A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Summary

This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.

Timeline

Start

2023-09-05

Primary completion

2028-04

Completion

2032-04

Drugs

Indications