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BioWorld - Wednesday, February 25, 2026
Home » Topics » Drugs » Gene therapy

Gene therapy
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Silhouette of child and brain

Rett bet set includes Neurogene, Taysha gene therapies

July 24, 2025
By Randy Osborne
No Comments
Neurogene Inc.’s disclosure June 30 of its registrational trial design in Rett syndrome (RS) added spice to the space, where Taysha Gene Therapies Inc. is another closely watched player. The New York-based firm said Embolden will test NGN-401, a gene therapy for the treatment of females age 3 and older with RS. U.S. regulators have signed off regarding key aspects of Embolden’s structure.
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Eye and DNA illustration
Ocular

Partnership to advance Opus Genetics’ RDH12 gene therapy

July 24, 2025
No Comments
Opus Genetics Inc. has entered a strategic partnership with the Global RDH12 Alliance to advance Opus’ gene therapy program for patients with vision loss due to retinol dehydrogenase 12 (RDH12) gene mutations.
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Sarepta yields to FDA request, pauses Elevidys shipments

July 22, 2025
No Comments
In an about-face, Sarepta Therapeutics Inc. said it would “voluntarily and temporarily” pause all shipments of Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec) in the U.S. The move comes only a few days after the firm publicly declined a U.S. FDA request to halt shipping of the therapy in the wake of a third patient death, this one linked to a gene therapy using the same adeno-associated virus (AAV) vector as Elevidys.
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Sarepta confronts FDA, won’t stop Elevidys shipments

July 21, 2025
By Lee Landenberger
No Comments
Sarepta Therapeutics Inc. is declining a U.S. FDA request to voluntarily halt shipping its gene therapy, Elevidys (delandistrogene moxeparvovec), in the U.S. On July 18, Sarepta said had it received “an informal request” from the FDA to stop the shipments following a third patient’s death, tied to the gene therapy SRP-9004, which uses the same vector as Elevidys.
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Sarepta CEO: Third same-vector gene therapy death ‘not material’

July 18, 2025
By Randy Osborne
No Comments
Sarepta Therapeutics Inc. made known a third gene therapy death, this time with SRP-9004 for limb-girdle muscular dystrophy. The patient was a late-stage, non-ambulatory 51-year-old man participating in the phase I Discovery trial, who expired about a month ago of acute liver failure, as did the two previous subjects who passed away after they were treated with Elevidys (delandistrogene moxeparvovec), Cambridge, Mass.-based Sarepta’s gene product for Duchenne muscular dystrophy.
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Sarepta cutbacks propel the stock forward

July 17, 2025
By Lee Landenberger
No Comments
Carving out $400 million in annual cost savings is going over well with Sarepta Therapeutics Inc.’s investors, as the company’s stock got a 19.6% boost on July 17. Its big seller, the gene therapy Elevidys (delandistrogene moxeparvovec), continued its revenue numbers decline, so Sarepta chopped operating expenses by letting about 500 employees go in a 36% cutback and pared its development path.
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Brain and DNA
Neurology/psychiatric

Voyager Therapeutics adds new gene therapy to Alzheimer’s program

July 17, 2025
No Comments
Voyager Therapeutics Inc. has expanded its Alzheimer’s disease (AD) pipeline with the addition of a wholly owned program that modulates the expression of apolipoprotein E (APOE). Using a proprietary intravenous-delivered, blood-brain barrier (BBB)-penetrant Tracer capsid, the product delivers a bifunctional payload.
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CMS’ gene therapy access model goes live, with 33 states on board

July 16, 2025
By Jennifer Boggs
No Comments
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
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Optogenetics illustration
Ocular

Aavantgarde Bio cleared to begin clinical study of Stargardt disease gene therapy in US

July 16, 2025
No Comments
The U.S. FDA has cleared Aavantgarde Bio Srl’s IND application for AAVB-039, the company’s gene therapy program for Stargardt disease, the most common inherited form of macular degeneration.
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Concept art for adeno-associated viral-based gene therapy.

JCR’s third deal with Alexion gives rights to AAV capsids

July 15, 2025
By Marian (YoonJee) Chu
No Comments
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
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