Rocket Pharmaceuticals Completes Enrollment for Danon Disease Gene Therapy Trial

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading biotechnology company focused on genetic therapies for rare disorders, has completed patient enrollment for its global Phase 2 clinical trial evaluating RP-A501, a gene therapy for Danon disease. The trial enrolled 12 male patients across the U.S. and Europe within three months, following a successful safety run-in phase.

Danon disease is a rare, inherited disorder that leads to heart failure, with few treatment options beyond heart transplantation. RP-A501, Rocket’s investigational therapy, aims to address the root cause of the disease by delivering functional LAMP2B genes directly to heart cells. If successful, this gene therapy could offer a single-dose, life-changing treatment for patients with this fatal disorder.

RP-A501 Phase 2 Trial Overview
The trial is a single-arm, multi-center study assessing the safety and efficacy of RP-A501 in male Danon disease patients. Key endpoints include improvements in LAMP2 protein expression, reductions in left ventricular mass, and changes in troponin levels. These markers could support accelerated approval of the therapy, with long-term follow-up for full regulatory approval. The study also includes a pediatric safety run-in and a pre-treatment observational period to better assess biomarker changes.

About RP-A501
RP-A501 is Rocket’s first-in-class gene therapy for Danon disease and the first gene therapy to show safety and efficacy in clinical studies for cardiovascular conditions. Administered via intravenous infusion, the therapy delivers the functional LAMP2B gene to the heart, aiming to restore normal cardiac function in patients with Danon disease. It has received advanced therapy designations from both the FDA and the European Medicines Agency.

About Danon Disease
Danon disease is an X-linked genetic disorder caused by mutations in the LAMP-2 gene, leading to heart failure, particularly in male patients, who often face death in adolescence or early adulthood. With limited treatment options, including heart transplants, there is a critical need for innovative therapies.

About Rocket Pharmaceuticals
Rocket Pharmaceuticals is a late-stage biotech company developing gene therapies for rare, life-threatening disorders. The company’s pipeline includes treatments for genetic diseases like Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency. Rocket’s cardiovascular portfolio includes its leading RP-A501 program for Danon disease and other heart-related genetic disorders.