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Archive for the ‘Gene therapy’ Category

Catalent introduces cryogenic capabilities to support growing demand for cell and gene therapy – gasworld

Saturday, April 17th, 2021

Sharing the news on Thursday (15th April), the company said the investment has enabled part of the facility to be dedicated to the safe handling of cell and gene therapy samples, including the installation of cryogenic storage, allowing biological materials to be preserved in liquid nitrogen vapor at temperatures of around -180C.

The facility also has the ability to package, label and distribute cryogenic materials, ensuring the integrity of the therapies being prepared for clinical trials, and has been designed so that capacity can be rapidly expanded further to meet growing clinical supply needs, as well as future commercial demand.

Establishing robust clinical supply chain services for cell and gene therapies is complex and challenging, and Catalent has undertaken an in-depth strategic review to evaluate how it can establish a safe, efficient and flexible approach to support this fast-growing area of the industry, said Ricci Whitlow, President, Catalent Clinical Supply Services.

The solution we have implemented at Philadelphia not only meets current needs, but also provides a template for us to easily replicate at other facilities in our global network, allowing incremental capacity expansion within the new infrastructure as demand grows.

With sites in the US, UK, Germany, Singapore, Japan and China, and an extended network of over 50 depots, Catalents clinical supply services can handle a broad range of international compliance and distribution requirements to support global clinical trials.

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Catalent introduces cryogenic capabilities to support growing demand for cell and gene therapy - gasworld


Bioprocessing and Bioproduction Trends in Cell and Gene Therapies – Technology Networks

Saturday, April 17th, 2021

Cell and gene therapies hold great promise for treating human diseases, for which current therapeutics are lacking. However, the research and development process for delivering these therapies to the clinic is complex, comprising various bioprocessing and bioproduction steps that must be fulfilled and optimized to ensure the safety and efficacy of the final product. The process continues to evolve to meet increasing demand and to overcome unexpected obstacles, such as the impact of the COVID-19 global pandemic on supply chains and manufacturing efforts in the biotech and biopharma industries.In this interview, Technology Networks spoke with Neal Goodwin, PhD, Chief Scientific Officer at Teknova, to learn more about current bioprocessing and bioproduction trends in the cell and gene therapy space. Goodwin also discusses the impact of the COVID-19 pandemic on the biopharmaceutical sector and highlights how the industry has united to contribute to the development of vaccines at this critical time.

Molly Campbell (MC): You have 20 years of experience working in biotechnology and therapeutics. Can you talk about the evolution of the field through this time?Neal Goodwin (NG):It has been a great 20 years! The human genome release is one of the most dramatic events during those 20 years. Once we had the human genome at our disposal, the field burgeoned. We've seen targeted therapeutics make a significant impact in personalized healthcare imatinib, erlotinib, and the new KRAS inhibitors, among other targeted therapeutics for oncology, come to mind. Also, the first immuno-oncology therapeutics are now positively affecting large numbers of patients which is terrific.

The ability to move into gene therapy and address severe genetic disorders and conduct gene replacement therapy is fantastic. The progress in gene delivery viral vectors and non-viral nanoparticle delivery contributed to the rapid advancement of prominent COVID-19 vaccines. The mRNA-based vaccines and non-viral nanoparticle-based gene delivery technologies have validated a promising approach to gene therapy that is being expanded to rare genetic diseases. The ability to mass-produce mRNA vaccines demonstrated the feasibility of mRNA treatments and gene therapies for more widespread use for treating broader therapeutic targets and conditions.

MC: Can you talk to us about some of the current trends you are seeing in the bioprocessing and bioproduction space?NG: First of all, there is a considerable demand for bioprocessing and commercial production of viral and non-viral vectors. These efforts must support what is now well over 1,000 clinical development programs in gene therapy alone. There is a need to develop better individualized processes that are readily scalable for GMP production matched to specific therapeutics. This effort will require the implementation of new strategies, new technologies, and increased global manufacturing capacity. It is an exciting place to be, but there is a great deal of development left to achieve streamlined processes and optimized capacity.

MC: Cell and gene therapies are key emerging markets. What are some of the key challenges that exist when developing these products from the lab, through to clinical testing and eventually authorization?NG:There is a wide breadth of requirements I will try to narrow them down to a few of the essential points. Some autologous cellular therapies require cells to be genetically modified ex vivo. This genetic modification is usually done with viral vectors, and a crucial aspect is the delivery of the gene, i.e., the active pharmaceutical ingredient, to the cells. There is a need for more advanced lentiviral vector systems that are safer for use in systemic gene delivery.

Among the non-cellular gene delivery segment, adeno-associated virus (AAV) vector gene delivery is the most widely used viral gene delivery platform. AAV production relies on the efficient expression in human cell lines of plasmids that encode viral packaging, capsid and replication genes, and expression plasmids encoding the therapeutic gene. One of the drawbacks of AAV vectors employed for gene therapy is they generally cannot deliver larger gene fragments required for optimal treatments. So, better viruses or non-viral gene delivery systems are needed.

In turn, choosing the best cell manufacturing conditions that provide the most efficient viral vector production is essential. A key challenge is how to scale up with reproducibility and efficiency.

MC: How can we look to overcome the challenge of scale-up in cell and gene therapies?NG: Because therapeutic programs are each different, having adaptable processes to individual programs is vital. Some strategies focus on gene delivery into autologous cells or allogeneic cells that become expanded and transplanted into patients other strategies require direct delivery of viral or non-viral gene therapy products into patients.In viral gene delivery, matching the most suitable virus to achieve the best target tissue tropism is essential. To date, AAV has been successfully used in two FDA-approved gene therapies, with many in advanced clinical trials. Traditionally, there have been few available AAV serotypes available for gene delivery. However, now we can use a directed evolution approach to manipulate and generate AAV capsids that have better target tissue tropism with reduced host immunogenicity.

Concerning AAV production, the purification processes of viruses need optimized, which is a focus of Teknova. We are barcoding different AAV serotypes with specific processes to achieve higher purity and viral vector yield.

MC: How has the COVID-19 impacted the biotech sector?NG: COVID-19 motivated and united the biotech sector. It remains a worldwide effort where enormous challenges are being addressed rapidly and collaboratively. The field appears more collaborative, almost cheering once competitive firms and laboratories. Teknova experienced this by retooling and supplying GMP viral transport media for COVID-19 testing and providing the global research community with research reagents and custom GMP manufactured products. This transformation is something that instils pride.

MC: How do you envision the biotech sector will change over the next few years? Are there any areas, aside from cell and gene therapies, that excite you?NG: The biotech sector's attitude is positive and more confident than 20 years ago, and this spirit enables us to tackle problems once considered unsolvable.

One of the most exciting biotech advancement areas is precision therapeutics. For example, a difficult hurdle to overcome was treating cancers linked to specific mutant KRAS alleles in a high percentage of cancers. Multiple firms have recently advanced precision therapeutics to late-stage clinical trials to treat this historically untreatable cancer type precisely. I think this can-do attitude will continue.

Another area where I expect to see progress is gene therapy delivery that will increasingly evolve non-viral delivery technologies. This evolution will allow precise targeting of therapeutics delivery and undoubtedly improve human health through this process.

Neal Goodwin was speaking to Molly Campbell, Science Writer for Technology Networks.

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Bioprocessing and Bioproduction Trends in Cell and Gene Therapies - Technology Networks


Alliance for Cancer Gene Therapy Summit 2021 Features World Renowned Cancer Researchers Advancing Solid Tumor Breakthroughs – WFMZ Allentown

Saturday, April 17th, 2021

STAMFORD, Conn., April 16, 2021 /PRNewswire-PRWeb/ --April 29 marks a pivotal moment in cancer research when the world's brightest minds come together to discuss how to translate the success of CAR T-cell therapies for blood cancers into successful cell and gene therapies for the most complex and deadly solid tumor cancers. The scientists and companies driving the latest advances in cancer cell and gene therapy will gather online for a virtual Summit with the Alliance for Cancer Gene Therapy, who envision a cancer free future and want to change the C-word from Cancer to Cure. The Summit is open to the public, to medical professionals, scientists and companies interested in cell and gene therapy to fight cancer. To register for the Summit, visit

Summit 2021 is being held online on Thursday, April 29, 2021, from 10:00 a.m. until 6:00 p.m (ET), and features eight (8) panel discussions with leading researchers developing the next generation cancer cell and gene therapies, biotech companies who are bringing new treatments through the clinic, and investors who are funding this burgeoning pipeline of solid tumor breakthroughs.

The panels include:

Keynote: Does Cancer Have a Future? What's Next? Where Will We Be in 2025?

A conversation with Nobel Laureate James P. Allison, PhD, executive director of the Immunotherapy Platform at MD Anderson Cancer Center, and Carl H. June, MD, director of the Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania. The conversation will be moderated by Siddhartha Mukherjee, MD, founder, Myeloid Therapeutics, Pulitzer-prize winning author of "The Emperor of All Maladies."

Evolving Cell and Gene Regulatory and Manufacturing Processes to Match the Speed of Innovation

Panel discussion with: Rafael Amado, MD, executive vice president, head of R&D and chief medical officer, Allogene Therapeutics; Andre Choulika, PhD, chairman, CEO and founder, Cellectis; Amy DuRoss, founder and CEO, Vineti; Bruce Levine, PhD, Barbara and Edward Netter professor in Cancer Gene Therapy, University of Pennsylvania; Ke Liu, MD, PhD, senior vice president and head of Regulatory Affairs & Strategy at Sana Biotechnology, former FDA Oncology Center of Excellence associate director for Cell and Gene Therapy; and moderated by Asthika Goonewardene, managing director, Equity Research - Biotech, Truist Securities.

Innovating Science, From the Lab to the Clinic

Panel discussion with: Brian Brown, PhD, professor and associate director of the Precision Immunology Institute, Icahn School of Medicine, Mount Sinai; Yvonne Chen, PhD, associate professor of Microbiology, Immunology & Molecular Genetics, UCLA, Founder Kalthera; Franco Marincola, PhD, senior vice president and global head of Cell Therapy Research at Kite, a Gilead Company; Clodagh O'Shea, PhD, professor, Molecular and Cell Biology Laboratory, Salk Institute, scientific founder IconOVir Bio; and moderated by Jenna Foger, senior vice president, Science & Technology, Alexandria Venture Investments/Alexandria Real Estate Equities.

Tackling the Toughest Challenges Pancreatic Cancer

Panel discussion with: Sidi Chen, PhD, associate professor, Yale Cancer Center; Joseph A. Fraietta, PhD, director, Solid Tumor Immunotherapy Laboratory, Center for Advanced Cellular Therapeutics, University of Pennsylvania; Michael T. Lotze, MD, professor of Surgery and Bioengineering, University of Pittsburgh, chief cellular officer, Nurix, chair of Alliance for Cancer Gene Therapy's Scientific Advisory Council; Mark H. O'Hara, PhD, assistant professor of Medicine at the Hospital of the University of Pennsylvania; and moderated by Andrew Rakeman, PhD, vice president of Research, Lustgarten Foundation.

Tackling the Toughest Challenges Glioblastoma

Panel discussion with: Nduka Amankulor, MD, department of Neurosurgery, University of Pittsburgh Medical Center; Samantha Bucktrout, PhD, senior director of Research, Parker Institute of Cancer Immunotherapy; Noriyuku Kasahara, MD, PhD, principal investigator, Brain Tumor Center, UCSF; Crystal Mackall, MD, founding director, Stanford Center for Cancer Cell Therapy; and moderated by Klaus Veitinger, MD, PhD, venture partner OrbiMed.

New Approaches to Solid Tumor Breakthroughs

Panel discussion with: Ken Drazan, MD, chief executive officer, ArsenalBio; Daniel Getts, PhD, chief executive officer & co-founder, Myeloid Therapeutics; Garry E. Menzel, PhD, president and chief executive officer at TCR Therapeutics; and moderated by Luke Timmerman, founder and editor, The Timmerman Report.

Patients, Caregivers, Doctors, Oncologists, Advocates, Researchers, You, Me We all Have Cancer in Common

Panel discussion with: Caroline Corner, PhD, managing director, Westwicke; Robert Levis, director CLL Society; Callum Miller, CAR-T recipient, patient advocate; Tom Whitehead, co-founder, The Emily Whitehead Foundation; and moderated by Gregory C. Simon, former president of the Biden Cancer Initiative.

Innovating Finance, Non-Traditional Funding Sources

Panel discussion with: Jay Campbell, managing director of Cancer Research Institute Clinical Accelerator and Venture Fund; Marc Hurlbert, PhD, chief science officer, Melanoma Research Alliance; Ken Schaner, partner Schaner & Lubitz; Anna Turetsky, PhD, principal, Venture Investments, The Mark Foundation for Cancer Research; moderated by Luke Evnin, PhD, co-founder and managing director MPM Capital.

"It's so rare to have the opportunity to hear from the world's top cancer researchers, biotechs, investors and patients all in the same room," noted Barbara Lavery, chief program officer for Alliance for Cancer Gene Therapy. "We're excited to have Alliance for Cancer Gene Therapy be a catalyst for these important conversations that will help not only other scientists working on new cell and gene therapy approaches, but companies seeking investment to advance their clinical pipelines, and patients and their families looking for potentially life changing therapeutic options in their fight against cancer."

To learn more, see the list of speakers and panels and register for the event, visit the website Alliance for Cancer Gene Therapy Summit 2021.

Alliance for Cancer Gene Therapy

For 20 years, Alliance for Cancer Gene Therapy has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Alliance for Cancer Gene Therapy funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. 100% of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit, call 203-358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube @acgtfoundation.

Media Contact

Jenifer Howard, J. Howard Public Relations, +1 (203) 273-4246,

Twitter, Facebook

SOURCE Alliance for Cancer Gene Therapy

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Alliance for Cancer Gene Therapy Summit 2021 Features World Renowned Cancer Researchers Advancing Solid Tumor Breakthroughs - WFMZ Allentown


Gene therapy startup StrideBio signs collaboration deal with Duke University – WRAL Tech Wire

Saturday, April 17th, 2021

RESEARCH TRIANGLE PARK StrideBio, an emerging startup focusing on gene therapies which closed on a big $81.5 million round of venture capital in March, is partnering with Duke University as it expands efforts to create next-generation therapies.

The agreement announced Wednesday is described as a multi-technology collaboration.

Specific financial terms were not disclosed but includeequity, upfront and milestone-driven payments, and sponsored research commitments from StrideBio to Duke University. Also included are royalties on future product sales.

We are very excited to partner with Duke University to advance these technologies that can improve and expand on the potential benefits of gene therapies for patients who desperately need them, stated Sapan Shah, StrideBios CEO. We look forward to working together with a fantastic group of Duke researchers and clinicians to bring next-generation AAV-based gene therapies to patients with rare CNS diseases and beyond, starting with Alternating Hemiplegia of Childhood.

StrideBio is focused on what it calls engineered viral vectors, or AAV, for gene therapy. The firm has already announced it has struck a deal with Crispr Therapeutics to develop in vivo gene delivery applications. As part of the deal, StrideBio will receive development funding, milestones and royalties on licensed vectors, and retain certain rights to use the novel AAV vectors for gene therapy applications.

Heres how StrideBio describes the agreement and the tecnologies involved:

The agreements announced today provide StrideBio an exclusive license to multiple technologies that will enable best-in-class next-generation gene therapies developed at Duke University. Included are novel engineered AAV vectors which complement StrideBios existing STRIVETMcapsid engineering platform, having been selected through a cross-species evolution that results in significantly enhanced tropism and potency versus AAV9 across a wide range of tissues such as CNS, skeletal and cardiac muscle. Data on these novel vectors were presented by Duke researcher and StrideBio co-founder, Aravind Asokan, Ph.D., at the American Society of Gene & Cell Therapy 23rdAnnual Meeting in an abstract titled Cross Species Evolution of Synthetic AAV Strains for Clinical Translation (Gonzalez et al., ASGCT 2020, Abstract 24). In addition, StrideBio has licensed exclusive rights covering a novel use of IgG-degrading enzyme IdeZ to clear neutralizing antibodies in conjunction with AAV gene therapy administration. This innovative approach was recently published by members of the Asokan Lab in a manuscript titled Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme (Elmore et al., JCI Insight, 2020, 5(19): e139881). Finally, StrideBio obtained license rights to a novel gene therapy approach for the treatment of AHC recently published by Duke researcher Mohamad Mikati, M.D., in a manuscript titled AAV Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood (Hunanyan et al., Human Gene Therapy, February 12, 2021).

Gene therapy startup StrideBio lands $81.5M in capital, new investors

This License and Master Sponsored Research Agreement will ensure that these innovative technologies receive the resources and expertise necessary to develop treatments that can ultimately benefit patients. We are delighted to have StrideBio as a partner on this important effort in the gene therapy area, said Robin Rasor, Executive Director of the Office of Licensing and Ventures at Duke, in the announcement.

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Gene therapy startup StrideBio signs collaboration deal with Duke University - WRAL Tech Wire


Cell and gene therapy services market forecast to see significant growth over the next decade –

Saturday, April 17th, 2021

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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Cell and gene therapy services market forecast to see significant growth over the next decade -


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

Saturday, April 17th, 2021

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) ( 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)

AGTC IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) or

AGTC Corporate Contacts:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617)

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617)

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


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

Saturday, April 17th, 2021

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


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

Saturday, April 17th, 2021

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


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

Saturday, April 17th, 2021

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

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


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

Saturday, April 17th, 2021

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


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

Saturday, April 17th, 2021

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


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

Saturday, April 17th, 2021

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

Saturday, April 17th, 2021

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



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 at

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

For media enquiries, please contact:

Alex Heeley or Abdul Khalifeh

De Facto Communications

T: +44 (0) 203 735 8168

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


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

Saturday, April 17th, 2021

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


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

Saturday, April 17th, 2021

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

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 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...


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

Saturday, April 17th, 2021

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.


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 (Study Identifier: NCT04561258).


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

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

Saturday, April 17th, 2021

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'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 Cell and Gene Therapy Manufacturing QC Product Testing Microbial Testing Identity Purity Potency Viability 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 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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Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell – STAT

Friday, February 19th, 2021

Novartis and the Bill and Melinda Gates Foundation are joining forces to discover and develop a gene therapy to cure sickle cell disease with a one-step, one-time treatment that is affordable and simple enough to treat patients anywhere in the world, especially in sub-Saharan Africa where resources may be scarce but disease prevalence is high.

The three-year collaboration, announced Wednesday, has initial funding of $7.28 million.

Current gene therapy approaches being developed for sickle cell disease are complex, enormously expensive, and bespoke, crafting treatments for individual patients one at a time. The collaboration aims to instead create an off-the-shelf treatment that bypasses many of the steps of current approaches, in which cells are removed and processed outside the body before being returned to patients.


Sickle cells cause is understood. The people it affects are known. But its cure has been elusive, Jay Bradner, president of the Novartis Institutes for BioMedical Research, told STAT.

We understand perfectly the disease pathway and the patient, but we dont know what it would take to have a single-administration, in vivo gene therapy for sickle cell disease that you could deploy in a low-resource setting with the requisite safety and data to support its use, he said. Im a hematologist and can assure you that in my experience in the clinic, it was extremely frustrating to understand a disease so perfectly but have so little to offer.


Sickle cell disease is a life-threatening inherited blood disorder that affects millions around the world, with about 80% of affected people in sub-Saharan Africa and more than 100,000 in the U.S. The mutation that causes the disease emerged in Africa, where it protects against malaria. While most patients with sickle cell share African ancestry, those with ancestry from South America, Central America, and India, as well as Italy and Turkey, can also have the hereditary disease.

The genetic mutation does its damage by changing the structure of hemoglobin, hampering the ability of red blood cells to carry oxygen and damaging blood vessels when the misshapen cells get stuck and block blood flow. Patients frequently suffer painful crises that can be fatal if not promptly treated with fluids, medication, and oxygen. Longer term, organs starved of oxygen eventually give out. In the U.S., that pain and suffering is amplified when systemic and individual instances of racism deny Black people the care they need.

Delivering gene therapy for other diseases has been costly and difficult even in the best financed, most sophisticated medical settings. Challenges include removing patients cells so they can be altered in a lab, manufacturing the new cells in high volume, reinfusing them, and managing sometimes severe responses to the corrected cells. Patients also are given chemotherapy to clear space in their bone marrow for the new cells.

Ideally, many of those steps could be skipped if there were an off-the-shelf gene therapy. That means, among other challenges, inventing a way to eliminate the step where each patients cells are manipulated outside the body and given back the in vivo part of the plan to correct the genetic mutation.

Thats not the only obstacle. For a sickle cell therapy to be successful, Bradner said, it must be delivered only to its targets, which are blood stem cells. The genetic material carrying corrected DNA must be safely transferred so it does not become randomly inserted into the genome and create the risk of cancer, a possibility that halted a Bluebird Bio clinical trial on Tuesday. The payload itself mustnt cause such problems as the cytokine storm of immune overreaction. And the intended response has to be both durable and corrective.

In a way, the gene delivery is the easy part because we know that expressing a normal hemoglobin, correcting the mutated hemoglobin, or reengineering the switches that once turned off normal fetal hemoglobin to turn it back on, all can work, Bradner said. The payload is less a concern to me than the safe, specific, and durable delivery of that payload.

For each of these four challenges delivery, gene transfer, tolerability, durability there could be a bespoke technical solution, Bradner said. The goal is to create an ensemble form of gene therapy.

Novartis has an existing sickle-cell project using CRISPR with the genome-editing company Intellia, now in early human trials, whose lessons may inform this new project. CRISPR may not be the method used; all choices are still on the table, Bradner said.

Vertex Pharmaceuticals has seen encouraging early signs with its candidate therapy developed with CRISPR Therapeutics. Other companies, including Beam Therapeutics, have also embarked on gene therapy development.

The Novartis-Gates collaboration is different in its ambition to create a cure that does not rely on an expensive, complicated framework. Novartis has worked with the Gates Foundation on making malaria treatment accessible in Africa. And in October 2019, the Gates Foundation and the National Institutes of Health said together they would invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV that would be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, Trevor Mundel, president of global health at the Gates Foundation, said in a statement about the Novartis collaboration. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought.

Asked which is the harder problem to solve: one-time, in vivo gene therapy, or making it accessible around the world, David Williams, chief of hematology/oncology at Boston Childrens Hospital, said: Both are going to be difficult to solve. The first will likely occur before the therapy is practically accessible to the large number of patients suffering the disease around the world.

Williams is also working with the Gates Foundation, as well as the Koch Institute for Integrative Cancer Research at MIT, Dana-Farber Cancer Institute, and Massachusetts General Hospital, on another approach in which a single injection of a reagent changes the DNA of blood stem cells. But there are obstacles to overcome there, too, that may be solved by advances in both the technology to modify genes and the biological understanding of blood cells.

Bradner expects further funding to come to reach patients around the world, once the science progresses more.

There is no plug-and-play solution for this project in the way that mRNA vaccines were perfectly set up for SARS-CoV-2. We have no such technology to immediately redeploy here, he said. Were going to have to reimagine what it means to be a gene therapy for this project.

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Novartis and Gates Foundation Team Up To Deliver Affordable Sickle Cell Gene Therapy – BioSpace

Friday, February 19th, 2021

Taljat David/Shutterstock

Novartis and the Bill & Melinda Gates Foundation have partnered on a single-dose, in vivo gene therapy for sickle cell disease (SCD). The Foundation will offer funding for development of the therapy.

Existing gene therapy approaches to sickle cell disease are difficult to deliver at scale and there are obstacles to reaching the vast majority of those affected by this debilitating disease, said Jay Bradner, a hematologist and president of the Novartis Institutes for BioMedical Research (NIBR). This is a challenge that calls for collective action, and we are thrilled to have the support of the Bill & Melinda Gates Foundation in addressing this global unmet medical need.

The announcement comes only a day after bluebird bio announced that it has placed its Phase I/II and Phase III trial of LentiGlobin gene therapy for sickle cell disease (SCD) on temporary suspension. The cause is a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).

HGB-206 is the companys ongoing Phase I/II trial of LentiGlobin for SCD. It includes three cohorts, A, B and C. In Group C, a refined manufacturing process designed to increase vector copy number was used.

Group C also received LentiGlobin for SCD manufactured from hematopoietic (blood) stem cells (HSCs) collected from peripheral blood after mobilization with plerixafor, instead of by way of bone marrow harvest, which was the method used in Groups A and B.

HGB-210 is their ongoing Phase III single-arm open-label trial. It is evaluating efficacy and safety of LentiGlobin for SCD in patients between two years and 50 years of age with sickle cell disease.

Which underlines that even though gene therapy is making headway, it is still a cutting-edge technology.

SCD is a hereditary blood disease that affects millions of people globally, with more than 300,000 born with it each year. It primarily affects people of African descent, and sub-Saharan Africa bears about 80% of the disease burden. It affects the structure of red blood cells, causing a distinct sickle shape, which decreases the ability of red blood cells to transport oxygen efficiently.

Gene therapies might help end the threat of diseases like sickle cell, but only if we can make them far more affordable and practical for low-resource settings, said Trevor Mundel, president of Global Health at the Gates Foundation. Whats exciting about this project is that it brings ambitious science to bear on that challenge. Its about treating the needs of people in lower-income countries as a driver of scientific and medical progress, not an afterthought. It also holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.

Novartis also announced today that the U.S. Food and Drug Administration (FDA) approved the expanded indication for Entresto (sacubitril/valsartan) to decrease the risk of cardiovascular death and hospitalization for heart failure in adults with chronic heart failure. The biggest benefit was for patients with left ventricular ejection fraction (LVEF) below normal.

The expansion was based on data in the PARAGON-HF Phase III trial.

This approval is a significant advancement, providing a treatment to many patients who were not eligible for treatment before because their ejection fraction was above the region we normally considered reduced, said Scott Solomon, professor of Medicine at Harvard Medical School and Brigham and Womens Hospital, and PARAGON-HF Executive Committee co-chair. We can now offer a treatment to a wider range of patients who have an LVEF below normal.

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Charles River Dives Deep Into Cell and Gene Therapy With Cognate BioServices Acquisition – BioSpace

Friday, February 19th, 2021

Igor Golovniov/SOPA Images/LightRocket via Getty Images

Charles River Laboratories will acquire Cognate BioServices, a cell and gene therapy contract development and manufacturing organization, in an $875 million cash deal that will significantly expand the companys capabilities in the high-growth cell and gene therapy sector.

In addition to expanding Charles Rivers capabilities in the cell and gene therapy space, the company said the acquisition of Memphis, Tenn.-based Cognate will also establish a comprehensive solution from discovery and non-clinical development through CGMP manufacturing in advanced drug modalities. Cognate has a global presence with more than 500 employees. In January, Cognate announced plans to significantly expand cell and gene therapy manufacturing capacity, laboratory space, warehousing capabilities, and increase office support at its facilities inthe United StatesandEurope.

The company has extensive experience producing various cell types and technologies used in cellular immunotherapy and immuno-oncology, regenerative medicine and advanced cell therapy. Its primary area of expertise is in CGMP cell therapy manufacturing, which processes a variety of cellular products and other starting materials to develop and produce allogeneic (donor-derived) and autologous (patient-derived) cell therapies. Cognate also produces plasmid DNA, which is a foundational tool used in the development of gene-modified cell therapies and gene therapies, as well as other CDMO inputs, Charles River said.

James C. Foster, president and chief executive officer of Charles River Labs, said Cognate provides a synergistic fit for Charles River. He said bringing Cognate under the companys umbrella presents a unique opportunity to expand the companys capabilities and also enhance its offerings to clients in emerging areas of scientific innovation.

Additionally, Charles River said the addition of Cognate will complement its existing Biologics Testing Solutions business. Cognates capabilities will enable clients to seamlessly conduct analytical testing, process development, and manufacturing for advanced modalities with the same scientific partner, enabling them to achieve their goal of driving greater efficiency, the company said.

The addition of Cognate is also expected to provide a significant financial boost to Charles River. Cognate is expected to generate annual revenue of approximately $140 million in 2021, and is expected to grow at least 25% annually over the next five years, the company said.

This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions and enabling us to achieve our goal of establishing a single scientific partner to provide biopharmaceutical clients with an integrated solution to help accelerate their cell and gene therapy programs from discovery and non-clinical development through commercialization, Foster said in a statement. Because of the synergistic fit with Charles River, the market growth potential, and the emerging role of advanced drug modalities as treatments for oncology and rare disease, we believe Cognate will meaningfully enhance our long-term revenue and earnings growth potential.

The acquisition is expected to move quickly. Charles River anticipates the closing of the deal by the end of the first quarter of 2021. Based on the anticipated completion of the acquisition by the end of the first quarter, Cognate is expected to add approximately $110 million to Charles Rivers 2021 consolidated revenue for the partial year.

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