07:40 AM EDT, 08/20/2025 (MT Newswires) -- Rocket Pharmaceuticals ( RCKT ) said Wednesday that the US Food and Drug Administration has lifted a clinical hold on its mid-stage trial of RP-A501 to treat Danon disease.

The biotech company said the regulator has authorized it to resume the study with a recalibrated dose of RP-A501 in three patients. The company also said it will work with FDA investigators to implement an immunomodulatory regimen that more closely reflects what was administered in the early-stage pediatric cohort.

Shares of Rocket Pharmaceuticals ( RCKT ) were up more than 16% in recent Wednesday premarket activity.