FDA asks Sarepta to stop shipping gene therapy
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Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies.
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.
Sarepta Therapeutics announced it has laid off more than one-third of its workforce, a drastic cost-cutting move following the deaths of two teenagers administered its gene therapy for Duchenne muscular dystrophy.
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News-Medical.Net on MSNNew cellular entry pathway improves gene therapy outcomesScientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy,
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new FDA leadership’s much-touted support for cell and gene therapies against rare diseases.