Gene Therapy
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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 -
Mario Guti via Getty Images
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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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 -
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 -
Jacob Bell/BioPharma Dive
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 -
Sedat Suna via Getty Images
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 -
Scott Olson via Getty Images
Gene editingLilly to acquire Verve in $1B bet on gene editing for heart disease
The deal centers around a medicine that could “shift the treatment paradigm for cardiovascular disease,” Lilly said, but also follows a yearslong decline in Verve’s share price amid skepticism over the need for such a therapy.
By Kristin Jensen • June 17, 2025 -
Jacob Bell/BioPharma Dive
Second Duchenne patient dies after receiving Sarepta gene therapy
Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain patients while it evaluates new safety precautions.
By Ben Fidler • June 15, 2025 -
Courtesy of EQT Life Sciences
SpliceBio lands $135M for a new kind of eye gene therapy
The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.
By Gwendolyn Wu • June 11, 2025 -
Sarah Silbiger via Getty Images
FDA meeting gives window into gene therapy field’s angst
Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.
By Ned Pagliarulo • June 6, 2025 -
Jacob Bell/BioPharma Dive
FDA to use new review tool on Sarepta’s gene therapy work
The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene therapy applications it later brings to the regulator.
By Ben Fidler • June 4, 2025 -
Permission granted by Intellia Therapeutics
Gene editingIntellia filing spurs safety concerns over CRISPR drug
Shares lost a quarter of their value after the company disclosed, in a regulatory filing, signs of liver stress in a study participant given one of its gene editing treatments.
By Kristin Jensen • May 29, 2025 -
Sinhyu via Getty Images
Patient dies in trial of Rocket gene therapy for heart condition
The FDA has placed a clinical hold on the study while Rocket works with trial monitors and experts to investigate the cause of the patient’s death.
By Ned Pagliarulo • May 27, 2025 -
Permission granted by Muldoon family
A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases
A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months.
By Ben Fidler • May 15, 2025
