Gene Therapy
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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 Ben Fidler • Oct. 7, 2025 -
Courtesy of Galapagos
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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TrendlineGene 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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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 -
Courtesy of UniQure
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 Jonathan Gardner • Sept. 24, 2025 -
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Sponsored by CencoraSetting 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 -
Andrew Harnik via Getty Images
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 DanaherTurning aspirational innovations into tangible impacts
Making tomorrow’s breakthroughs a reality for the patients who need therapies today.
Sept. 2, 2025 -
Jacob Bell/BioPharma Dive
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 -
Jacob Bell/BioPharma Dive
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 -
Courtesy of Iovance Biotherapeutics
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 Ben Fidler • 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 Jonathan Gardner • 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 Ben Fidler • 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 -
Permission granted by Dispatch Bio
Startup launchesDispatch 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 Ben Fidler • July 23, 2025 -
Jacob Bell/BioPharma Dive
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 -
Courtesy of Sarepta
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 Ben Fidler , Ned Pagliarulo • Updated July 22, 2025 -
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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 -
Jacob Bell/BioPharma Dive
Sarepta reports patient death in limb-girdle trial, compounding concerns on gene therapy risks
The death of a 51-year-old man in the study follows two other deaths of Duchenne patients treated with Sarepta’s marketed gene therapy Elevidys.
By Ben Fidler • Updated July 18, 2025 -
Jacob Bell/BioPharma Dive
Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks
On a conference call, CEO Doug Ingram said the decision to cut 36% of the company's staff and halt several drug programs was essential to ensuring Sarepta's "long-term viability."
By Ben Fidler • Updated July 16, 2025 -
Ned Pagliarulo/BioPharma Dive
33 states pick up CMS program to pay for sickle cell gene therapies
States representing about 84% of Medicaid beneficiaries are participating in an initiative that enables the U.S. government to negotiate pay-for-performance deals with the manufacturers of products like Casgevy and Lyfgenia.
By Ben Fidler • July 16, 2025 -
Sarah Silbiger via Getty Images
FDA turns back Capricor’s Duchenne cell therapy
The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the agency during deramiocel’s review.
By Ben Fidler • July 11, 2025 -
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Trump administrationGene therapy faces fresh uncertainty as two more top FDA officials depart
The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.
By Ben Fidler • June 20, 2025
