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Archive for the ‘Gene therapy’ Category

Avacta JV raises $7.3m for cell and gene therapy push | Business Weekly – Business Weekly

Wednesday, February 3rd, 2021

A Cambridge UK-Korea joint venture promising great things in nextgen cell and gene therapy technology has been rewarded with major cash backing in a Series A round.

Avactas JV with Daewoong Pharmaceutical AffyXell Therapeutics has secured $7.3 million to further develop its pipeline of next generation cell and gene therapies.

AffyXell was established in January 2020 to develop novel mesenchymal stem cell therapies. The business is combining Avactas Affimer platform with Daewoongs MSC platform such that the stem cells are genetically modified to produce and secrete therapeutic Affimer proteins in situ in the patient.

The Affimer proteins are designed to enhance the therapeutic effects of the MSC creating a novel, next generation cell therapy platform.

The Series A funding has been raised from a group of venture funds including Samsung Venture Investment Corporation, Shinhan Venture Investment, Smilegate Investment, Shinhan Investment Corporation, Kolon Investment, Stonebridge Ventures and Gyeongnam Venture Investment.

The proceeds will be used by AffyXell to further the development of MSCs engineered to produce Affimer molecules generated by Avacta that suppress immune response and restore immune balance.

While initially focusing on inflammatory and autoimmune diseases and prevention of organ transplant rejection, longer term goals could also include applications in regenerative medicine, infectious diseases and oncology.

Avacta's R & D costs associated with the generation of the Affimer proteins are funded by AffyXell whilst Avacta retains the rights to commercialise the Affimer proteins outside the field of cell therapies.

Avacta CEO Dr Alastair Smith said: The potential for AffyXells new class of MSC therapies to deliver improved treatments for a wide range of inflammatory and autoimmune diseases is significant, in a market estimated to be worth $16 billionn by 2025.

We expect these novel engineered MSCs to show a more powerful therapeutic effect than existing antibodies and stem cells and they therefore have the potential to lead the rapidly growing field of cell and gene therapy.

AffyXell is uniquely positioned to develop novel and powerful cell therapies through the combination of two world-class technologies: Avactas Affimer platform and Daewoongs proprietary technology for generating off-the-shelf allogeneic MSC therapies.

Completion of the Series A funding is a strong validation of this concept and moves us closer to providing these new therapies to the patients who need them.

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Taysha Gene Therapies Highlights Strategic Priorities and Provides 2021 Business Outlook – BioSpace

Wednesday, February 3rd, 2021

Feb. 1, 2021 12:00 UTC

Expects Phase 1/2 biomarker and preliminary clinical data for TSHA-101 in GM2 gangliosidosis in second half of 2021 and by year-end 2021, respectively

Plans to initiate a U.S. Phase 1/2 trial for TSHA-101 in GM2 gangliosidosis in second half of 2021 as well as Phase 1/2 trials for TSHA-118 in CLN1, TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome by year-end 2021

Anticipates advancement of four programs into IND/CTA-enabling studies: SLC13A5 haploinsufficiency, Adult Polyglycosan Body Deficiency (APBD), Lafora disease and GM2 AB variant

Expects IND/CTA submission from one of the following programs: SLC13A5 haploinsufficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency

Anticipates advancement of four new undisclosed programs into preclinical development focused on neurodevelopmental disorders, genetic epilepsies and neurodegenerative diseases

Intends to advance the development of next-generation technologies including miRARE platform, redosing strategy, mini-gene payloads and novel capsids, to optimize key components of the companys AAV-based gene therapies

Continues to make progress on internal 187,000 square-foot, 2,000-liter capacity, multi-product cGMP facility located in Durham, NC

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today highlighted its strategic priorities and provided a business outlook for 2021.

We enter 2021 having built a strong foundation on which to execute our corporate and pipeline objectives. Notably, we expanded our seasoned leadership team and esteemed board of directors steeped in gene therapy development and commercialization expertise, successfully raised funds in our initial public offering, transitioned from a preclinical- to a clinical-stage company, and achieved important progress on R&D initiatives and our three-pillar manufacturing strategy, said RA Session II, President, Founder and CEO of Taysha. 2021 will be a transformational year as we intend to rapidly advance multiple drug candidates to clinical proof-of-concept, further expand our platform-enabled pipeline and advance next-generation technologies. Specifically, we expect to report clinical data for our GM2 gangliosidosis program in the second half of this year and have multiple ongoing clinical studies by year end. We also anticipate several IND/CTA submissions across three CNS franchises and have multiple therapies in IND/CTA-enabling studies while advancing four new programs into preclinical development. In addition, we are excited to advance our next-generation platform technologies and further our efforts in redosing, transgene regulation and capsid development. We believe that our platform will drive future sustained innovation and value creation and look forward to highlighting the productivity of our platform in an R&D day later this year. Lastly, we continue to make progress on cGMP facility and process development capabilities with the completion of the design phase and initiation of procurement of long lead equipment.

Anticipated Milestones by Program

TSHA-101 for infantile GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes HEXA and HEXB intrathecally for the treatment of infantile GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease

TSHA-118 in CLN1: a self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments

TSHA-102 in Rett syndrome: a self-complementary AAV9 gene therapy in development for one of the most common genetic causes of severe intellectual disability, designed to deliver MECP2 as well as a novel miRARE platform that regulates transgene expression on a cell-by-cell basis

TSHA-104 in SURF1-associated Leigh syndrome: a self-complementary AAV9 viral vector with a codon optimized transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments

Pipeline programs advancing into IND/CTA-enabling studies

Discovery programs

Next-generation technology platform

Anticipated Corporate Milestones in 2021

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 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 and early-stage programs, 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, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. 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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Unlock the Long-Term Genomics Runway with ‘ARKG’ – ETF Trends

Wednesday, February 3rd, 2021

Ground zero for disruptive growth in the healthcare sector is genomics through the ARK Genomic Revolution Multi-Sector Fund (CBOE: ARKG).

ARKG holds equity securities of companies across multiple sectors, including health care, information technology, materials, energy, and consumer discretionary, that are relevant to the funds genomics theme. The active management team behind the ARKG strategy combines a top-down and bottom-up research methodology to identify innovative companies and convergence across markets.

The second generation of cell and gene therapy is one of multiple frontiers ARKG provides exposure to. Its also lacking in many old-school biotechnology ETFs.

New cell and gene therapy innovations could increase the total addressable market for oncology therapeutics by more than 20-fold, according to ARK Research.

The actively managed ARKG offers investors a thematic multi-capitalization exposure to innovative elements that cover advancements in gene therapy bio-informatics, bio-inspired computing, molecular medicine, and pharmaceutical innovations.

ARKG includes companies that merge healthcare with technology and capitalize on the revolution in genomic sequencing. These companies try to better understand how biological information is collected, processed, and applied by reducing guesswork and enhancing precision.

Interestingly, ARKG marries one disruptive technology with others.

The US Food and Drug Administration (FDA) approved Gleevec, an oral chemotherapy, after ten years of trials, seven years of which were in solid tumors. This timeline suggests that the FDA could approve the first CAR-T therapy for solid tumors in 2025, notes ARK. Because of artificial intelligence (AI), gene-editing, and next generation sequencing (NGS), failure rates and time-to-market should fall, accelerating approval rates.

The evolution of gene therapies from ex vivo to in vivo is another scenario worth monitoring in the coming years.

Unlike ex vivo, in vivo therapies cannot check edited cells before transduction. That said, in vivo gene therapy is more cost effective and easier to manufacture and scale. It also enables more access to the liver, eye, central nervous system (CNS), and muscles, concludes ARK.

For more on disruptive technologies, visit our Disruptive Technology Channel.

The opinions and forecasts expressed herein are solely those of Tom Lydon, and may not actually come to pass. Information on this site should not be used or construed as an offer to sell, a solicitation of an offer to buy, or a recommendation for any product.

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4BIO Capital’s review of AAV gene therapy clinical trials published in Nature Reviews Drug Discovery – GlobeNewswire

Monday, January 25th, 2021

Press Release

4BIO Capitals review of AAV gene therapy clinical trials published in Nature Reviews Drug Discovery

Systematic review and meta-analysis of 149 clinical trials of AAV-based gene therapies demonstrates high safety and efficacy rates

Data reinforces potential of AAV-based gene therapies to become a mainstream and potentially curative treatment modality

25 January 2021

LONDON & BOSTON 4BIO Capital (4BIO or the Group), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a systematic review paper entitled The Clinical Landscape for AAV Gene Therapies in Nature Reviews Drug Discovery.

The review, led by Dr. Dima Kuzmin in collaboration with academics from the University of Oxford and Childrens Hospital of Philadelphia as well as several members of 4BIOs management and advisory teams, analyses 149 unique adeno-associated virus (AAV) gene therapy clinical trials, determining current key trends and depicting the impact of clinical trials on the gene therapy field. This is the first systematic review and meta-analysis that establishes transition probabilities, cumulative safety, and efficacy data for AAV gene therapies.

Gene therapy using AAV as a vector has emerged as a novel therapeutic modality with significant clinical developments made over the past 20 years, including the treatment of over 3000 patients, showing its potential for substantial disease modification in many monogenic disorders, and perhaps even cures. The most commonly used serotype, AAV2, has produced vast amounts of safety and efficacy evidence, indicating its ability to be readily used within the gene therapy space. In addition, decreasing clinical trial duration, which reflects increased comfort from regulators, and the growing number of successful clinical trials focusing on treating diseases of the central nervous system (CNS), reiterates the potential of AAV gene therapy as a safe, well tolerated and efficacious therapeutic modality.

The authors show that AAV gene therapy is generally safe and well tolerated, with no clinical trials failing to reach their primary safety endpoints prior to the cut-off date of the review. Additionally, whilst sample sizes are still relatively small, the existing data indicates the success rates of gene therapy development are significantly higher than the average for all modalities. For example, the transition probability for a drug progressing from an investigative new drug (IND) to a new drug application (NDA) is 31% for ophthalmology gene therapies as compared to 24% across all modalities; 43% vs. 16% in metabolic diseases; 56% vs 47% in haematology; and 30% vs. 19% in neurology.

However, the authors conclude that drawbacks remain and further innovations, such as better manufacturing, an ongoing switch to engineered capsids and synthetic promoters could lead AAV gene therapies to advance from the primary targets of the retina, liver, muscle and brain into other major organs and therefore into the next stage of clinical significance.

Dr. Dima Kuzmin, Managing Partner at 4BIO Capital, said: The analysis of vast amounts of clinical trial data has allowed us to establish the safety, tolerability and efficacy of AAV gene therapy, and consider it as a potential curative modality. Whilst certain challenges, such as uncertainty with regards to durability of response to gene therapy and potential viral vector liver toxicity are still yet to be overcome, it is clear that AAV gene therapy has the potential to become an effective mainstream therapeutic option.

-Ends-

Contacts

About 4BIO Capital

4BIO Capital is an international venture capital firm focused solely on the advanced therapies sector.

4BIOs objective is to invest in, support, and grow early stage companies developing treatments in areas of high unmet medical need, with the ultimate goal of ensuring access to these potentially curative therapies for all patients. Specifically, it looks for viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome.

The 4BIO team comprises leading advanced therapy scientists and experienced life science investors who have collectively published over 250 scientific articles in prestigious academic journals including Nature, The Lancet, Cell, and the New England Journal of Medicine. 4BIO has both an unrivalled network within the advanced therapy sector and a unique understanding of the criteria that define a successful investment opportunity in this space.

For more information, please visit http://www.4biocapital.com

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Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency – FDA.gov

Monday, January 25th, 2021

Docket Number: FDA-2020-D-1137 Issued by:

Guidance Issuing Office

Center for Biologics Evaluation and Research

FDA plays a critical role in protecting the United States from threats such as emerging infectious diseases, including the Coronavirus Disease 2019 (COVID-19) pandemic. FDA is committed to providing timely guidance to support response efforts to this pandemic.

FDA is issuing this guidance to provide manufacturers of licensed and investigational cellular therapy and gene therapy (CGT) products with risk-based recommendations to minimize potential transmission of the novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). This guidance is intended to supplement the recommendations to drug and biological product manufacturers provided in FDAs Good Manufacturing Practice Considerations for Responding to COVID-19 Infection in Employees in Drug and Biological Products Manufacturing; Guidance for Industry issued in June 2020 (Ref. 1) (June 2020 GMP Guidance). The recommendations in this guidance specifically consider the source material (cells and/or tissues) recovered from donors and how the CGT product will be manufactured (e.g., cell expansion in culture, viral reduction steps, formulation).

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets ManagementFood and Drug Administration5630 Fishers Lane, Rm 1061Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2020-D-1137.

01/19/2021

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Nanoscope Therapeutics Receives Orphan Drug Designation for gene therapy of blindness – PRNewswire

Monday, January 25th, 2021

BEDFORD, Texas, Jan. 25, 2021 /PRNewswire/ --NANOSCOPE THERAPEUTICS Inc., a clinical-stage biotechnology company determined to change lives through the ocular gene therapy, today announced that it has received an Orphan drug designation from FDA for gene therapy-based treatment of Stargardt disease, a form of inherited retinal degenerative disease caused by gene mutation and passed on to children.

Central vision loss due to poor functioning RPE cells and loss of photoreceptors in the macula is the hallmark of Stargardt disease.According to Federation for Fighting Blindness, Stargardt disease is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S.The loss of vision is devastating to both children and adults, and significantly impacts their quality of life. There is an immense need to restore vision in these patients.

"Currently, there are no approved therapies for Stargardt Disease"said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope."We are excited by the potential of ambient light activatable Multi-Characteristic Opsin (MCO) based photosensitization of retinal neurons for treating Stargardt disease in a gene agnostic manner."

"vMCO-010 expands the portfolio of orphan drug designations obtained in multiple pipeline programs, expanding its therapeutic potential from Retinitis Pigmentosa (RP) with peripheral retinal degeneration to macular degeneration as in Stargardt disease", said Al Guillem, Chairman of Nanoscope Therapeutics Board.

Sai Chavala, Chief Medical Officer of Nanoscope, commented "We look forward to developing our innovative optogenetic platform technologies for visually-challenged patients through continued interaction with the FDA. The orphan designation will aid in accelerating our clinical development program."

The Orphan Drug Act encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset clinical development costs, as well as eligibility for seven years of post-approval market exclusivity.

"We are extremely excited and pleased to announce a second orphan drug designation for MCO based treatment for retinal diseases. Since the pathology of degenerated macula in Stargardt disease is similar to that of dry-AMD, we are very excited about the opportunity to learn from our Stargardt program and advance MCO-based dry-AMD program"said Samarendra Mohanty, President and Chief Scientific officer of Nanoscope that received an orphan designation in 2017 for MCO for treatment of RP.

AboutNANOSCOPE THERAPEUTICS Inc.

Nanoscope Therapeutics is advancing gene therapy using light-sensitive molecules for giving sight to the millions of blind individuals suffering from retinal degenerative disease, for which no cure exists. We utilize an ambient light-sensitive MCO molecule to re-sensitize the retina toward ambient light level. Our pipeline includes optogenetics based retinal regeneration therapy for vision restoration in patients with RP, Stargardt disease, and dry-AMD.

Contact:

Sulagna BhattacharyaInvestor Relations and Corporate Communications817-719-2692[emailprotected]

http://www.nanostherapeutics.com

SOURCE Nanoscope Therapeutics

http://www.nanostherapeutics.com

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How 2 scientific pioneers teamed up to run AskBio, Bayer’s new gene therapy division – BioPharma Dive

Monday, January 25th, 2021

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.

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2021 Payer Strategies Build on Telehealth, Gene Therapy Lessons – HealthPayerIntelligence.com

Monday, January 25th, 2021

January 22, 2021 -In 2021, payers will expand upon lessons learned about telehealth and gene therapy in 2020 as they implement their strategies in the new year.

Listen to the full podcast to hear more details. And dont forget to subscribe on iTunes, Spotify, or Google Podcasts.

Now that telehealth utilization has become more widespread, payers will work toward integrating virtual care and telehealth.

Over the course of the pandemic, payers had the opportunity to use telehealth and virtual care in a variety of contexts. As a result, the healthcare system has more data on what uses are best suited to telehealth.

For example, the data reinforced the fact that telehealth and virtual care are excellent modes of care for behavioral and mental health needs. One payer saw over 1 million telehealth claims between February and May 2020 and 50 percent of those telehealth visits were related to mental healthcare.

However, beyond behavioral and mental healthcare utilization, diagnostic work and some primary care services have also emerged as areas that can benefit from telehealth utilization. Virtual cares role in chronic disease management and specialty care is expanding, according to some payer experts.

Payers will also need to grapple with how to approach gene therapy coverage in 2021. Throughout the pandemic, the healthcare industry and the public at large have been clinging to the hope of a gene therapy that would cure the coronavirus.As a result, the pandemic underscored the demand and the impact of these types of treatments.

In 2021, payers will now want to return to creating innovative, new payment models for expensive gene therapy treatments. CVS Health and Aetna reported that care coordination is key to lowering costs around gene therapies.

Payers can also implement value-based care contracts in which pharmaceutical companies can be held accountable for drug therapies that do not meet certain quality metrics.

One of the biggest takeaways from 2020, however, is that the future is unpredictable and that at times historical data cannot prepare healthcare organizations for coming events.

As a result, experts are predicting that real-time data collection will become more essential to forecasting the future in healthcare.

A little less than 75 percent of health executives told PricewaterhouseCoopers that they planned to increase investments in predictive technology in 2021.

Working off of recent data can help organizations both on a macro scale and a micro scale as payers work toidentify widespread potential crises more quickly and to prevent health crises in individual members lives, PricewaterhouseCoopers pointed out.

Additionally, collaboration with other healthcare industry stakeholders can help inform payers efforts to observe trends and respond quickly.

Healthcare Strategies explored these and other trends in its recent special episode on 2021 expectations across the healthcare industry.

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Advarra Announces New Gene Therapy Ready Site Network – PRNewswire

Monday, January 25th, 2021

This will address an accelerating gene therapy market that is expected to grow globally by 16.6 percent from 2020-2027.

"The Gene Therapy Ready network demonstrates our commitment to empowering sites and supporting our industry partners as they pursue advanced genetic engineering to find cures for the world's most pressing health conditions," said Scott Uebele, President and Chief Research Services Officer at Advarra. "Our commitment to efficient study activation is unwavering, and this is another example of how Advarra bringslife sciences companies,CROs, research sites, investigators,andacademiatogether at the intersection of safety,compliance,technology, and collaboration."

All Gene Therapy Ready sites stand ready to help industry sponsors conduct clinical trials that advance cures, develop vaccines, and find treatments for rare disease. By placing clinical trials with a Gene Therapy Ready site, research sponsors can save significant time during study startup.

"This innovative network is truly the first of its kind. We constantly look for ways to support our sponsors in rapidly starting trials in a safe, compliant, and quality manner. With the Gene Therapy Ready network, we can improve study startup times by a month or more, potentially placing cures in the hands of patients faster," said James Riddle, Vice President of Research Services and Strategic Consulting at Advarra. "The Gene Therapy Ready site network charts a course to success by providing our sponsor clients with a clear choice for IBC review services."

About Advarra

Advarra advances the way clinical research is conducted: bringing life sciences companies, CROs, research sites, investigators, and academia together at the intersection of safety, technology, and collaboration. With trusted IRB and IBC review solutions, innovative technologies, experienced consultants, and deep-seated connections across the industry, Advarra provides integrated solutions that safeguard trial participants, empower clinical sites, ensure compliance, and optimize research performance. Advarra is advancing clinical trials to make them safer, smarter, and faster. For more information, visit advarra.com.

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

Monday, January 25th, 2021

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.

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Covance boosts Franklin to lead its cell and gene therapy unit – FierceBiotech

Monday, January 25th, 2021

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.

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.

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.

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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 -…

Monday, January 25th, 2021

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

Read the full report: https://www.reportlinker.com/p05060878/?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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Neurogene and University of Edinburgh Announce Research Collaboration to Advance Next Generation Gene Therapies – Business Wire

Monday, January 25th, 2021

NEW YORK--(BUSINESS WIRE)--Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, and University of Edinburgh, a world leader in biomedical and translational research for neurodevelopmental diseases, today announced a research collaboration to advance development of multiple platform approaches to enable next generation gene therapies.

The collaboration provides comprehensive research capabilities to Neurogene, enabling the company and the University to expedite a multiple platform approach to improve upon existing gene therapy technologies. Under the terms of the collaboration, Neurogene 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.

This partnership provides Neurogene with preeminent neurological research expertise and capabilities. Dr. Cobbs lab has contributed significant scientific expertise to improve the quality of our current rare disease pipeline and generated promising early data to allow us to tackle complex neurological diseases not addressable with conventional gene therapy, said Rachel McMinn, Ph.D., Founder and CEO of Neurogene. With this collaboration, I look forward to advancing our mission to provide safe and effective genetic therapies to patients and families as quickly as possible.

Neurogene is a science-driven company committed to investing in innovation and is the right partner for us to build upon the early successes in gene therapy technology, Stuart Cobb, Ph.D., Simons Fellow and Reader in Neuroscience at the Patrick Wild Centre and Centre for Discovery Brain Sciences, University of Edinburgh, stated. We are excited to collaborate with Neurogene on the critically-important endeavor of improving upon current gene therapy technologies. Gene therapy is a very promising yet complex development area, and we are privileged to help address the unmet needs that exist within rare neurological diseases.

In addition to Dr. Cobbs position at the University of Edinburgh, he serves as Chief Scientific Officer of Neurogene.

This Collaboration has been supported by Edinburgh Innovations, the University of Edinburghs commercialization service.

About Neurogene Inc.

Neurogene Inc. is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring therapies to patients that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options currently exist. Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address. For more information, visit http://www.neurogene.com.

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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…

Monday, January 25th, 2021

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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Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701, a New Gene Therapy for the Treatment of Wilson Disease |…

Monday, January 25th, 2021

DetailsCategory: DNA RNA and CellsPublished on Thursday, 21 January 2021 17:52Hits: 651

Clinical trial will utilize a single-protocol Phase 1/2/3 design

UX701 manufacturing complete at commercial quality and scale using HeLa PCL technology

First patient to be dosed in the first half of 2021

NOVATO, CA, USA I January 21, 2021 I Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX701, an investigational AAV9 gene therapy being evaluated for the treatment of Wilson Disease. Enrollment in a seamless single-protocol Phase 1/2/3 study is expected to begin in the first half of 2021. This will be the companys third in-house clinical gene therapy program and the second program in the clinic with the HeLa producer cell line manufacturing system.

FDA IND clearance allows for the advancement of this new gene therapy into the clinic and brings forward the hope for a new treatment for patients with Wilson Disease. UX701 has the potential to directly address the underlying basis of disease by restoring the normal transport and excretion of copper, said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. The seamless Phase 1/2/3 clinical trial design will allow us to efficiently evaluate safety and efficacy of UX701 before studying an optimal dose in a larger number of patients to support registration. We appreciate the agencys support for a novel clinical trial design that could bring this important potentially new treatment to the greatest number of patients as efficiently as possible.

Study Design

UX701 will be studied in a seamless, single-protocol Phase 1/2/3 clinical trial. Manufacture and testing of GMP-grade drug product to supply the clinical study are complete using the companys proprietary HeLa 2.0 producer cell line (PCL) process at the 2,000 liter scale.

Stage 1 (evaluation of initial safety and dose finding)

In the first stage of the study, the safety and efficacy of three dose levels of UX701 will be evaluated in 27 patients (nine per cohort), randomized 2-to-1 (gene therapy versus placebo). The dose cohorts will be enrolled sequentially using ascending doses. The patients will be followed for 52 weeks before transitioning to long-term follow-up and selecting a pivotal dose. The dose will be determined based on the safety profile, changes in biomarkers of copper metabolism (e.g. 24-hr urinary copper, ceruloplasmin concentration, ceruloplasmin activity, non-ceruloplasmin bound copper, and total serum copper), and the reduction in the use of the current standard (SOC), copper chelator and/or zinc.

Stage 2 (optimal dose evaluation for pivotal clinical data generation)

The second and pivotal stage will use the dose selected from Stage 1 and enroll an additional 63 patients, randomized 2-to-1 (gene therapy versus placebo). The co-primary endpoints in Stage 2 will evaluate the effect of UX701 on copper regulation based on 24-hour urinary copper concentration and percent reduction in SOC at Week 52. Key secondary endpoints in Stage 2 include the effect of UX701 on additional biomarkers of copper metabolism and patient- and clinician-reported outcomes from a modified Wilson Disease Functional Rating Scale.

Stage 3 (long-term follow-up)

All patients in Stage 1 and Stage 2 who are randomized to receive placebo may be eligible to receive UX701 at the Stage 2 dose. Patients receiving UX701 will be continued to be monitored for long term safety and durability of response.

About Wilson Disease

Wilson disease is a rare inherited disorder caused by mutations in the ATP7B gene, which results in deficient production of ATP7B, a protein that transports copper. Loss of function of this copper-binding protein results in the accumulation of copper in the liver and other tissues, most notably the central nervous system. Patients with Wilson disease experience hepatic, neurologic and/or psychiatric problems. Those with liver disease can experience such symptoms as fatigue, lack of appetite, abdominal pain and jaundice, and can progress to fibrosis, cirrhosis, life-threatening liver failure and death. Wilson disease can be managed by reducing copper absorption or removing excess copper from the body using life-long chelation therapy, but unmet needs exist because some treated patients experience clinical deterioration and debilitating side effects. Wilson disease affects more than 50,000 individuals in the developed world.

About UX701

UX701 is an investigational AAV type 9 gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion. It has been shown in preclinical studies to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease. UX701 was granted Orphan Drug Designation in the United States and European Union.

About Ultragenyx

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the companys website at http://www.ultragenyx.com.

SOURCE: Ultragenyx Pharmaceutical

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Chinese scientists develop gene therapy that could delay ageing and extend lifespan – Geo News

Monday, January 25th, 2021

BEIJING: Chinese Scientists have developed a new gene therapy that showed some reversal effects of ageing in mice and extend their lifespans.

However, scientists presume that the findings 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 an 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.

Read more: Latest on coronavirus: South Africa strain may resist antibody treatments

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.

We just tested the function of the gene in different kinds of cell types, in the human stem cell, the mesenchymal progenitor cells, in the human 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.

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Chinese scientists develop gene therapy that could delay ageing and extend lifespan - Geo News

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Chinese scientists develop new gene therapy that can delay the aging process – National Post

Monday, January 25th, 2021

Article content continued

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.

We just tested the function of the gene in different kinds of cell types, in the human stem cell, the mesenchymal progenitor cells, in the human 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 aging 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 aging even by a very minor percentagein the future.

(Reporting by Martin Quin Pollard; Editing by Kim Coghill)

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Chinese scientists develop new gene therapy that can delay the aging process - National Post

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Cell and Gene Therapy Consumables Market Analysis with Increasing Business Opportunities, Outstanding Investment Rate and Growth Opportunities Up To…

Monday, January 25th, 2021

Jan. 25, 2021, FNF Research (fnfresearch.com) offering a comprehensive analysis of the Updated Latest Report 2020 [2020-2026] Cell and Gene Therapy Consumables Market Report by Quantitative Research Incorporating Impact Of Economic And Non-economic Aspects where users can benefit from the complete market research report with all the required useful information about this market. Based on the Cell and Gene Therapy Consumables market development status, competitive landscape, and development model in different regions of the world, this report is dedicated to providing niche markets, potential risks, and comprehensive competitive strategy analysis in different fields. From the competitive advantages of different types of products and services, the development opportunities and consumption characteristics and structure analysis of the downstream application fields are all analyzed in detail.

The report also focuses on the major driving trends and challenges that affect the market and the vendor landscape. The report explains the competitive landscape and current trends in the Cell and Gene Therapy Consumables market. The report concludes with an analysis of the Cell and Gene Therapy Consumables vendor landscape and includes detailed profiles of the major players in the global Cell and Gene Therapy Consumables market.

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Key Questions Answered in this Report

This report highlights the current and future market potential for Cell and Gene Therapy Consumables and provides a detailed analysis of the competitive environment, regulatory scenario, drivers, restraints, opportunities, and trends in the market. The report also covers market projections through 2026, as well as key market players.

What benefits does FNF research study is going to provide?

Cell and Gene Therapy Consumables Market Study Coverage: It includes key market segments, key manufacturers covered, the scope of products offered in the years considered, global Cell and Gene Therapy Consumables Market, and study objectives. Additionally, it touches on the segmentation study provided in the report on the basis of the type of product and applications.

Key Highlights of the Table of Contents:

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Segment Analysis:

Analysis of the market segment includes the two major product and service categories as well as the end-user. Such a segmentation enables a granular view of the market that is needed to understand the finer nuances.

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.**

The report also includes a discussion on the major players in each regional market for Cell and Gene Therapy Consumables. It explains the main market drivers of the global Cell and Gene Therapy Consumables market, current trends within the industry; and the regional dynamics of the Cell and Gene Therapy Consumables market.

The report concludes with detailed profiles of major global vendors in the Cell and Gene Therapy Consumables industry.

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

Report Includes:

A comprehensive analysis of the global markets for the report within the industry and its sub-segments

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Competitive landscape featuring major market participants of the global Cell and Gene Therapy Consumables industry, and Porters Five Forces analysis considering both the micro and macro environmental factors prevailing in the market

Detailed insights into factors driving and restraining the growth of the global Cell and Gene Therapy Consumables market

Company profiles of the market-leading participants

Scope of the Cell and Gene Therapy Consumables Market Report:

Cell and Gene Therapy Consumables Market Historic Data (2020-2026):

Cell and Gene Therapy Consumables Market Forecast (2020-2026):

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Research Coverage:

The Cell and Gene Therapy Consumables market has been segmented based on offering, technology, end-use application, and end-user. It also provides a detailed view of the market across four main regions: North America, Europe, APAC, and RoW.

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Abeona Therapeutics Announces Successful Type B Meeting with FDA for Pivotal Phase 3 VIITAL Study of EB-101 in Recessive Dystrophic Epidermolysis…

Monday, January 25th, 2021

NEW YORK and CLEVELAND, Jan. 25, 2021 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the company held a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to align with the Agency on the companys proposal regarding co-primary endpoints for the pivotal Phase 3 VIITAL study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB). Following the meeting, Abeona is proceeding with its plan to enroll between 10 to 15 patients with RDEB, comprising approximately 35 large chronic wound sites treated in total.

We appreciate the clarity provided by the FDA and we are pleased to be aligned with the Agency on the co-primary endpoints for the Phase 3 VIITAL study, said Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona. Following the successful completion of the FDA meeting,we continue with all necessary steps to enroll our next patient in the VIITAL study and aim to complete enrollment in 2021.

The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6).

As previously announced, data from a Phase 1/2a clinical trial presented at the 2020 Society for Pediatric Dermatology Annual Meeting showed that wound healing of 50% or greater following EB-101 treatment in patients with RDEB was associated with no pain at treated sites at three-, four- and five-years post-treatment, compared with presence of pain in 53% of wound sites at baseline.

Jodie Gillon, Vice President and Chief Patient Officer of Abeona commented, We greatly appreciate the level of clarity we received from the FDA as we continue to work with our clinical study partners at Stanford University Medical Center and patient advocacy groups to enroll additional patients in the VIITAL study.

Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITAL study. Additional information about the trial, including eligibility criteria, is available at https://www.abeonatherapeutics.com/clinical-trials/rdeb and https://clinicaltrials.gov/ (Identifier: NCT04227106).

About Recessive Dystrophic Epidermolysis Bullosa Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About EB-101 EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITAL study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL study is a randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patients own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITAL study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking 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, and that involve risks and uncertainties. These statements include statements about the Companys aim to complete enrollment of patients in our VIITAL study in 2021. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of our announced strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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

Monday, January 25th, 2021

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.

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.

For More Insights Get FREE Detailed TOC @ https://www.databridgemarketresearch.com/toc/?dbmr=global-cancer-gene-therapy-market&pm

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.

About Data Bridge Market Research:

An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

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