Clinical Trials:

Danon Disease

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RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart.

An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. and EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months.

Natural History Study​

Rocket is also sponsoring a multi-center, global, non-interventional natural history study designed to collect longitudinal prospective and retrospective clinical information on patients with Danon Disease. The overarching goal of the study is to characterize the natural history of Danon Disease by collecting de-identified information from male patients living with the condition. Initial trial sites for the natural history study in the U.S. and EU have been identified, and patient enrollment will begin pending approval from each center’s institutional review board.

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