Rocket Pharma's Shares Soar After US FDA Lets Gene Therapy Trial Resume

US FDA Lifts Clinical Hold on Rocket Pharmaceuticals' Gene Therapy ...

Rocket Pharma's shares soar after US FDA lets gene therapy trial resume

The drugmaker's shares were up 19% in midday trading after soaring more than 30% earlier Wednesday morning.

Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease

CRANBURY, N.J.--(BUSINESS WIRE)--Aug 20, 2025-

Rocket can resume gene therapy trial after FDA lifts hold

The company plans to continue with a lower dose and a change in the pre-treatment regimen after a trial participant died earlier this year.

Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on ...

FDA lifts hold on Rocket’s pivotal study for its gene therapy, which is now a lower dose

The FDA gave Rocket Pharmaceuticals the green light to resume a clinical trial of its gene therapy for Danon disease, a rare genetic disorder that affects multiple organs, including the heart. Rocket is