News

Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
Sarepta fails to win EU backing for muscle disorder gene therapy
Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...
FDA Says Sarepta Can Resume Gene Therapy in Some Patients
In a major win for Sarepta Therapeutics Inc., US regulators are recommending that patients who can walk be allowed to take ...
FDA opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
Medscape5d
EMA Says No to Duchenne Gene Therapy Elevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
493 employees laid off at biopharmaceutical company with operations in Andover
A biopharmaceutical research company with ties in Andover has laid off a third of its workforce as one of the industry leaders in gene therapy goes through a “strategic restructuring.” Sarepta ...
Chemistry World1d
Sarepta gene therapy deaths highlight tragic rare disease dilemma
Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...
FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy
The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...
Fierce Pharma9h
Vinay Prasad's exit signals FDA's probable slip toward more permissive regulation of cell and gene therapy: analyst
But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...
What’s behind Sarepta’s gene therapy deaths?
Three patient deaths linked to gene therapies developed by Sarepta Therapeutics (SRPT) have drawn FDA scrutiny on the viral ...
The American Journal of Managed Care8h
FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients
Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
GEN - Genetic Engineering and Biotechnology News2d
Sarepta to Resume Shipping DMD Gene Therapy to Ambulant Patients
Sarepta Therapeutics said it will resume shipments of its gene therapy Elevidys® (delandistrogene moxeparvovec-rokl) “imminently” to ambulant patients with Duchenne muscular dystrophy (DMD), ending a ...