RP-A501 is Rocket Pharmaceuticals’ first adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease.
Danon disease is a life-threatening heart-failure syndrome that develops during childhood and adolescence. The rare neuromuscular and cardiovascular disease is associated with severe cardiomyopathy, skeletal myopathies, and mild cognitive impairment.
Between 15,000 and 30,000 patients in the U.S. and Europe are estimated to have Danon disease. It is often fatal by the time patients reach their twenties or thirties as a result of progressive heart failure unless it is treated with a cardiac transplantation. However, this is not curative.
Rocket Pharmaceuticals plans to publish a comprehensive literature review of Danon cases and conduct a retrospective chart review in conjunction with the phase 1 clinical trial, which is expected to begin after June 2019.
Source: Rocket Pharmaceuticals, January 22, 2019