Archive for the ‘Gene Therapy Research’ Category

Reuters | Published: January 21, 2021 10:37:09 | Updated: January 21, 2021 14:27:09

Scientists in Beijing have developed a new gene therapy which can reverse some of the effects of ageing in mice and extend their lifespans, findings which may one day contribute to similar treatment for humans.

The method, detailed in a paper in the Science Translational Medicine journal earlier this month, involves inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing.

The specific therapy they used and the results were a world first, said co-supervisor of the project Professor Qu Jing, 40, a specialist in ageing and regenerative medicine from the Institute of Zoology at the Chinese Academy of Sciences (CAS).

These mice show after 6-8 months overall improved appearance and grip strength and most importantly they have extended lifespan for about 25%, Qu said.

The team of biologists from different CAS departments used the CRISPR/Cas9 method to screen thousands of genes for those which were particularly strong drivers of cellular senescence, the term used to describe cellular ageing.

They identified 100 genes out of around 10,000, and kat7 was the most efficient at contributing to senescence in cells, Qu said.

Kat7 is one of tens of thousands of genes found in the cells of mammals. The researchers inactivated it in the livers of the mice using a method called a lentiviral vector.

n liver cell and the mouse liver cell and for all of these cells we didnt see any detectable cellular toxicity. And for the mice, we also didnt see any side effect yet.

Despite this, the method is a long way from being ready for human trials, Qu said.

Its still definitely necessary to test the function of kat7 in other cell types of humans and other organs of mice and in the other pre-clinical animals before we use the strategy for human ageing or other health conditions, she said.

Qu said she hopes to be able to test the method on primates next, but it would require a lot of funding and much more research first.

In the end, we hope that we can find a way to delay ageing even by a very minor percentage...in the future.

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Gene therapy developed to delay ageing - The Financial Express

REGULATED INFORMATION

Clinical programs running on schedule, including Phase III trial of JTA-004 and Phase IIb trial of ALLOB

Extensive achievements in collaborations and partnerships to enhance development, manufacturing and commercialization capabilities

Strong financial position following fundraising, licencing agreement and optimisation of manufacturing assets

Gosselies, Belgium, 20January 2021, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today provides a business update for the fourth quarter, ending 31 December 2020 as well as a business outlook for 2021.

Bone Therapeutics managed to achieve a highly productive period in the last few months. We have made major advances in nearly all aspects of our activities while executing on our business strategy, said Miguel Forte, Chief Executive Officer, Bone Therapeutics. We recently started treating patients in the Phase IIb study with our allogeneic bone cell therapy product ALLOB. We also completed patient recruitment in the pivotal Phase III trial with the enriched viscosupplement, JTA-004, ready to provide topline data in the third quarter of this year. This will be a critical milestone in the history of Bone Therapeutics. In addition to our clinical programs, we have strongly improved our manufacturing capabilities with a partnership with Catalent, a leading global cell and gene CDMO, and extended the geographic reach of our bone cell therapies into Asia thanks to the licensing agreement with our Chinese partners Link Health and Pregene. We also have explored new grounds in the collaborations with Rigenerand and BioWin consortium. These collaborations will further expand the application of our innovative cell therapy platform and broaden our advanced clinical pipeline with potential new breakthrough developments. Building on the strong foundation of these recent achievements and a strengthened cash position, we are confident for 2021, continuing the progress we have already made and moving our allogeneic cell therapy and advanced biological products through clinical development while exploring new innovations that meet critical needs of patients.

Clinical highlights Q4 2020 to date

Corporate highlights Q4 2020 to date

Financial highlights Q4 2020 (1)

Outlook for 2021

Financial Calendar 2021

29 April Full Year Results & Annual Report 202026 May Q1 2021 Business and Financial Highlights9 June Annual General Meeting 20218 September Half Year Results 202126 October Q3 2021 Business and Financial Highlights

The financial calendar is communicated on an indicative basis and may be subject to change.

(1) Unaudited number

About Bone Therapeutics

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

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

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

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

For further information, please contact:

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

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

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

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

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

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Bone Therapeutics provides fourth quarter 2020 business update and 2021 outlook - GlobeNewswire

Large pharmaceutical companies have made gene therapy a priority with a series of acquisitions over the past several years, a stamp of validation for a field that's pushed through decades of ups and downs.

One of the latest buyers is German healthcare conglomerate Bayer, which in October inked a $2 billion deal for North Carolina gene therapy developer Asklepios Biopharmaceuticals, also known as AskBio.

For Bayer, the acquisition is part of a broader effort to build a gene and cell therapy division. But the deal is just as noteworthy for AskBio, an unusually large, privately held biotech based on the work of one of gene therapy's pioneers, Jude Samulski.

AskBio chose the security of a wealthy backer over independence and the chance to go public like several of its peers. And the deal helped the biotech quickly hire Katherine High, one of the few executives with experience shepherding a gene therapy through to regulatory approval. All of which makes the efforts of AskBio, now operating as an independent arm of Bayer, worth watching.

"I think we have the right people, the right chemistry, and the right amount of experience to make a difference here," Samulski said in an interview.

As 2019 drew to a close, so did a chapter in High's career. A hematologist by training, High, 69, has spent three decades working in gene therapy, a large portion of which was as a founder, president and chief scientific officer of Philadelphia biotech Spark Therapeutics.

At Spark, High had helped make history by steering the development of Luxturna, a treatment for a genetic form of blindness. When cleared by the Food and Drug Administration in late 2017, Luxturna became the first gene therapy for an inherited disease approved in the U.S. Roche swooped in soon after to acquire Spark, and closed the deal in December 2019.

Katherine High, president of therapeutics at AskBio

Permission granted by AskBio

High decided to take a year off from biopharma. But the coronavirus pandemic dashed her plans to conduct research at Rockefeller University. The institution reduced its staff to essential personnel, and the Harvard Club of New York City, where High, a Philadelphia resident, planned to stay during the week, closed its doors.

"My sabbatical turned into a virtual event, which was good; I got a lot of things done review articles written, book chapters written, things like that," High said in an interview. "I really needed a break."

She spent time with her first grandchild, swam, and, fulfilling a longtime desire, remotely studied German at Middlebury College's storied language program.

But High couldn't keep away from drug research. During periodic visits to North Carolina, where she has family, High dropped in on Samulski and fellow AskBio co-founder Sheila Mikhail.

High has over the years both collaborated and competed with Samulski, a University of North Carolina researcher and expert in gene therapy delivery tools known as adeno-associated viruses, or AAVs. He formed AskBio in 2001 with another gene therapy researcher, Xiao Xiao, and CEO Sheila Mikhail, a life sciences attorney.

"Our paths have crossed, our students have crossed, our sciences [have] definitely cross-pollinated," Samulski said, describing High's academic work at University of Pennsylvania and his at UNC.

By the time of their meeting, AskBio had grown to become one of the gene therapy field's most unique. Originally bootstrapped with angel investing and backing from the Muscular Dystrophy Association, AskBio had spun multiple gene therapy programs into companies that were later acquired. The returns from those buyouts were then used by AskBio to build its own manufacturing capabilities, a crucial step for gene therapy products.

During High's visits, Samulski and Mikail shared some of the progress the company had made advancing its technology. Among them: the acquisition of a Scottish biotech whose technology may allow the company to more tightly control how much protein a gene therapy can produce. Doing so could help overcome a critical limitations of gene therapy, which can have widely varying effects from patient to patient.

"We have a roadmap, how to get from A to B," Samulski told High. "If you want to come in and champion that, we would love to have you."

As AskBio was courting High, Bayer was eyeing AskBio, which had put in motion plans for an initial public offering a typical step for a biotech of its size.

Bayer had already announced plans to develop a cell and gene therapy division, acquiring Bluerock Therapeutics, a maker of "off-the-shelf" cell-based treatments, in 2019.

But the large pharma didn't have an anchor for its gene therapy ambitions. Marianne De Backer, Bayer's head of business strategy and development, had assembled a list of developers to pursue. AskBio was at the top.

"If you look at the [gene therapy] assets that are on the market today, like Zolgensma from Novartis, part of the technology is based on technology from AskBio," she said in an interview, referring to the Swiss company's spinal muscular atrophy treatment. A Duchenne muscular dystrophy treatment in late-stage testing at Pfizer also originated within AskBio, as did a Takeda program being studied in hemophilia.

De Backer faced two obstacles, though. Bayer, for one, didn't know the AskBio team, and couldn't meet them in person because of the travel restrictions that began during the pandemic.

"It was really almost a cold call," she said.

Bayer was also competing against the draw of a deep market for public stock offerings, which helped a record number of biotechs to IPO in 2020. De Backer said she needed to show AskBio that she could get the deal done quickly. So she and Mikhail spent six weeks hammering out terms, including an agreement the company could continue to operate independently an "arm's length" arrangement like one Bayer made with BlueRock.

Such promises are often made, and eventually broken, when a larger company acquires a smaller one. But Samulski's concerns that AskBio's work might be stifled within such a massive company were eased after speaking with BlueRock CEO Emile Nuwaysir.

Jude Samulski, co-founder and chief scientific officer at AskBio

Permission granted by AskBio

"[Nuwaysir] said, they have left me alone, they've encouraged me to do what I'm doing,' and I said, OK, that's what I needed to hear,'" Samulski said.

The acquisition allows the company to spend more time on science and less on raising money, he added.

"If I go back and write a grant today, it'll be three years before we can start the project," Samulski said. "In this setting, when we have our meeting ... the decision-makers are at the table and the science starts that afternoon."

For High, AskBio represents a return to a similar role as the one she had left: helping run an advanced gene therapy business newly acquired by big pharma. At AskBio, she's been named president of therapeutics.

The role, however, lines up with High's current career ambitions. AskBio has the manufacturing capabilities, breadth of clinical-stage programs and financial backing to take on diseases like Parkinson's and congestive heart failure the types of complex, common conditions gene therapy hasn't yet been proven in.

"There are great strengths in pharma, and there are great strengths in biotech, and the ideal situation is one that will let you employ the strengths of both types of organizations," she said.

High considered other options, such as working with a different and unproven drugmaking technology. But as someone who's spent much of her life living the story of gene therapy, she knows more than most the challenges of pioneering a new technology and convincing regulators of its worth.

Sometimes people "may underestimate the amount of time it takes to build all the tools that you need to enable regulators to say 'yes, this is safe,'" she said.

By sticking with gene therapy, much of the groundwork has been laid. She's just looking to take it a step further.

"I'm probably not going to work for another three decades," High said, with a laugh.

Read this article:
How 2 scientific pioneers teamed up to run AskBio, Bayer's new gene therapy division - BioPharma Dive

However, the partners were not willing to disclose, as of today, which diseases exactly are being targeted under this alliance.

The collaborative project combines Neurogenes manufacturing and drug development capabilities with the University of Edinburghs novel platform and neurodevelopmental disease expertise.

Under the terms of the collaboration, the US company will provide financial support for Dr Stuart Cobbs laboratory at the University of Edinburgh, in exchange for the right to license any applicable intellectual property at agreed-upon economic terms.Neurogene will be responsible for late stage preclinical and all clinical development of any products generated under the collaboration.

Dr Cobbs lab uses a broad range of technologies to develop novel treatments for neurodevelopmental disorders based on a deep understanding of the molecular pathology.

In addition to Dr Cobbs position at the university, where he is a Simons fellow and reader in neuroscience, he is also Neurogenes chief scientific officer (CSO).

Neurogenes lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Its product pipeline of gene therapy candidates addresses distinct monogenic neurological diseases.

Neurogene is trying to find treatments for, among others, Batten disease - a group of rare, inherited diseases of the nervous system also called neuronal ceroid lipofuscinoses (NCLs).The company is focusing on CLN5 and CLN7, two rare, late infantile and rapidly progressive subtypes of Batten disease.Children with CLN5 or CLN7 typically develop signs and symptoms of the diseases at a young age, including seizures, progressive deterioration in intellectual and motor capabilities, and loss of vision.CLN5 is caused by a variant in the CLN5 gene, which leads to disruption of normal CLN5 protein function. The CLN7 subtype of Batten disease is caused by a variant in the CLN7 gene, also called the MFSD8 gene, which leads to disruption of normal CLN7 protein function.

Another disorder Neurogene is targeting is Charcot-Marie-Tooth disease (CMT) a group of inherited diseases that affect the peripheral nervous system (PNS). CMTs are the most common inherited motor and sensory nerve disorders - neuropathies.

It is also working to determine and address the root cause of diseases such as aspartylglucosaminuria (AGU) a rare, neurodegenerative lysosomal storage disorder (LSD).

In December 2020, Neurogene announced the completion of a US$115m Series B financing, which was led by EcoR1 Capital, with participation from existing investors Redmile Group, Samsara BioCapital, Cormorant Asset Management and an undisclosed leading healthcare investment fund.

New investors included funds and accounts managed by BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, and Alexandria Venture Investments.

The company said proceeds from the financing would be used to advance Neurogenes portfolio of multiple gene therapy programs into the clinic, as well as accelerate investment in novel gene therapy product designs and Neurogenes technology platform addressing key limitations in conventional gene therapy, while building out its AAV vector GMP manufacturing capabilities.

Oleg Nodelman, portfolio manager, EcoR1 Capital, said then: Neurogene is establishing itself as a leader in the gene therapy arena for neurological diseases. We are impressed by the companys innovation and accomplishments to date and are pleased to provide our support to Neurogene to advance medical research in this rapidly evolving area.

Visit link:
Neurogene in tie up with university to advance gene therapy technologies - BioPharma-Reporter.com

Jan. 22, 2021, FNF Research (fnfresearch.com) published the latest study on [2020-2026] Cell and Gene Therapy Consumables Market Report by Quantitative Research Incorporating Impact Of Economic And Non-economic Aspects was recently released. It uses exploratory techniques like qualitative and quantitative analysis to uncover and present data on the target market. Efficient sales strategies have been mentioned that would business and multiply customers in record time.

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Amgen Inc.

ATLANTA BIOLOGICALS

bluebird bio Inc.

Cook

Dendreon Pharmaceuticals LLC

Fibrocell Science Inc.

General Electric

Kolon TissueGene Inc.

Orchard Therapeutics plc.

Pfizer Inc.

PromoCell GmbH

RENOVA THERAPEUTICS

Sibiono GeneTech Co. Ltd.

Spark Therapeutics Inc.

Vericel

Helixmith Co. Ltd.

Vitrolife

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Cell and Gene Therapy Consumables Market 2020 Key Manufacturers, Development Trends and Competitive Analysis 2026 KSU | The Sentinel Newspaper - KSU...

Researchers have implicated the pro-inflammatory cytokine interleukin-1 (IL-1) in a wide variety of diseases such as osteoarthritis, rheumatoid arthritis (RA), diabetes, and obesity. Steven Abramson, MD, the Frederick H. King Professor of Internal Medicine, professor of pathology, and chair of the Department of Medicine at NYU Langone Health, has long studied how IL-1 can propagate and exacerbate the disease process. That research effort has more recently expanded to include investigations into how the anti-inflammatory IL-1 receptor antagonist, IL-1Ra, can counter IL-1 and modulate the inflammatory response. Based on intriguing findings about how certain gene variants may influence osteoarthritis risk and severity, a new National Institutes of Health (NIH) research grant will help Dr. Abramson and collaborators seek out IL-1related targets for inflammatory disease prevention and treatment.

To help clarify the inflammatory process, Dr. Abramson and collaborators including Mukundan G. Attur, PhD, associate professor of medicine, and Jonathan Samuels, MD, associate professor of medicine, examined several variants of the IL-1Raencoding IL1RN gene in the knee joints and cells of osteoarthritis and rheumatoid arthritis patients. In particular, a haplotype designated TTG predicted which at-risk patients would go on to develop knee osteoarthritis and was associated with more severe radiographic osteoarthritis as well as new onset RA. Its a marker of both severity and increased risk for incident osteoarthritis, Dr. Abramson says.

Their 2019 study in osteoarthritis patients, published in Annals of the Rheumatic Diseases, suggested that the IL1RN TTG haplotype produced less IL-1Ra protein. So one explanation for the finding is that these people with the gene are deficient in the endogenous inhibitor of IL-1, which is driving the disease, Dr. Abramson says. Conversely, a separate haplotype called CTA yields more IL-1Ra protein production and may be protective.

In collaboration with Jef D. Boeke, PhD, professor of biochemistry and molecular pharmacology and director of the Institute for Systems Genetics, a new NIH grant may help clarify how each gene haplotype modulates inflammation, influences the associated gene regulatory networks, and contributes to the mechanics of disease pathogenesis. In particular, the research will focus on a haplotype block, or a section of DNA including multiple genes adjacent to the IL1RN gene. The researchers hope to learn whether any of the neighboring genes have inflammatory properties of their own, a synergistic effect on IL1RN, or even a more dominant effect on the underlying inflammatory pathway. One reason to do that is if youre developing a drug, you might find that one of these other genes is a better target than IL1RN, Dr. Abramson says.

One key to the unique research effort is Dr. Boekes expertise in using CRISPR-Cas9 gene editing technology to construct a series of what his lab calls assemblons, or precisely altered haplotype blocks. Led by Dr. Attur, the collaborators will then transfect embryonic stem cells with the manipulated DNA and use in vitro assays to gauge the effects of the putative risk and protective IL1RN haplotypes. The genetic manipulation is very technical. But if we can succeed, it allows us to really define the role of these haplotypes, not just in osteoarthritis but in other IL-1driven diseases, Dr. Abramson says.

After differentiating the engineered embryonic stem cells into macrophage cells, the researchers will measure production of the IL-1Ra protein. Well also be stimulating the macrophages in an inflammatory way and looking at the profile of inflammatory mediators that they produce, Dr. Abramson says. Experiments may reveal whether stimulated macrophages that carry the protective IL1RN CTA haplotype, for example, produce more IL1-Ra protein and fewer pro-inflammatory mediators such as IL-1, cyclooxygenase-2 (COX-2), and tumor necrosis factor (TNF). In the same way, sequential knockouts of other genes in the assemblon may clarify their own contributions to each haplotypes effects.

If the researchers can zero in on the principal drivers of disease through their in vitro experiments, they plan to inject the engineered embryonic stem cells into mice models of osteoarthritis and RA. The in vivo studies of the gene regulatory network may help determine how specific gene variants influence disease outcomes.

The research could have broad implications for understanding IL-1associated inflammatory diseases and for personalizing antiIL-1 therapies. It might be that in personalized medicine, antiIL-1 treatments will be more effective in patients who have a deficiency of IL-1 receptor antagonist, Dr. Abramson says. A patient who produces abundant IL-1Ra, on the other hand, may not benefit from receiving more of it as a therapy. Alternatively, the research may suggest that the IL1RN haplotypes are exerting their influence mainly by modulating other genes with key roles in the disease pathogenesis. It may be that they will emerge as targets that people hadnt even thought about in those diseases, he says.

Link:
New Research Grant Seeks to Clarify the Role Genes Play in Modulating Inflammation - NYU Langone Health

INDIANAPOLIS, Jan. 22, 2021 /PRNewswire/ --Eli Lilly and Company (NYSE:LLY) today announced the successful completion of its acquisition of Prevail Therapeutics Inc. (NASDAQ: PRVL). The acquisition establishes a new modality for drug discovery and development at Lilly, extending Lilly's research efforts through the creation of a gene therapy program that will be anchored by Prevail's portfolio of clinical-stage and preclinical neuroscience assets.

"We are pleased to complete the acquisition of Prevail and establish a gene therapy program at Lilly that has the potential to deliver transformative treatments for patients with neurodegenerative diseases such as Parkinson's, Gaucher and dementia," said Mark Mintun, M.D., vice president of pain and neurodegeneration research at Lilly.

The impact of this transaction will be reflected in Lilly's 2021 financial results according to Generally Accepted Accounting Principles (GAAP). There will be no change to Lilly's 2021 financial guidance for research and development expense or non-GAAP earnings per share as a result of this transaction.

The Offer and the MergerThe tender offer for all of the outstanding shares of common stock of Prevail at a price of (i) $22.50 per share, net to the seller in cash, without interest and less any applicable tax withholding, plus (ii) one non-tradable contingent value right (a "CVR"), which CVR represents the contractual right to receive a contingent payment of up to $4.00 per share, net to the seller in cash, without interest and less any applicable tax withholding, which amount (or such lesser amount, as further described below) will become payable, if at all, if a specified milestone is achieved prior to December 1, 2028 (the "Offer"), expired as scheduled at one minute past 11:59 p.m., Eastern time, on January 21, 2021. Computershare Trust Company, N.A., the depositary and paying agent for the Offer, has advised Lilly that27,374,689 shares of Prevail common stock were validly tendered and not properly withdrawn in the Offer, representing approximately79.8 percent of the shares of Prevail common stock outstanding. All of the conditions to the Offer have been satisfied, and on January 22, 2021, Lilly and its wholly-owned subsidiary, Tyto Acquisition Corporation, accepted for payment, and will promptly pay for, all shares validly tendered and not properly withdrawn in the Offer.

Following completion of the Offer, Lilly completed the acquisition of Prevail through the merger of Tyto Acquisition Corporation with and into Prevail, without a vote of Prevail's stockholders pursuant to Section 251(h) of the General Corporation Law of the State of Delaware, with Prevail surviving the merger as a wholly-owned subsidiary of Lilly. In connection with the merger, each share of common stock of Prevail not validly tendered in the Offer (other than (1) shares owned by Prevail (or held in Prevail's treasury) immediately prior to the effective time of the merger, (2) shares owned by Lilly, Tyto Acquisition Corporation or any other wholly owned subsidiary of Lilly immediately prior to the effective time of the merger or (3) shares held by any stockholder that was entitled to and has properly demanded statutory appraisal of such shares pursuant to, and who complied in all respects with, Section 262 of the Delaware General Corporation Law (the "DGCL") and who, as of the effective time of the merger, had neither effectively withdrawn nor lost its rights to such appraisal and payment under the DGCL with respect to such shares) has been cancelled and converted into the right to receive the same (i) $22.50 per share in cash, without interest and less applicable tax withholding, plus (ii) one CVR, as will be paid for all shares that were validly tendered and not properly withdrawn in the Offer. Prevail's common stock will be delisted from the NASDAQ Stock Market.

Under the terms of the agreement, Prevail stockholders were awarded one non-tradable CVR worth up to $4.00 per share in cash payable (subject to certain terms and conditions) upon the first regulatory approval for commercial sale of a Prevail product in one of the following countries: United States, Japan, United Kingdom, Germany, France, Italy or Spain. To achieve the full value of the CVR, such regulatory approval must occur by December 31, 2024. If such regulatory approval occurs after December 31, 2024, the value of the CVR will be reduced by approximately 8.3 cents per month until December 1, 2028 (at which point the CVR will expire). There can be no assurance any payments will be made with respect to the CVR.

About Eli Lilly and CompanyLilly is a global healthcare leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at http://www.lilly.com. C-LLY

Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements about Lilly's acquisition of Prevail Therapeutics Inc. ("Prevail"), regarding contingent consideration amounts and terms, regarding Prevail's product candidates and ongoing preclinical development, regarding Lilly's development of a potential gene therapy program, and regarding Lilly's expected 2021 financial guidance and the impact of the acquisition on research and development expense and non-GAAP earnings per share. It reflects current beliefs and expectations; however, as with any such undertaking, there are substantial risks and uncertainties in integration of acquisitions, in drug research, development and commercialization, and in Lilly's evaluation of its estimated financial results for 2021 and the impact of the acquisition. Actual results could differ materially due to various factors, risks and uncertainties. Among other things, there can be no guarantee that Lilly will realize the expected benefits of the acquisition, that product candidates will be approved on anticipated timelines or at all, that Lilly will be successful in building a gene therapy program, that any products, if approved, will be commercially successful, that all or any of the contingent consideration will become payable on the terms described herein or at all, that Lilly's financial results will be consistent with its expected 2021 guidance or that Lilly can reliably predict the impact of the acquisition on its 2021 financial guidance and results. For further discussion of these and other risks and uncertainties, see Lilly's most recent Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission (the "SEC"). Except as required by law, Lilly does not undertake any duty to update forward-looking statements to reflect events after the date of this press release.

Refer to:

Mark Taylor; mark.taylor@lilly.com; (317) 276-5795 (Media)

Kevin Hern; hern_kevin_r@lilly.com; (317) 277-1838 (Investors)

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Lilly Completes Acquisition of Prevail Therapeutics - BioSpace

Databridgemarketresearch.com Present Cancer Gene Therapy Market Industry Trends and Forecast to 2027 new report to its research database. This report is always helpful to business or organization in every subject of trade for taking better decisions, solving the toughest business questions and minimizing the risk of failure. The studies of this report carefully analyzes the market status, growth rate, future trends, market drivers, opportunities, challenges, risks, entry barriers, sales channels, and distributors. The most advanced tools and techniques have been used to structure this Cancer Gene Therapy Market report such as SWOT analysis and Porters Five Forces Analysis. Moreover, different segments of the market taken into consideration in this market research report give better market insights with which reach to the success gets extended.

Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

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The major players covered in the cancer gene therapy market report are Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc, Merck & Co., Inc., CELGENE CORPORATION, Anchiano Therapeutics, Achieve Life Sciences, Inc among other domestic and global players.

Competitive Landscape and Cancer Gene Therapy Market Share Analysis

Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Cancer gene therapy market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for cancer gene therapy market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the cancer gene therapy market. The data is available for historic period 2010 to 2018.

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Cancer Gene Therapy Market : Future Prospects With Covid-19 Impact Analysis 2027 | Top Players- Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc -...

GlobalGene TherapyMarket Research Report and Forecast 2020-2025is the latest report byFior Marketswhich is the fastest growing market research company. The report provides a comprehensive scope of the market which includes future supply and demand scenarios, changing market trends, high growth opportunities, and in-depth analysis of the future market prospects. The report features real-time developments in the globalGene Therapymarket encompasses a highly structured and comprehensive outlook of the market. It shows market types and applications that are categorized as ideal market segments. The report covers the competitive data analysis of the emerging and prominent players of the market. Along with this, it provides comprehensive data analysis on the risk factors, challenges, and possible new market avenues.

The report has viewed the current top players and the forthcoming contenders. Business procedures of the vital participants and the new entering market ventures are concentrated in detail in this report. The report also encompasses SWOT investigation, income offer, and contact data. The report throws light on specific drivers, restraints, opportunities, challenges, and other determinants that tremendously favor and oppose normal growth in the globalGene Therapymarket. It also covers the product pricing factors, growth, emerging and dominant trends, overall market dynamics, and market size. The report includes a wide spectrum of the market to provide insightful data for the forecast period 2020-2025.

NOTE:Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post the COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

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The report gives the market segments that have been fragmented into sub-segments. The study gives a transparent view of the global market and includes a thorough competitive scenario and portfolio of the key players functioning in it. The report offers fundamental opinions regarding the market landscape, emerging and high-growth sections of the globalGene Therapymarket, high-growth regions, and market drivers, restraints, and also market chances. It targets estimating the current market size and growth potential of the global market across sections such as also applications and representatives.

Competitive Analysis:

Te report accurately profiles key vendors and players functioning in the globalGene Therapymarket, in terms of their ranking and core competencies, together with determining the competitive landscape. It also studies competitive developments such as partnerships and collaborations, mergers, and acquisitions (M&A), research and development (R&D) activities, product developments, and expansions in the global market.

The top key players profiled in this report are:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

Other Segment Analysis:

Segment classification of the market structure has been encouraged by our research experts to allow readers to comprehend the versatility of the market in terms of product and service variation. The market has been examined with vital market-specific developments across segment categories. Market segments such as type and application are also determined by quantitative and qualitative review. Type market size bifurcated into its product typeGermline Gene Therapy and Somatic Gene Therapyin terms of Volume (K Units) and Value (USD Million). Market segment by application, split into:Cardio Vascular Diseases, Infectious Diseases, Genetic Disorders, Neuro Disorders, Cancer, Others

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Country-Wise Assessment:

The report presents an understanding of the regional, country, and even local developments. Overview of globalGene Therapy market dynamics such as industry outlook, value chain developments, SWOT and PESTEL assessment as well as Porters Five Point analysis. The report also encompasses crucial analytical reviews on key elements, trends, current, and future perspectives. By regional analysis, the report covers:North America, Europe, Asia Pacific, South America, and the Middle East and Africa.

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Global Gene Therapy Market Worth $38.41 Million by 2025- Exclusive Report by Fior Markets - PharmiWeb.com

LabCorps contract research organization business Covance has promoted Maryland Franklin, Ph.D., to vice president and head of its cell and gene therapy unit.

She moves up from being Covances site lead and executive director of scientific development at the Ann Arbor, Michigan, facility, which focuses on preclinical oncology.

Now, she steps up to run its cell and gene therapy business, a major element in any CRO's portfolio these days as more and more biopharmas look to tap the therapies for potentially curative treatments for a range of diseases.

It remains a tricky proposition to pull off, but cell and gene therapy are very much the current course for R&D across the life sciences as well as a major part of Covances business. Under her new role, Franklin will oversee these offerings.

These solutions aim to help sponsors reduce risk, transition programs within and between phases of development faster and create a more patient-centric experience, Covance said in a statement, as Franklin will also be tapped to further extend Covance by Labcorps industry leading position.

RELATED: Covance to 'transform' into a decentralized CRO

We are thrilled to welcome Dr. Franklin to Covance by Labcorp. Her experience and expertise will bring perspective and insight to cell and gene therapy at Covance, said Bill Hanlon, Ph.D., president of clinical, therapeutic and regulatory sciences for Covance.

Dr. Franklin joins us at a critical juncture in our ability to support sponsors needs throughout the drug development process. She will guide our highly experienced scientists across functional disciplines to seamlessly develop and commercialize a cell or gene therapy. With Dr. Franklins expertise, we hope to further grow and advance our cell and gene therapy programs.

Cell and gene therapy approaches continue to show great promise in treating a variety of diseases that range from extremely debilitating rare diseases to applications in oncology, added Franklin. With several approved advanced therapies to date and many, many more in development, Im excited to join Covance by Labcorp to and help sponsors in their mission to improve the lives of patients.

.

Link:
Covance boosts Franklin to lead its cell and gene therapy unit - FierceBiotech

Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer. Due to the high success rate during the preclinical and clinical trial, cancer gene therapy is gaining popularity. There are many techniques used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.

The global Cancer Gene Therapy market is expected to expand at a CAGR of +32% over the forecast period 2019-2025.

The report, titled Global Cancer Gene Therapy market defines and briefs readers about its products, applications, and specifications. The research lists key companies operating in the global market and also highlights the key changing trends adopted by the companies to maintain their dominance. By using SWOT analysis and Porters five force analysis tools, the strengths, weaknesses, opportunities, and threats of key companies are all mentioned in the report. All leading players in this global market are profiled with details such as product types, business overview, sales, manufacturing base, competitors, applications, and specifications.

Top Key Vendors in Market:

Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology, Shenzhen SiBiono GeneTech, and Altor Bioscience.

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Cancer Gene Therapy market has been studied in terms of all parameters such as applications, types, products and many other. Each and every data leading to growth or fall of the respective segments have been explained. Entire supply chain with respect to market is studied in depth and is conveyed in the most comprehensive way possible. The reasons there is going to be an increasing trend to this market are studied and are elaborated. Driving forces, restraints and opportunities are given to help give a better picture of this market investment for the forecast period.

Different global regions such as North America, Latin America, Asia-Pacific, Europe, and India have been analyzed on the basis of the manufacturing base, productivity, and profit margin. This Cancer Gene Therapy market research report has been scrutinized on the basis of different practical oriented case studies from various industry experts and policymakers. It uses numerous graphical presentation techniques such as tables, charts, graphs, pictures and flowchart for easy and better understanding to the readers.

The reports conclusion leads into the overall scope of the global market with respect to feasibility of investments in various segments of the market, along with a descriptive passage that outlines the feasibility of new projects that might succeed in the global Cancer Gene Therapy market in the near future.

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Key questions answered in the report include:

Table of Content:

Global Cancer Gene Therapy Market Research Report 2019-2025

Chapter 1: Industry Overview

Chapter 2: Cancer Gene Therapy Market International and China Market Analysis

Chapter 3: Analysis of Revenue by Classifications

Chapter 4: Analysis of Revenue by Regions and Applications

Chapter 5: Analysis of Cancer Gene Therapy Market Revenue Market Status.

Chapter 6: Sales Price and Gross Margin Analysis

Continue for TOC..

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Explore why Cancer Gene Therapy Market is thriving by 2025 with top key players like Genelux Corporation, Cell Genesys, Advantagene, GenVec,...

New York, Jan. 22, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Transient Protein Expression Market Forecast to 2027 - COVID-19 Impact and Global Analysis By Product Type ; Application ; End User, and Geography." - https://www.reportlinker.com/p06010110/?utm_source=GNW However, high cost of products are likely to pose a negative impact on the market growth.Transient protein expression procedure has been widely in use for animal and plant cells for the last three decades.However, in the recent years, significant evolution in proteomics has resulted in the development of recombinant proteins.

The effective results of transient protein expression in animals and plants have increased research and product development for human applications.Various companies, including biopharmaceuticals and contract research and development organizations have channelized their efforts toward the development of products based on transient protein expression.The adoption of transient protein expression allow companies to get various genes to develop recombinant proteins without delaying cell line generation.

Thus, the quick process of cell line development with required gene expression, companies are widely attracted towards uniform proteins that have drug-like properties, which allows production of vaccines and viral vectors. In addition, the transient protein expression process is widely being used in the production of monoclonal antibodies, modified human proteins, growth factors and cytokines, hormones, and blood products.The COVID19 pandemic has resulted in rise in the use of transient protein expression in vaccine development.Various researchers have started studying the novel coronavirus extensively, with the use of transient protein expression.

For instance, in MarchApril 2020, Absolute Antibody (UK) increased the production of multigram quantities of multiple anti-SARS-CoV-2 spike proteins to develop neutralizing antibodies. Similarly, the transient protein expression was widely used to produce a positive control protein in the development of in-vitro diagnostics kits.Product Type InsightsThe transient protein expression market by product type is segmented into instruments, reagents, vectors, and competent cells.In 2019, instruments segment held a largest market share in the transient protein expression market, by product type.

This segment is also expected to dominate the market in 2027 as they are the reducing human input is that it enables continuous cell maintenance and protein production. Moreover, the similar segment is anticipated to also witness the fastest growth rate during the forecast period.

Application InsightsBased on application, the global transient protein expression market is segmented into genomic research, gene therapy, bio production, cancer research, and drug development.In 2019, the genomic research segment held the largest market share in the transient protein expression market.

This segment is also expected to dominate the market by 2027 as it increases DNA sequencing performance. Moreover, transient protein expression has helped in the study of all the genes of a person (the genome), including their interactions with each other as well as the environment.

End User InsightsIn terms of end user, the global transient protein expression market is segmented into pharmaceutical and biotechnology companies, academic and research institutes, and clinical research organizations.In 2019, the pharmaceutical and biotechnology companies segment held the largest market share.

This segment is also expected to dominate the market during the forecast period as pharmaceutical and biotechnology firms are increasing their spending on research and R&D activities. Moreover, transient protein expression has helped the recent improvements in existing technologies and it is moving toward industrial production of plant-based vaccines, antibodies, and biopharmaceuticals.Major primary and secondary sources for transient protein expression included in the report are National Research Council Canada, UK BioIndustry Association, Australian Cluster Observatory and McKell Institute, UAE Federal Customs Authority, and Alpen Capitals report, among others.Read the full report: https://www.reportlinker.com/p06010110/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global transient protein expression market is expected to reach US$ 983.10 million by 2027 from US$ 660.00 million in 2019 - GlobeNewswire

New York, Jan. 18, 2021 (GLOBE NEWSWIRE) -- Increased investment in advanced technologies for treatment of genetic and chronic diseases is driving growth of the regenerative medicine market.Market Size USD 7.34 Billion in 2019, Market Growth - CAGR of 15.6%, Market TrendsApplications in COVID-19 vaccine.

The global regenerative medicine market is forecast to reach a market size of USD 23.57 Billion by 2027, and register a robustly incline revenue growth, according to a new report by Reports and Data. Primary factors driving demand for regenerative medicines are advancements in surgical technology and monitoring devices, and major increase in prevalence of complex and degenerative diseases. Upsurge in incidence of cancers has been resulting in increasing research into stem cell therapy. Growth in research and development activities in emerging countries and rising focus on stem cell research is resulting in significant growth in the global revenue of regenerative medicine market.

Stem cell technology is growing rapidly and continues to play a crucial role in regenerative medicine and the related field. This technology opens up the possibility of treating Parkinsons Disease, arthritis, and spinal cord injury. Increase in demand for stem cell technology is a major factor driving growth of the regenerative medicine market.

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Recent developments in regenerative medicine for 3D bioprinting, stem cell treatment for heart repair, and vision loss has created demand for additional investments in the R&D of the technology to help with other diseases.

The COVID-19 impact:

Demand for regenerative medicine has witnessed increased demand during the COVID-19 pandemic. Regenerative medicine helps in understanding a mechanism of infection and to develop ways to prevent the spread of the virus. It is also being used to create advanced treatments to treat persons infected by the COVID-19 virus. Private companies are also using it to develop an effective vaccine for COVID-19.

Regenerative Medicine Market Size, Share & Industry Demand By Product (Tissue-Engineered Products, Cell Therapies, Gene Therapies, Progenitor & Stem Cell Therapies), By Application (Musculoskeletal Disorders, Oncology, Wound Care, By Material), and Region, Segment Forecast to 2027, To identify the key trends in the industry, click on the link below: https://www.reportsanddata.com/report-detail/regenerative-medicine-market

Further key findings from the report suggest

List of Key Companies Identified in the Regenerative Medicine Market Report:

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For the purpose of this report, Reports and Data has segmented into the global regenerative medicine market on the basis of product, application, material, and region:

Browse similar research reports:Cell Therapy Market By Therapy Type (Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy), By Therapeutic Area (Malignancies, Autoimmune Disorders, Musculoskeletal Disorders), By Cell Type, And By End User, Forecasts To 2027

Tissue Engineering Market Size, Growth & Analysis, By Material, By Application (Cancer, Urology, Neurology, Dental, Cell Banking & Cord Blood, Gynecology, Integumentary/Skin, Spine, Musculoskeletal, & Orthopedics, Vascular & Cardiology), And Region, Segment Forecasts To 2027

Gene Expression Market By Product And Services (Equipment, Consumables, And Services), By Capacity (Low- To Mid- Plex Gene Expression Analysis And High-Plex Gene Expression Analysis), By Application (Diagnostic, Drug Discovery, Research), And Segment Forecasts To 2027

About Reports and Data

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Regenerative Medicine Market Size Worth $23.57 Bn By 2027; High demand for 3D bioprinting of tissues and organs to better understand their mechanism...

Databridgemarketresearch.com Present Cancer Gene Therapy Market Industry Trends and Forecast to 2027 new report to its research database. This analysis offers an examination of a range of segments that are relied upon to witness the quickest development amid the estimate forecast frame. The company profiles of all the key players and brands that are dominating the Cancer Gene Therapy Market with moves like product launches, joint ventures, mergers and acquisitions which in turn is affecting the sales, import, export, revenue and CAGR values are mentioned in the report. A complete discussion about numerous market related topics in this research report is sure to aid the client in studying the market on competitive landscape. This report also gives you an idea about consumers demands, preferences, and their altering likings about particular product.

Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

Download Exclusive Sample Report (350 Pages PDF with All Related Graphs & Charts) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cancer-gene-therapy-market&pm

The major players covered in the cancer gene therapy market report are Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc, Merck & Co., Inc., CELGENE CORPORATION, Anchiano Therapeutics, Achieve Life Sciences, Inc among other domestic and global players.

Competitive Landscape and Cancer Gene Therapy Market Share Analysis

Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Cancer gene therapy market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for cancer gene therapy market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the cancer gene therapy market. The data is available for historic period 2010 to 2018.

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Cancer Gene Therapy Market Segmentation, Parameters, Prospects 2021 And Forecast Research Report To 2027 - The Courier

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received both rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease, said RA Session II, President, Founder and CEO of Taysha. We are encouraged by the early evidence of TSHA-105s disease-modifying approach and believe these designations will help us potentially accelerate the development of this exciting program. We look forward to working with the FDA to make TSHA-105 available to patients as expeditiously as possible.

SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. This rare form of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life.

We are pleased that the FDA recognizes TSHA-105s potential as an innovative therapeutic option for SLC13A5 deficiency, said Rachel Bailey, Ph.D., Assistant Professor in Pediatric Neurology at UT Southwestern. This disease is a debilitating form of genetic epilepsy in children that significantly impacts movement, motor control, cognition and quality of life, and there remains a need to alter the course of this disease early in life.

As a mother of two children with SLC13A5 deficiency, I have witnessed firsthand the devastating impact that numerous seizures and comorbidities accompanying the disease has on those affected by this disease, said Kim Nye, Founder of TESS Research Foundation. Tayshas commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years post approval.

About Taysha Gene Therapies

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-105, 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 benefits of rare pediatric disease designation and orphan drug designation to 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. 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5...

New York, Jan. 19, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Cancer Gene Therapy Market 2021-2025" - https://www.reportlinker.com/p05060878/?utm_source=GNW Our report on cancer gene therapy market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the side effects of traditional cancer treatments, benefits associated with gene therapy for cancer treatment and the rising prevalence rate of cancer boosting the demand for cancer therapeutics. In addition, the side effects of traditional cancer treatments is anticipated to boost the growth of the market as well. The cancer gene therapy market analysis includes application segments and geographical landscapes.

The cancer gene therapy market is segmented as below: By Application Oncolytic virotherapy Gene transfer Gene-induced immunotherapy

By Geographical Landscapes North America Europe Asia ROW

This study identifies the rising partnerships and collaborations as one of the prime reasons driving the cancer gene therapy market growth during the next few years. Also, favorable government regulations for gene therapy programs and rapid growth potential in developing economies will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our report on cancer gene therapy market covers the following areas: Cancer gene therapy market sizing Cancer gene therapy market forecast Cancer gene therapy market industry analysis

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About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Cancer Gene Therapy Market is expected to grow by $ 2.96 bn during 2021-2025 progressing at a CAGR of 20% during the forecast period -...

Gene Therapy Technologies Market Report recently published by Worldwide Market Reports company focuses mostly on required solutions to the users. The study includes analysis, forecast, and revenue from 2021 to 2026. The advancement rate is evaluated dependent on insightful examination that gives credible information on the worldwide market. Imperatives and advancement points are merged together after a significant comprehension of the improvement of this market.

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The top players covered in Gene Therapy Technologies Market are: Bluebird bio, Adaptimmune, GlaxoSmithKline, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology

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The international Gene Therapy Technologies market has been characterized by several primary factors, with each factor tends to play a crucial role in the boom of the market. The growth in the products has doubled with the smoother availability of the customer base that has been helping the company flourishing globally. On the other hand, the presence of a dynamic supply chain has helped the company to grow exponentially.

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Research Methodology:

The Gene Therapy Technologies market report has been prepared after thorough market research being conducted. It has been prepared as per Porters Five Force Model. In terms of timeline, the market takes the period between 2021-2026 into account for assessment. Apart from this, a comprehensive SWOT analysis has been provided for swift business decision making.

Enumerating some of the fundamental parameters encompassed in the report:

Global Gene Therapy Technologies Market Report includes Detailed TOC points:

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Gene Therapy Technologies Market Estimated to Experience a Hike in Growth by 2021 2026: Bluebird bio, Adaptimmune, GlaxoSmithKline - KSU | The...

Drugs that target the cancer-promoting proteins MDM2 and BET have been tried in acute myeloid leukemia (AML) and haven't been all that effective on their own. But what if they were combined?

Researchers at the Sanford Burnham Prebys Medical Discovery Institute and the University of Glasgow have early evidence a combination strategy may, in fact, work in AML.

Combining MDM2 and BET inhibitors improved the killing of AML cell lines in lab studies and was more effective than solo treatment in eradicating the cancer in mouse models, the researchers reported in the journal Nature Communications. The combination seems to work by activating the tumor-suppressing protein p53, they reported.

The results were surprising because previous research had shown that each drug on its own had modest benefit against AML, said senior author Peter Adams, Ph.D., a professor at Sanford Burnham Prebys, in a statement. The new research provides scientific rationale to advance clinical studies of the drug combination in patients with AML.

The gene TP53 produces the protein p53, a known tumor suppressor. TP53 is frequently mutated across a range of cancers, which is why targeting the gene is a popular pursuit in oncology research.

Until now, the popular thinking was that MDM2 inhibitors activate p53. BET inhibitors, on the other hand, suppress leukemia-associated genes but dont affect p53, researchers believed.

Adams and his team tested MDM2 and BET inhibitors in AML cell lines and samples from patients. They were surprised to discover that BET inhibitors actually do activate p53by suppressing another protein called BRD4. Combining MDM2 and BET inhibition produces a 'double whammy' effect that fully unleashes the anti-cancer activity of p53, Adams said.

RELATED: How novel combos could overcome resistance to targeted drugs in leukemia, solid tumors and more

The Sanford Burnham Prebys-led team went on to test the combination in two mouse models of AML. In both cases, inhibiting BET and MDM2 together outperformed either mechanism on its own in eradicating the cancer and extending survival, the researchers reported.

The biopharma industry continues to show an interest in both BET and MDM2 inhibitors, though development efforts have run into some obstacles.

In 2019, Roche dropped a phase 1 BET inhibitor from its pipeline. And last April, the Swiss pharma giant stopped testing MDM2 inhibitor idasanutlin in a phase 3 AML trial after a combination of the drug with cytarabine proved disappointing. Early trials of idasanutlin in combination with Roches AML drug Venclexta are underway.

Meanwhile, other early-stage BET and MDM2 inhibitors have driven some deal-making in biopharma. In 2018, Aptose Biosciences teamed up with Ohm Oncology to advance a BET inhibitor in hematologic cancers. And last September, Rain Therapeutics licensed an MDM2-targeted drug from Daiichi Sankyo and raised $63 million to take it into pivotal trials in differentiated or dedifferentiated liposarcoma.

The Sanford Burnham Prebys and University of Glasgow researchers noted in their study that the heterogeneity of AML makes it a particularly difficult disease to address with targeted treatments. While many different genes can be mutated to drive the disease, no single mutation is dominant in a majority of patients.

But 90% of AML tumors have TP53, suggesting that human AML subtypes employ alternative mechanisms to inactivate the p53 pathway.

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Unleashing the cancer-fighting gene TP53 in leukemia with a novel combination treatment - FierceBiotech

Rising Demand for Gene Therapy Market 2021

Gene Therapy Market analysis on global market is a thorough study that offers a select combination of skillful market realities. The study shows changing market trends as well as the size of individual segments in this market. This report mentions various top players involved in this market. Analysis of the Global Gene Therapy Market begins with a market-based outline and underlines the current information on the global market, complemented by data on the current situation.

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Top Key Players Profiled in This Report

Novartis AG, Gilead Sciences, Inc., UniQure N.V., Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline PLC, Celgene Corporation, Shire PLC,

The key questions answered in the report:

Across the globe, different regions such as North America, Latin America, Asia-Pacific, Europe, and Africa have been examined on the basis of productivity and manufacturing base. Researchers of this report throw light on different terminologies of the Global Gene Therapy Market.

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Big Boom in Gene Therapy Market Detailed Analysis of Current and Future Industry Figures till 2028 |Novartis AG, Gilead Sciences, Inc., UniQure N.V.,...

Reuters | Published: January 21, 2021 10:37:09 | Updated: January 21, 2021 14:27:09

Scientists in Beijing have developed a new gene therapy which can reverse some of the effects of ageing in mice and extend their lifespans, findings which may one day contribute to similar treatment for humans.

The method, detailed in a paper in the Science Translational Medicine journal earlier this month, involves inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing.

The specific therapy they used and the results were a world first, said co-supervisor of the project Professor Qu Jing, 40, a specialist in ageing and regenerative medicine from the Institute of Zoology at the Chinese Academy of Sciences (CAS).

These mice show after 6-8 months overall improved appearance and grip strength and most importantly they have extended lifespan for about 25%, Qu said.

The team of biologists from different CAS departments used the CRISPR/Cas9 method to screen thousands of genes for those which were particularly strong drivers of cellular senescence, the term used to describe cellular ageing.

They identified 100 genes out of around 10,000, and kat7 was the most efficient at contributing to senescence in cells, Qu said.

Kat7 is one of tens of thousands of genes found in the cells of mammals. The researchers inactivated it in the livers of the mice using a method called a lentiviral vector.

n liver cell and the mouse liver cell and for all of these cells we didnt see any detectable cellular toxicity. And for the mice, we also didnt see any side effect yet.

Despite this, the method is a long way from being ready for human trials, Qu said.

Its still definitely necessary to test the function of kat7 in other cell types of humans and other organs of mice and in the other pre-clinical animals before we use the strategy for human ageing or other health conditions, she said.

Qu said she hopes to be able to test the method on primates next, but it would require a lot of funding and much more research first.

In the end, we hope that we can find a way to delay ageing even by a very minor percentage...in the future.

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Gene therapy developed to delay ageing - The Financial Express BD

Earlier this week, Biogen and ViGeneron signed a global collaboration and licensing agreement to develop gene therapies for inherited eye diseases. The companies will use Munich-based ViGenerons proprietary adeno-associated virus (AAV) technology platform to efficiently transduce target retinal cells via intravitreal injections.

Under the agreement, ViGeneron will develop in vitro therapeutic candidates for an undisclosed target. Biogen has the right to add an additional target within two years. The companies will jointly conduct a proof-of-concept study, but then Biogen will be responsible for all further development, human trials, and commercialization.

In exchange for use of its AAV gene therapy vector, ViGeneron will receive an undisclosed up-front payment as well as R&D funds from Biogen. ViGeneron is also entitled to development milestone payments and royalties on commercial sales of any products arising from the collaboration.

The joint research effort is part of Biogens overarching plan to diversify its drug pipeline by focusing on ophthalmology, which CEO Michel Vounatsos considers to be an emerging growth area for Biogen. In March 2019, Biogen entered the field of retinal gene therapy through the $800 million acquisition of Nightstar Therapeutics, and in July 2020, the company signed an agreement with Massachusetts Eye and Ear that focused on developing treatment for inherited retinal degeneration due to mutations in the PRPF31 gene.

Here are some reports relating to this deal for your reference: GlobeNewswire; S&P Global; EndPoints News

See the article here:
Biogen and ViGeneron to Collaborate on Ophthalmic Gene Therapy Development - JD Supra

Ronald Crystal began working in gene therapy in the 1980s, long before the first wave of approvals shook the industry. He took his ideas to Weill Cornell Medicine in 1993, where he helped build a large gene therapy program and spent more than a decade developing potential candidates.

Now, the gene therapy long hauler is launching his own company, Lexeo Therapeutics, with an $85 million Series A to drive three of the companys AAV-administered candidates to market, it said Thursday. Crystal will take the role of chief scientific adviser with Pfizer veteran Nolan Townsend joining as CEO.

These three clinical programs are really the focus of the company and the Series A financing, Townsend told Endpoints News.However, Lexeo has even more candidates waiting in the wings.

Townsend, who hails from Pfizers rare disease program, was introduced to Crystal via a mutual contact. He served in a variety of roles in his 12 years at the pharma giant, including as president of rare disease in North America and other developed markets, country manager in several nations, and director of business operations for Asia-established products. But what attracted him to Lexeo was the opportunity to go after both rare and common diseases.

I saw the potential of this research platform to address a number of rare diseases that do not have adequate therapies today, but also the potential of this platform to address non-rare diseases, he said.

To lead the fledgling team, Crystal and Townsend assembled a seasoned brain trust, including chairman Steven Altschuler, who previously served as chairman of gene therapy pioneer Spark Therapeutics, and PTC Therapeutics vet Jay Barth as executive VP and CMO.

The New York City-based biotech, whose name is a nod to its Lexington Avenue roots, already has two candidates in the clinic: LX1004, which has completed a Phase I/II study and is headed for a pivotal trial in 2022, in CLN2 Batten disease, an autosomal recessive lysosomal storage disease; and LX100, currently in Phase I for APOE4-associated Alzheimers disease. Lexeos third highlighted candidate, an IV-administered treatment for Friedreichs ataxia (FA) dubbed LX2006 is expected to enter Phase I this year. FA is a rare, degenerative multi-system disorder caused by a gene mutation that disrupts the normal production of the protein frataxin, critical to the function of mitochondria in a cell.

The upstart has up to 15 more undisclosed gene therapy programs at different stages of development, according to Townsend and Crystal. Plus, they intend to maintain an ongoing research collaboration with Weill Cornell to bolster their preclinical pipeline.

Lexeos AAV mediated gene therapy programs have the potential for broad applicability across a range of therapeutic indications, and in a single company pipeline present an opportunity for the natural evolution of gene therapy from rare genetic conditions to more common diseases, Crystal said in a statement.

The founder still serves as chairman of Weills Department of Genetic Medicine and will look to continue building Lexeos team in the near future.

We have less than 10 people in the company today. I think were expanding rapidly, so that that number will increase significantly over the next 12 months, Townsend said.

The Series A was led by Longitude Capital and Omega Funds, and joined by Lundbeckfonden Ventures, PBM Capital, Janus Henderson Investors, Invus, Woodline Partners, the Alzheimers Drug Discovery Foundation and Alexandria Venture Investments.

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With decades in gene therapy under his belt, Ronald Crystal launches new venture with up to 18 candidates in the pipe - Endpoints News

GeneOne Life Science Inc. said Friday it has signed a contract to supply plasmid DNA therapy worth $2 million to a U.S. biopharmaceutical company.

GeneOnes subsidiary VGXI, a leading plasmid DNA manufacturer located in Texas, will supply the drugs to the American market under the contract. The name of the U.S. firm is confidential due to contractual reasons, a GeneOne official told Korea Biomedical Review.

A plasmid is a small, extrachromosomal DNA molecule in a single cell, physically separated from chromosomal DNA, which replicates independently. It may be used for gene transferring as a potential treatment in gene therapy.

Due to the strikingly noticeable growth in gene therapy research and development worldwide, the demand for cGMP of Plasmid DNA, a raw material for producing gene therapy drugs, is increasing at a rapid pace, a GeneOne official said. These DNAs are used to manufacture adeno-specific virus gene therapy, CAR-T gene therapy, and genetic scissors therapy.

GeneOne is constructing new facilities at its production facility sites in Texas, such as quality assessment laboratories, refining and charging, packaging facilities, and working offices and convenient facilities to increase the quality of its products, according to the company.

We expect our partner, VGXI, to supply more of our high-quality plasmids in large quantity, as a leading manufacturer, GeneOne CEO Park Young-keun said.

Continued here:
GeneOne to supply plasmid DNA therapy worth $2 million to US - Korea Biomedical Review

The massive growth of gene therapy research and development over the last few years has boosted demand for viral vectors, the engineered virusesused to deliver therapeutic genes into patients bodies.

Tokyo-based Fujifilm Diosynth Biotechnologies is stepping up to meet that demand.

Fujifilm will invest 4 billion yen ($40 million) to build a new manufacturing facility for viral vectors in Watertown, Massachusetts, the company said Monday. It will be Fujifilms third viral-vector manufacturing site, joining similar facilities the company has opened in Texas and the U.K.

Virtual Clinical Trials Summit: The Premier Educational Event Focused on Decentralized Clinical Trials

In this virtual environment, we will look at current and future trends for ongoing virtual trials, diving into the many ways companies can improve patient engagement and trial behavior to enhance retention with a focus on emerging technology and harmonized data access across the clinical trial system.

We are strategically establishing this facility in the greater-Boston area where there is a high concentration of biopharmaceutical companies and academia innovating in the field of advanced therapies, Martin Meeson, CEO of Fujifilm Diosynth Biotechnologies, said in a statement.

RELATED: The top 10 manufacturers in the fight against COVID-19 Fujifilm Diosynth Biotechnologies

Fujifilm Diosynth Biotechnologies planted a flagin viral vectors back in 2014, when it established its Texas site in College Station. As the market started to grow, the company invested an additional 13 billion yen ($120 million) in the site. It announced in October that it would add viral-vector manufacturing capabilities to its U.K. site, expecting those services to be online this spring.

The growth of gene therapy R&D has boosted demand for advanced manufacturing capabilities to the point that investors from all facets of biopharma are stepping in to provide services. Last January, for example, Nationwide Childrens Hospitals Abigail Wexner Research Institute (AWRI) in Columbus, Ohio, revealed a planto builda commercial-scale gene therapy manufacturing site.

That news came just months after Harvard University said it would invest $50 million in a not-for-profit manufacturing and training facility focused on cell and viral vectors. Fujifilm Diosynth Biotechnologies has a seat on the board of that organization, called Advanced Biological Innovation and Manufacturing.

RELATED: $50M cell and viral vector manufacturing operation backed by Harvard

Fujifilm is far from the only contract manufacturer answering the demand for viral vectors, either. Novartis and Pfizer ramped up their investments in gene therapy manufacturing last year. Contract manufacturers such as Catalent and Thermo Fisher Scientific are also expanding operations aimed at supporting gene therapy R&D.

Fujifilms Watertown site will start process development this fall, the company said. It expects to start offering contract manufacturing for early-stage clinical trials of therapies that use viral vectors in the fall of 2023.

Read more here:
Fujifilm triples down on viral vector manufacturing with new $40M Boston site - FiercePharma

ROCKVILLE, Md., Jan. 5, 2021 /PRNewswire/ --REGENXBIO Inc. (Nasdaq: RGNX) today provided an update on the RGX-314 programs, including the announcement that the pivotal program for RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) is now active. In addition, REGENXBIO announced a new program, RGX-202, a novel, potentially best-in-class, one-time gene therapy for the treatment of Duchenne Muscular Dystrophy (DMD).

"2020 was a very productive year at REGENXBIO, and we are excited to move into 2021, which we expect to be another year of clinical execution. The initiation of our first pivotal program for RGX-314 for the treatment of wet AMD is a great step forward for the field as we look to broaden the applicability of gene therapy to larger patient populations. In addition, we are excited to announce RGX-202, a potential one-time gene therapy for the treatment of DMD. RGX-202 is the first gene therapy program in the REGENXBIO pipeline to be developed under the leadership of our Chief Scientific Officer, Olivier Danos. We look forward to filing an IND for this program later this year," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We continue to advance our pipeline of innovative therapies in the clinic as well as our manufacturing capabilities. I would also like to express my deep gratitude to our employees and clinical partners as well as patients and their families for their ongoing commitment and support despite the challenges posed by the global COVID-19 pandemic."

Pivotal Program for RGX-314 for the Treatment of wet AMD

REGENXBIO today announced that ATMOSPHERE, the first of two planned pivotal trials to evaluate RGX-314, is active and patient screening is ongoing. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.

REGENXBIO completed an End of Phase 2 meeting with the FDA to discuss the details of a pivotal program to support a Biologics License Application (BLA). Based on discussions with the FDA, REGENXBIO plans to conduct two randomized, well-controlled clinical trials to evaluate the efficacy and safety of RGX-314 in patients with wet AMD, enrolling approximately 700 patients total. In addition, REGENXBIO and the FDA aligned on a clear path to support manufacturing plans in the pivotal program. REGENXBIO expects to submit a BLA based on these trials in 2024.

"We are pleased to have reached alignment with the FDA on key elements of our pivotal program for the treatment of wet AMD. Our plan allows us to further accelerate the clinical development of RGX-314 towards the goal of a BLA filing in 2024 and we have already begun site activation and patient screening for our first planned pivotal trial," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "We have strengthened the key design elements for the planned trials based on the long-term data from our dose-escalation Phase I/IIa trial of RGX-314 and believe that we are well-positioned to execute on this pivotal program."

Suprachoroidal Delivery of RGX-314 for the Treatment of Wet AMD and Diabetic Retinopathy (DR)

New Program for the Treatment of Duchenne Muscular Dystrophy

REGENXBIO also announced today the development of a potential one-time gene therapy for the treatment of DMD, which is based on a novel microdystrophin construct.

"DMD is a severe, degenerative disease affecting thousands of children worldwide. It is caused by mutations of the gene which encodes dystrophin, a protein necessary for muscle cell strength and function, and innovation and development of potential new treatment options for patients with DMD has been a goal for the gene therapy field for many years," said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. "Since I joined REGENXBIO, we have been working to develop this gene therapy candidate using our proprietary AAV8 vector, with a focus on including the C-Terminal Domain of dystrophin, which may potentially bolster the key cell signaling pathways and muscle membrane integrity, leading to improved muscle strength and resistance. We look forward to completing the IND-enabling studies and bringing this program into the clinic."

The design of the new RGX-202 microdystrophin transgene is based on innovative vector engineering by REGENXBIO scientists and incorporates learnings from the laboratory of George Dickson, Emeritus Professor of Molecular Cell Biology at Royal Holloway, University of London, a pioneering figure in dystrophin research.

"The data from dystrophic laboratory trials suggest that a gene therapy delivering a microdystrophin gene incorporating an extended coding region from the C-Terminal Domain such as RGX-202 may provide substantial added muscle function for patients with DMD. A blend of the innovative science applied to microdystrophin gene design, and an AAV vector that is well-established, makes this new approach very promising," said Professor George Dickson from Royal Holloway. "I am pleased to see this important science developing from Royal Holloway's research is now being advanced under the leadership and gene therapy expertise of Olivier Danos and the team from REGENXBIO. I look forward to seeing this program enter the clinic."

Financial Guidance

REGENXBIO expects to report that as of December 31, 2020, it had between $515 million and $530 million in cash, cash equivalents and marketable securities, including the $200 million upfront payment from REGENXBIO's royalty monetization agreement with entities managed by Healthcare Royalty Management, LLC. REGENXBIO expects these resources to fund its operations, including the completion of its internal manufacturing capabilities and clinical advancement of its product candidates, until late 2022.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About ATMOSPHERE

ATMOSPHERE is a multi-center, randomized, active-controlled trial to evaluate the efficacy and safety of a single-administration of RGX-314 versus standard of care in patients with wet AMD. The trial is designed to enroll 300 patients at a 1:1:1 ratio across two RGX-314 dose arms (6.4x1010 genome copies (GC)/eye and 1.3x1011 GC/eye delivered subretinally) and an active control arm of monthly intravitreal injections of ranibizumab (0.5 mg/eye). The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks. Secondary endpoints of the trial include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections. Patient selection criteria will include patients with wet AMD who are responsive to anti-VEGF treatment and will be independent of preexisting neutralizing antibody status. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The trial will be conducted at approximately 60 clinical sites based in the United States, with over 100 retinal surgeons.

About AAVIATE

AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation trial that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector, a targeted, in-office route of administration. The trial is expected to enroll approximately 40 patients with severe wet AMD across two cohorts. Patients in each cohort will be randomized to receive RGX-314 versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio, and two dose levels of RGX-314 will be evaluated: 2.5x1011GC/eye and 5x1011GC/eye. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the trial is mean change in vision in patients dosed with RGX-314, as measured by best corrected visual acuity (BCVA), at Week 40 from baseline, compared to patients receiving monthly injections of ranibizumab. Other endpoints include mean change in central retinal thickness (CRT) and number of anti-VEGF intravitreal injections received following administration of RGX-314.

About ALTITUDE

ALTITUDE is a multi-center, open label, randomized, controlled dose-escalation trial that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314. The trial is expected to enroll approximately 40 patients with DR across two cohorts. Patients will be randomized to receive RGX-314 versus observational control at a 3:1 ratio, and two dose levels of RGX-314 will be evaluated: 2.5x1011GC/eye and 5.0x1011GC/eye. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the trial is the proportion of patients that improve in DR severity based on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (ETDRS-DRSS) at 48 weeks. Other endpoints include safety and development of DR-related ocular complications.

About Duchenne Muscular Dystrophy

DMD is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. DMD is caused by mutations in the DMD gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death.

About RGX-202

RGX-202 is designed to deliver a novel microdystrophin transgene which retains key elements of the dystrophin protein, including an extended coding region of the C-Terminal (CT) domain found in naturally-occurring dystrophin, as well as other fundamental improvements to the transgene. Presence of the CT domain has been shown to recruit several key proteins to the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Additional design features, including codon optimization and reduction of CpG content, may potentially improve gene expression, increase translational efficiency and reduce immunogenicity. RGX-202 is designed to use the NAV AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle specific promoter (Spc5-12) to support the delivery and targeted expression of genes throughout skeletal and heart muscle.

Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to accurately predict how long REGENXBIO's existing cash resources will be sufficient to fund its anticipated operating expenses, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at http://www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements,whether as a result of new information, future events or otherwise.

SCS Microinjector is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Preliminary Financial Information

REGENXBIO reports its financial results in accordance with U.S. generally accepted accounting principles. All financial data in this press release for the year ended December 31, 2020 is preliminary, as financial close procedures for the year ended December 31, 2020 are not yet complete. These estimates are not a comprehensive statement of the financial position of REGENXBIO for the year ended December 31, 2020. Actual results may differ materially from these estimates as a result of the completion of normal year-end accounting procedures and adjustments, including the execution of REGENXBIO's internal control over financial reporting, the completion of the preparation and management's review of REGENXBIO's financial statements for the year ended December 31, 2020 and the subsequent occurrence or identification of events prior to the filing of the financial results for the year ended December 31, 2020 on Form 10-K with the SEC.

Contacts:

Tricia TruehartInvestor Relations and Corporate Communications347-926-7709[emailprotected]

Investors:Eleanor Barisser, 212-600-1902[emailprotected]

Media:David Rosen, 212-600-1902[emailprotected]

1Koo, Taeyoung et al. "Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of 1-syntrophin and -dystrobrevin in skeletal muscles of mdx mice." Human gene therapy vol. 22,11 (2011): 1379-88. doi:10.1089/hum.2011.020

SOURCE REGENXBIO Inc.

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REGENXBIO Announces Update on RGX-314 and Pivotal Program for the Treatment of Wet AMD and New Gene Therapy Program for the Treatment of Duchenne...