Gene Therapy

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    Hemophilia gene therapies are struggling on the market, even as innovation soars

    The business case for hemophilia gene therapy still isn’t adding up due to persistent market barriers.

    By Kelly Bilodeau • Dec. 5, 2025
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    Courtesy of UniQure
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    UniQure slides further on outlook for Huntington’s gene therapy

    Finalized minutes from a late-October meeting with the FDA confirm the agency doesn't see the data collected so far as enough to support an approval filing.

    By • Dec. 4, 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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    Courtesy of Regeneron Pharmaceuticals
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    Gene editing

    Regeneron inks gene editing deal with startup Tessera

    The big biotech, which has made genetic medicine a focus in recent years, is paying Tessera $150 million for rights to a potential one-and-done treatment for alpha-1 antitrypsin deficiency.

    By • Dec. 1, 2025
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    Novartis wins approval to use SMA gene therapy in older patients

    Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor function.

    By • Nov. 25, 2025
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    Sponsored by Pearson

    APOE4 and CRISPR are reframing Alzheimer’s Risk

    New research on APOE4 and CRISPR brings hope for slowing Alzheimer’s progression.

    Nov. 24, 2025
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    Sponsored by MilliporeSigma

    Securing quality in every dose: 5 essential capabilities for CGT developers to look for in a fill-finish CDMO

    With their promise to transform disease trajectories, cell and gene therapies (CGTs) are bringing new hope to patients with an ever-expanding array of high-burden and chronic conditions as well as rare genetic disorders.

    Nov. 17, 2025
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    Gene editing

    Korro to cut staff, shift strategy as RNA editing drug misses mark in early testing

    Korro’s lead program for alpha-1 antitrypsin deficiency wasn’t as potent as anticipated, leading the company to change course and lay off a third of its workforce.

    By Kristin Jensen • Nov. 13, 2025
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    Gene editing

    FDA unveils new regulatory roadmap for bespoke drug therapies

    The “plausible mechanism” pathway, outlined by Martin Makary and Vinay Prasad, is designed to help accelerate treatments custom-made for individuals with rare and serious diseases.

    By • Updated Nov. 13, 2025
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    Lilly picks up an eye gene therapy in deal with MeiraGTx

    The alliance extends a deal streak in genetic medicine for Lilly and hands the company rights to an experimental treatment for an ultra-rare form of childhood blindness.

    By • Nov. 10, 2025
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    Caribou results suggest renewed promise for ‘off-the-shelf’ cancer cell therapy

    While early, the data support Caribou’s plan to boost its therapies’ effectiveness by using donor cells that are carefully matched to a patient’s immune system. 

    By • Nov. 3, 2025
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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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    Courtesy of Intellia Therapeutics
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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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    Courtesy of UniQure
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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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    Permission granted by Hussey Photography, Tom Hussey
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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