Gene Therapy


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    Image courtesy of Ultragenyx.

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    Ultragenyx says Angelman therapy is working, but safety questions remain

    Treatment appeared to result in functional and cognitive gains in people with the neurological disorder. Three participants experienced lower extremity weakness, however.

    By April 15, 2024
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    Nemes Laszlo via Getty Images
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    Roche, Adaptimmune part ways on cell therapy research

    The two companies have ended a collaboration that was struck in 2021 and focused on “off-the-shelf” cell therapies for cancer.

    By April 12, 2024
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    Trendline

    Gene Therapy

    Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, researchers and drugmakers still face major challenges.

    By BioPharma Blog staff
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    Seth Babin/BioPharma Blog
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    Verve pauses base editing trial, shifts strategy after treatment side effect

    Lab abnormalities in one study participant led the company to halt enrollment and prioritize a different version of its genetic medicine for heart disease.

    By April 2, 2024
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    iStock via Getty Images
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    Orchard sets out to sell world’s priciest gene therapy

    Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25 million list price, the highest of any genetic medicine to come to market.

    By Kristin Jensen • March 20, 2024
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    Nemes Laszlo via Getty Images
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    Emerging biotech

    Capstan heats up ‘in vivo’ cell therapy chase with $175M fundraise

    The high-profile startup, co-founded by CAR-T innovator Carl June, will use the funds to bring an autoimmune disease treatment into proof-of-concept studies.

    By March 20, 2024
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    Orchard follows buyout with FDA approval of rare disease gene therapy

    The U.S. clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

    By Kristin Jensen • March 19, 2024
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    Bristol Myers cell therapy wins first-of-its-kind approval

    Sold as Breyanzi, the therapy is now cleared for use in certain adults with hard-to-treat forms of relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma.

    By March 15, 2024
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    Design Cells via Getty Images
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    J&J, Novo support Asgard’s push to make ‘in vivo’ cell therapies

    The Swedish biotech will use the investment to advance a personalized cancer therapy that reprograms tumor cells inside the body.

    By March 14, 2024
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    FDA to focus on ‘early deaths’ in meeting on broader CAR-T use in myeloma

    Briefing documents posted ahead of Friday’s advisory panel zeroed in on increased rates of early death in tests of cell therapies from Bristol Myers Squibb and Johnson & Johnson.

    By March 13, 2024
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    Nemes Laszlo via Getty Images
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    Allogene taps Arbor in pursuit of ‘off-the-shelf’ CAR-T therapies for autoimmune disease

    The partnership is designed to help the company stand out in what’s quickly become one of the industry’s hottest areas of drug research.

    By March 12, 2024
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    metamorworks via Getty Images
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    Regenxbio data suggest ‘niche’ in Duchenne gene therapy

    Study results showed early signs of promise in Duchenne patients who are currently ineligible to receive Sarepta’s Elevidys, triggering a stock surge.

    By Kristin Jensen • March 6, 2024
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    Courtesy of Cure Ventures
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    Cure Ventures backs a cell therapy startup targeting Parkinson’s

    The startup, Kenai Therapeutics, has raised $82 million and will be chaired by Jeff Jonas, a new Cure partner and former CEO of Sage Therapeutics.

    By Feb. 29, 2024
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    ismagilov via Getty Images
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    BioMarin preaches patience amid slow sales for hemophilia gene therapy

    The company earned only $3.5 million last year from its Roctavian treatment, far below the $50 million to $150 million range it had forecast eight months ago.

    By Feb. 23, 2024
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    Love Employee via Getty Images
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    NEJM paper fills in details on ‘remarkable’ CAR-T result in autoimmune disease

    The full results spotlight cell therapy’s potential to treat lupus and other inflammatory conditions, but also the problems drugmakers must still solve.

    By Feb. 21, 2024
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    Courtesy of Iovance Biotherapeutics
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    Iovance, with approval of ‘TIL’ cell therapy, readies for complex launch

    The biotech set a price of roughly $515,000 per patient for its therapy Amtagvi, the first to be approved based on a decades-old technique of using tumor-infiltrating lymphocytes.

    By Feb. 20, 2024
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    Courtesy of Sarepta
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    FDA to review expanded use of Sarepta Duchenne gene therapy

    The agency will decide by June 21 whether to broaden eligibility for Elevidys, and won’t convene a group of outside experts beforehand.

    By Feb. 16, 2024
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    Mohammed Haneefa Nizamudeen via Getty Images
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    Intellia, ReCode partner on genetic medicines for cystic fibrosis

    The partnership will use Intellia's "DNA writing” technology, and focus on people with the lung disease who have limited or no available treatments

    By Feb. 15, 2024
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    Dr_Microbe via Getty Images
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    New CMS pilot to test payment scheme for pricey sickle cell gene therapies

    The agency is planning a pilot program across states to help sickle cell patients access treatments like the newly approved Casgevy and Lyfgenia.

    By Jan. 31, 2024
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    jetcityimage via Getty Images
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    Lilly gene therapy finding puts focus on hearing loss treatment pipeline

    An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.

    By Updated Jan. 25, 2024
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    Gene therapy biotech Jaguar spins out manufacturing company

    Called Advanced Medicine Partners, the new company will offer specialized cell and gene therapy manufacturing services to biotech and pharma clients.

    By Updated Jan. 18, 2024
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    Courtesy of Vertex Pharmaceuticals
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    FDA widens approval of Vertex’s CRISPR medicine to treat beta thalassemia

    The agency’s decision to expand use of Casgevy, which won a landmark OK for sickle cell disease in December, comes two months ahead of schedule.

    By Jan. 16, 2024
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    Courtesy of Intellia Therapeutics
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    Intellia to lay off staff, cut some early research

    The CRISPR specialist joins some of its genetic medicine peers in trimming staff, announcing an “organizational streamlining” to focus resources on prioritiy drug programs.

    By Jan. 4, 2024
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    Grigorii Yalukov via Getty Images
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    Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy

    The hemophilia B therapy, which Pfizer will sell in Canada as Beqvez, is also under review in the U.S., where the FDA expects to make a decision by the second quarter.

    By Jan. 3, 2024
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    Permission granted by Novartis
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    Novartis deepens ties with Voyager via $100M gene therapy deal

    The deal builds on an existing alliance and includes tools Novartis will use in gene therapies for Huntington’s disease and spinal muscular atrophy.

    By Jan. 2, 2024
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    J&J bets bigger on MeiraGTx’s eye gene therapy

    Ahead of a Phase 3 readout that could come next year, the pharma is paying $130 million in upfront and near-term cash for rights to the retinitis pigmentosa treatment that it didn’t already own.

    By Dec. 21, 2023