Sarepta reports 2nd patient death
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Sarepta Therapeutics (NasdaqGS:SRPT) SRP-9003 Gene Therapy Earns FDA Platform Technology Designation
Sarepta Therapeutics (NasdaqGS:SRPT) recently announced that its SRP-9003 gene therapy received a platform technology designation from the FDA, and updated safety protocols for ELEVIDYS in the UK. Despite these developments,
Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other parents still push for the therapy.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy,
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.
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At an FDA roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
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