FDA Gene Therapy Concerns

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FDA, Sarepta and Gene Therapy

FDA’s Prasad Exits After Sarepta Gene Therapy Controversy

Vinay Prasad, a top regulator at the US Food and Drug Administration, has left the agency after a controversy over his handling of Sarepta Therapeutics Inc.’s gene therapy.

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FDA opens probe into mysterious death linked to gene therapy

Fierce Pharma · 1d

FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients

· 18h

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

Sarepta Therapeutics (SRPT.O) shares surged more than 30% before the bell on Tuesday, as analysts said the resumption of U.S. shipments for its muscular gene therapy partially removes financial headw...

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FDA Probes Death Of 8-Year-Old Who Received Sarepta's Elevidys

· 1d

Sarepta resumes shipping of gene therapy Elevidys to patients who can walk

Just The News3h

FDA allows distribution of muscular dystrophy drug again after public criticism

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy raised questions about a key FDA official who broke with Trump on regulation.

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 muscular dystrophy (LGMD) trials, while also revoking the biopharma’s gene therapy platform technology designation.

pharmaphorum18h

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 safety labelling process and risk-mitigation approach for non-ambulatory patients."

9don MSN

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.

5don MSN

Sarepta drops as report suggests FDA needs new trials for Elevidys

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its gene therapy, Elevidys. Read more here.

PharmExec6d

Recapping the Sarepta Saga—And Implications for Industry

A timeline of key developments surrounding Sarepta Therapeutics' Duchenne muscular dystrophy drug Elevidys, and the potential future impacts for gene therapy regulation and market access.