Gene Therapy: Page
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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 -
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 -
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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 -
Courtesy of AbbVie
AbbVie stakes $335M on a startup’s RNAi drugs
ADARx Pharmaceuticals will work with AbbVie to make RNA interference medicines for a variety of disease areas, including neurology, immunology and cancer.
By Gwendolyn Wu • May 15, 2025 -
Mario Guti via Getty Images
Abeona sells speedy drug review voucher for $155M
The biotech has quickly turned a cell therapy approval into one of the more lucrative recent sales for a priority review voucher, which companies can use to fast track drug reviews.
By Ben Fidler • May 12, 2025 -
Jacob Bell/BioPharma Dive
Sarepta shares plummet as lower sales outlook adds to gloom over FDA’s new biologics hire
Already facing headwinds caused by a patient’s death and treatment delays, the biotech now may have to contend with a less friendly FDA.
By Kristin Jensen • May 7, 2025 -
Sarah Silbiger via Getty Images
Prasad’s FDA appointment pressures cell and gene therapy stocks
A vocal opponent of his predecessor Peter Marks, Vinay Prasad will now lead the office tasked with reviewing some genetic medicines, adding more uncertainty to an already struggling field of research.
By Ben Fidler • May 6, 2025 -
Mario Guti via Getty Images
Vinay Prasad, a physician and FDA critic, to lead agency center overseeing vaccines
Prasad has gained a reputation for questioning U.S. health policy and accelerated approvals in oncology, and has also opposed some vaccine mandates and the use of COVID shots in children.
By Ben Fidler , Jonathan Gardner • May 6, 2025 -
Sarah Silbiger via Getty Images
Abeona cell therapy approved by FDA for rare skin condition
Zevaskyn, which Abeona priced at $3.1 million, treats epidermolysis bullosa. It will compete with a gene therapy from Krystal Biotech that is off to a strong sales start.
By Ben Fidler • April 29, 2025 -
del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
Immune resetCaribou, in reversal, scraps autoimmune cell therapy and cuts staff
The CRISPR company is laying off about a third of its employees and stopping work on an experimental lupus treatment in a bid to focus resources on a pair of cancer cell therapies.
By Ben Fidler • April 25, 2025 -
Permission granted by Bluebird bio
Bluebird says Ayrmid missed deadline for rival takeover bid
The company’s board reiterated its recommendation to instead pursue an offer from SK Capital and Carlyle that’s worth about $29 million upfront.
By Gwendolyn Wu • April 16, 2025 -
Seth Babin/BioPharma Dive
Gene editingVerve’s second swing at gene editing for heart disease shows early promise
Wall Street analysts said the results for Verve’s therapy appeared competitive on efficacy, while avoiding any major safety concerns — at least so far.
By Ben Fidler • April 14, 2025 -
Permission granted by Qiagen
Sponsored by QIAGEN Digital InsightsThe critical role of evidence-based databases in pharmacogenomics: Ensuring accuracy, consistency and relevance
Discover how QIAGEN PGXI transforms pharmacogenomics with faster, more accurate insights!
By QIAGEN Digital Insights • April 14, 2025 -
Permission granted by Roche
Roche halts testing of Sarepta Duchenne gene therapy in Europe
The Swiss drugmaker, which holds European rights to Elevidys, suspended three trials while researchers investigate the death of a young man who died following treatment.
By Kristin Jensen • April 3, 2025 -
Permission granted by Landmark Bio
New genetic medicine CDMO acquires Landmark Bio
Artis BioSolutions joins a host of startups trying to improve development and manufacturing capacity for cutting-edge gene and cell therapies.
By Kristin Jensen • April 2, 2025 -
Sarah Silbiger via Getty Images
Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks
Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.
By Ben Fidler • March 31, 2025 -
Courtesy of Bluebird bio
Bluebird gets rival takeout offer from Ayrmid
The new bid surpasses an offer SK Capital and Carlyle Group made last month for the gene therapy developer, which Bluebird’s board still supports.
By Ben Fidler • March 28, 2025
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