Rocket Pharmaceuticals
CompanyUnited States
Rocket Pharmaceuticals develops 2 assets, the furthest in phase 2 (RP-A501 in Danon disease). Mostly gene therapy acting on autophagy, proteostasis and LAMP2; based in the United States.
- 2
- Assets
- Furthest stage
- Phase 2
- 1
- Trial
- 0
- Signals
Pipeline
2Delivers functional LAMP2B to restore lysosomal function and autophagy in Danon disease.
Phase 2, Gene therapy, Autophagy, Proteostasis, Danon disease
AAVrh.74 gene therapy delivering functional BAG3 transgene to rescue chaperone-assisted autophagy and proteostasis
Phase 1, Gene therapy, Autophagy, BAG3, dilated cardiomyopathy
Full company profiles: pipeline, trials, funding and people.
In the full profile
- Evidence behind the mechanisms4
- Trials1
- Similar companies6