Gene Therapy

  • Intellia Therapeutics
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    Gene editing

    FDA places formal hold on two Intellia CRISPR trials

    Two days after Intellia voluntarily paused dosing due to safety concerns, the regulator stepped in with an official hold that makes a longer halt more likely. 

    By • Oct. 30, 2025
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    BioMarin, following sluggish sales, to offload hemophilia gene therapy

    The company intends to divest Roctavian and “remove it from our portfolio,” CEO Alexander Hardy said, after nearly three years of slow uptake for a medicine once viewed as a future blockbuster.

    By • Oct. 27, 2025
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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 Dive staff
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    Gene editing

    Intellia pauses two CRISPR drug studies after safety scare

    A study volunteer receiving Intellia’s transthyretin amyloidosis treatment was hospitalized due to severe signs of liver stress, prompting the company to pause enrollment and dosing while evaluating a new safety protocol. 

    By • Oct. 27, 2025
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    Adverum, hammered by gene therapy downturn, sells to Lilly in unusual deal

    Lilly’s upfront offer for Adverum, the developer of a gene therapy for age-related macular degeneration, is less than the company’s previous closing share price.

    By • Oct. 24, 2025
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    Regeneron, with ‘game-changing’ new data, to seek approval of hearing loss gene therapy

    Study results published in The New England Journal of Medicine show the therapy significantly improved hearing in nearly a dozen children with a rare, genetic form of deafness.

    By • Oct. 12, 2025
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    Lexeo says FDA open to speedier approval of rare disease gene therapy

    The agency will consider a submission that includes pooled data from ongoing studies, a decision analysts viewed as a notable, additional sign of regulatory flexibility for gene therapies.

    By • Oct. 7, 2025
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    Galapagos to decide on fate of cell therapy business ‘within weeks’

    The company has received a “limited number” of non-binding proposals, mostly from groups of financial investors, for a business that was once its primary focus.

    By Kristin Jensen • Oct. 2, 2025
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    Capricor readies revised FDA pitch for Duchenne cell therapy

    Two months after its treatment for Duchenne-related cardiomyopathy was rejected, the company aligned with the agency on a framework that could support a clearance if upcoming study results are positive.

    By Kristin Jensen • Sept. 25, 2025
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    UniQure to seek approval of Huntington’s gene therapy after trial win

    New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have “massive effects on patients’ lives,” an investigator said.

    By • Sept. 24, 2025
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    Sponsored by Cencora

    Setting cell and gene therapies up for success with a smarter CMC strategy

    Being proactive — especially when it comes to testing, process development and preparing for commercialization — can greatly decrease the risk that your CGT will receive a CRL.

    Sept. 22, 2025
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    Child dies in Capsida study of rare disease gene therapy

    Capsida is investigating the root cause of the death, which occurred in the first patient treated in a trial of the company’s therapy for a rare neurodevelopmental disorder.

    By Ned Pagliarulo • Updated Sept. 11, 2025
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    FDA outlines new review pathway for drugs treating ultra-rare diseases

    The agency's two main review offices will work together to flexibly evaluate medicines for serious conditions that affect fewer than 1,000 people in the U.S.

    By Ned Pagliarulo • Sept. 4, 2025
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    Sponsored by Danaher

    Turning aspirational innovations into tangible impacts

    Making tomorrow’s breakthroughs a reality for the patients who need therapies today.

    Sept. 2, 2025
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    Sarepta pushes off debt payments in bid to regain financial footing

    A refinancing deal will delay the maturity of $700 million in debt until 2030, allowing the company to “fully fund” its pipeline and meet its near-term financial obligations, CEO Doug Ingram said. 

    By Kristin Jensen • Aug. 21, 2025
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    Rocket can resume gene therapy trial after FDA lifts hold

    The company plans to continue with a lower dose and a change in the pre-treatment regimen after a trial participant died earlier this year.

    By Kristin Jensen • Aug. 20, 2025
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    Sarepta sells Arrowhead shares as partnership, debt payments loom

    Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as “additional breathing room” ahead of a debt maturity in 2027.

    By Kristin Jensen • Aug. 14, 2025
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    As gene therapy sales sputter, one biotech aims to defy the odds

    With a strong launch underway for a bladder cancer gene therapy, Ferring is finding the kind of commercial success that’s eluded many of its peers.

    By Meagan Parrish • Aug. 11, 2025
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    Iovance cuts staff amid slow sales start for ‘TIL’ cell therapy

    In its latest earnings report, Iovance confirmed plans to reduce its workforce by 19% in a bid to save $100 million in yearly costs.

    By • Updated Aug. 8, 2025
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    Adaptimmune, seeking to stay afloat, sells off cell therapies

    A deal with US WorldMeds includes the FDA-approved TCR therapy Tecelra and two clinical-stage cancer medicines.

    By • July 28, 2025
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    Sarepta woes mount as Duchenne gene therapy knocked back in Europe

    The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could further jeopardize Sarepta’s financial future.

    By • Updated July 25, 2025
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    Roche stops shipping Sarepta gene therapy in some countries outside US

    The Swiss company, which markets Elevidys abroad, said it believes the benefit-risk balance to treatment remains positive in Duchenne patients who can still walk, however.

    By Ned Pagliarulo • July 23, 2025
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    Startup launches

    Dispatch emerges with $216M and plans for a ‘universal’ solid tumor therapy

    The startup is using elements of gene and cell therapy to create medicines that could be useful against an array of tough-to-treat solid tumors.

    By • July 23, 2025
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    Sarepta stops Elevidys shipments after standoff with FDA

    Company CEO Doug Ingram said the pause was necessary for Sarepta to maintain a "productive and positive working relationship" with the regulator.

    By Ned Pagliarulo • July 21, 2025
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    5 questions on Sarepta, the FDA and a Duchenne gene therapy crisis

    While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency could still carry major consequences for the Duchenne community.

    By , Ned Pagliarulo • Updated July 22, 2025
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    Alamy
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    FDA asks Sarepta to stop shipping Duchenne gene therapy

    The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still walk. A pause on shipments to older, non-ambulatory patients remains in place.

    By Ned Pagliarulo • Updated July 19, 2025