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Archive for the ‘Gene Therapy Research’ Category

Taysha Announces Exclusive Option from UTSW to License Worldwide Rights to Clinical-Stage AAV9 Gene Therapy Program for CLN7 Disease, a Research…

CLN7 program currently in Phase 1 clinical proof-of-concept trial with preliminary data anticipated by year-end 2021

Intrathecal dosing of the high dose first-generation construct resulted in nearly complete normalization of impaired open field and motor function and more than doubled median life expectancy in MSFSD8 knockout mice; data to be presented at upcoming 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis

Taysha also enters into a research collaboration with UT Southwestern to develop next-generation construct for CLN7 disease, which is expected to improve potency, safety profile, packaging efficiency and manufacturability over first-generation construct

Initiation of a planned pivotal CLN7 clinical trial with next-generation construct anticipated in 2022, with reference to human proof-of-concept data generated from first-generation construct

Provides a grant to Batten Hope, the leading CLN7 patient advocacy group, to support patient awareness, disease education and newborn screening initiatives

Estimated prevalence of CLN7 disease is 4,000 patients worldwide

Taysha expected to have five clinical stage programs by year-end 2021

Webcast today at 8:00 AM Eastern Time

DALLAS, October 05, 2021--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has obtained an exclusive option from UT Southwestern (UTSW) to license worldwide rights to a clinical-stage AAV9 gene therapy replacement program for the treatment of CLN7 disease. The company has also entered into a research collaboration with UTSW to develop a next-generation construct for the treatment of CLN7 disease, which is expected to improve potency, safety profile, packaging efficiency and manufacturability over the first-generation construct. Completion of the next-generation construct design is anticipated by year-end 2021, with commercial-grade GMP material expected in 2022.

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The first-generation construct for the CLN7 program was developed in the laboratory of Steven Gray, Ph.D., Associate Professor at UT Southwestern Medical Center and Chief Scientific Advisor for Taysha, with financial support from Milas Miracle and Batten Hope, the leading CLN7 patient advocacy groups. The CLN7 program is currently in a Phase 1 clinical proof-of-concept trial run by UTSW, and Taysha expects the availability of preliminary human proof-of-concept clinical safety and efficacy data from the first-generation construct by year-end 2021. Taysha intends to initiate a planned pivotal trial using a next-generation construct in 2022, with reference to the human proof-of-concept clinical data being generated from the first-generation construct.

In addition, Taysha has provided a grant to Batten Hope to support patient awareness, disease education and newborn screening initiatives.

CLN7 disease is a rare, fatal and rapidly progressive neurodegenerative disease that is a form of Batten disease. CLN7 is caused by autosomal recessive mutations in the MFSD8 gene that results in lysosomal dysfunction. Disease onset occurs around two to five years of age, with death often ensuing in young adolescence. Patients experience gradual nerve cell loss in certain parts of the brain and typically present with seizures, vision loss, speech impairment and mental and motor regression. Currently, there are no approved therapies to treat CLN7 disease, which impacts an estimated 4,000 patients globally.

Preclinical data in rodents supported advancement of the first-generation construct into a Phase 1 clinical proof-of-concept study in patients with CLN7 disease. In an in vivo efficacy study, intrathecal (IT) administration of the first-generation construct to MFSD8 knockout mice with high or low doses resulted in clear age and dose effects with early intervention and high dose achieving the best therapeutic benefits. IT high dose of the first-generation construct in younger knockout mice resulted in: 1) widespread MFSD8 mRNA expression in all tissues assessed; 2) nearly complete normalization of impaired open field and rotarod performance at 6 and 9 months post injection; 3) more than doubled median life expectancy (16.82 months versus 7.77 months in untreated knockout mice); and 4) maintained healthy body weight for a prolonged period of time. Toxicology studies in wild type rodents demonstrated safety and tolerability of IT administration of the first-generation construct. These preclinical data will be presented by Xin Chen, Ph.D., Assistant Professor, Department of Pediatrics at UT Southwestern, in an oral presentation at the 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis on October 8, 2021.

"The CLN7 program is a strategic addition to our gene therapy pipeline focused on monogenic CNS diseases. Encouraging preclinical data generated in relevant rodent models suggest that the first-generation construct has the potential to reduce overall disease pathology, preserve motor function and ultimately prolong survival," said RA Session II, President, Founder and Chief Executive Officer of Taysha. "The first-generation construct is currently in a Phase 1 clinical proof-of-concept trial with two patients dosed to date, and we look forward to the availability of preliminary data by year-end. With human proof-of-concept clinical data to reference, we expect to advance a next-generation construct into a planned pivotal trial in 2022, which we anticipate should improve potency, safety, packaging efficiency and manufacturability over the first-generation construct. Importantly, we are also pleased to announce our grant to Batten Hope, the leading CLN7 disease nonprofit patient advocacy organization, to support patient awareness, disease education and newborn screening initiatives. We believe a gene therapy approach has the potential to address a significant unmet need in an estimated 4,000 patients globally."

Gina Hann, Batten Hope Founder, President and Treasurer, added, "Our mission is to support families with children suffering from terminal and rapidly progressive neurodegenerative diseases like CLN7. We are honored to receive Tayshas support to raise awareness, increase newborn screening and help patients gain access to potentially transformative treatments that offer hope and therapeutic advancement for conditions with significant unmet needs."

UTSW is currently enrolling patients in an investigator-sponsored Phase 1 open-label, dose escalation clinical proof-of-concept trial at Dallas Childrens Hospital for an intrathecally dosed AAV9-based gene replacement therapy for the treatment of infantile CLN7 disease. The primary endpoint of the trial is safety and tolerability by incidence and severity of treatment related serious adverse events. Secondary efficacy endpoints include the Clinical Global Impression, neuropsychological, ataxia and motor function assessments and quality of life. The design rationale and a discussion of outcome measures for this clinical trial will be presented as a poster at the upcoming 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis. To date, one patient has been dosed at 5x1014 total vg and a second patient was dosed at 1x1015 total vg as measured by the qPCR method. UTSW continues to enroll patients in this Phase 1 study at 1x1015 total vg and expects to dose additional patients in the near term. Preliminary safety and efficacy data are expected by the end of 2021.

With the addition of the CLN7 program, Taysha expects to have five clinical stage programs by year-end. As such, the TSHA-104 program for the treatment of SURF1-associated Leigh syndrome will transition to the companys collaborators at UTSW to complete IND-enabling studies, followed by a planned investigator-initiated clinical trial by the end of 2022. Taysha will continue to support the SURF1 natural history study in partnership with UTSW.

Financial terms of the agreements were not disclosed.

Webcast Information

Taysha management will host a webcast today at 8:00 am ET / 7:00 am CT to discuss todays news. To participate, please access the following link: http://lifesci.rampard.com/WebcastingAppv5/Events/eventsDispatcher.jsp?Y2lk=MTQ1MQ==. The live webcast and replay may also be accessed by visiting Tayshas website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 60 days.

About Taysha Gene Therapies

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, as well as the CLN7 program, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our expectations regarding the benefits of a next-generation construct for CLN7, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211005005463/en/

Contacts

Company Contact: Kimberly Lee, D.O.SVP, Corporate Communications and Investor RelationsTaysha Gene Therapiesklee@tayshagtx.com

Media Contact: Carolyn HawleyCanale Communicationscarolyn.hawley@canalecomm.com

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Taysha Announces Exclusive Option from UTSW to License Worldwide Rights to Clinical-Stage AAV9 Gene Therapy Program for CLN7 Disease, a Research...

Amicus, looking for a boost, sells its gene therapy work to a SPAC – BioPharma Dive

Dive Brief:

When Amicus acquired a privately held biotech called Celenex in 2018, it appeared to signal a new direction for a company that had already been around for almost two decades. Amicus spent all that time developing chemical drugs known as "pharmacological chaperones," and after a series of ups and downs, had finally brought the first of them, Galafold, to market.

Acquiring Celenex, however, made Amicus a gene therapy developer too. Celenex had several research programs that originated within Nationwide Children's Hospital, one of the nation's top gene therapy hubs. Amicus has since bought several others from the University of Pennsylvania, further establishing gene therapy research as a top priority and quietly giving the New Jersey biotech one of the larger portfolios in the industry.

But Amicus has paid a price, as it still isn't profitable despite having an approved product. The company spent nearly $500 million on expenses last year, largely to fund its pipeline. Its shares also continue to hover around $11 apiece, almost exactly where they were when the biotech began its gene therapy work three years ago.

The value of those assets "has largely gone unrecognized to date," Crowley said on a conference call with analysts. Executives believe housing those assets in a separate company "is the best way to unlock that value for shareholders," he said.

To do this, Amicus has sold its gene therapy business into a special purpose acquisition company, or SPAC, a popular tool of late for biotech investors to take their startups public or acquire stakes in emerging biotechs. The business has been renamed Caritas and will trade on the Nasdaq stock exchange when the deal closes either later this year or early next.

Crowley, who has led Amicus from the start, will join Caritas, which will start with two gene therapies for Batten's disease in clinical testing, several others behind it, more than 115 employees and $400 million in cash. But Amicus will also be able to benefit from Caritas' progress. According to Bradley Campbell, Amicus' chief operating officer and soon-to-be replacement CEO, the company will still hold a 36% stake in its spinout, partial rights to preclinical gene therapies for Fabry and Pompe, and "rights of first negotiation" to others for certain muscular dystrophies.

The companies also believe splitting in two will help each move faster, with Caritas investing solely in gene therapy development and manufacturing and Amicus working to broaden use of Galafold and secure approvals of AT-GAA. Amicus is also getting a $200 million investment from Perceptive and other investors in the deal.

The transaction makes sense given it could "enhance investor interest" in Amicus's gene therapy work, wrote SVB Leerink analyst Joseph Schwartz. Even so, the deal may raise questions about Amicus's long-term growth prospects beyond its two top drugs, wrote Stifel analyst Dae Gon Ha.

"We think investors may question the timing, and its potential significance in the grand scheme of AT-GAA and [Amicus] shares," he wrote.

Amicus shares briefly jumped 10% pre-market before those gains were erased.

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Amicus, looking for a boost, sells its gene therapy work to a SPAC - BioPharma Dive

Aruvant to Participate in 2021 Cell & Gene Meeting on the Mesa to be Held Online and in Person October 12 to 14, 2021 – Yahoo Finance

Company to sponsor SCDAA 49th Annual National Convention to be held October 12 to 16, 2021

NEW YORK and BASEL, Switzerland, Oct. 5, 2021 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, today announced that members of the Aruvant leadership team will participate in the annual Cell & Gene Meeting on the Mesa which is taking place in person in Carlsbad, CA and online from October 12 to October 14, 2021. In addition, Aruvant will be a corporate sponsor of the upcoming 49th Annual National Sickle Cell Disease Association of America (SCDAA) Convention to be held October 12 to October 16, 2021, online.

(PRNewsfoto/Aruvant Sciences)

At the Meeting on the Mesa conference, Dr. Palani Palaniappan, Aruvant's chief technology officer, will participate in the panel titled, "What's Next for Advanced Therapies", taking place live Tuesday, October 12 from 7:15 to 8:45 AM PST. In addition, Dr. Will Chou, chief executive officer, will give a company presentation and provide an update on the development of ARU-1801, an investigational lentiviral gene therapy for sickle cell disease (SCD), and ARU-2801, a one-time, adeno-associated virus gene therapy designed to deliver potentially curative efficacy to patients with hypophosphatasia without the limitations of chronic administration. The presentation will include ARU-2801 preclinical data and clinical data from the ongoing Phase 1/2 MOMENTUM study of ARU-1801 in patients with severe SCD.

In addition, Aruvant is a sponsor of the SCDAA annual meeting. SCDAA's Annual National Convention is a four-day conference designed to address the multifactorial aspects of SCD and sickle cell trait. This year's theme is "Unstoppable: Working Together for Sickle Cell". Click here to view the full program. The Aruvant Meeting on the Mesa presentation and panel discussion will be available for registered participants at http://www.meetingonthemesa.com on October 12, 2021.

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About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit http://www.momentumtrials.com and for more on the company, please visit http://www.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciences and on Instagram @Aruvant_Sciences.

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Aruvant to Participate in 2021 Cell & Gene Meeting on the Mesa to be Held Online and in Person October 12 to 14, 2021 - Yahoo Finance

New Culture Medium Supports Expansion of Natural Killer Cells for Cell and Gene Therapies – PRNewswire

CARLSBAD, Calif., Oct. 5, 2021 /PRNewswire/ -- Thermo Fisher Scientific today announced the launch of Gibco Cell Therapy Systems (CTS) NK-Xpander Medium, a GMP-manufactured cell culture medium that supports large-scale growth and culture of functional natural killer (NK) cells with or without the use of feeder cells. This is the first medium from Thermo Fisher specifically designed to support expansion of NK cells for cell therapy applications and is supported by raw material traceability and regulatory documentation.

"Cell therapy developers are increasingly turning to NK cells because they do not elicit the kinds of active immune responses that trigger conditions such as graft versus host disease," said Mark Powers, vice president, research and development at Thermo Fisher Scientific. "With NK-Xpander Medium, manufacturers can reach the necessary scale they need for NK cell therapies while minimizing regulatory burden and risk."

NK cells grown in NK-Xpander Medium exhibit a greater rate of expansion when compared to NK cells grown in other media and demonstrate cell killing in in vitro and in vivo models. In addition to their low immunogenicity, the ease of availability and sourcing of NK cells, coupled with efficient in vitro expansion, make them ideally suited for the development of allogeneic cell therapies. Unlike autologous therapies, which are produced using a patient's own cells, allogeneic cell therapies are derived from healthy donor tissue, and are more conducive to cost-effective scale-up of cell therapy manufacturing.

"NK cell therapies hold promising applications in treating solid tumors, which haven't been well served by therapies derived from other cell types," said Richard Eckert, professor and chairman at University of Maryland School of Medicine. "To capitalize on the promise of these therapies, our lab used human NK cells grown in Gibco CTS NK-Xpander Medium to study their impact on solid tumor-derived cancer cells. The NK cells cultured in NK-Xpander Medium displayed robust and potent cancer cell killing activity."

NK-Xpander Medium is part of Thermo Fisher's CTS product line, a comprehensive portfolio of GMP-manufactured products backed by regulatory documentation and designed to work synergistically, from cell isolation/activation to gene transfer and cell expansion, to address cell therapy developers' manufacturing workflow needs.

To learn more about Thermo Fisher's cell and gene therapy solutions, please visit http://www.thermofisher.com/CGT. To learn more about Gibco CTS NK-Xpander Medium, please visit http://www.thermofisher.com/nkcelltherapy.

* For Research Use or Manufacturing of Cell, Gene, or Tissue-Based Products. This product is not intended for direct administration into humans or animals.

About Thermo Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $35 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. Our global team of more than 90,000 colleagues delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services and Patheon. For more information, please visitwww.thermofisher.com.

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Media Contact Information:

Mauricio Minotta Phone: 760-805-5266 Email: [emailprotected]

Jessika Parry Phone: 419-266-4016Email: [emailprotected]

SOURCE Thermo Fisher Scientific

http://www.thermofisher.com

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New Culture Medium Supports Expansion of Natural Killer Cells for Cell and Gene Therapies - PRNewswire

Castle Creek Biosciences Expands its Innovative Gene Therapy Platform for Rare Genetic Connective Tissue Disorders through Research Collaboration with…

EXTON, Pa., Sept. 29, 2021 /PRNewswire/ --Castle Creek Biosciences, Inc., a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options, today announced a research collaboration with Mayo Clinic to advance discovery and pre-clinical development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta (OI) and classical Ehlers-Danlos syndrome (EDS), which are rare genetic connective tissue disorders that currently have no treatments approved by the U.S. Food & Drug Administration (FDA). The research will be led by principal investigator David R. Deyle, M.D., a board-certified medical geneticist with the department of medical genetics at Mayo Clinic and a leader in the field of connective tissue disorders.

"We are honored to be working with Dr. Deyle and his highly regarded research team at Mayo Clinic to identify and evaluate gene therapy candidates that hold promise for treating debilitating, rare connective tissue disorders with high unmet medical needs," said Matthew Gantz, president and chief executive officer of Castle Creek Biosciences. "We expect this initiative will be the first of multiple Castle Creek strategic collaborations with leading medical research institutions that have the potential to expand our innovative gene therapy discoveries for rare diseases and offer hope to underserved patient communities impacted by these devastating conditions."

Osteogenesis imperfecta, also known as brittle bone disease, is caused by genetic mutations that affect the synthesis of Type I collagen and can lead to fragile bones, scoliosis, short stature, dental disorders, and laxity of skin ligaments. OI is estimated to affect one in 6,600 people in the U.S. and may be diagnosed at any age. Classical EDS results from genetic mutations affecting synthesis of Type V collagen and is associated with skin hyperextensibility and fragility, hypotonia, joint instability, chronic pain, and fragile blood vessels. Vascular and pulmonary complications have also been reported. EDS is estimated to affect one in 20,000 people in the U.S.

"Castle Creek is leveraging its proven expertise and experience in rare diseases and late-stage clinical development of cell and gene therapies to establish strategic collaborations with world-class research organizations for studying early-stage novel treatments to address critical, unmet medical challenges of patients suffering from rare genetic conditions," said Jeff Aronin, founder and chairman of Castle Creek Biosciences, and founder, chairman and chief executive officer of Paragon Biosciences, LLC. "We commend Matthew and his leadership team for their strategic insight and guidance that have long-term potential to fuel discovery and advancement of innovative gene therapy candidates and enhance the depth of Castle Creek's pipeline."

For this research collaboration, Castle Creek will contribute its proficiency in rare diseases and gene therapy development and has licensed intellectual property related to OI and classical EDS from Mayo Clinic. Following completion of the discovery through pre-clinical development phases at Mayo Clinic, Castle Creek anticipates moving into clinical development of selected gene therapy candidates at its in-house, commercial-scale current good manufacturing practices (cGMP) manufacturing facility located in Exton, Pa.

About Castle Creek Biosciences, Inc.

Castle Creek Biosciences, Inc. is a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options. The company's proprietary autologous fibroblast platform potentially allows for the development ofpersonalized, targeted and redosable cell-based gene therapy product candidates formonogenic and chronic disorders. The company's most advanced product candidate, dabocemagene autoficel (D-Fi), is currently being evaluated in a Phase 3 clinical trial for the localized treatment of chronic wounds due to recessive dystrophic epidermolysis bullosa (RDEB). The company is also currently evaluating FCX-013 in a Phase 1/2 clinical trial for the treatment of moderate to severe localized scleroderma. In addition, Castle Creek Biosciences is pursuing discovery and potential development of early-stage novel product candidates with the goal of expanding its robust pipeline into other rare diseases and broader indications where there are significant unmet needs. The company operates an in-house, commercial-scale manufacturing facility in Exton, Pennsylvania that benefits from the validated systems and processes previously implemented at the site for manufacture of an FDA-approved cell therapy product.Castle Creek Biosciences, Inc. is a portfolio company of Paragon Biosciences, LLC. For more information, visit https://castlecreekbio.com/or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon Biosciences, LLC

Paragon is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, adaptive biology and advanced biotechnology. The company's current portfolio includesCastle Creek Biosciences,CiRC Biosciences,Emalex Biosciences,Evozyne,Harmony Biosciences,Qlarity Imaging,Skyline Biosciences, and a consistent flow of incubating companies created and supported by the Paragon Innovation Capital model. Paragon stands at the intersection of human need, life science, and company creation. For more information, please visithttps://paragonbiosci.com/.

Media Contacts

Jenna Urban Berry & Company Public Relations 212.253.8881 [emailprotected]

Karen CaseyCastle Creek Biosciences302.750.4675 [emailprotected]

SOURCE Castle Creek Biosciences, Inc.

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Castle Creek Biosciences Expands its Innovative Gene Therapy Platform for Rare Genetic Connective Tissue Disorders through Research Collaboration with...

Sarepta Therapeutics Opens Genetic Therapies Center of Excellence in Columbus, Ohio – Stockhouse

CAMBRIDGE, Mass., Oct. 04, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today celebrated the grand opening of the Genetic Therapies Center of Excellence (GTCOE), its new research facility in Columbus, Ohio.

The 85,000 square foot state-of-the-art facility expands Sarepta’s research and development capabilities and footprint, which includes sites in Cambridge, Andover and Burlington, Mass. With more than 70 employees today and plans to double the number of employees by the end of 2022, the Center is focused on discovery, pre-clinical and clinical development supporting Sarepta’s pipeline of genetic medicines which includes RNA, gene therapy and gene editing programs. The Center also supports process development and optimization work that enables the transition from clinical-scale to commercial-scale manufacturing, a critical task facing companies developing gene therapies.

Advances in the science of genetic medicine are creating incredible opportunities to develop medicines with the potential to transform the lives of people with rare diseases. Sarepta’s Genetic Therapies Center of Excellence complements and enhances our existing research and development expertise and will play a central and strategic role in our future as the leader in precision genetic medicine,” said Doug Ingram, president and chief executive officer, Sarepta.

Among the guests joining the Sarepta team today for a dedication, ribbon-cutting ceremony and facility tours: The Honorable Jon Husted, Ohio’s Lieutenant Governor; Pat Furlong, president and chief executive officer, Parent Project Muscular Dystrophy (PPMD); Jessica Evans, assistant director, The Speak Foundation; local officials; and luminaries from Columbus’ growing biotechnology sector. At the event, Sarepta also announced a $20,000 donation to the Ronald McDonald House Charities of Central Ohio, with Dee Anders, chief executive officer and executive director, Ronald McDonald House Charities of Central Ohio, present to accept.

Sarepta has operated in Columbus since 2018 and we’re proud to be at the forefront of Columbus’ emergence as a leading hub for biotechnology committed to the local community and the patients and families we serve,” said Louise Rodino-Klapac, Ph.D., Sarepta’s Columbus-based executive vice president and chief scientific officer. Our growing presence in Ohio will help us strengthen our close working relationships with long-standing local partners such as Nationwide Children’s Hospital, while we work with the greatest urgency to advance our pipeline, further the science of genetic medicine and create an environment where future generations of scientific talent will thrive.”

Sarepta Therapeutics’ decision to expand in Ohio is the latest example that Ohio is a great state to grow a business,” said Lt. Governor Jon Husted. When we created the Columbus Innovation District last year, we were focused on cultivating the right environment in central Ohio to attract new investments and jobs in gene and cell therapy. This new facility is a victory, as it builds on our strategy, creating jobs and producing some of the most advanced research and development of precision genetic medicine, further solidifying Ohio as a leader in gene therapy.”

About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit http://www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at http://www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking Statements This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding potential opportunities in the rare disease space; the potential transformative benefits of medicines in the rare disease space; our plans to double the number of employees in Columbus, Ohio by the end of 2022; and the potential for our growing presence in Ohio to help strengthen our close working relationships with long-standing local partners while we work with the greatest urgency to advance our pipeline, further the science of genetic medicine and create an environment where future generations of scientific talent will thrive.

These forward-looking statements involve risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Many of these risks and uncertainties are beyond our control. Known risk factors include, among others: we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, many of which are outside of our control, including possible limitations on company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; the impact of the COVID-19 pandemic; and those risks identified under the heading Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2020, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings we make, which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties we face, we encourage you to review our SEC filings. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. We undertake no obligation to update forward-looking statements based on events or circumstances after the date of this press release, except as required by law.

Source: Sarepta Therapeutics, Inc.

Investor Contact: Ian Estepan, 617-274-4052 iestepan@sarepta.com

Media Contact: Tracy Sorrentino, 617-301-8566 tsorrentino@sarepta.com

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Sarepta Therapeutics Opens Genetic Therapies Center of Excellence in Columbus, Ohio - Stockhouse

Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market to Witness Huge Growth by 2028 | BioReliance, Cobra Biologics, Oxford BioMedica…

Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market report focused on the comprehensive analysis of current and future prospects of the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing industry. It describes the optimal or favourable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period. An in-depth analysis of past trends, future trends, demographics, technological advancements, and regulatory requirements for the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market has been done in order to calculate the growth rates for each segment and sub-segments.

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Top Key Vendors of this Market are:

BioReliance, Cobra Biologics, Oxford BioMedica, UniQure, FinVector, MolMed, MassBiologics, Richter-Helm, FUJIFILM Diosynth Biotechnologies, Lonza, Aldevron, Eurogentec, Cell and Gene Therapy Catapult, Biovian, Thermo Fisher Scientific (Brammer Bio), VGXI, PlasmidFactory, bluebird bio, Novasep, Spark Therapeutics, Vigene Biosciences.

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Segmentation:

Product Type Segmentation:

CancersInherited DisordersViral InfectionsOthers

Industry Segmentation:

CancersInherited DisordersViral InfectionsOthers

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market. This report is a consolidation of primary and secondary research, which provides market size, share, dynamics, and forecast for various segments and sub-segments considering the macro and micro environmental factors. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market.

The influence of the latest government guidelines is also analysed in detail in the report. It studies the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing markets trajectory between forecast periods. The cost analysis of the Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market has been performed while keeping in view manufacturing expenses, labour cost, and raw materials and their market concentration rate, suppliers, and price trend.

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The report provides insights on the following pointers:

Market Penetration: Comprehensive information on the product portfolios of the top players in the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market.

Competitive Assessment: In-depth assessment of the market strategies, geographic and business segments of the leading players in the market.

Product Development/Innovation: Detailed insights on the upcoming technologies, R&D activities, and product launches in the market.

Market Development: Comprehensive information about emerging markets. This report analyzes the market for various segments across geographies.

Market Diversification: Exhaustive information about new products, untapped geographies, recent developments, and investments in the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing market.

Regions Covered in the Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Report 2021: The Middle East and Africa (GCC Countries and Egypt) North America (the United States, Mexico, and Canada) South America (Brazil etc.) Europe (Turkey, Germany, Russia UK, Italy, France, etc.) Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

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Table of Contents

Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Research Report 2021 2027

Chapter 1 Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market Forecast

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Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market to Witness Huge Growth by 2028 | BioReliance, Cobra Biologics, Oxford BioMedica...

Transforming Growth Factor Sales Gaining Traction & to reach US$ 709.9 Mn with Significant Development in Cell Therapy Research – PRNewswire

Cell therapy plays an important role in vascular and hematopoietic, neural, skeletal, pancreatic, periodontal, and mucosal tissue regeneration. Platelet-derived growth factors and bone morphogenetic factors are gaining high demand for diabetic neuropathic ulcers and periodontal defects, and tissue regeneration at sites of tibia fractures. Increasing demand for transforming growth factors in regenerative medicine is expected to propel market growth substantially over the coming years.

Stem cell research continues to expand due to high adoption of stem cell treatment. Therefore, bone morphogenetic proteins (BMPs) and TGF-beta proteins are gaining demand and supporting the clinical development of cellular therapies. Cell culture has helped the most in oncology research as cancer cells are more responsive to culture in vitro, which, in turn, is drive demand in research areas.

Increasing healthcare R&D expenditure further improves the chances of breakthrough treatment options. Rapidly growing healthcare expenditure is resulting in new molecule discovery, evaluation of various molecules for possible disease treatment, and adoption of innovative approaches in these studies.

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Key manufacturers of transforming growth factor are focusing on the development of technology to cater to specific research requirements.

The COVID-19 pandemic affected health services for other diseases such as hypertension, cancer, diabetes, and cardiovascular diseases. Selective procedures such as orthopedic joint replacement were impacted. Shifted focus of healthcare professionals from these diseases to address the COVID-19 crisis negatively impacted the overall healthcare industry.

However, the transforming growth factor space did not see any long-term adverse effect on its business. A short-term impact of COVID-19 has been seen on the market due to disruptions in the supply chain and research activities during lockdowns. The market is projected to experience smooth growth over the coming years.

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Key Takeaways from Market Stud

By grade, the GMP segment is fast-growing at CAGR of 9.3%, on the back of rise is demand for supreme quality growth factors across regions.

Based on product, bone morphogenetic proteins (BMPs) is leading with over 43% market share.

Oncology research is estimated to lead the market by application. This segment accounted for approximately 22% share of the market.

Pharmaceutical and biotechnology companies lead demand for transforming growth factors with a market share of 48%.

By region, North America is set dominate the global market with a value share of around 41%. Europe is slated to be the second-largest leading region with a value share of 32%.

"Increasing drug discovery and stem cell research is expected to drive market expansion of transforming growth factors over the next ten years," says an analyst of Persistence Market Research.

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Market Competition

Agreements, collaborations, and partnerships have emerged as the key growth strategy adopted by industry players. By focusing on these strategies, key stakeholders are expanding their geographic footprints and strengthening their existing product portfolios.

In February, 2019, Roche entered into a definitive merger agreement to acquire Spark Therapeutics.

In 2021, Bio-Techne Corporation) and Catamaran Bio expanded their collaboration for the development of cell engineering and cell process technologies for use by Catamaran in the manufacturing of CAR-NK cell therapy products.

What Does the Report Cover?

Persistence Market Research offers a unique perspective and actionable insights on the transforming growth factor market in its latest study, presenting historical demand assessment of 2016 2020 and projections for 2021 2031.

The research study is based on the product (activin, bone morphogenetic proteins (BMPs), TGF-beta proteins), grade (GMP grade, NON-GMP grade), application (oncology research, haematology research, wound healing research, dermatology research, cardiovascular disease & diabetes, cell therapy and ex vivo manufacturing, others), end user (pharmaceutical and biotechnology companies, research centres & academic institutes, CMOs and CDMOs), across seven key regions of the world.

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Persistence Market Research (PMR), as a 3rd-party research organization, does operate through an exclusive amalgamation of market research and data analytics for helping businesses ride high, irrespective of the turbulence faced on the account of financial/natural crunches.

Overview:

Persistence Market Research is always way ahead of its time. In other words, it tables market solutions by stepping into the companies'/clients' shoes much before they themselves have a sneak pick into the market. The pro-active approach followed by experts at Persistence Market Research helps companies/clients lay their hands on techno-commercial insights beforehand, so that the subsequent course of action could be simplified on their part.

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Transforming Growth Factor Sales Gaining Traction & to reach US$ 709.9 Mn with Significant Development in Cell Therapy Research - PRNewswire

Longeveron Successfully Advancing its Cell-Based Therapy Studies in a Growing Industry Segment – Yahoo Finance

Research and development of stem cell-based therapies, where a patients own cells, or those from a donor, are used to fight injury and disease, is one of the fastest growing areas in the biotech space. Longeveron Inc. (NASDAQ: LGVN), a clinical-stage biotechnology company in the thick of clinical development, continues to advance its investigational therapeutic, Lomecel-B, for chronic, aging-related and life-threatening conditions.

The company recently announced the results of its randomized, blinded and placebo-controlled Phase 2 trial to evaluate the safety and efficacy of its proprietary Lomecel-B infusion in frail, older patients between 70 and 85 years old. The trial, which was partially funded by the National Institute on Aging, evaluated a single intravenous infusion of 4 different doses of Lomecel-B cell therapy compared to placebo on the change in the distance a person could walk in 6 minutes (a test known as the 6-minute walk test). Results showed that a single infusion of Lomecel-B resulted in an increase in walk distance of approximately 50 meters (164 feet) at 6 and 9 months after infusion, while the placebo-treated subjects showed minimal improvement at 6 months and a deterioration by 9 months.

Lomecel-B is a proprietary allogeneic product comprised of medicinal signaling cells (MSCs) from the bone marrow of adult donors, which are culture-expanded in Longeverons current good manufacturing practice cell-processing facility. According to trial results so far, Lomecel-B, and MSCs in general, may be injected or infused into an unrelated recipient without triggering a harmful reaction (rejection) due to the biochemical properties of these specialized cells. This is in part what makes this class of biologic so intriguing for use as a regenerative therapeutic.

A growing global trend is for biotech companies to direct their services to the cell and gene therapy industry and moving to expand into a new branch of the pharmaceutical contract development and manufacturing organization world.

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The U.S. Food and Drug Administration (FDA) has approved a small number of cell and gene therapy drugs. Still, a new product pipeline is fighting for the agencys attention with approximately 1,200 experimental therapies more than half in Phase 2 clinical trials. The annual sales growth estimates for cell therapies are projected to reach 15%.

Longeveron has also initiated a Phase 2 trial evaluating the safety and efficacy of Lomecel-B injection into the heart of children born with hypoplastic left heart syndrome (HLHS), a rare and often fatal congenital heart defect.

Longeveron believes that using the same cells that promote tissue repair, organ maintenance and immune system function can develop safe and effective therapies for some of the most challenging diseases and conditions associated with aging.

We continue to make steady progress advancing our Lomecel-B clinical research programs forward, Longeveron CEO Geoff Green said. We have encouraging top-line results from our Aging Frailty program, and anticipate initiating a Phase 2 trial in Alzheimers disease later this year.

Longeveron shared their review of the Aging Frailty trial data with independent frailty experts, with the objective of planning the next steps for the program. The company presented clinical data at the 2021 International Conference on Frailty & Sarcopenia Research on Sept. 29 during a round-table presentation.

Learn more about Longeveron at http://www.longeveron.com.

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2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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Longeveron Successfully Advancing its Cell-Based Therapy Studies in a Growing Industry Segment - Yahoo Finance

Global Hemoglobinopathies Markets, 2021-2028 – High Unmet Needs / Increasing R&D Investment / Initiatives to Improve Disease Awareness -…

DUBLIN--(BUSINESS WIRE)--The "Global Hemoglobinopathies Market Size, Share & Trends Analysis Report by Type (Thalassemia, Sickle Cell Disease, Other Hemoglobin (Hb) Variants), by Diagnosis, by Therapy, by Region, and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global hemoglobinopathies market size is expected to reach USD 15.7 billion by 2028, expanding at a CAGR of 10.8%

Increasing awareness regarding hemoglobinopathies and government initiatives to diagnose the diseases at an early stage are expected to propel market growth over the forecast period. Moreover, increasing R&D investment, the presence of a promising drug pipeline, and technologically advanced diagnostics platforms are expected to boost the growth of the market.

The development of novel curative technologies, such as CRISPR/Cas9 and hematopoietic stem cell transplantation, coupled with a promising pipeline, is expected to propel market growth. Moreover, the presence of regulatory agencies, such as the FDA, which are working toward improving drug approval rate by granting accelerated approval for hemoglobinopathies drugs, is expected to drive the market.

Initiatives such as the Sickle Cell Awareness Initiative (SCAI) are working toward educating the people about the disease, which will increase the diagnosis and treatment rate. SCAI also raises funds for individuals affected with sickle cell diseases (SCDs) and provides research funding.

Moreover, governments of Middle Eastern and Asian countries provide funds for R&D of hemoglobinopathies treatment. For instance, various programs undertaken by governments, such as thalassemia screening in neonates and providing medicines, are impacting the market growth positively.

The high patient population in low-income countries has encouraged market players and non-profit organizations to launch several initiatives to improve the access to the therapy.

For instance, in February 2019, the Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL) was launched by Global Blood Therapeutics, Inc. in order to provide research funding for novel SCD projects, with an aim to expand the access to optimal healthcare for sickle cell disease.

Biopharmaceutical companies are collaborating with nonprofit organizations to promote public awareness about hemoglobinopathies.

An increase in investment and funding for the development of novel therapies to treat hemoglobinopathies will further boost the market growth over the forecast period. The National Heart, Lung, and Blood Institute (NHLBI) has significantly invested in research & development in sickle cell disease.

The NHLBI supports research work through various initiatives. Furthermore, private funding such as the Bronx Blood Research Fund (BBRF) provides a platform for research and management of thalassemia and other hemoglobinopathies.

Gene therapy has emerged as a promising treatment option for managing hemoglobin disorder as it targets the underlying genetic cause of the condition through the administration of one-time gene therapy and significantly reduces the need for blood transfusions.

The current pipeline of gene therapy products includes CTX001 (CRISPR Therapeutics), BIVV003 (Sangamo Therapeutics, Inc. & Bioverativ Inc), and HGB-206 (bluebird bio, Inc).

Hemoglobinopathies Market Report Highlights

Global Hemoglobinopathies Market Variables, Trends & Scope

Penetration and Growth Prospect Mapping

Epidemiology Assessment of Hemoglobinopathies

Market Variable Analysis

Market Restraint Analysis

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/8dphl0

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Global Hemoglobinopathies Markets, 2021-2028 - High Unmet Needs / Increasing R&D Investment / Initiatives to Improve Disease Awareness -...

BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa – WSAZ-TV

Published: Oct. 4, 2021 at 6:00 AM EDT

NEW YORK, Oct. 4, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced today that Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the2021 Cell & Gene Meeting on the Mesa, being held as a hybrid conferenceOctober 12-14, and October 19-20, 2021.

Dr. Lindborg's presentation highlights the expansion of Brainstorm's technology portfolio to include autologous and allogeneic product candidates, covering multiple neurological diseases. The most progressed clinical development program, which includes a completed phase 3 trial of NurOwn in ALS patients, remains the highest priority for Brainstorm. Brainstorm is committed to pursuing the best and most expeditious path forward to enable patients to access NurOwn.

Dr. Lindborg's presentation will be in the form of an on-demand webinar that will be available beginning October 12. Those who wish to listen to the presentation are required to registerhere. At the conclusion of the 2021 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website underEvents and Presentations.

About the 2021 Cell & Gene Meeting on the Mesa

The meeting will feature sessions and workshops covering a mix of commercialization topics related to the cell and gene therapy sector including the latest updates on market access and reimbursement schemes, international regulation harmonization, manufacturing and CMC challenges, investment opportunities for the sector, among others. There will be over 135 presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

The conference will be delivered in a hybrid format to allow for an in-person experience as well as a virtual participation option. The in-person conference will take place October 12-14 in Carlsbad, CA. Virtual registrants will have access to all content via livestream during program dates. Additionally, all content will be available on-demand within 24 hours of the live program time. Virtual partnering meetings will take place October 19-20 via Zoom.

About NurOwn

The NurOwntechnology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwntechnology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing and clinical development plans, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect,""likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwntreatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwntreatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwntreatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

ContactsInvestor Relations:Eric GoldsteinLifeSci Advisors, LLCPhone: +1 646.791.9729egoldstein@lifesciadvisors.com

Media:Paul TyahlaSmithSolvePhone: + 1.973.713.3768Paul.tyahla@smithsolve.com

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SOURCE Brainstorm Cell Therapeutics Inc

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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BrainStorm to Present at the 2021 Cell & Gene Meeting on the Mesa - WSAZ-TV

DTx Pharma to Present at the OIS Retina Innovation Showcase – Business Wire

SAN DIEGO--(BUSINESS WIRE)--DTx Pharma, Inc. (DTx), a privately-held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease, announced that Dr. Arthur Suckow, Co-Founder and CEO, will present at the Ophthalmology Innovation Summits Retina Innovation Showcase on October 7, 2021, during the Annual Scientific Meeting of the American Society of Retina Specialists.

Dr. Suckow will make a presentation during the Spotlight on Cell and Gene Therapy session and discuss DTx Pharmas most advanced program for retinitis pigmentosa (RP). RP is a rare disorder that affects roughly 1 in 4,000 people, both in the United States and worldwide. People with the disease tend to initially develop night blindness, followed by total blindness, as a result of the death of their photoreceptor cells. No therapeutic options exist for this condition that can be caused by more than 300 mutations of 100 different genes.

DTx Pharmas proprietary FALCON (Fatty Acid Ligand Conjugated Oligonucleotide) delivery platform is designed to improve the efficacy of RNA therapies by using fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. Using this novel technology platform, DTx Pharma can improve the cellular uptake of targeted RNA therapies and apply therapies to multiple cell types in the eye, with the goal of developing a gene agnostic therapy for RP.

There are no effective therapeutics for this debilitating disease. We look forward to presenting our preclinical data this week and to working with the experts in retinitis pigmentosa to evaluate a game-changing therapeutic in patients over the next several years, says Dr. Suckow.

About DTx Pharma

DTx Pharma, Inc. is a privately held biotechnology company based in San Diego, CA creating novel RNA-based therapeutics to treat the genetic drivers of disease. The companys proprietary delivery technology platform utilizes fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. In preclinical studies, DTx has demonstrated cellular uptake and broad activity of oligonucleotides in the retina, muscle, heart, neurons, T cells, and other specialized cell types. To advance the FALCON platform toward and into clinical development, DTx has raised more than $100M in combined investment from several of the worlds leading healthcare investors including RA Capital Management and Access Biotechnology, pharmaceutical companies such as Eli Lilly and Company, the National Institute of Health (NIH), and research foundations such as the CMT Research Foundation (CMTRF). To learn more about DTx Pharma, please visit http://www.dtxpharma.com and follow DTx on LinkedIn and Twitter @DTxPharma.

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DTx Pharma to Present at the OIS Retina Innovation Showcase - Business Wire

Glioma subtype may hold the secret to the success of immunotherapies – Michigan Medicine

Bench-to-bedside

Armed with this knowledge, further experiments showed that giving G-CSF, which is already used clinically as an immune system booster in cancer patients to mice with non-mutant IDH1 also increased their survival. And giving it in combination with the immune-stimulating gene therapy had an even bigger impact.

The team also confirmed that patients who have gliomas with mutated IDH1 also have higher levels of G-CSF circulating in their blood a clue that the findings will be applicable beyond the mouse models.

The next step, says Lowenstein, will be to work on moving these findings into a clinical trial, building on the current, ongoing trial using the immunotherapy/gene therapy combination.

Our study shows two main things: Patients with the IDH1 mutation may benefit from immunotherapy due to the G-CSF their tumors are producing, he said. And patients without the mutation may benefit from combining treatment with G-CSF and immunotherapy.

Additional authors include Brandon L. McClellan, Ruthvik P. Avvari, Rohit Thalla, Stephen Carney, Margaret S. Hartlage, Santiago Haase, Maria Ventosa, Ayman Taher, Neha Kamran, Li Zhang, Syed Mohammed Faisal, Felipe J. Nez, Mara Beln Garcia-Fabiani, Wajd N. Al-Holou, Daniel Orringer, Jason Heth, Parag G. Patil, Karen Eddy, Sofia D. Merajver, Peter J. Ulintz, Joshua Welch, Chao Gao, Jialin Liu and Gabriel Nez all of U-M; Shawn Hervey-Jumper of University of California, San Francisco; and Dolores Hambardzumyan of the Tisch Cancer Institute, Mount Sinai School of Medicine, New York.

Funding for the work was provided by National Institutes of Health and National Institute of Neurological Disorders & Stroke (R37-NS094804, R01-NS105556, R21- NS107894, R01- NS076991, R01-NS082311, R01-NS096756; the U-M Department of Neurosurgery; the Pediatric Brain Tumor Foundation, Leahs Happy Hearts Foundation, Ians Friends Foundation, Chad Tough Foundation, Pediatric Brain Tumor Foundation, and Smiles for Sophie Forever Foundation, National Cancer Institute (T32-CA009676), American Brain Tumor Association Basic Research Fellowship and a Rogel Cancer Center Scholar Award.

Paper cited: G-CSF secreted by mutant IDH1 glioma stem cells abolishes myeloid cells immunosuppression and enhances the efficacy of immunotherapy, Science Advances. DOI: 10.1126/sciadv.abh3243

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Glioma subtype may hold the secret to the success of immunotherapies - Michigan Medicine

Viral Vectors and Plasmid DNA Manufacturing Market 2021; Growing Number of Gene Therapy Candidates, Coupled with their Rapid Progression through…

DUBLIN, September 30, 2021--(BUSINESS WIRE)--The "Viral Vectors And Plasmid DNA Manufacturing Market Size By Product Type, By Application, By End Product, By Geographic Scope And Forecast" report has been added to ResearchAndMarkets.com's offering.

The Global Viral Vectors and Plasmid DNA Manufacturing Market was valued at USD 583.71 Million in 2020 and is projected to reach USD 1,866.90 Million by 2028, growing at a CAGR of 15.40% from 2021 to 2028.

A growing number of patients opting for gene therapy is a major factor propelling the growth of the Viral Vectors and Plasmid DNA Manufacturing market. Gene therapy is a leading field in medical science, which promises new treatment development for patients suffering from various disease. Genetically modified therapies have emerged as a promising treatment approach for various diseases (primarily ones that currently have no cure), including inherited disorders and certain viral infections. Demand for plasmid DNA is rising steeply because of a boom in gene therapy development.

This report provides an all-inclusive environment of the analysis for the Viral Vectors And Plasmid DNA Manufacturing Market. The market estimates provided in the report are the result of in-depth secondary research, primary interviews and in-house expert reviews. These market estimates have been considered by studying the impact of various social, political and economic factors along with the current market dynamics affecting the Viral Vectors And Plasmid DNA Manufacturing Market growth.

Along with the market overview, which comprises of the market dynamics the chapter includes a Porter's Five Forces analysis which explains the five forces: namely buyers bargaining power, suppliers bargaining power, threat of new entrants, threat of substitutes, and degree of competition in the Viral Vectors And Plasmid DNA Manufacturing Market. It explains the various participants, such as system integrators, intermediaries and end-users within the ecosystem of the market. The report also focuses on the competitive landscape of the Viral Vectors And Plasmid DNA Manufacturing Market.

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The report will provide a valuable insight with an emphasis on the global market including some of the major players such as Merck KGaA, Lonza, FUJIFILM Diosynth Biotechnologies U.S.A., Inc., Cobra Biologics Ltd., Brammer Bio, Waisman Biomanufacturing, Genezen, YPOSKESI, Advanced BioScience, Laboratories, Inc. (ABL, Inc.), Novasep Holding S.A.S, ATVIO Biotech Ltd, and Others.

Key Topics Covered:

1 Introduction

2 Research Methodology

3 Executive Summary

3.1 Market Overview

3.2 Global Viral Vectors and Plasmid DNA Manufacturing Market Regional Insights

3.3 Global Viral Vectors and Plasmid DNA Manufacturing Market Geographical Analysis

3.4 Global Viral Vectors and Plasmid DNA Manufacturing Market, by Product Type

3.5 Global Viral Vectors and Plasmid DNA Manufacturing Market, by Application

3.6 Global Viral Vectors and Plasmid DNA Manufacturing Market, by End Product

3.7 Future Market Opportunities

3.8 Global Market Split

4 Market Outlook

4.1 Global Viral Vectors and Plasmid DNA Manufacturing Market Outlook

4.2 Market Drivers

4.2.1 Increasing Number of Patients Opting for Gene Therapy

4.2.2 Rising Prevalence of HIV/Aids and Growing R&D Funding from Several Organizations

4.3 Restraints

4.3.1 Manufacturing Challenges Pertaining to Large Scale Production of Vectors

4.4 Opportunities

4.4.1 Growing Healthcare Infrastructure and Government Support

4.4.2 Growing Number of Gene Therapy Candidates, Coupled With Their Rapid Progression Through Various Phases of Clinical Development

4.5 The Impact of Covid-19

4.6 Porters Five Force Model

4.7 Product Life Line

5 Market, by Product Type

5.1 Overview

5.2 Viral Vector

5.3 Plasma DNA

5.4 Non-Viral DNA Vectors

6 Market, by Application

6.1 Overview

6.2 Cancer

6.3 Inherited Disorder

6.4 Infectious Diseases

6.5 Others

7 Market, by End Product

7.1 Overview

7.2 DNA Vaccines

7.3 Gene Therapy

7.4 Immunotherapy

7.5 Others

8 Market, by Geography

8.1 Overview

8.2 North America

8.3 Europe

8.4 Asia-Pacific

8.5 Row

9 Competitive Landscape

10 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/cnmete

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Viral Vectors and Plasmid DNA Manufacturing Market 2021; Growing Number of Gene Therapy Candidates, Coupled with their Rapid Progression through...

Celsion Corporation Adds Key Resources to its Vaccine Development Initiative and Clinical Trial Capabilities – Yahoo Finance

LAWRENCEVILLE, N.J., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, announces the strengthening of its management team with a new hire and a promotion in its vaccine development program, and the hiring of a veteran clinical trial project manager for its Phase II GEN-1 immunotherapy study in advanced ovarian cancer. These changes all are effective immediately and are as follows:

Carlo Iavarone, Ph.D. joins as Senior Director, Non-Clinical Research

Subeena Sood, Ph.D. promoted to Senior Manager, Biology and Preclinical Studies

Beth J. Llewellyn joins as Director of Clinical Operations

Dr. Iavarone will serve as project leader for the PLACCINE vaccine initiative. He will be based in Huntsville, Ala. and brings to Celsion more than 15 years of experience investigating and leading the development of vaccines, including molecular target identification and characterization of RNA vaccines. Most recently, from 2019 until 2021 he was a science advisor for both Guidepoint and Clora, providing input for a viral target and RNA vaccine delivery system. Dr. Iavarone joined GlaxoSmithKline in 2015 as a senior scientist studying small molecules and RNA vaccines in animal and human cell lines. From 2007 until 2015 he held positions of increasing responsibility at Novartis, including as a principal scientist for a melanoma vaccine project.

Dr. Iavarone has authored more than 15 papers on oncology and vaccine research that were published in peer-reviewed journals. He holds a Ph.D. in Molecular Pathology and Physiopathology from Federico Il University in Naples, Italy, and did his post-doctoral work at Novartis in Siena, Italy.

Dr. Sood is responsible for assay development and in vivo experiments for the PLACCINE DNA vaccine and gene therapy program, and also is based in Huntsville. She has experience with several pharmaceutical companies in experiment design, pharmacological and biochemical assays, manufacturing process design and development, and optimization and implementation of Quality by Design. Dr. Sood joined Celsion as manager of animal research in 2019, where she has designed and conducted all preclinical research. Prior to Celsion, since 2017 she was a Formulation Scientist II at Novocol Healthcare. From 2016 to 2017 Dr. Sood was a Research Associate II at Nektar Therapeutics, and from 2015 to 2016 she was a Quality Control Chemist I at Par Pharmaceuticals. She also worked in regenerative medicine as a Research Fellow at Medstar Heart Institute, Washington Hospital Center in Washington, D.C. from 2010 to 2013.

Story continues

Dr. Sood has authored more than 25 articles published in peer-reviewed journals, mainly in the area of cardiology and oxidative stress. She received her Ph.D. in Pharmacology from the All India Institute of Medical Sciences in New Delhi, and was a post-doctoral associate at the Baylor College of Medicine.

Ms. Llewellyn is responsible for the management of the ongoing Phase II OVATION 2 Study with GEN-1 in advanced ovarian cancer and will be based at our corporate office in Lawrenceville. Previously she was the President of 2L Pharma, a clinical operations consulting firm she founded in 2014. Her work included preparing protocols for Investigational New Drug submissions to the U.S. Food and Drug Administration, clinical trial site qualification and compliance and functioning as a liaison between clinical trial sites, contract research organizations and study sponsors. From 2011 to 2014 she was a Clinical Operations Management Consultant for Alba Therapeutics with oversight for all clinical activities related to a Phase IIb protocol investigating the use of a novel pharmaceutical agent for celiac disease. From 2010 to 2011 she was a Clinical Research Associate for Nabi Biopharmaceuticals, where she was responsible for providing in-house and field study monitoring, operational guidance and general assistance for multiple protocols investigating the use of a novel vaccine.

Ms. Llewellyn has been involved in over 30 clinical trials in a variety of therapeutic areas including oncology and infectious disease. She received a B.A. in psychology from Ohio University. Her graduate training in experimental psychology included studies in research design and statistical analysis.

As we continue to advance the development of our PLACCINE DNA-mediated vaccine platform and or Phase II study of GEN-1, we are delighted to deepen our bench strength with these talented and experienced professionals, said Michael H. Tardugno, Chairman, President and Chief Executive Officer of Celsion. Dr. Iavarone brings impressive clinical development experience particularly in RNA vaccines, which is so important Celsions work to develop a SARS-CoV-2 vaccine utilizing a DNA plasmid that encodes for multiple viral antigens.

Dr. Sood has been instrumental in the successful development of assays used to evaluate biological activity of our first generation of vaccines. She has proven herself to be a capable scientific leader, whose expertise will be relied upon as we complete our preclinical work to establish proof of concept using for the PLACCINE platform Covid-19 as a benchmark vaccine.

Lastly, as we advance our Phase 2 study with GEN-1 in advanced ovarian cancer, we welcome Ms. Llewellyn to Celsion. She is charged with ensuring that trial enrollment proceeds as planned, and we are confident she will capably address any protocol issues that might arise, Mr. Tardugno continued. Overall, we believe that with this strengthened team we are better able to realize the promise of Celsions technologies for the benefit of patients and our stockholders.

About Celsion Corporation

Celsion is a fully integrated, clinical stage biotechnology company focused on advancing a portfolio of innovative cancer treatments, including immunotherapies, DNA-based therapies and directed chemotherapies through clinical trials and eventual commercialization. The companys product pipeline includes GEN-1, a DNA-based immunotherapy for the localized treatment of ovarian cancer. ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, is under investigator-sponsored development for several cancer indications. Celsion also has two feasibility stage platform technologies for the development of novel nucleic acid-based immunotherapies and other anti-cancer DNA or RNA therapies. Both are novel synthetic, non-viral vectors with demonstrated capability in nucleic acid cellular transfection. For more information on Celsion, visit http://www.celsion.com.

Forward-looking Statements

Forward-looking statements in this news release are made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon current beliefs, expectation, and assumptions and include statements regarding the platform having the potential to provide broad protection against coronavirus disease 2019 (COVID-19), and possible future mutations of SARS-CoV-2 or other coronaviruses. These statements are subject to a number of risks and uncertainties, many of which are difficult to predict, including the ability of the Companys platform to provide broad protection against COVID-19, and possible future mutations of SARS-CoV-2 or other coronaviruses, the issuance of a patent to the Company for use of its technology platform for treating or preventing infection with the SARS-CoV-2 virus that causes COVID-19, unforeseen changes in the course of research and development activities and in clinical trials; the uncertainties of and difficulties in analyzing interim clinical data, particularly in small subgroups that are not statistically significant; FDA and regulatory uncertainties and risks; the significant expense, time and risk of failure of conducting clinical trials; the need for Celsion to evaluate its future development plans; possible acquisitions or licenses of other technologies, assets or businesses; possible actions by customers, suppliers, competitors or regulatory authorities; and other risks detailed from time to time in the Celsion's periodic filings with the Securities and Exchange Commission. Celsion assumes no obligation to update or supplement forward-looking statements that become untrue because of subsequent events, new information or otherwise.

Celsion Investor ContactJeffrey W. ChurchExecutive Vice President and CFO609-482-2455jchurch@celsion.com

LHA Investor RelationsKim Sutton Golodetz212-838-3777kgolodetz@lhai.com

# # #

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Celsion Corporation Adds Key Resources to its Vaccine Development Initiative and Clinical Trial Capabilities - Yahoo Finance

Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis -…

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been granted orphan drug designation from the European Commission for TSHA-101, an AAV9-based bicistronic gene replacement therapy in development for GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease.

GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A. The prognosis is devastating, with infantile forms often leading to death within the first four years of life and juvenile onset patients rarely surviving beyond mid-teens, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. Residual Hex A activity correlates with the severity of GM2, and based on our understanding of this correlation, small increases in Hex A activity are likely to lead to significant improvements in clinical outcomes and quality of life. Based on dose-dependent improvements in survival in preclinical models, we are highly encouraged that our novel bicistronic gene therapy approach with TSHA-101 has the potential to be a life changing therapy for patients suffering from this rapidly progressive disorder with no current treatment options.

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A (Hex A) enzyme. These genetic defects result in progressive dysfunction of the central nervous system. Residual Hex A enzyme activity determines the severity of the disease. The infantile form of the disease has an onset of symptoms usually before six months of age with residual Hex A enzyme activity of less than 0.1%. Juvenile onset occurs between 1.5 and five years of age with residual Hex A enzyme activity of approximately 0.5%. Early adult onset of the disease has residual Hex A enzyme activity of between 2% to 4%. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

TSHA-101 is an investigational gene therapy that delivers the HEXA and HEXB genes that make up the -hexosaminidase A enzyme. The two genes are driven by a single promoter within the AAV9 bicistronic vector ensuring that the 2 sub-units of Hex A are produced in a one-to-one ratio within each cell, which is important to ensure efficient production of the transgene. TSHA-101 is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA). TSHA-101 is administered intrathecally and is currently being evaluated in a single arm, open-label Phase 1/2 clinical trial for the treatment of infants with GM2 gangliosidosis sponsored by Queens University. Preliminary clinical safety and biomarker data are expected by year-end 2021.

The European Commission grants orphan drug designation for medicines being developed for the diagnosis, prevention or treatment of treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

About Taysha Gene Therapies

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including TSHA-101, 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, TSHA-101s eligibility for accelerated approval in the United States and Europe, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, both of which are 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 Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis -...

Ottawa biotech startup Virica gets multimillion-dollar funding injection for gene therapy-boosting technology – Ottawa Business Journal

An Ottawa biotech startup has raised millions of dollars to scale up its technology that aims to supercharge production of a key component in emerging gene therapies for cancer and other diseases.

Virica Biotech announced the series-A round, which was led by New York-based Dynamk Capital, on Monday. The company did not reveal the exact value of the deal, which is expected to close later this year with additional contributions from follow-on investors, but co-founder and CEO Jean-Simon Diallo said it was a multimillion-dollar investment.

Founded in 2018, Virica Biotech makes specialized compounds that help boost production of viral vectors, which deliver material into infected cells thats designed to fix defective genes responsible for diseases such as cancer and hereditary blindness.

Diallo used a cooking analogy to describe the process, likening viral vectors to kernels of popcorn that need the catalyst his firm provides to rapidly reproduce.

If you dont apply some heat, youre going to get maybe a few popped kernels, but thats it, he said. Adding our (product) basically allows you to fully pop the popcorn. Were kind of supercharging the microwave, if you will.

Diallo began working on the technology alongside pioneering Ottawa Hospital cancer researcher Dr. John Bell a decade ago as a way to help cancer-fighting viruses bypass the human immune systems defences.

When financing proved hard to come by due to the lengthy approvals process for such therapies, Diallo found another use for the compound that didnt require the same stringent testing: an additive to boost the effectiveness of traditional vaccines that contain virus particles.

Investors soon took note, and the company raised nearly a million dollars in seed funding by early 2020, just as the pandemic triggered a worldwide R&D effort to develop a new vaccine against COVID-19.

While his compound doesnt work on mRNA vaccines that have widely used in the fight against the coronavirus, Diallo says the pandemic gave his firm a proverbial shot in the arm nonetheless.

It forced us to get set up very quickly, he explained. There was certainly a lot of interest in the technology. COVID kind of got us moving. We realized that the opportunity for improving the manufacturing yield of gene therapies in particular was quite enormous.

Diallo says Virica now has contracts with more than 20 manufacturers of gene therapies that hope his products will make their treatments cheaper and more effective.

The firms revenues have more than doubled in the past 18 months, and Diallo expects Viricas headcount to grow from 16 to at least 30 by next year as demand for its technology ramps up. In addition, he says he still sees a big upside in the vaccine market, which now accounts for about 15 per cent of the firms sales.

Historically, the vaccine industry has always been very slow to move, he said. Hopefully, (the COVID) crisis will change things a little bit for the better because everybody had to kind of roll up their sleeves and innovate.

Virica was initially based out of Bayview Yards as part of Invest Ottawas pre-accelerator program. But the growing startup struggled to find suitable lab space in the region until the new Ottawa of Ottawa Health Innovation Hub helped it secure a 3,000-square-foot facility at the University of Ottawa Heart Institute that officially opened this week.

Diallo, a biochemistry professor at uOttawa and a senior scientist at the Ottawa Hospital Research Institute, said he considered moving the company to Montreal and Toronto. He called on the citys business and political leaders to beef up support for biotech ventures like his that are in the early stages of commercializing their products.

We were really glad to be able to stay in Ottawa, Diallo said. There is a biotech sector in Ottawa, it is growing and we need to work together to foster it. Otherwise, people like me are going to leave. Its that simple.

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Ottawa biotech startup Virica gets multimillion-dollar funding injection for gene therapy-boosting technology - Ottawa Business Journal

Global Nucleic Acid Therapeutics CDMO Market to Reach $4463.7 Million by 2030 – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Nucleic Acid Therapeutics CDMO Market - A Global and Regional Analysis: Focus on Product, Technology, and End User - Analysis and Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

The global nucleic acid therapeutics CDMO market was valued at $1,546.4 million in FY2020 and is estimated to reach $4,463.7 million by 2030.

The completion of human genome sequencing and the elucidation of the molecular pathways that are critical in the disease molecule interaction have offered an unprecedented opportunity and growth for the development of nucleic acid-based therapeutics. However, to keep with the manufacturing and development of such therapies, the pharmaceutical companies have established partnerships with the contract development and manufacturing company (CDMO) which are the viable alternatives to the in-house development of the drugs. Moreover, the success of the respective business model has also led these CDMOs to become an integral part of such pharmaceutical companies' value chain.

The increasing willingness to outsource drug development to the CDMOs, and the rising need for pharmaceuticals have resulted in the expansion of the global market for nucleic acid therapeutics CDMO.

In the past decade, there has been a vast increase in the amount of gene sequence information that has the potential to revolutionize the way diseases are categorized and treated. Traditional diagnoses, largely anatomical or descriptive in nature, are likely to be superseded by the molecular characterization of the disease. The fact that certain genes drive key disease processes will also enable the rational design of gene-specific therapeutics. Antisense oligonucleotides represent a technology that can play multiple roles in this process. Further, at present, there are 16 nucleic acid therapies approved by the FDA and EMA and many more in the pipeline implying the reliance and acceptance over the usage of such therapies in the market.

Market Growth Drivers

Market Challenges

Market Opportunities

Key Questions Answered in this Report:

Key Topics Covered:

1 Markets

1.1 Industry Outlook

1.2 Product Definition

1.3 Global Nucleic Acid Therapeutics CDMO Market Footprint, ($ Million), 2020-2030

1.4 Current Nucleic Acid Therapeutic CDMOs Landscape

1.5 Significant Usage of Nucleic Acid in Therapeutics

1.6 Types of Nucleic Acid Synthesized for Therapeutics

1.7 Market Dynamics

1.7.1 Market Drivers

1.7.1.1 Accelerating Shift of the Pharmaceutical Market Toward Innovative Biologic and Cell and Gene Therapy Products

1.7.1.2 Reduction in Overall Manufacturing Cost at CDMOs

1.7.1.3 Rising Approvals of Nucleic Acid Therapeutics

1.7.2 Restraints

1.7.2.1 Lack of Expertise in Nucleic Acid Manufacturing

1.7.2.2 Supply Chain and Logistical Challenges

1.7.2.3 Difficult Therapeutic Classification Due to Wide Variety of Nucleic Acids

1.7.3 Opportunities

1.7.3.1 Increasing Outsourcing Trend Among Pharmaceutical Companies

1.7.3.2 Accelerating Research and Development Along with Technology

1.7.3.3 Growth in Developing Countries

2 Competitive Landscape

3 Chemical Synthesis Method

4 Technology

5 End-User

6 Products

7 Regions

8 Markets - Competitive Benchmarking & Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/1x5210

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Nucleic Acid Therapeutics CDMO Market to Reach $4463.7 Million by 2030 - ResearchAndMarkets.com - Business Wire

Seattle biotech firm Shape Therapeutics inks gene therapy deal with Roche worth up to $3B – GeekWire

Shape Therapeutics CEO Francois Vigneault. (Shape Photo)

Shape Therapeutics has signed a deal potentially exceeding $3 billion with pharma giant Roche to support the development of gene therapies for Alzheimers and Parkinsons disease, the Seattle company announced Wednesday.

Shapes RNA editing technologies can change the sequence of RNA, which encodes the bodys protein building blocks. The company will deploy this technology with Roche in preclinical studies against targets relevant for these neurological conditions as well as certain rare diseases.

The collaboration may also leverage the biotech companys technologies for gene delivery. This system is designed to deliver RNA editing technology or other payloads directly to specific areas of the body, such as the nervous system or muscle.

Shape aims to unlock the next breakthrough in RNA technologies in the gene therapy space across a wide range of therapeutic areas, said Francois Vigneault, co-founder and CEO, in a statement.

Shapes RNA editing technology could potentially be used to change the amount of a key regulatory protein in the body or treat genetic diseases. The companys gene delivery technology is based on AAV vectors, a platform currently used to treat several rare conditions.

Under the agreement, Shape is eligible to receive an initial payment as well payments for hitting development, regulatory and sales milestones potentially exceeding $3 billion in aggregate value. Any products from the collaboration will be developed and commercialized by Roche.

The collaboration comes on the heels of a recent $112 million Series B investmentin June for the biotech, founded in 2018 with RNA editing technology spun out of the lab of co-founderPrashant Mali, a bioengineer at the University of California, San Diego.

Vigneault was former VP of research at Juno Therapeutics, a Seattle cell therapy company acquired by Celgene in 2018 for $9 billion. Other Juno veterans include Shapes head of platform technologies Adrian Briggs and vice president and head of research David Huss.

The new collaboration may reflect growing drug company interest in neurosciences, which is on the upswing after a lull several years ago. The recent FDA approval of Biogens Alzheimers drug Aduhelm may also be propelling interest in the field.

This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy, said James Sabry, Head of Roche Pharma Partnering, in a statement.

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Seattle biotech firm Shape Therapeutics inks gene therapy deal with Roche worth up to $3B - GeekWire

How to Prevent Parkinson’s Disease: Tips, Medications, and Research – Healthline

Parkinsons disease is the second most common neurodegenerative disorder in the United States, affecting at least 500,000 people. Its a progressive disease, but can be managed with treatment. For now, the condition is diagnosed by symptoms rather than specific lab tests.

By the time a formal diagnosis is made, the National Institute of Neurological Disorders and Stroke (NINDS) notes that the disease has usually progressed to a point where people have difficulty controlling bodily movements.

Thats why its important to know whether its possible to prevent this disease.

Currently, there is no therapy or treatment that can slow the progression of Parkinsons or effectively relieve advanced symptoms, according to the NINDS.

By the time classic motor symptoms of Parkinsons disease show up, a significant loss of brain cells and function have already occurred. Scientists are investigating ways to detect early signs of the disease, to potentially stop or slow the progression.

Researchers aim to learn more about biomarkers of the early stages of the disease. Finding reliable biological identifiers might help doctors diagnose and treat Parkinsons earlier. Identifying these signs would give them more time to try therapeutic treatments before the disease has progressed.

For example, research indicates that it may be useful to study the activity of a neuronal protein in the brain known as -synuclein, or alpha-synyclein.

A 2019 study noted that measurements of -synuclein have shown encouraging preliminary results with regard to potential early diagnosis. Another 2019 study also examined how -synuclein accumulates in the brains of people suspected of having Parkinsons disease.This information could be used to develop therapies, perhaps antibody therapy, to prevent that accumulation from happening.

Scientists are also working to learn more about environmental factors and genetic factors that might contribute to the risk of developing Parkinsons. One recent genetic research breakthrough is the development of a DNA chip called NeuroX, which could potentially determine a persons risk, but more research is needed.

Parkinsons disease is the result of complicated combination of interconnected events, as one 2016 study described it. Since aging is the most common risk factor, future treatments may need to take degeneration of certain neurons into account.

While its not yet known if there are surefire ways to prevent Parkinsons disease, there are a few things experts recommend.

For example, you might try incorporating physical activity into your routine and eating a healthy and balanced diet for a variety of health reasons. So far, research into nutritional supplements is lacking. However, if you have specific dietary needs, talk to your doctor to see if supplementation is appropriate.

Could CBD oil help? Its possible, but we dont know for sure yet. Some research, including a 2018 review of studies, suggest that cannabidiol (CBD) might help prevent Parkinsons disease. However, the studies are mostly animal studies and there is not yet a body of research involving humans and CBD.

Some experts suggest that you may be able to delay some of the effects of Parkinsons disease through regular physical activity. Ideally this would include a combination of exercise that includes:

The NINDS has funded a number of studies to learn more about the impact of exercise, including whether exercise might help people delay the need for medication.

There are a variety of options for treating and managing Parkinsons symptoms, most of which involve medications that address the brains low levels of dopamine. Dopamine is a chemical in your brain that affects movement, and Parkinsons causes your brain to lose neurons that produce this chemical.

Medications that address this include levodopa, or levodopa combined with carbidopa. Or your doctor might prescribe a dopamine agonist, which mimics the action of dopamine in your brain. Other drugs used to treat Parkinsons include:

Another possible treatment option is deep brain stimulation (DBS). DBS was approved by the U.S. Food and Drug Administration in 1997. Many people have found that this treatment, which involves sending electrical impulses into the brain via tiny electrodes, helps control tremors once treatment with levodopa is no longer effective.

A small 2018 study found that DBS seemed to slow the progression of tremors in people with Parkinsons disease. It also found that DBS could be used effectively in people with an earlier disease stage than previously thought.

Scientists hope that more treatments may become available in the future, as they learn more about which drug may or may not be effective at slowing or halting the progression of the disease.

For example, a randomized, double-blind trial of 62 patients found that people with Parkinsons who took a drug usually used to treat diabetes seemed to stop the progression of the Parkinsons symptoms. They received weekly injections of exenatide for 48 weeks.

It was a relatively small study, and longer-term trials are needed, according to the researchers. A larger study involving more patients is currently ongoing.

If you are already living with Parkinsons disease, here are some tips to manage it:

There are medications that can help treat the symptoms of Parkinsons disease, and scientists are currently conducting research that could result in new treatment and therapies.

For example, you might one day have the option to take a medication used to treat prostate gland enlargement if youre at risk for developing Parkinsons disease.

The results of a study published in early 2021 suggest that certain medications often used to treat enlarged prostates are associated with a decreased risk of developing Parkinsons disease. Specifically, the researchers compared terazosin, doxazosin, and alfuzosin, which enhance energy metabolism, to tamsulosin, which is also used to treat benign enlarged prostates. They found that the latter did not seem to have the same effect.

The findings built on their previous research, which suggested that the use of terazosin, doxazosin, and alfuzosin was associated with slower progression and fewer complications in people with Parkinsons disease.

Researchers are also looking into the potential of stem cells to create new neurons to produce dopamine. They are also researching a protein called glial cell-derived neurotrophic factor, or GDNF, to potentially slow the progression of Parkinsons.

Ongoing research into a gene called LRRK2 or LARK2 and how it may interact with other genes related to Parkinsons disease is also promising, as it may shed light on how the disease progresses and how it might be halted.

For now, the symptoms of Parkinsons disease can be managed with medication and potentially deep brain stimulation. But research is underway to look for earlier methods of detection, as well as better treatments. Eventually, we might even have a way to prevent it from developing in the first place.

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How to Prevent Parkinson's Disease: Tips, Medications, and Research - Healthline

Returning Vision to The Fully Blind – Interview with Dr. Botond Roska – Hungary Today

We sat down for an interview with Professor Botond Roska, MD PhD, one of the worlds leading experts in the study of vision and the retina and last years recipient of the Krber Prize, to discuss the ground-breaking therapy he and his team have developed and already successfully used to return some vision to a handful of blind patients.

Can you tell us about the exact nature of your research for which you received the Krber Award?

It is sometimes difficult to know exactly what you get an award for, but I think I received it for two distinct but related scientific pursuits: firstly, my research on understanding vision, and secondly, for developing a treatment called optogenetic vision restoration therapy, through which a completely blind person can be sensitized to light, allowing them to see again.

FactProfessor Botond Roska, MD PhD, is a biomedical researcher and one of the foremost experts on vision, the retina, and treating diseases that cause blindness. He is a founding director of the Institute for Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Professor at the University of Basel. In 2019, he was awarded the Order of Saint Stephen, the highest national honor bestowed by Hungary, as well as the Louis-Jeantet Prize for Medicine. In 2020, he won the Krber European Science Prize for his research on a gene therapy that could be utilized to restore some vision to the fully blind, which he successfully implemented with his team for the first time this May.

What exactly does this procedure you developed entail?

It is a so-called two-component therapy. We inject a gene therapy vector into the eye, which is a small, virus-like particle with a DNA that encodes a light-sensitive protein. We target this protein at some elements of the blind retina. The vector also has a goggle that records the world, and then projects a picture onto the retina in a very specific way, in a particular color.

How effective is it now, and how effective can it potentially become?

The therapy restores a certain amount of vision, but not full sight. Based on the few patients we have had so far, the first of whom we recently published a paper about, the therapy allows patients to recognize objects, but it does not enable them to read, for instance.

However, there is a lot of room to improve. This is only the very first step, the very first optogenetic therapy.

What kinds of blindness or visual impairment can it help or cure?

It is useful in cases when someone is fully blind as a result of photoreceptor dysfunction, but their retina is still connected to their brain via an intact optic nerve.

How profound an effect do you think being able to heal peoples vision at a large scale might have on society?

Blindness is one of the conditions that people name as the worst affliction to have. In fact, in a recent survey conducted in the U.S., participants named it as the worst condition they could suffer from out of a whole host of common health problems, ranking it above cancer, Alzheimers, and other truly devastating diseases.

Our whole life is spent looking at phones and computers. Particularly during the pandemic, we could have almost no social interactions or opportunities to work without these, so that blind people were essentially cut off from the world apart from what little interaction they could have with it through hearing.

Therefore, we hope that such therapies, once they become widely available since they are in the clinical trial phase at the moment will improve many peoples lives tremendously.

Where do you think your research might lead you in the future?

There are three main directions. The first one is simple; as we discussed, we need to keep researching and improving optogenetic vision therapies, expanding them to target other cell types in the retina.

The second one is to do with the fact that if the optic nerve is missing, we cannot provide any therapies at the moment. This is something we are working on at my institute together with researcher Dniel Hillier, who is leading these efforts. We aim to find ways of restoring vision when there is no optic nerve.

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Hungarian-born researcher Dr. Botond Roska has been awarded the 2019 Louis-Jeantet Prize for Medicine for the discovery of basic principles of visual information processing and the development of therapeutic strategies, such as gene therapy, to restore vision in retinal disorders. Within the last few months, the neurobiologist (who celebrates his 50th birthday this year) received []Continue reading

The third is that most visual impairment is partial blindness, in which the method we developed cannot be used, so we are interested in looking into very large diseases affecting a lot more people. We want to try to slow down the degeneration or restore more vision to those with partial sight loss.

Why did you choose to dedicate your career to understanding human vision at the hardware level?

It was a chain of random events. I did not plan to be a vision researcher. I just wanted to understand things. When I finished my medical school, I was quite sure I did not want to treat patients for a living, I was more interested in understanding the human body. I decided to go into research, and I met someone who was researching the retina. I started my research in this field, and came up against an increasing number of questions that I found fascinating.

I went from topic to topic, physiology, virology, the molecular biology of the eye, then towards the end of my graduate studies, I read a paper that said it was possible to make cells light-sensitive using molecules form other organisms. At that time, I understood the retina quite well, and thought that I could combine my knowledge with these findings to try to design a therapy. My lab and myself are both interested in gaining scientific insight as well as designing therapies, and that is the path I plan to continue on for the rest of my career.

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The findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible, Botond Roska said.Continue reading

What is the worst and best part of the publicity youve received as a result of your success?

The best part is certainly that blind people can become informed that we are working on a therapy, which can provide them with some hope. It is also important for my institute, which can more easily recruit brilliant scientific minds who will come up with even better therapies down the line.

On the other hand, while there is nothing really bad about publicity per se, it can sometimes be time-consuming. Our paper about our first patient came out in May, and it has been downloaded around 90,000 times, with just about every country in the world reporting on our findings. And of course, all of the press outlets in all these countries bombarded our inboxes with requests. We were basically paralyzed for almost a month.

As someone who is called upon with increasing frequency to explain your cutting edge research, how do you approach the issue of science communication?

Communication is very important. It is part of our lives. In Switzerland specifically, it is taken very seriously, and it is part of our jobs as researchers to explain things to the public. We also have professionals to help us navigate the world of public relations.

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President Jnos der decorated neurobiologist Botond Roska with the Hungarian Saint Stephen Order on Tuesday, Hungarys national holiday. Hungarian-born Researcher Awarded for Helping People Regain Their Sight Each year on the holiday of the founder of our state we celebrate those Hungarians whose outstanding talent and achievements serve the well-being and prosperity of many others, []Continue reading

The difficulty today is finding the balance between our work and its communication to the world. Because our research reaches a tremendous number of people extremely quickly thanks to the global nature of social media and other platforms, we get a lot of requests for interviews and articles. The difficulty for us is balancing our responsibility to inform the public of our progress with actually making some. Sometimes, it feels very much like we are in the eye of the storm.

Many people consider you a likely future Nobel laureate. Do you believe you might receive the award?

I dont think about any of that. It does not occupy any of my time or of my imagination. At this moment, there are still great challenges ahead of us if we want to make the therapies as good as they can be. That is what I concentrate on. We also need to focus on innovating and coming up with new tools to help realize our scientific vision. This is also a costly and time-consuming endeavor.

What would you tell aspiring scientists, how can they best succeed in their scientific endeavors?

I think that the key is to come to science with an incredible desire to solve interesting problems. I never wanted to be successful in the public eye, just accomplished at solving scientific questions. For some reason, some scientists are more present in the media, and some of them win prizes, while others win prizes and choose not to have a public presence. Often, the most brilliant and prolific scientists do not even win prizes.

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Returning Vision to The Fully Blind - Interview with Dr. Botond Roska - Hungary Today

Ryder’s Story: Middleton baby with rare genetic condition gets a $2.1 million infusion to give him a fighting chance – KTVB.com

Ryder Comer was born with SMA, the best treatment option was an infusion that cost $2.1 million. Now, his mom is working to add SMA to Idaho's newborn screening.

MIDDLETON, Idaho When Jake and Haley Comer of Middleton welcomed their new baby boy Ryder into the world, they were completely overjoyed. But, they quickly realized something might not be right.

"The whole pregnancy was completely healthy, there were no signs of anything." said Haley Comer, Ryder's proud mom. "As soon as he was born though, he was diagnosed with low muscle tone. No parent wants to think that something is wrong with their perfect little baby."

The Comers went to their pediatrician to get some answers. The doctor agreed that newborn Ryder was "floppy" and had low muscle tone. He recommended genetic testing to see if there was a reason for that. While waiting for the results in the following weeks, the unthinkable happened. Ryder stopped breathing.

"The day after Ryder turned a month old, I found him unresponsive at home, I had him in a little baby carrier. So, I called 911 and started CPR and luckily was able to revive him. We were rushed to the ER, and that initiated a month-long stay in the hospital," said Haley Comer with tears in her eyes.

While they were at St. Luke's, the genetic testing results came back. The news was not what the Comers were hoping for.

"We got our test results back and Ryder was diagnosed with SMA, Spinal Muscular Atrophy Type 1," said Haley. "It was hard, there were a lot of tears, but at the same time we want to be brave. You have to put on a brave face for your baby."

SMA is a progressive, rare genetic disease that impacts the muscles. Babies with SMA Type 1 face many physical challenges, including muscle weakness and trouble breathing, coughing, and swallowing.

Doctors at St. Luke's in Boise told the Comers about a promising new treatment option. A gene therapy infusion that could save baby Ryder's life.

"They told us that they would be transferring him to Salt Lake City to Primary Children's Hospital, because no hospitals in Idaho performed this infusion," said Haley. "A couple days later, we met with a neurologist in Salt Lake and learned about our treatment options. This gene therapy option seemed like the best one. It's a once in his lifetime, one-hour long, gene therapy infusion. He is missing a very important gene that produces survival motor neuron protein. That helps our body move our muscles. It basically injects this gene into his body, and then his body learns to keep producing this survival neuron protein to keep his muscles alive."

#7sHero Tonight at 10 p.m. on KTVB youll meet this precious baby boy, Ryder Comer of Middleton. His parents Jake &...

It sounded like the best option for Ryder, but the Comers were stunned when they found out just how much the infusion treatment would cost.

"It's called Zolgensma, and it costs $2.1 million for one single infusion," said Haley.

Zolgensma is FDA-approved for patients with all forms and types of SMA who are under 2 years of age. Research shows babies dosed with Zolgensma as soon after diagnosis as possible have had better results than those who waited to begin treatment. It's given through an IV infusion that takes about an hour, and it's a one-time treatment.

The Comers weren't even sure their insurance would cover it, but they moved forward with trying to get the pricey infusion procedure preauthorized.

"It was terrifying when they told us," said Jake Comer, Ryder's dad. "I was like I hope you take monthly payments for the rest of my life."

Incredibly, the approval from their insurance company, United Healthcare, went smoothly and quickly.

"We paid our deductible, and our out-of-pocket, and they took care of the rest," said Jake in disbelief. "That definitely took the stress of our shoulders during such a traumatic time, so we are very grateful."

The Comers say it was all a whirlwind. Ryder received the infusion within 24 hours.

"We feel pretty fortunate because a lot of families had to fight for weeks and months to get their insurance to cover this," said Haley. "For Ryder, his disease had progressed so much by the time he got it, he was at the point where every day mattered."

After the $2.1 million infusion, Ryder started making strides within days. The changes they've seen over the past several months have been so wonderful for the Comers.

"His breathing got stronger, his voice got stronger, his cry got louder, he had a really weak cry at first," said Haley. "Now he can lift his arms above his head and grab toys! He can't quite hold his head up yet, but we are working on it. He is in feeding and physical therapy, and we are doing everything we can to get him stronger."

So far, Ryder is the only baby in Idaho with SMA to receive this gene therapy infusion. Haley has a new passion. She is now dedicated to helping other SMA families like theirs.

"What we are really working toward right now is to have SMA added to Idaho's newborn screening. Idaho is one of the last remaining states that does not include SMA on its newborn screening test. It's just a simple blood test. Before the approval of his gene therapy two years ago, the life expectancy for babies diagnosed with SMA Type 1 was not much past two years old. That is just devastating for families. So having SMA added to Idaho's newborn screening will allow these babies to have access to treatment so much sooner. The sooner you get a diagnosis and have treatment, the better outcomes you will have in life."

Haley has been in contact with the Idaho Department of Health and Welfare, and with the organization CURE SMA, which has been working to add this disease to every state's newborn screening. She's in the early stages, but this mom's goal is to make that happen here in Idaho, too.

"We just think that every baby deserves to be treated as soon as they possibly can," said Haley.

August is SMA Awareness Month. Baby Ryder and his parents are paving the way for other Idaho babies born with SMA.

"He's expected to get stronger and hopefully meet those milestones of walking and talking and crawling and all those things, but we just don't know, we are working hard in therapy to give him the best possible chance. One day a time."

For more information on SMA, click here. If you'd like to help the Comer family with their medical expenses, there is a GO FUND ME set up to do so.

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Ryder's Story: Middleton baby with rare genetic condition gets a $2.1 million infusion to give him a fighting chance - KTVB.com

Ulcerative Colitis Study Analyzes Gene Expression to Measure Risk of Progression to Surgery – GenomeWeb

NEW YORK An international team of researchers has used transcriptomic data from ulcerative colitispatients to develop a predicted polygenic transcriptional risk score, or PPTRS,that can identify UC-affected individuals at fivefold elevated risk of progressing to surgical resection of the large bowel.

In a paper published on Thursday in the American Journal of Human Genetics, the Georgia Institute of Technology-led team noted that 5 percent to 10 percent of people with UC require bowel resection, or colectomy, within five years of diagnosis, but that polygenic risk scores based on genome-wide association studies generally don't provide meaningful prediction of progression to surgery. However, studies of Crohn's disease have shown that gene expression profiling of GWAS-significant genes provides some stratification of risk of progression to complicated disease through transcriptional risk scoring, or TRS.

In their paper, the researchers demonstrated that a measured TRS based on bulk rectal gene expression in a cohort of UC patients had a positive predictive value approaching 50 percent for colectomy. Single-cell profiling demonstrated that the disease-associated genes were active in multiple diverse cell types from both the epithelial and immune compartments, and expression quantitative trait locusanalysis identified genes with differential effects at baseline and the one-year follow-up, the researchers said. But for the most part, they found that differential expression associated with colectomy risk was independent of local genetic regulation.

Overall, their data suggested that prediction of gene expression from relatively small transcriptome datasets can be used in conjunction with transcriptome-wide association studies for stratification of risk of disease complications.

The researchers began by performing differential expression analysis between baseline rectal RNA-seq biopsies of individuals in the PROTECT multicenter pediatric inception cohort study of response to standardized colitis therapy. Analyses were done on 21 affected individuals who progressed to colectomy and 310 who did not. They identified downregulation of 783 transcripts in the individuals who underwent colectomy and upregulation of 1,405 transcripts overall.

They also obtained rectal biopsy RNA-seq data for 92 affected individuals at week 52 and observed a marked shift in gene expression at follow-up, prompting them to ask whether local regulation of the gene expression might contribute to this effect. They found that there were 72 SNPs that were significantly regulating 308 genes at both time points.

Further examination of the expression of colectomy-associated genes in a single-cell RNA-seq dataset obtained from rectal biopsies provided strong evidence that both epithelial and immune cells contributed to the risk of disease progression, the researchers said.

The researchers then performed a TWAS to capture the effects of all polymorphisms within 1 Mb of each transcript expressed in the PROTECT rectal biopsies and then used the weights to predict gene expression in a validation cohort from the UK Biobank. They tested for differential predicted gene expression in 70 percent of the validation samples and discovered about 800 genes either upregulated or downregulated in UC-affected individuals relative to non-IBD control individuals. They then derived a PPTRS for UC based on the effect sizes of the minor alleles and applied it to the remaining 30 percent of the validation samples, as well as to the PROTECT genotypes, and found that the PPTRS efficiently discriminated UC-affected individuals from non-IBD control individuals.

Significantly, it also discriminated the individuals who underwent colectomy versus those who didn't in both the UK Biobank and PROTECT.

"More extensive single-cell profiling, combined with cell-type-specific genetic analysis of gene expression, is likely to lead to the development of even better transcriptional risk signatures," the authors concluded. "It is also likely that such focused and personalized analysis may highlight specific pathological mechanisms active in particular affected individuals."

They did note, however, that these results were limited by the relatively small sample size of colectomies in the PROTECT study, and that validation of cross-ancestry assessments and the evaluation of the consistency of gene expression prediction across populations should be a high priority.

In an email, corresponding author and GIT researcher Greg Gibson noted that while the study's multiple layers of replication show that transcriptional profiling of the rectum greatly enhances risk stratification for risk of colectomy, this was not a clinical trial, so the approach is not yet approved for evaluation of patients.

"We hope that it will progress to implementation in the near future," he added."The prediction from genotypes alone is less likely to have clinical utility since the precision is still quite low, so that aspect is more research oriented."

He further noted that the approach he and his colleagues used could also be applied to a wide range of diseases, and that they are pursuing that research.

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Eight ways scientists are unwrapping the mysteries of the human brain – MIT Technology Review

Brain development that occurs after birth is also important. Rebecca Saxe at MIT is working to understand the brain structures and activities responsible for social cognition, which allows us to consider the mental states of other people.

Saxe has discovered a particular brain region that is key; by studying how activity in this region and others changes over the course of childhood, she may be able to understand how social abilities develop. She has also found that these brain activity patterns are altered in people with autism spectrum disorders.

Even though researchers are starting to understand some of the processes that govern development and have identified things that can derail it, were far from being able to intervene when such problems occur. But as we gain insights, we could someday test therapies or other ways to address these developmental issues.

Computational neuroscientists use mathematical models to better understand how networks of brain cells help us interpret what we see and hear, integrate new information, create and store memories, and make decisions.

Understanding how the activity of neurons governs cognition and behavior could lead to ways to improve memory or understand disease processes.

Terry Sejnowski, a computational neurobiologist at the Salk Institute, has built a computer model of the prefrontal cortex and analyzed its performance on a task in which a person (or machine) has to sort cards according to a rule thats always changing. While humans are great at adapting, machines generally struggle. But Sejnowskis computer, which imitates information flow patterns observed in the brain, performed well on this task. This research could help machines think more like humans and adapt more quickly to new conditions.

Aude Oliva, the MIT director of the MIT-IBM Watson AI Lab, uses computational tools to model and predict how brains perceive and remember visual information. Her research shows that different images result in certain patterns of activity both in the monkey cortex and in neural network models, and that these patterns predict how memorable a certain image will be.

Research like Sejnowskis may inspire smarter machines, but it could also help us understand disorders in which the function of the prefrontal cortex is altered, including schizophrenia, dementia, and the effects of head trauma.

Researchers are trying to determine the genetic and environmental risk factors for neurodegenerative diseases, as well as the diseases underlying mechanisms.

NHUNG LE

Improving prevention, early detection, and treatment for diseases like Alzheimers, Parkinsons, Huntingtons, chronic traumatic encephalopathy, and ALS would benefit millions of people around the world.

Yakeel Quiroz, at Massachusetts General Hospital, studies changes in brain structure and function that occur before the onset of Alzheimers symptoms. Shes looking for biomarkers that could be used for early detection of the disease and trying to pinpoint potential targets for therapeutics. One potential biomarker of early-onset Alzheimers that shes founda protein called NfLis elevated in the blood more than two decades before symptoms appear. Quiroz has also identified a woman with a protective genetic mutation that kept her from developing cognitive impairments and brain degeneration even though her brain showed high levels of amyloid, a protein implicated in Alzheimers development. Studying the effects of this beneficial mutation could lead to new therapies.

Researchers at the Early Detection of Neurodegenerative Diseases initiative in the United Kingdom are analyzing whether digital data collected by smartphones or wearables could give early warnings of disease before symptoms develop. One of the initiatives projectsa partnership with Boston Universitywill collect data using apps, activity tracking, and sleep tracking in people with and without dementia to identify possible digital signatures of disease.

As we learn more about the underlying causes of neurodegenerative diseases, researchers are trying to translate this knowledge into effective treatments. Advanced clinical trials targeting newly understood mechanisms of disease are currently under way for many neurodegenerative disorders, including Alzheimers, Parkinsons, and ALS.

Connectomics researchers map and analyze neuronal connections, creating a wiring diagram for the brain.

Understanding these connections will shed light on how the brain functions; many projects are exploring how macro-scale connections are altered during development, aging, or disease.

Mapping these connections isnt easythere may be as many as 100 trillion connections in the human brain, and theyre all tiny. Researchers need to find the best ways to label specific neurons and track the connections they make to other neurons in remote parts of the brain, refine the technology to collect these images, and figure out how to analyze the mountains of data that this process produces.

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Eight ways scientists are unwrapping the mysteries of the human brain - MIT Technology Review

NeuExcell Therapeutics Raises Over $ 10 Million Series Before Round To Keep The Company Growing | Around The Web-Pennsylvania – Pennsylvania News…

State College, Pennsylvania., August 30, 2021 / PRNewswire / -NeuExcell Therapeutics (www.neuexcell.com), A gene therapy company focused on neurodegenerative diseases has announced a Series Pre-A funding round of over $ 10 million. The round was led by Co-Win Ventures and was attended by other institutional investors Yuan Bio, Oriza Seed, Tsingyuan and Inno Angel.

We are honored to join this very reputable group of investors, he said. Peter Tombros, Chairman of the Board of Directors of NeuExcell Therapeutics. Investor experience and support will enable us to leverage our unique neuroregenerative gene therapy platform across multiple neurodegenerative indications. This funding strength validates our strategy and biotechnology. Further examine our science in the industry.

Professor Gong Chen, co-founder and chief scientific advisor of the company, said: There is an urgent need for breakthrough therapies like us.

I think this is a great opportunity to invest in experienced leadership, he said. Xin Huang, Managing Partner of Co-Win Ventures. NeuExcells unique technology has the potential to act as a platform for treating many neurodegenerative diseases, providing hope for breakthrough new therapies for patients who do not have the right choices today.

With the end of this successful pre-A round, we welcome him. Xin Huang Jonathan Sun attended the board meeting.

About NeuExcellTherapeutics

NeuExcell is a privately held early stage genetic engineering company headquartered in Pennsylvania, USA When Shanghai, China.. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and damage to the central nervous system. Based on Professor Gong Chens scientific research, we have developed a potentially destructive nerve repair technique through the conversion of astrocytes to neurons. In vivo By introducing neural transcription factors through adeno-associated virus (AAV) -based gene therapy. NeuExcells pipeline covers major neurodegenerative diseases such as stroke, Huntingtons disease, amyotrophic lateral sclerosis (ALS), Alzheimers disease, Parkinsons disease, traumatic brain injury, spinal cord injury, and glioma. increase.

About Co-Wof Venture

Founded in 2009, Co-Win Ventures is an early stage investor in healthcare and TMT with a focus on equality, transparency, sharing and innovation. Co-Wins business network China When USA..Total AUM is about US $ 1 billion, Co-Win aims to be a reliable partner for great entrepreneurs to build breakthrough technologies and businesses. Co-Win Ventures has helped more than 140 portfolio companies, including leading leaders in their respective sub-sectors, including Cytek, Connect, Thrive (acquired by Nasdaq-listed company EXAS), Taimei Technology, Genecast, Sinovation and Augta. ..

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