FDA lifts clinical hold on Rocket's gene therapy for heart failure after 3-month delay

Kyle Blankenship

New Jersey’s Rocket Pharmaceuticals hit a major roadblock for its AAV-delivered gene therapy for heart failure in May after the FDA came asking for more details on target patients. Three months down the road, Rocket is clear for liftoff once again.

The FDA on Monday lifted its clinical hold on Rocket’s Phase I trial for gene therapy candidate RP-A501 for Danon disease, an ultra-rare genetic disorder that can lead to heart failure in children and young adults, the company said in a release.

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FDA lifts clinical hold on Rocket's gene therapy for heart failure after 3-month delay

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