Rocket Pharmaceuticals to Present Updated Data from the Phase 1 Clinical Trial of RP-A501 for Danon Disease at the American Heart Association’s 2024 Late-Breaking Science Sessions

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CRANBURY, N.J., November 12, 2024--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the company will be presenting new data on the results of the RP-A501 Phase 1 trial to treat patients with Danon disease at the American Heart Association’s 2024 Late-Breaking Science sessions being held from November 16-18, 2024 in Chicago, Illinois.

Details for the presentation are as follows:

Title: Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy
Session: Amyloid, Hypertrophic and Danon Cardiomyopathies: Targeted Therapies and Specific Populations
Presenter: Joseph Rossano, M.D., M.S., FAAP, FACC, Co-Director of the Cardiac Center and Chief of the Division of Cardiology at Children's Hospital of Philadelphia
Date and Time: Monday, November 18, 2024, from 9:45-9:57 a.m. CT

Rocket plans to host an investor webinar on November 18, 2024, at 12:00 p.m. ET. To join the investor webinar, please register at https://www.webcaster4.com/Webcast/Page/3046/51498. Webinar details will be posted on the Events and Presentations page of the Rocket website at www.RocketPharma.com.

About RP-A501

RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. Danon disease is caused by mutations in the LAMP2 gene.

RP-A501 consists of a recombinant adeno-associated virus serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene (AAV9.LAMP2B) which, when inserted into cardiac cells (cardiomyocytes) harboring mutations in the endogenous LAMP2 gene, has the potential to substantially restore cardiac function by addressing the root cause of Danon disease. RP-A501 is a single dose treatment administered as an intravenous infusion. In preclinical and clinical studies, AAV9.LAMP2B has been generally well tolerated and shown to target cardiomyocytes and deliver the functional LAMP2B gene to heart tissue, which ultimately leads to improvement in cardiac structure and overall clinical function in patients.

About Danon Disease

Danon disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe.