RP-A501

BIOLOGICAL2 trials

Sponsors

Rocket Pharmaceuticals Inc.

Conditions

Danon Disease

Phase 1

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Rocket Pharmaceuticals Inc.Danon Disease

Start: 2019-04-17End: 2025-03-24Updated: 2023-03-03

Phase 2

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

RecruitingNCT06092034

Rocket Pharmaceuticals Inc.Danon Disease

Start: 2023-09-05End: 2032-04-01Target: 14Updated: 2026-05-04

Related Papers

An International Longitudinal Natural History Study of Patients With Danon Disease: Unique Cardiac Trajectories Identified Based on Sex and Heart Failure Outcomes

Journal of the American Heart Association2025-03-213 citations

Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease

New England Journal of Medicine2024-11-1849 citations