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RP-A501

Gene therapy (AAV vector) · Gene replacement · developed by Rocket Pharmaceuticals

Phase 1 Most advanced trial (none active)

RKTO Developer

1 Registered trials

What is RP-A501?

RP-A501 is a recombinant adeno-associated virus 9 (rAAV9) gene therapy delivering the human LAMP2B transgene. It is administered as a single intravenous infusion and is studied in Danon disease, a lysosomal storage disorder.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Gene therapy (AAV vector)
Mechanism: Gene replacement
Therapy area: Cardiovascular, Neurology
Developer: Rocket Pharmaceuticals Inc. (RKTO)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT03882437	Phase 1	Danon Disease	Unknown	RKTO

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Danon Disease

More from Rocket Pharmaceuticals

RP-L201 FDA approved Mozafancogene Autotemcel Phase 2 RP-L301 Phase 2 RP-L401 Phase 1

Sources

ClinicalTrials.gov NCT03882437

Updated Oct 3, 2026. Something wrong or missing? Report it on the RKTO page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z