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RP-L401

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

Phase 1 Most advanced trial (none active)

RKTO Developer

1 Registered trials

What is RP-L401?

RP-L401 is a lentiviral gene therapy that delivers the TCIRG1 gene into CD34+ hematopoietic stem cells ex vivo to restore function in infantile malignant osteopetrosis, a genetic disorder affecting bone resorption.

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

At a glance

Drug type: Gene therapy (Lentiviral vector)
Mechanism: Gene replacement
Therapy area: Hematology
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
NCT04525352	Phase 1	Infantile Malignant Osteopetrosis	Terminated	RKTO

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

Conditions studied

Infantile Malignant Osteopetrosis

More from Rocket Pharmaceuticals

RP-L201 FDA approved Mozafancogene Autotemcel Phase 2 RP-A501 Phase 1 RP-L301 Phase 2

Sources

ClinicalTrials.gov NCT04525352

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