FDA Gene Therapy Concerns

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Sarepta Will Resume Gene Therapy Shipments

Sarepta shares rebound after shipments of gene therapy Elevidys resume in US

By Bhanvi Satija and Siddhi Mahatole (Reuters) -Sarepta Therapeutics shares snapped a three-day losing streak on Tuesday, as analysts said resumption of U.S. shipments for its muscular gene therapy soothes immediate concerns about liquidity and the therapy's withdrawal from the market.

Bloomberg on MSN · 19h

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 its gene therapy Elevidys again.

cleveland.com · 1d

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 muscular dystrophy. U.S. distribution has been paused amid safety concerns.

pharmaphorum5h

FDA gives the go-ahead to restart Elevidys shipments

In a statement, the Cambridge, Massachusetts-based company said that shipments of Elevidys (delandistrogene moxeparvovec) will restart while it continues dialogue with the FDA "on next steps in the ...

Fierce Biotech8d

Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb-girdle ...

14d

FDA Delays Approval For Ultragenyx's Gene Therapy, But Not A Dealbreaker, Says Analyst

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

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