Gene Therapy
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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 Delilah Alvarado • Oct. 30, 2025 -
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 Ben Fidler • Oct. 27, 2025 -
Explore the Trendline➔
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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 -
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 Jonathan Gardner • Oct. 27, 2025 -
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 Jacob Bell • Oct. 24, 2025 -
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 Jacob Bell • Oct. 12, 2025 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 -
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 Ben Fidler • July 23, 2025 -
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 -
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 -
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