the round were Arch Venture Partners, co-founded by Robert Nelsen and one of the largest early-stage technology venture firms in the United States; Goldman Sachs; and Nolan Capital, the investment fund of former AveXis CEO and current Jaguar Executive Chairman Sean P. Nolan.

Jaguar Gene Therapy is a company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including galactosemia, genetic causes of autism spectrum disorder, Type 1 diabetes and Bardet-Biedl syndrome.

"We are thrilled to have attracted this prestigious group of visionary investors, which affirms our strategic approach, promising pipeline programs, and unique collaboration with Deerfield Management," said Joe Nolan, chief executive officer of Jaguar Gene Therapy. "With this capital infusion, we are well positioned to execute on our mission to accelerate breakthroughs in gene therapy and bring them to children and adults with severe genetic diseases."

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Medically Necessary: Growth of biologics and gene therapies means more cold chain – FreightWaves

April 17th, 2021 1:52 am

This is an excerpt from the April 13, 2021 edition of Medically Necessary, a health care supply chain newsletter.Subscribe here.

The trend: The number of biologic drugs and gene therapies is growing, and logistics companies are increasing their capacity for temperature-controlled transportation to support them.

Biologics are drugs created from biological sources, such as blood or other cells. Theyre usually much more complex than drugs produced chemically.

Gene therapies, which can be biologics, are drugs that replace or modify a patients gene to treat a disease.

Drugs in both categories require temperature to be carefully controlled during transportation.

The future: About a quarter of all drugs currently require some kind of temperature control during transportation, according to RanjeetBanerjee, CEO of the pharmaceutical logistics company Cold Chain Technologies.

He expects that number to jump to about a third of all drugs within the next two years.

With the launch of biologics, with the launch of personalized medicine and certain gene therapy formulations, that are happening now, the need for temperature and other condition monitoring is becoming even more acute, he said.

Biologics: The market for temperature-sensitive biologics is growing faster than that of other types of drugs.

Spending on biologics through Medicare Part B, which covers drugs that patients recieve at a doctors office or hospital, jumped from about 56% of total drug spending in 2006 to 77% in 2017, according to a Health and Human Services Department report.

In large part, that growth is because biologic drugs tend to be very expensive. The report found that biologics accounted for almost all of the programs drug spending growth during those years.

The number of patients receiving biologics also appears to be increasing.

Data from Medicare Part D, which covers prescription drugs, shows that the number of beneficiaries receiving a group of seven common biologics increased by more than 75% from 2011 to 2018.

A 2017 report from The Biosimilars Council predicts that the growth of biosimilars cheaper alternatives that have the same clinical effects as biologics will further expand access to these types of treatments.

Like biologics, many biosimilars require temperature control during transportation and storage.

Gene therapies: The cold chain logistics company Cryoport expects the value of the gene therapy market to increase sevenfold between 2019 and 2024.

FDA approved the first gene therapy in the U.S. in 2017 and has approved six more treatments since then.

The agency approved two of those seven treatments in the first quarter of 2021 alone.

In 2019, former FDA Commissioner Scott Gottlieb said he expected the agency to be approving 10 to 20 cell and gene therapy products every year by 2025.

This trend has a couple of logistics implications, Cedric Picaud, CEO of the Cryoport company CRYOPDP told FreightWaves. The value of each shipment is very high. The cost of managing such medication is high. In terms of logistics, the lead time requirement is very challenging.

The response: As the number of biologic drugs and gene therapies continues to grow, logistics companies are increasing their capacity for temperature-controlled transportation.

In March, Cold Chain Technologies, which previously focused on packaging, expanded the scope of its business.

The company will now offer last-mile delivery services, warehousing and point-of-care storage for temperature-controlled drugs. The goal is to provide logistics from the manufacturer to the patient, Banerjee said.

Over the past two years, Cryoport has acquired several cold chain logistics companies to expand its reach.

Cryoport acquired an Australian logistics company in April. That follows the acquisition of two cold chain logistics companies in August 2020 and another acquisition in May 2019.

The company grew from 57 employees at the end of 2018 to more than 625 by the end of 2020.

Tribe Transportation, a temperature-controlled trucking company based in Georgia, recently added more than 100 trailers to its fleet to meet demand from the life sciences industry.

There are still life sciences customers out there looking for capacity to move shipments, Executive Vice President Matt Handte told FreightWaves. Post-vaccine, weve seen that continue to rise.

The past three years have been the companys best years for growth of both revenue and fleet size, Handte said.

The virus: The coronavirus vaccine rollout has put the cold chain in the spotlight, but companies dont expect this to be a flash in the pan.

Cold Chain Technologies provides packaging for COVID-19 vaccines made by Moderna and Johnson & Johnson. The company opened a new manufacturing plant in Tennessee last year to meet the needs of the vaccine rollout, but Banerjee doesnt expect that capacity to go to waste.

If we plan this carefully, we could use this capacity to support COVID vaccinations outside the U.S., which will have a longer ramp down as well as taking care of the new drugs and biologics, he said.

Whats next? Banerjee said he hopes the vaccine rollout will help logistics companies do a better job of transporting temperature-controlled drugs after the pandemic.

How do we make sure we transform the cold chain so that the next time this happens we are way ahead? he said. And use all that learning for the day-to-day shipments of drugs and pharmaceuticals.

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Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day – BioSpace

April 17th, 2021 1:52 am

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced new data for multiple preclinical programs and a planned R&D Day, which will be held in June 2021.

Collectively, these new preclinical data highlight Tayshas next wave of novel gene therapies that have the potential to impact meaningful patient populations. The promising data underscore our ability to rapidly and reproducibly investigate disease biology, design innovative gene therapies and efficiently advance the development of these drug candidates, said RA Session II, President, Founder and Chief Executive Officer of Taysha. Among the compelling new data, for the first time, we have shown that TSHA-113, an AAV9 gene therapy that utilizes AAV-mediated gene silencing, reduced tau expression in mouse models of human tauopathies. The potential implications of these data are far reaching, and we intend to further evaluate TSHA-113 in additional preclinical studies. The totality of the preclinical data presented today support the fundamental elements of our scientific approach of coupling validated technology with novel targeted payload design while utilizing a proven HEK293 suspension manufacturing process. We believe our deep pipeline and innovative scientific engine hold tremendous potential, and we are poised to continue delivering meaningful value to patients with monogenic CNS diseases.

Todays data demonstrate the breadth, depth and velocity of our development engine as a sustainable pivotal-stage gene therapy company. There are no approved disease modifying therapies for any of the programs in our portfolio and we are encouraged by the results of our gene therapy approach of vectorized RNA and gene replacement therapies across our portfolio, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. We are very excited to further develop TSHA-113 in tauopathies, including Alzheimers disease, MAPT-associated frontotemporal dementia and progressive supranuclear palsy, based on the significant reduction in tau expression demonstrated in transgenic mouse models of human tauopathies. In addition, to date, we have advanced five programs into IND/CTA-enabling studies, including TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN in Lafora disease, TSHA-111-MALIN in Lafora disease, TSHA-112 in APBD and TSHA-119 in GM2 AB variant. We intend to file an IND/CTA for one of these five named programs by the end of 2021. By mid-year, we intend to select a development candidate for Angelman syndrome and obtain interim expression and safety data from confirmatory non-human primate studies by year-end. We remain on track to report Phase 1/2 biomarker data for TSHA-101 in GM2 gangliosidosis in the second half of this year and to provide a clinical and regulatory update for TSHA-120 in giant axonal neuropathy by year-end. Finally, in the second half of the year, we continue to expect dosing of the first patient with CLN1 disease in a Phase 1/2 trial for TSHA-118 under an already open IND, filing an IND/CTA for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome, and filing an IND for TSHA-101 in GM2 gangliosidosis in the U.S. These anticipated clinical and regulatory milestones are expected to be followed by the initiation of Phase 1/2 clinical trials for each of these indications. We look forward to providing additional updates at our R&D Day in June.

TSHA-113 for Tauopathies

Taysha is developing tau-specific microRNA (miRNA) shuttles designed to target tau mRNA for all six isoforms found in the human brain and/or mouse brain. TSHA-113 is an AAV9 capsid that packages these miRNA shuttles and is delivered in the CSF for the treatment of tauopathies.

TSHA-105 for SLC13A5 deficiency

TSHA-105 is a recombinant self-complementary AAV9 vector that expresses the human SLC13A5 protein under the control of a ubiquitous promoter. The drug candidate is being developed for the treatment of SLC13A5 deficiency.

TSHA-106 for Angelman syndrome

TSHA-106 is an intrathecally delivered AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS, the antisense transcript governing the expression of UBE3A through the paternal allele.

TSHA-112 for Adult Polyglucosan Body Disease (APBD)

TSHA-112 is an intrathecally delivered AAV9 viral vector designed for miRNA-mediated knockdown of the GYS1 gene to treat APBD.

TSHA-111-LAFORIN for EPM2A and TSHA-111-MALIN for EPM2B for Lafora disease

TSHA-111-LAFORIN and TSHA-111-MALIN are intrathecally delivered AAV9 viral vectors designed for miRNA-mediated knockdown of the GYS1 gene to treat Lafora disease.

TSHA-119 for GM2 AB variant

TSHA-119 is a self-complementary AAV9 vector designed to deliver a functional copy of the GM2A gene to treat GM2 AB variant.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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From gene therapies to medical treatments, cutting-edge research finds a home in NC – WRAL.com

April 17th, 2021 1:52 am

By Abbey Slattery, WRAL Digital Solutions

This article was written for our sponsor, the North Carolina Biotechnology Center.

For decades, North Carolina has been known as a nerve center for the life sciences industry. In fact, after the creation of the Biotech Center in the 1980s, North Carolina became the first state in the country to have a state-supported entity dedicated to growing the industry.

N.C. Biotech runs a number of programs to support technology and company development, to recruit companies and to connect North Carolina residents to high-paying jobs.

In the years since then, numerous life sciences companies have established a base in North Carolina, including major names like Biogen, BASF, LabCorp and Merck, among others. By committing to the industry early and cultivating a strong university system, the state established itself as a hub and prepared a workforce that rivals that of anywhere else in the country. Now, it's the site of global scientific breakthroughs and game-changing medical advancements.

"North Carolina has really done a good job with creating a flow from the workforce to the industry, and we've continued to grow and expand because of that. We now have a lot of research and development tools, and we have a tremendous amount of manufacturing here," said Laura Gunter, executive vice president at NCBIO. "All of the amenities of North Carolina and climate and education attract people, but then we've done a good job of educating folks and giving North Carolinians an opportunity to work and thrive here."

With a highly trained workforce of over 67,000, 775 diverse bioscience companies and nearly 2,500 companies, the state has created a thriving ecosystem for the industry. In the Triangle region alone, agricultural and biopharma manufacturing companies are developing exciting new advancements with potential for global impacts.

"A company called Pairwise in Durham is focused on using the gene-editing technology called CRISPR, but with plants. They're working on developing more nutrients or better growing capabilities with plants using that CRISPR technology," said Gunter. "They've developed a new variety of leafy green called brassica that's related to Brussels sprouts, cauliflower and kale. It's nutrient-dense and naturally resistant to pests, but apparently, it has a very pungent flavor. What they've done is work on the flavor, while keeping the nutrients and resistance."

"Another major company is Novozymes, and they specialize in industrial enzymes and microorganisms. If you've ever toured any of these facilities where they're doing fermentation, like a beer brewing facility, Novozymes does that on a massive scale with industrial enzymes," continued Gunter. "Those enzymes can be used in everything from pharmaceuticals to food to detergent. They're also working on some things on the agriculture side to improve crop yields through microbes."

Biogen founded in 1978 and one of the world's first global biotechnology companies is another major player that finds a home in Durham. While Pairwise and Novozymes are more focused on the agricultural side of biotech, Biogen is making advancements in pharmaceutical manufacturing and medical research programs. Its active research includes treatments for conditions like multiple sclerosis, spinal muscular atrophy and other serious neurological conditions.

The company announced a $200 million expansion in March to produce gene therapies and other treatments in their project pipeline. It joins Novartis Gene Therapies, which is producing its Zolgensma therapy for spinal muscular atrophy in Durham, alongside Adverum, Audentes, Beam Therapeutics, bluebird bio, Cellectis, Pfizer, Taysha Gene Therapies and more.

The benefit of cell and gene therapies is the treatments are potentially curative. Early applications are for diseases caused by single-gene mutations, and those that are life-threatening. Thanks to these companies, North Carolina will be the place these treatments are produced. The treatments would likely have a global impact on improving the lives of those who suffer.

One specific example is spinal muscular atrophy, which is the loss of nerve cells and the weakening of muscles near the spine. This degenerative disease has previously been a fatal diagnosis for infants.

"The fascinating thing about spinal muscular atrophy treatments, in the case of Biogen and Novartis, they're both game-changing and potentially curative treatments versus just trying to alleviate a symptom," said Gunter. "With the gene replacement therapy that Novartis is using, if it works, then that child could potentially no longer have the disease. If it ends up being a long-term fix, it's pretty miraculous and it's made right here."

The list of companies that develop products and treatments in North Carolina goes on from endoscopy devices at Cook Medical to in vitro products at bioMerieux to injectable drugs at Hospira. Thanks to forward-thinking from the state and organizations like N.C. Biotech, North Carolina is well-equipped to continue being the site of life sciences products and advancements.

"North Carolina has paid attention to reinforcing the entire pipeline starting with idea and proof of concept by a university researcher. Small companies are created from the research, and we help them navigate a bumpy road to success. Then, we work with companies all around the world looking for the right place to manufacture their products," said Doug Edgeton, president and CEO of N.C. Biotech.

"We continue to invest in this innovation infrastructure, and train the talent to do these important jobs. We think it's why North Carolina is a global leader in this field."

This article was written for our sponsor, the North Carolina Biotechnology Center.

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University of Cambridge research suggests gene therapies offer hope in treating glaucoma and dementia – Cambridge Independent

April 17th, 2021 1:52 am

Promising research at the University of Cambridge suggests gene therapies could help repair some of the damage caused in chronic neurodegenerative conditions such as glaucoma and dementia, writes editor Paul Brackley.

Their studies in animals offer hope that such therapies could be effective in polygenic conditions, which are complex and do not have a single genetic cause.

Gene therapies involve replacing a missing or defective gene with a healthy version.

They have become increasingly common, but typically for rare and monogenic conditions those caused by a single defective gene, such as Lebers congenital amaurosis, spinal muscular atrophy and Lebers hereditary optic neuropathy.

There have been limited applications of such therapies to polygenic conditions, which include the majority of neurodegenerative conditions.

In their new research, Cambridge scientists delivered two candidate molecules simultaneously to nerve cells using a single virus to achieve a strong effect on axonal growth.

Dr Tasneem Khatib, from the John van Geest Centre for Brain Repair at the University of Cambridge, the studys first author, explained: The axons of nerve cells function a bit like a railway system, where the cargo is essential components required for the cells to survive and function.

In neurodegenerative diseases, this railway system can get damaged or blocked. We reckoned that replacing two molecules that we know work effectively together would help to repair this transport network more effectively than delivering either one alone, and that is exactly what we found.

This combined approach also leads to a much more sustained therapeutic effect, which is very important for a treatment aimed at a chronic degenerative disease.

Rather than using the standard gene therapy approach of replacing or repairing damaged genes, we used the technique to supplement these molecules in the brain.

Axons are long fibres that transmit electrical signals, allowing nerve cells to communicate with one another and with muscles.

Axonal transport is a cellular process that moves key molecules and cellular building blocks such as mitochondria, lipids and proteins to and from the body of a nerve cell.

It has been suggested that stimulating this process by enhancing intrinsic neuronal processes in the diseased central nervous system could be a way of repairing damaged nerve cells.

The Cambridge team explored this idea using two candidate molecules for improving axonal function brain-derived neurotrophic factor (BDNF) and its receptor, tropomyosin receptor kinase B (TrkB).

They tested the idea in two models of neurodegenerative disease known to be associated with reduced axonal transport glaucoma and tauopathy, a degenerative disease associated with dementia.

Glaucoma involves damage to the optic nerve and is typically associated with abnormally high pressure in the eye.

Using rats, researchers deployed a tracer dye to show axonal transport between the eye and brain was impaired in glaucoma, while a reduction in electrical activity in the retina in response to light suggested vision was also impaired.

Dr Khatib and colleagues then used viral vectors which are gene therapy delivery systems to deliver the two molecules to the retina of rats. The movement of the dye showed this restored axonal transport between the retina and the brain and the retinas also showed an improved electrical response to light, which is a prerequisite for visual restoration.

Next they used transgenic mice bred to model tauopathy, which is the build-up of tangles of tau protein in the brain.

Tauopathy is seen in neurodegenerative diseases, including Alzheimers disease and frontotemporal dementia.

Injecting the dye again enabled the team to show that axonal transport was impaired between the eye and the brain and that it was restored using the viral vectors.

They even found preliminary evidence of possible improvement in the mices short-term memory using an object recognition task.

Before the treatment, a mouse was placed at the start of a Y-shaped maze and left to explore two identical objects at the end of the two arms.

Shortly after, the mouse was once again placed in the maze, but this time one arm contained a new object, while the other contained a copy of the repeated object.

The researchers measured the amount of the time the mouse spent exploring each object to see whether it had remembered the object from the previous task.

After the viral vector had been injected into the mouses brain, the test was repeated and suggested a small improvement in short-term memory. The result here was not deemed statistically significant, but the researchers were encouraged and plan a larger study to confirm it.

Professor Keith Martin, from the Centre for Eye Research Australia and the University of Melbourne, who led the study while at Cambridge, said: While this is currently early stage research, we believe it shows promise for helping to treat neurodegenerative diseases that have so far proved intractable.

Gene therapy has already proved effective for some rare monogenic conditions, and we hope it will be similarly useful for these more complex diseases which are much more common.

In the study, published in Science Advances, the researchers write: We feel that these findings do have implication for clinical practice.

The research was supported by Fight for Sight, Addenbrookes Charitable Trust, the Cambridge Eye Trust, the Jukes Glaucoma Research Fund, Quethera Ltd, Alzheimer's Research UK, Gates Cambridge Trust, Wellcome and the Medical Research Council.

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Gene therapy: Discussing the worlds most expensive treatments at Health Horizons Future Healthcare Forum

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University of Cambridge research suggests gene therapies offer hope in treating glaucoma and dementia - Cambridge Independent

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What is AAV Gene Therapy and What Makes it Safe? – Freethink

April 17th, 2021 1:52 am

Genetic diseases were thought to be incurable letters carved in stone (or on a double helix). But gene therapy could change all that.

From treating Duchenne muscular dystrophy, autism, or glaucoma, there has been a surge in the news surrounding gene therapy (type "gene therapy" into the Freethink search tool, and you'll see endless stories about medical breakthroughs).

The total number of completed or ongoing gene therapy clinical trials reached 4000 in February 2020. This type of treatment is on its way toward addressing some of the most severe and complicated ailments known to man. Gene therapy even shows promise for treating diseases like cancer or Huntington's disease longstanding mysteries of medical science.

Instead of using medications or surgery, doctors can use gene therapy to fight diseases at their source. Gene therapy attempts to cure a disease, or boost the body's ability to combat a disease, by replacing a defective gene or adding a new gene.

The success of gene therapy all hinges on the ability to deliver that precious genetic cargo into the patient's cells safely. To do that, scientists use a "vector" as a delivery vessel for the genetic material. In most cases, that vector is a virus.

Scientists engineer an unwitting virus to act as a vector to deliver a therapeutic genetic payload.

We don't typically think fondly of viruses. Usually, they don't enter the human body to help. Instead, viruses like the flu or coronavirus make humans sick by inserting their genetic material into human cells and hijacking them. The cell reads the viral DNA (or RNA) and produces virus offspring, infecting other cells, repeating the process.

But, since viruses naturally deliver genetic material into a human cell, they are the most widely used vectors in gene therapy.

Scientists engineer an unwitting virus to act as a vector to deliver a therapeutic genetic payload. They strip the virus of its own genes and replace it with new genes. Once the virus transports its genetic cargo into tissues, the altered genes get to work producing further instructions to treat the disease.

But the human body can recognize the virus as a foreign invader and launch an immune response attempting to target and reject the virus.

Enter the adeno-associated viruses (AAV) a small virus that has emerged as the most promising platform for gene therapy.

These small, versatile viruses have several advantages over other gene therapy vectors.

First, despite their small size, they can carry a sizeable genetic payload. They are not known to cause many human diseases, and they can be designed to target particular types of cells or tissues. They cannot replicate on their own, so scientists can control how much gene therapy is delivered to the body.

The best part: they don't trigger a very large immune response like other viruses. A gene therapy trial in the 1990s that used an adenovirus (a kind of cold virus, different from AAVs) caused a deadly immune overreaction in one patient and effectively ended gene therapy research in humans for a decade.

After that, researchers went back to the drawing board, looking for safer vectors. AAVs, because they don't cause disease and can't replicate, generally fly under the immune system's radar. These characteristics make them perfect vessels for gene therapy. And clinical trials have since shown that AAV gene therapy is safe and, for some diseases, effective.

Now that AAVs are established as gene therapy vectors, scientists are working to improve their delivery and create the next generation of AAV gene therapy.

Researchers have been successful with AAV gene therapy in the clinical setting. Currently, two FDA-approved gene therapy treatments use AAVs: Luxturna, which was approved in 2017 for a rare vision disorder, and Zolgensma, which was approved last year for spinal muscular atrophy.

But there are challenges to overcome. Because AAVs are viruses that people are naturally exposed to, many people have already developed antibodies against them. When a patient who has been previously exposed to an AAV receives AAV gene therapy, these antibodies could block the therapy.

Recently, a team of researchers figured out how to cloak the AVV to sneak past the immune system and deliver its gene therapy payload without triggering any immune response.

With the recent surge in AAVs as viral vectors and more clinical studies in the pipeline, we can expect to see new gene therapies that are safe and effective.

We'd love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at [emailprotected]

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What is AAV Gene Therapy and What Makes it Safe? - Freethink

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ATMPS and Ori Biotech Collaborate on an Integrated Digital Platform for Cell and Gene Therapy Developers – BioSpace

April 17th, 2021 1:52 am

The collaboration will improve visibility and reporting of cell and gene therapy manufacturing through a pre-integrated data solution

London, UK and Woodcliff Lake, NJ, US, April 14, 2021 ATMPS Ltd, a leader in blockchain-based vein to vein cell orchestration platforms for advanced therapies, and Ori Biotech, an innovator in cell and gene therapy (CGT) manufacturing platforms, today announce a global collaboration to create seamless integration between their respective data platforms to support the development of Advanced Therapies.

The non-exclusive partnership will result in the integration of Oris proprietary CGT manufacturing platforms data architecture with ATMPS HataaliTM, a secure data sharing platform, specially designed as a scalable system for advanced therapies. The combination of the two proprietary platforms will provide improved product tracking, scheduling as well as live monitoring of the manufacturing status across the entire CGT supply chain from vein to vein.

Ori selected ATMPS Hataali technology for integration with our platform to allow the secure sharing of relevant process data with therapy developers, contract development and manufacturing organizations (CDMOs) and even clinicians or patients in real-time. This collaboration will provide increased visibility into the treatment process for those involved at each stage of the treatment process, said Jason C. Foster, CEO, Ori Biotech. The addition of established technologies from companies like ATMPS allows Ori to further its mission of enabling partners across the CGT industry to bring therapies as safely, cost effectively and quickly as possible to large numbers of patients with few alternative therapeutic options.

Hataali encrypts and stores data using distributed ledger technology through a process known as hashing. This process ensures data security is protected through a system of private keys and cryptography. Hataali was the first cell orchestration platforminstalled in a hospital globally and ATMPS the first blockchain company to integrate with the UK National Health Service for the delivery of advanced therapies.

Ori is an ideal partner for ATMPS, as both companies share a common mission to lower the costs and improve processes for cell and gene therapies. Oris work in helping innovators scale from preclinical to commercial is a massive step towards reducing complexity and bringing these vital therapies to patients more quickly, said Raja Sharif, CEO, ATMPS. We all know how challenging and expensive the development and manufacturing of CGTs can be. With the help of Hataali, Ori can securely leverage vein-to-vein data to advance their manufacturing solutions much more efficiently. This will enable real-time therapy tracking, improved patient trust and expediated clinical development.

Ori continues to experience significant growth since closing a $30 million Series A round of funding as it prepares to bring its platform to market. The company is actively recruiting the best talent in cell and gene therapy technology through its networks and through http://www.oribiotech.com.

###

About ATMPS

ATMPS Ltd provides patent pending advanced technical solutions for healthcare companies in the Advanced Therapy Medicines Products sector, including CAR-T treatments, stem cell and gene treatments. ATMPS have a unique distributed ledger solution which provides immutable and incorruptible data and automation capabilities. The solution is operational, configurable and can provide data analytics modules. It can integrate with all LIMS, MES and legacy solutions. It provides full chain of custody, identity and condition of samples, treatments and assets which it tracks. It also schedules and co-ordinates the supply chain participants, as well as giving them information at the same time, in real time, from a "single source of truth. For more information, please visit ATMPS website athttps://www.atmps.net/.

About Ori Biotech

Ori is a London and New Jersey based cell and gene therapy (CGT) manufacturing technology company. Ori has developed a proprietary, flexible manufacturing platform that closes, automates and standardizes CGT manufacturing allowing therapeutics developers to further develop and bring their products to market at commercial scale. The promise of the full stack Ori platform is to fully automate CGT manufacturing to increase throughput, improve quality and decrease costs by combining hardware, software and data services.

Ori was founded by Professor Chris Mason (AVRO) and Dr. Farlan Veraitch (UCL) and has brought together a seasoned Board and executive management team with over 100 years of pharmaceutical, CGT and venture building experience including CEO Jason C. Foster (Indivior), CBO Jason Jones (Miltenyi Biotec), VP Tom Heathman (Minaris), VP David Smith (Minaris) alongside expert advisers like Bruce Levine, Anthony Davies and Annalisa Jenkins. For more information, visit http://www.oribiotech.com.

For media enquiries, please contact:

Alex Heeley or Abdul Khalifeh

De Facto Communications

T: +44 (0) 203 735 8168

E:a.heeley@defacto.co.uk/a.khalifeh@defacto.co.uk

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ATMPS and Ori Biotech Collaborate on an Integrated Digital Platform for Cell and Gene Therapy Developers - BioSpace

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CRISPR Therapies Pipeline Insights 2021: Analysis of Key Companies, Emerging Therapies, Recent Happenings and Futuristic Trends – GlobeNewswire

April 17th, 2021 1:52 am

Los Angeles, USA, April 12, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapies Pipeline Insights 2021: Analysis of Key Companies, Emerging Therapies, Recent Happenings and Futuristic Trends

The leading gene-editing companies looking at commercializing CRISPR-based therapeutics are CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine. CRISPR Therapeutics has the largest market cap of the three, at $10.9B, with a clinical development program that is more advanced than those of Intellia and Editas. Editas Medicine has the smallest market cap of the three companies. Intellia has established high profile collaborations with Regeneron and Novartis.

DelveInsights CRISPR Therapies Pipeline Insight report offers a broad view of available CRISPR therapies in the market, pipeline CRISPR therapies, their MoA, RoA, key companies working in the domain and competitive assessment.

Some of the key takeaways from the CRISPR Therapies Pipeline Report:

Interested in knowing more? Request for the sample @ CRISPR Therapies in the Pipeline

The report underlines the present unmet needs in the market, driving factors and market constraints, along with the holistic view of the inactive therapeutics (comprising dormant and terminated products) with the reasons behind their downfall, detailed insights into the structure and gene editing tool of the pipeline CRISPR therapies to help clients gauge the opportunities and risks in the market.

In the News

Know what is happening in the CRISPR Pipeline Therapies @ CRISPR Pipeline Recent Happenings

What is CRISPR?

CRISPRs (Clusters of Regularly Interspaced Short Palindromic Repeats) are specialized stretches of DNA and are a shorthand for CRISPR-Cas9, which are transcribed by the bacteria to RNA stretches during viral infections. The same CRISPR technology can be leveraged to identify, alter and modify the DNA sequences and genomes.

The technique is used to correct genetic defects, prevent the spread of disease by altering the genetic sequence, improving crop viability and durability, and so on without affecting the functions of other genes.

Want to learn more about the leading candidates in different clinical stages of trials? Reach out @ CRISPR Emerging Therapies and Key Companies

At a Glance: Emerging CRISPR Therapies, RoA, MoA and Companies

Know more about budding CRISPR therapies projected to transform the landscape @ Emerging CRISPR Therapeutics and Market Scenario

CRISPR Therapeutic Assessment The CRISPR Therapies Pipeline report proffers comprehensive insights into active pipeline assets segmented by Stage, Product Type, Route of Administration, Molecule Type, Target and Indications of various drugs.

By Product Type

By Stage

By Route of Administration

By Mechanism of Action

By Targets

By Stage and Route of Administration

By Stage and Product Type

To know more, Visit CRISPR CAS-9 Technology and Emerging Trends

Scope of the report

Learn more about the scope and highlights of the report @ CRISPR Pipeline Emerging Drug Pipeline

Key Questions Answered in the Report

Got queries? Get in touch @ CRISPR Technology and Pipeline Therapies

Table of Contents

Know more about report offerings @ CRISPR Pipeline Insights

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

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CRISPR Therapies Pipeline Insights 2021: Analysis of Key Companies, Emerging Therapies, Recent Happenings and Futuristic Trends - GlobeNewswire

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Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet’s Gene Therapy Programs Targeting…

April 17th, 2021 1:52 am

FOSTER CITY, Calif. & PARIS--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Vivet Therapeutics (Vivet) announced today an agreement whereby Mirum has the exclusive option to develop and subsequently commercialize Vivets two proprietary AAV gene therapy programs for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company.

Under the terms of the agreement, Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs. Until that time, Mirum will provide funding to support the continued research and development costs associated with the two gene therapy programs.

This gene therapy collaboration will work to address the root cause of PFIC3 and PFIC2 and provide an option for patients who do not respond to ASBT inhibition, said Chris Peetz, president and chief executive officer at Mirum. We are encouraged by the expertise and dedication of the Vivet team to develop next-generation gene therapies that have the potential to transform the lives of patients and their families. There are clear synergies in our combined missions to help the people who need it the most.

Mirums dedication to the treatment of cholestatic liver diseases and those rare diseases for which there are limited medications make them a great partner to potentially develop and bring to market VTX-803 and VTX-802, said Jean-Philippe Combal, chief executive officer and co-founder of Vivet. Their leadership in rare liver disease and in particular PFIC, is important as they not only understand the urgent need for patients but also that todays treatment options will never be enough. Gene therapy is a transformative approach, potentially benefiting more patients.

VTX-803 and VTX-802 are two proprietary AAV gene therapy programs of Vivet currently being evaluated in preclinical studies for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. It is thought that successful correction by gene therapy of the defective MDR3 transporter and bile salt export pump (BSEP) functions for PFIC3 and PFIC2, respectively, may ultimately provide a cure for patients living with these rare liver diseases. Such an approach carries the potential to overcome the main limitations of current standard of care for PFIC3 and PFIC2. It may provide long-lasting benefits by restoring physiological bile secretion and preventing severe hepatic complications of the diseases and outweighing its related significant costs.

Vivet has received Orphan Drug Designation for VTX-803 by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Additionally, preclinical proof-of-concept studies highlighting VTX-803 were published in Nature Communications in 2019. The data demonstrated sustained and significant reversal of PFIC3 disease biomarkers in a model of PFIC3.

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS. The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021. Additionally, Mirums marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency. Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.

To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics, following preclinical evaluation and IND/CTA-enabling studies.

Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

About Vivet Therapeutics

Vivet Therapeutics is a clinical stage emerging biotechnology company developing novel gene therapy treatments for rare, inherited metabolic diseases.

Vivet is building a diversified gene therapy pipeline based on novel recombinant adeno-associated virus (rAAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundacin para la Investigacin Mdica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigacin Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain.

Vivets lead program, VTX-801, currently under IND clinical development with the GATEWAY clinical trial, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damages, neurologic symptoms and potentially death.

Vivets second gene therapy product, VTX-803 for PFIC3, received US and European Orphan Drug Designation in May 2020.

Vivet is supported by international life science investors including Novartis Venture Fund, Roche Venture Fund, HealthCap, Pfizer Inc., Columbus Venture Partners, Ysios Capital, Kurma Partners and Idinvest Partners.

Please visit us on http://www.vivet-therapeutics.com and follow us on Twitter at @Vivet_tx and LinkedIn.

About PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe. Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease The PFIC2 patient population accounts for approximately 60% of the PFIC patient population. PFIC2 is caused by a mutation in the ABCB11 gene, which normally encodes a bile salt export pump protein that moves bile acids out of the liver.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the option and license agreement between Mirum and Vivet and the potential development of VTX-802 and VTX-803. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as plans, will, may, anticipates, expects, potential and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirums current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirums business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirums filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on managements assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet's Gene Therapy Programs Targeting...

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LGBT+ Investment Firm Gaingels Joins American Gene Technologies’ Mission to Cure HIV, and Other Serious Human Diseases – PRNewswire

April 17th, 2021 1:52 am

ROCKVILLE, Md., April 15, 2021 /PRNewswire/ --AmericanGene Technologies(AGT),an emerging gene and cell therapy company, announced today that Gaingels, a leading venture investment syndicate in service of the LGBT+ community and its allies, made an investment in AGT to support the Phase 1 clinical trial of an HIV cure. The Phase 1 human trial is investigating the safety of AGT103-T, a single dose, autologous cell therapy intended to cure the disease.

This investment places Gaingels alongside private investors who believe deeply in AGT's mission, rapid drug development platform, and gene and cell therapy programs for HIV, cancer, and PKU.

Gaingels is an investment network comprised of 1000+ investors focused on high-growth venture-backed companies who embrace LGBT+ leadership. Gaingels works to support its portfolio companies in identifying and recruiting diverse leadership talent, fostering a vibrant global community of industry leaders, investors, operators, and entrepreneurs who share a common goal for positive social change through business and successful investments.

"Diversity in every aspect of society and business leads to the best outcomes for all of humanity," said Jeff Galvin, CEO and Founder of American Gene Technologies. "AGT is a place where smart, motivated people of every background thriveour team, investors, and the populations we serve are all diverse. We're proud to partner with Gaingels to accelerate solutions to the market and to achieve success for patients and investors alike. This investment is symbolic to us at AGT. Our science, mission, and team present opportunities and products that uplift everyone without regard to background, lifestyle, or identity," Galvin added. "AGT's one-and-done cell therapy is intended to create a durable natural immunity to HIV, returning HIV+ persons to a life without daily antiretroviral medication, and free of the risks of AIDS, transmitting HIV to others, or becoming reinfected. Gaingels support will put us another step closer to achieving this goal."

"As the largest investor network focused on supporting and investing in the best venture-backed companies that embrace and value diverse leadership, including LGBTQ+, Gaingels is proud of participating in AGT's financing," said Lorenzo Thione, Managing Director of Gaingels. "Its mission hits uniquely close to home for the LGBTQ+ community given AGT's focus on creating functional immunity and thus an effective cure for HIV. We are resolved on helping the company grow and scale and achieve this uniquely ambitious and important goal, while strengthening its prospects of success by building a culture that reflects the diversity of its executive teams, staff and customers."

About Gaingels Gaingelsis the leading investment syndicate in support of and representing the LGBTQ community and allies in the venture capital space. With over $150,000,000 deployed into a portfolio of over 300 companies, Gaingels seeks to directly and indirectly influence the venture ecosystem towards greater diversity, inclusion and equity of access. Gaingels invests in companies resolved on building diverse and inclusive teams who are aligned with our mission and seek to expand and embrace LGBTQ leadership into their companies and boards. We actively support our portfolio companies in identifying and recruiting diverse leadership talent, and we strive to foster a vibrant global community of industry leaders, investors, operators, and entrepreneurs who share a common goal for positive social change through business and successful investments. Gaingels is a founding co-signer of the Diversity Term Sheet Rider Initiative to increase access to venture funding events for non-traditional check writers, and regularly co-invests with select VC leads across a variety of sectors, from technology, to B2B, healthcare and consumer, in competitive and over-subscribed rounds from Seed to Growth/pre-IPO.

Website: https://gaingels.com/

About American Gene Technologies (AGT) AmericanGene Technologies(AGT)is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. AGT's mission is to transform people's lives through genetic medicines that rid the body of disease. AGT has three patents for the technology used to make the AGT103-T cell product which is currently in a clinical trial to test its ability to functionally cure HIV+ individuals. AGT also has received ten patents for its uniqueimmuno-oncology approachto stimulategamma-delta () T cellsto eliminate a variety of human solid tumors, such as breast, prostate, and liver cancer. The company has developed a synthetic gene for treatingPhenylketonuria (PKU), a debilitating inherited disease, and has been grantedOrphan Drug Designationby the Food and Drug Administration (FDA).

For more information on AGT's Phase 1 clinical trial, visit clinicaltrials.gov (Study Identifier: NCT04561258).

Website: https://www.americangene.com

American Gene Technologies Contact:C. Neil Lyons, Chief Financial OfficerPhone:(301) 337-2269Email:Contact Requests

For media inquiries, please contact:Sasha Whitaker, Digital Marketing and CommunicationsPhone:(301) 337-2100Email:Contact Requests

SOURCE American Gene Technologies

http://www.americangene.com

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LGBT+ Investment Firm Gaingels Joins American Gene Technologies' Mission to Cure HIV, and Other Serious Human Diseases - PRNewswire

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Global Regenerative Medicines Market 2020-2030: Opportunities in Drug Approvals and Strong Pipeline of Cell and Gene Therapies & Multiple…

April 17th, 2021 1:52 am

DUBLIN--(BUSINESS WIRE)--The "Global Regenerative Medicines Market 2020-2030: Focus on Products, Applications, 17 Countries' Data and Competitive Landscape" report has been added to ResearchAndMarkets.com's offering.

The global regenerative medicines market is projected to reach $87.03 billion by 2030 and set to witness a CAGR of 13.99% from 2020 and 2030.

The market is driven by certain factors, including increasing consolidation among healthcare juggernauts, rising number of clinical trials for regenerative therapies, a favorable regulatory environment to accelerate approvals and market entry, and rising awareness for stem cell therapeutics, that are fueling the growth of the global regenerative medicines market.

Regenerative medicine is challenging the current healthcare practices by targeting the treatment of the root causes of disease and disorders, transforming it into an incredibly exciting space. However, these advancements have not come without significant challenges and uncertainties, which will also further need to be overcome to realize the full potential of regenerative medicine.

Cell and gene therapies, under the regenerative umbrella, are slowly and steadily becoming a healthcare standard, particularly in developed regions such as the U.S. and Europe, where already established markets for tissue engineering and stem cell have largely been responsible for driving the global regenerative medicine phenomenon. Cell and gene therapies, though advancing at a rapid pace, have primarily been targeted for hematological malignancies.

However, the same potential has not been replicated in solid tumor applications, consequently creating demand for cell therapies focusing on solid tumors. As a result, the industry is now moving toward the research and subsequent clinical translation of next-generation therapies, led by natural killer (NK) cells, which offer a viable option for solid tumors as well. Such trends are consistently propelling the industry toward realizing the true potential of precision medicine.

Within the research report, the market is segmented on the basis of products, applications, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Competitive Landscape

The global regenerative medicines market is currently witnessing several developments, primarily aimed toward bringing new products to support clinicians for the treatment of life-threatening disorders. Major manufacturers of regenerative medicine products are actively involved in undertaking significant business strategies in order to translate success in research and development into the commercial clinical setting. Although tissue engineering and stem cells remain the dominating product segments, the advent of cell and gene therapy has revolutionized the regenerative medicine phenomenon, moving it more toward the precision medicine space.

Key Topics Covered:

Executive Summary

1 Product Definition and Market Scope

1.1 Inclusion and Exclusion

1.2 Scope of Work

1.3 Key Questions Answered in the Report

2 Research Methodology

3 Market Overview

3.1 Clinical Importance of Regenerative Medicine

3.2 Market Footprint

3.3 Impact of COVID-19

4 Market Dynamics

4.1 Impact Analysis

4.2 Market Drivers

4.2.1 Increasing Consolidation in the Regenerative Medicines Market

4.2.2 Rising Number of Clinical Trials

4.2.3 Favorable Regulatory Environment

4.2.4 Rising Awareness for Stem Cell Therapeutics

4.3 Market Restraints

4.3.1 Lack of Reliable Vector Production for Cell and Gene Therapy

4.3.2 Exorbitant Cost of Next-Generation Therapies

4.4 Opportunities

4.4.1 Drug Approvals and Strong Pipeline of Cell and Gene Therapies

4.4.2 Multiple Investments in Expansion of cGMP Units

4.4.3 Innovations in Regenerative Medicine

5 Industry Insights

5.1 Overview

5.2 Legal and Regulatory Framework in the U.S.

5.2.1 Cell and Gene Therapy

5.2.1.1 Cell and Gene Therapy Manufacturing QC

5.2.1.1.1 Product Testing

5.2.1.1.1.1 Microbial Testing

5.2.1.1.1.2 Identity

5.2.1.1.1.3 Purity

5.2.1.1.1.4 Potency

5.2.1.1.1.5 Viability

5.2.1.1.1.6 Cell Number/Dose

5.2.2 Stem Cell Therapy

5.2.3 Tissue-Engineered Products

5.3 Legal and Regulatory Framework in Europe

5.4 Legal and Regulatory Framework in Asia-Pacific

5.4.1 China

5.4.2 Japan

6 Global Regenerative Medicines Market: Competitive Insights

6.1 Overview

6.2 Synergistic Activities

6.3 Product Approval

6.4 Mergers and Acquisitions

6.5 Business Expansion and Funding

6.6 Product Launches and Upgradations

6.7 Market Share Analysis, 2019-2020

6.7.1 Market Share Analysis for Global Regenerative Medicines Market (Tissue Engineering), 2019-2020

6.7.2 Market Share Analysis for Global Regenerative Medicines Market (Cell and Gene Therapy), 2019-2020

6.7.3 Market Share Analysis for Global Regenerative Medicines Market (Stem Cell Therapy), 2019-2020

6.8 Growth Share Analysis

6.8.1 Growth Share Analysis (by Company)

6.8.2 Growth Share Analysis (by Product)

6.8.3 Growth Share Analysis (by Application)

7 Global Regenerative Medicines Market (by Product Type), $Million, 2019-2030

7.1 Overview

7.2 Tissue Engineering

7.2.1 Allogeneic Tissue

7.2.2 Autologous Tissue

7.2.3 Xenogeneic Tissue

7.2.4 Synthetic Tissue

7.2.5 Other Tissues

7.3 Stem Cells

7.3.1 Stem Cell Services

7.3.2 Stem Cell Therapy

7.4 Cell and Gene Therapy

7.4.1 Cell Therapy

7.4.1.1 Cell Therapy (by Type)

7.4.2 Gene Therapy

8 Global Regenerative Medicines Market (by Application), $Million, 2019-2030

8.1 Overview

8.2 Orthopedic and Musculoskeletal

8.3 Oncology

8.4 Wound Care

8.5 Cardiology

8.6 Immunology

8.7 Dermatology

8.8 Other Applications

9 Global Regenerative Medicines Market (by Region), $Million, 2019-2030

10 Company Profiles

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Being bionic: the future of regenerative medicine – Toronto Star

April 17th, 2021 1:51 am

Six decades ago, two researchers at the Ontario Cancer Institute at Princess Margaret Hospital made a startling discovery. James Till and Ernest McCulloch had found transplantable stem cells, special building block cells that have the ability to grow into any kind of human tissue.

Till and McCulloch were studying the effects of radiation at the time, but their work set off an explosion of research aimed at harnessing stem cells to treat all kinds of diseases and conditions. Subsequent breakthroughs in stem cell therapy have been used to treat more than 42,000 patients for hemophilia, restore sight to blind mice and even help a 78-year-old man regrow the end of a sliced-off fingertip. And researchers are still unlocking what might be possible.

The potential of regenerative medicine is astounding, says Michael May, president of the Centre for Commercialization of Regenerative Medicine (CCRM), a Toronto non-profit that helps bring new stem cell therapies and other regenerative medicine technologies to market. Researchers are harnessing stem cells to repair, replace or regenerate human cells, tissues and organs with the aim of improving treatments for conditions ranging from diabetes to blindness to heart failure and cancer.

More recent advances most notably Shinya Yamanakas Nobel Prize-winning 2012 discovery that regular adult tissue cells can be reprogrammed to become stem cells again, therefore endowing them with the ability to become any type of cell in the body have also ushered in a new wave of regenerative medicine research and what May calls a global race to bring newly possible cell therapies to market.

As president of CCRM, Mays job is to help move some of that research from the laboratory into the real world. Over the last decade, his organization has helped 11 companies come to market with regenerative medicine technologies, such as Montreals ExCellThera, which provides new therapeutic options for patients who suffer from myeloid leukemia and lack a traditional bone marrow donor.

While the last decade was defined by research and technological breakthroughs, May says the next decade will be all about lowering manufacturing costs and tackling patient access bottlenecks. Last November, CCRM announced that it would partner with McMaster Innovation Park in Hamilton to create Canadas first commercial-scale factory for making cells, which will be able to produce billions of cells enough to treat thousands of patients per week.

Weve just scratched the surface of whats possible in regenerative medicine, May says. He envisions a time when well eventually use these techniques not just to cure and fix human bodies, but also make them better. Now we can make cells, we can design them by genetically engineering them to do things that they naturally do, but that can be more than nature designed, says May. He says the editing of human traits in this way could eventually augment human abilities to such an extent that theyre unrecognizable.

Biomaterials are another technology that could transform regenerative medicine. Before joining CCRM, May himself helped found a Toronto biomaterials startup called Rimon Therapeutics, which developed a smart dressing for chronic wounds that used special polymers to support the bodys natural healing process. Similar advanced biomaterials could eventually be used in combination with cell therapies to not just fight aging and degeneration, but to also prevent it entirely, and even improve upon the human bodys natural baseline health.

Fifty years from now if theres some sort of blindness, well have a lens on the eye that will automatically focus and react or change as the eye ages, he says.

Nick Zarzycki is a freelancer who writes about technology for MaRS. Torstar, the parent company of the Toronto Star, has partnered with MaRS to highlight innovation in Canadian companies.

Disclaimer This content was produced as part of a partnership and therefore it may not meet the standards of impartial or independent journalism.

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Russell Health Honored in Global Business Leaders Magazine’s ’20 Leading Companies of the Year 2021′ – PRNewswire

April 17th, 2021 1:51 am

WILLOWBROOK, Ill., April 14, 2021 /PRNewswire/ --Based in Willowbrook, Illinois, Russell Health is a national marketer and distributor of specialty medical products and services. Russell Health, Inc., was recently awarded as #2 in Global Business Leaders Magazine's "20 Leading Companies of the Year 2021." Based in Atlanta, Georgia, Global Business Leaders Magazine's mission 'focuses on exalting the contribution of leaders who have been the emissary for their respective industries.' Their 2021 Top 20 list features a collection of leaders across industries like medical technology, finance, marketing, blockchain solutions, industrial fabrication, and more. Read more here

Russell Health's full-page feature presents an article titled, "Russell Health: A Mini Amazon for Regenerative Medicine." It discusses the history of Russell Health Inc., ongoing research and benefits of Stem Cell Recruitment Therapy, and well-defined commentary about how Russell Health has redefined the medicine market, even during a global pandemic. Read Russell Health's featured article here

About Russell Health: Russell Health and its partners have distributed regenerative therapy products nationwide and achieved profound clinical outcomes in multiple therapeutic areas including cosmetics, wound care, pain management, podiatry, orthopedic, dentistry and gynecology. With their partners and suppliers, they work to provide innovative life-changing and sustaining products and therapies to patients and healthcare providers around the world.

Russell Health's Stem Cell Recruitment Therapyproducts are intended for homologous use to help repair, reconstruct or supplement the patient's joints or soft tissue as well as help to increase mobility while decreasing pain. These responsibly sourced acellular tissue allografts are helping people of all ages to recover from injuries and get their life back.

Pull Quotes:

"We have built a mini-Amazon for regenerative medicine." (Ryan Salvino, CEO of Russell Health)

"Our ultimate goal from the beginning has been to help people by providing safe alternatives to risky procedures and expensive treatments while offering an alternative to synthetic drugs and embracing more holistic and organic products. We want to continue to become the number one supplier of regenerative medicine in the U.S." (Jonathan Benstent, Vice President of Russell Health)

"While the pandemic caused major disruption throughout the industry, it managed to pivot patients and physicians toward alternative treatments such as Stem Cell Recruitment Therapy. This demand can help in further enhancing the discovery of new applications for Stem Cell Recruitment Therapy products. As a result, Russell Health is working with some of the top leaders in the regenerative medicine field to continue to grow and provide innovative products to customers and their patients." (Global Business Leaders Magazine)

Visit Russell Health online to learn more about Stem Cell Recruitment Therapy. For media inquiries or to contact the Russell Health team directly. Please visit http://www.russellhealth.comor email [emailprotected].

Contact: Veronica Bennett

Address & Phone: 621 Plainfield Rd., Willowbrook, IL 60527; 844-249-6200

Email: [emailprotected]

Online: http://www.russellhealth.com

Social Media: http://www.linkedin.com/company/russell-health:: https://www.facebook.com/russellhealthinc:: https://www.instagram.com/russellhealth:: https://twitter.com/health_russell

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Cellino Biotech developing tech to help scale stem cell therapies – MedCity News

April 17th, 2021 1:51 am

In response to emailed questions, Cellino Biotech CEO and Co-founder Dr. Nabiha Saklayen, talked about the formation of the company and its goal to make stem cell therapies more accessible for patients.

Why did you start this company?

I see a huge need to develop a technology platform to enable the manufacture of cell therapies at scale. We recently closed a $16 million seed financing round led by Khosla Ventures and The Engine at MIT, with participation from Humboldt Fund. Cellino is on a mission to make personalized, autologous cell therapies accessible for patients. Stem cell-derived regenerative medicines are poised to cure some of the most challenging diseases within this decade, including Parkinsons, diabetes, and heart disease. Patient-specific cells provide the safest, most effective cures for these indications. However, current autologous processes are not scalable due to extensive manual handling, high variability, and expensive facility overhead. Cellinos vision is to make personalized regenerative medicines viable at large scale for the first time.

How did you meet your co-founders?

Nabiha Saklayen.

I met my co-founder Marinna Madrid in my Ph.D. research group. We had worked together for many years and had a fantastic working relationship. I then met our third co-founder Matthias Wagner through a friend. Matthias had built and run three optical technology companies in the Boston area and was looking to work with a new team. I was thrilled when we decided to launch the startup together at our second meeting. Matthias built the first Cellino hardware systems in what I like to call Matthias garage. In parallel, I was doing hundreds of expert interviews with biologists in academia and industry, and it started to narrow down our potential applications very quickly. Marinna was doing our first experiments with iPSCs. We iterated rapidly on building new versions of the hardware based on the features that were important to industry experts, such as single-cell precision and automation. Its incredible to witness our swift progress as a team.

What specific need or pain point are you seeking to address in healthcare/life sciences?

In general, autologous therapies are safer for patients because they do not require immunosuppression. The next iteration of cell therapies would use patient-specific stem cells banked ahead of time. Anytime a patient needs new cells, such as blood cells, neurons, or skin cells, we would generate them from a stem cell bank.

Today, patient-specific stem cell generation is a manual and artisanal process. A highly skilled scientist sits at a bench, looks at cells by eye, and removes unwanted cells with a pipette tip. Many upcoming clinical trials are using manual processes to produce stem cells for about ten to twenty patients.

At Cellino, we are converging different disciplines to automate this complex process. We use an AI-based laser system comes to remove any unwanted cells. By making stem cells for every human in an automated, scalable way, we are working towards our mission at Cellino to democratize personalized regenerative medicine.

What does your technology do? How does it work?

Cellinos platform combines label-free imaging and high-speed laser editing with machine learning to automate cell reprogramming, expansion, and differentiation in a closed cassette format, enabling thousands of patient samples to be processed in parallel in a single facility.

In general, autologous, patient-specific stem cell-derived therapies do not require immunosuppression and are safer for patients. Today, patient-specific stem cells are made manually, by hand. To scale the stem cell generation process, Cellino converges different disciplines to automate this complex process. We train machine learning algorithms to characterize cells before our AI-based laser system removes any unwanted cells. By making stem cells for every human in an automated, scalable way, our mission at Cellino is to democratize personalized regenerative medicine. Thats why our vision statement is Every human. Every cell.

Whats your background in healthcare? How did you get to where you are today?

When I arrived at Harvard University for my Ph.D. in physics, I wanted to be closer to real-world applications. Biology is inherently complex and beautiful, and I was interested in developing new physics-based tools to engineer cells with precision. During my Ph.D., I invented new ways to edit cells with laser-based nanomaterials. I collaborated with many brilliant biology groups at Harvard, including the Rossi, Scadden, and Church labs. Working closely with them convinced me that lasers offer a superior solution to editing cells with high precision. That realization compelled me to launch Cellino.

Do you have clinical validation for your product?

Our immediate goal for the next year is to show that our platform can produce personalized, high-quality, R&D-grade stem cells for different patients, which has not been established in an automated manner in the regenerative medicine industry so far. There is significant patient-to-patient variability in manual cell processing, which we eliminate with our platform.

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Lessons learned: W&M students working in healthcare reflect on a year of pandemic – William & Mary News

April 17th, 2021 1:51 am

Scribe and student: For the past five years, Brooke Ford has worked her way through college as an emergency room scribe at a few local hospitals. In her senior year at William & Mary, as the COVID-19 pandemic ravaged the nation, she managed medical scribes at two hospitals. Courtesy photo

by Adrienne Berard | April 16, 2021

For the better part of the past year, Brooke Fords return home from work has been an exercise in mitigating the spread of a potentially deadly pathogen.

She pulls off all her personal protective equipment, washes her hands, showers off, changes clothes, throws out any food or drink she may have touchedand greets her family.

I know a lot of people who were scared to even live with their families when they're working in a hospital room, because they didn't want to bring it home, said Ford, a William & Mary health sciences major who graduated in December.

For the past five years, Ford has worked her way through college as an emergency room scribe at a few local hospitals. In her senior year, as the COVID-19 pandemic ravaged the nation, she managed medical scribes at hospitals in Hampton and Newport News.

A scribe is responsible for recording the details of a patient's clinical history, current health issues, diagnoses and any medical procedures that are performed or prescribed, Ford explained. The scribe serves a vital role of being the ears and eyes in the room, so that a physician can focus squarely on treating the patient.

I lead teams at two hospitals, Ford said. During the pandemic, that has been really difficult because I had people that didn't want to come to work, which, I mean, it's scary. I dont blame them for that, but we had to learn to balance the fear and the need to do our jobs.

That balance between fear and the vital work ahead is one that has been familiar to healthcare professionals throughout the world this past year. For students graduating into the industry, the landscape has shifted dramatically, revealing fault lines in Americas healthcare system that these newest professionals are uniquely positioned to witness, study and address.

Matthew Tucker 22 started volunteering as an EMT with the Williamsburg Volunteer Fire Department his freshman year at William & Mary. Hes now the collegiate lieutenant, responsible for managing and training college students on the operations of emergency medical services provided by the local fire station.

I help train incoming college students on operations of the fire station, the ambulance, the equipment, all of those sorts of thing, he said.

This winter, as COVID-19 case numbers surged nationwide, Tucker was one of a handful of student volunteers who worked as first responders, supporting the career EMT staff at the fire station.

I know that the staff paramedics were especially burnt out. They couldnt trade shifts, they couldn't utilize their vacation time, so we were able to provide resources to support them in getting some relief, Tucker said. As students, we were able to see things that we haven't seen for 100 years. The amount of learning that can be done in that environment is astounding, and it has given me tremendous perspective on my future medical path.

Tucker said, as a student concentrating in public health, hes been particularly aware of the health disparities in the community he serves. It was immediately clear to him that the vast majority of emergency calls were coming from low-income neighborhoods.

I believe that we're often responding to places where the healthcare system has failed, where we are their primary care provider, a call to 911, taking them to the emergency room, he said. These disparities have always been there, but we're seeing them very plainly now, how socioeconomic status, job accessibility, food insecurity, all these different things affect someone's risk to COVID and ultimately affect people's health status. Weve not always been in tune to seeing that before the pandemic.

Sophie Kopec 21 spent this past summer analyzing data on experimental, early-stage clinical trials for the cell and gene medicine division of the non-profit Alliance for Regenerative Medicine. Explaining what she does is a key part of the job.

Its such an up-and-coming field that there are negative trigger words, all this stigma, especially around cell therapy, she said. So, part of my job was to look at data from all of these regenerative medicine clinical trials to identify the most promising ones. Then we translate that data into the commercial field to help show consumers that this is real, valid, verified science. Its not some kind of scam.

Kopec describes regenerative medicine as using the body's own functions to train it to fix itself.

For example, take gene therapy, in that case you have editing technology like CRISPR, where you can go in and actually edit the bodys genes and reprogram them to respond to the treatment and then body essentially heals itself, she said.

Kopec entered William & Mary with her sights hard-set on chemistry. It wasnt until her junior year, when she was working her way down the list of general education requirements, that she enrolled in a Foundations of Epidemiology course with Assistant Professor of Kinesiology and Health Sciences Carrie Dolan.

After taking that class, everything changed and I shifted my focus to public health, Kopec said. Ill always be grateful to William & Mary for that. I came into school with tunnel vision. I was going to go to med school and work in scrubs or in a lab, but my education broadened my perspective. Actually, it did more than that, it revealed my passion to me. Had I not been forced off track by the general education requirements, I would have never found this incredible spark and pursued this passion that I have now.

Graduation can be an anxiety-inducing milestone, even in the best of times. For those entering careers in the healthcare industry, the uncertainty of the pandemic has added another layer of stress and hope for overcoming the challenges of the past.

Next month, Kopec starts her new job as a clinical research coordinator at Children's National Hospital in D.C. Shell be serving in the neuro-oncology unit, working with children who have not responded to traditional therapy measures and providing experimental treatment that is often the last and only resort left. Her first in-person day at the hospital is scheduled for June 1.

Well be doing clinical trials that offer more therapeutic approaches to treating these late-stage tumors, she said. Even if the end result for some of these patients may be death, at least we know that we did everything in our power to help them and give them another chance to keep fighting and have another opportunity at life.

When Ford began her studies at William & Mary, she was interested in emergency medicine. She wanted to learn to respond in crisis to keep people alive. Now, after all she has witnessed in this year of pandemic, she hopes to study palliative care. She wants to learn how to help people live and die with dignity.

In medical school, they teach you how to save people, but what happens when you cant save them, when there is nothing else you can do? she said. Its not a conversation were often prepared for, but its so important. In a lot of ways, how can you know how you want to live if you don't know how you want to die?

So many families this year have had to make those choices for family members and they don't know what choices those family members would want to make for themselves, she added. Theyve had to make those choices, because those conversations haven't been had. I want to be there to help have those hard conversations.

After graduating from William & Mary, Tucker plans to go to medical school to obtain Master of Public Health and Medical Doctor degrees. He plans to work primarily with people experiencing homelessness and be able to treat them holistically.

If theres one main thing that I think that weve all learned this pandemic, its that health is incredibly complex, Tucker said. Truly, everything that we do impacts our health, every societal factor, institutional factor, personal factor that's put into place influences somebodys health.

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Organicell and Oklahoma State University Enter Into Agreement To Study Zofin in Respiratory Diseases Caused By Virus Infections Of Pandemic Potential…

April 17th, 2021 1:51 am

MIAMI--(BUSINESS WIRE)-- Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR), a clinical-stage biopharmaceutical company dedicated to the development of regenerative therapies, today announced that it has entered into an agreement with Oklahoma State University to evaluate Zofin for the treatment of respiratory diseases caused by virus infections of pandemic potential. This study site follows the Companys earlier announcement of the agreement reached with the Centers for Disease Control and Prevention (CDC) to conduct research to determine the anti-inflammatory and anti-infective effectiveness of Zofin in experimental models of influenza infection.

The ongoing COVID-19 pandemic has infected over 5 million people globally and caused over 300,000 case fatalities in 188 countries. Additionally, the CDC estimates between 39-55 million influenza related illnesses and more than 60,000 deaths due to influenza. The observed illnesses and case fatalities are primarily due to the lack of known effective antivirals and prophylactic vaccines to attenuate the viruses.

In this agreement, Organicell will supply Oklahoma State University with its lead compound, Zofin, which is an acellular material derived from human amniotic fluid. This extracellular vesicle-derived nanoparticle-based therapeutic will be assessed on its ability to induce antiviral and/or immunomodulatory activity against virus infections of pandemic potential as there is an unmet need for non-toxic and effective therapeutic approaches to deal with current and imminent pandemics.

Organicell is dedicated to continuing regenerative therapy research for diseases with unmet needs. We are thrilled to be partnering with Oklahoma State University and the CDC on the study of Zofin for viral infections of pandemic proportions. A year into the pandemic has shown us the importance of research aimed at the development of biologics, said Albert Mitrani, CEO of Organicell.

We are excited to partner with Oklahoma State University and the CDC to investigate how the extracellular vesicle-derived nanoparticles, in Zofin, will impact antiviral and/or immunomodulatory activity of viral infections with pandemic potential. Dr. Mari Mitrani, Chief Science Officer of Organicell.

About Zofin:

Zofin is an acellular biologic therapeutic derived from perinatal sources and is manufactured to retain naturally occurring microRNAs, without the addition or combination of any other substance or diluent. This product contains over 300 growth factors, cytokines, and chemokines as well as other extracellular vesicles/nanoparticles derived from perinatal tissues. Zofin is currently being tested in a phase I/II randomized, double blinded, placebo trial to evaluate the safety and potential efficacy of intravenous infusion of Zofin for the treatment of moderate to SARS related to COVID-19 infection vs placebo.

ABOUT ORGANICELL REGENERATIVE MEDICINE, INC.

Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR) is a clinical-stage biopharmaceutical company that harnesses the power of exosomes to develop innovative biological therapeutics for the treatment of degenerative diseases. The Companys proprietary products are derived from perinatal sources and manufactured to retain the naturally occurring exosomes, hyaluronic acid, and proteins without the addition or combination of any other substance or diluent. Based in South Florida, the company was founded in 2008 by Albert Mitrani, Chief Executive Officer and Dr. Mari Mitrani, Chief Scientific Officer. To learn more, please visit https://organicell.com/.

FORWARD-LOOKING STATEMENTS

Certain of the statements contained in this press release should be considered forward-looking statements within the meaning of the Securities Act of 1933, as amended (the Securities Act), the Securities Exchange Act of 1934, as amended (the Exchange Act), and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are often identified by the use of forward-looking terminology such as will, believes, expects, potential or similar expressions, involving known and unknown risks and uncertainties. Although the Company believes that the expectations reflected in these forward-looking statements are reasonable, they do involve assumptions, risks and uncertainties, and these expectations may prove to be incorrect. We remind you that actual results could vary dramatically as a result of known and unknown risks and uncertainties, including but not limited to: potential issues related to our financial condition, competition, the ability to retain key personnel, product safety, efficacy and acceptance, the commercial success of any new products or technologies, success of clinical programs, ability to retain key customers, our inability to expand sales and distribution channels, legislation or regulations affecting our operations including product pricing, reimbursement or access, the ability to protect our patents and other intellectual property both domestically and internationally and other known and unknown risks and uncertainties, including the risk factors discussed in the Companys periodic reports that are filed with the SEC and available on the SECs website (http://www.sec.gov). You are cautioned not to place undue reliance on these forward-looking statements All forward-looking statements attributable to the Company or persons acting on its behalf are expressly qualified in their entirety by these risk factors. Specific information included in this press release may change over time and may or may not be accurate after the date of the release. Organicell has no intention and specifically disclaims any duty to update the information in this press release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210414005207/en/

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First in the nation, FDA-approved Phase II mesenchymal stem cell therapy for Parkinson’s disease begins – Newswise

April 17th, 2021 1:51 am

Newswise A Phase II clinical trial to assess mesenchymal adult stem cells as a disease-modifying therapy for Parkinson's disease has been launched at The University of Texas Health Science Center at Houston (UTHealth).

"Studies have shown mesenchymal stem cells can migrate to the sites of injury and respond to the environment by secreting several anti-inflammatory and growth factor molecules that can restore tissue equilibrium and disrupt neuronal death," said Mya C. Schiess, MD, professor in the Department of Neurology and director and founder of the movement disorder subspeciality clinic and fellowship program at McGovern Medical School at UTHealth. "The stem cells interact directly with the immune cells, leading to an anti-inlammatory state that allows a restorative process to take place."

Safety and tolerability results, assessed on a previous trial, were recently published in the journal Movement Disorders. The Phase I study showed that there were no serious adverse reactions related to the stem cell influsion and no immunological reactions to the cells, which come from the bone marrow of a healthy adult donor. The study enrolled 20 patients with mild to moderate disease, who were infused with one of four different dosages and monitored for a year. Additionally, researchers reported a reduction in preripheral inflammatory markers and a reduction in motor symptoms.

Parkinson's diease is the second most common neurodegenerative disease, affecting more than a million Americans. It is also the fastest-growning of the neurodegenerative diseases, with more than 60,000 new cases identified every year. It is predicted that by 2040, Parkinson's disease will affect 17.5 million people worldwide.

Research has shown that one of the forces playing a critical role in the diease's development and progression is a chronic neuroinflammatory process that damages the brain's microenvironment and alters its healthy equilibrium. Inflammatiion perpetuates the neurodegenration in the brain areas that control movement, causing the tremors, imbalance, loss of speech, slowness, and other motor impairments.

The randomized, double-blind, placebo-controlled Phase II trial will investigate the safest and most effective number of repeat doses of stem cells to slow the progression of Parkinson's disease. The study will enroll 45 patients, ages 50 to 79, who will receive three infusions of either placebo or stem cell therapy at three-month intervals and will be followed for a year after the last infusion.

"Currently, there is no approved therapy that can delay the degenerative process in Parkinson's disease," Schiess said. "By investigating a treatment that can slow or stop the progression, we hope to improve the quality of life of those suffering from the disease. The ultimate goal is to use this treatment in individuals with a prodromal condition, meaning they are showing early signs of Parkinson's disease but are not yet clinically symptomatic. We hope to be able to potentially stop the diease's conversion or clinical manifestation in patients who are high-risk."

The Phase II trial, approved by the U.S. Food and Drug Administration, is supported with funding from the Michael J. Fox Foundation, John S. Dunn Foundation, and John and Kyle Kirksey.

Other McGovern Medical School faculty co-authors on the paper included Jessika Suescun, MD, Christopher Adams, MD, and Sean Savitz, MD, in the Department of Neurology. Marie-Francoise Doursout, PhD, Department of Anesthesiology; Charles Green, PhD, Department of Pediatrics; and Jerome G. Saltarrelli, PhD, Department of Surgery. Timothy M. Ellmore, PhD, Department of Psychology at the City College of New York, N.Y., was senior author.

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QC Kinetix (Myrtle Beach) is the Stem Cell Therapy and Regenerative Medicine Clinic Myrtle Beach – Press Release – Digital Journal

April 17th, 2021 1:51 am

Myrtle Beach, SC - QC Kinetix (Myrtle Beach) takes a different healthcare approach to most clinics in Myrtle Beach. It is a regenerative medicine clinic that focuses on non-surgical methods of managing pain. Regenerative medicine focuses on whole-body healing by using blood, cells, and tissues to help repair damaged areas and lessen inflammation and pain. Whether the pain is orthopedic or deep in the muscles and tendons, the clinics regenerative medicine technique is a non-invasive way to heal the body.

The physicians at QC Kinetix (Myrtle Beach) believe that whether a patient is suffering from persistent pain caused by an injury, disease, or age can permanently solve the problem. They do this by focusing on several different medical methods to help their patients regain their lives pain-free, to perform daily tasks without difficulty. The clinic treatments include the use of lasers, ultrasounds, regenerative cells, and platelet-rich plasma.

For the success of platelet-rich plasma injections or PRP therapy, the team mixes the patients platelets with plasma before injecting it into the injured area. Another area of regenerative therapy methods the clinic uses is called stem cell therapy. The stem cell therapy technique involves the team taking stem cells from a patients bone marrow and injecting them into inflamed or damaged areas. This injection of stem cells prompts the body to generate more red blood cells, reducing inflammation and pain.

QC Kinetix (Myrtle Beach) guarantees that all the patients who trust them will feel increased pain relief from one of their regenerative medicine treatments. The pain clinic ensures that they alleviate pain on several parts of the body, including the shoulder, elbow, wrist, ankle, low back, hip, knee, and feet.

To alleviate the pain from these areas, the clinic offers an array of services for Myrtle Beach residents. They include QC Knee that treats knee pain, cracking/popping, knee arthritis, and injuries such as torn ACL, MCL, LCL, and meniscus. QC Injury for patients of acute sports-related and musculoskeletal injuries and joint pain. QC 2M takes care of joint pain due to arthritis in the knees, shoulder, ankle, wrist, feet, hands, elbow, lower back, hips, and more.

Patients who visit the Myrtle Beach knee pain doctoralso enjoy QC Per4M, a treatment to improve energy, muscle strength, endurance, and appropriate weight loss with hormone replacement therapy. And QC Medical for patients with non-sports-related musculoskeletal pain resulting from sciatica, plantar fasciitis, or tendonitis.

The entire QC Kinetix staff (Myrtle Beach) believes that regenerative medicine, including stem cell therapy, works on several conditions. They include arthritis, plantar fasciitis, muscle damage, orthopedic pain, and tendon issues all over the body.

To learn more about QC Kinetix (Myrtle Beach) - Myrtle Beach, joint pain doctor, call (843) 310-2703 to schedule an appointment. Or visit their location, 8210 Devon Ct Suite A, Myrtle Beach, SC, 29572, US. For any inquiries about the services they offer, visit their website for more information.

Media ContactCompany Name: QC Kinetix (Myrtle Beach)Contact Person: Adam RoseEmail: Send EmailPhone: (843) 310-2703Address:8210 Devon Ct Suite A City: Myrtle BeachState: SCCountry: United StatesWebsite: https://qckinetix.com/myrtle-beach/

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