Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

But this time, the unknown is hitting the industry in a different way from when Prasad was tapped to lead CBER less than ...

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 ...

Prasad, a longtime critic of the FDA’s leniency in drug approvals, joined the agency in May. WASHINGTON — The Food and Drug ...

Niemann-Pick Disease Type C (NPC) is a rare genetic disorder caused by mutations in the NPC1 or NPC2 genes, leading to harmful lipid accumulation in organs like the liver and brain. Symptoms, often ...

Two major health agencies are facing leadership reshuffles, with the resignations of the FDA’s Vinay Prasad and NICE’s Dr Sam Roberts.

Vinay Prasad’s ouster as the Food and Drug Administration’s top vaccine and gene therapy regulator came after frustration ...

Indian-origin scientist Vinay Prasad resigned as the FDA's vaccine and gene therapy chief in just three months after backlash ...

This week on "The Readout LOUD," STAT's biotech reporters parse what Vinay Prasad's ouster means for biotech and the FDA.

PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...

Tidmarsh will temporarily guide CBER’s regulatory activities while the FDA searches for a permanent department lead.