Rocket Pharmaceuticals reported positive long-term follow-up data for RP-A501, its gene therapy for Danon disease, a rare genetic heart disorder. The results from the company’s pivotal Phase 1 study were presented following an investor webinar.
After approximately three to seven years of follow-up, left ventricular mass index—a measure of heart muscle thickness—was below baseline in five of six patients. Four patients maintained a reduction of at least 10%.
The company also outlined its plan for the ongoing FDA-aligned pivotal Phase 2 trial. Initial safety data were encouraging for the first three patients treated under a modified protocol.