Story perspectives
FDA Lifts Hold on Rocket's Gene Therapy Trial, Shares Surge
8/22/2025
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Story summary
- The FDA has lifted the clinical hold on Rocket Pharmaceuticals' gene therapy trial for Danon disease.
- The trial was paused after a patient's death related to treatment complications.
- Rocket will implement a lower dose and adjust pre-treatment regimens for improved safety.
- The genetic disorder affects fewer than 1,000 individuals in the U.S.
- Following the FDA's decision, Rocket's shares increased by over 30%.
