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RP-L301

Cell therapy · Gene replacement · developed by Rocket Pharmaceuticals

Phase 2 Most advanced trial (none active)

RKTO Developer

1 Registered trials

What is RP-L301?

RP-L301 is an autologous cell therapy consisting of genetically modified CD34+ hematopoietic stem cells containing a corrected PKLR gene. It is being studied for pyruvate kinase deficiency, a genetic disorder affecting red blood cell metabolism.

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

At a glance

Also known as: Intervention/Treatment
Drug type: Cell therapy
Mechanism: Gene replacement
Therapy area: Hematology
Developer: Rocket Pharmaceuticals Inc. (RKTO)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 2

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT06422351	Phase 2	Pyruvate Kinase Deficiency	Suspended	RKTO

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

Conditions studied

Pyruvate Kinase Deficiency

More from Rocket Pharmaceuticals

RP-L201 FDA approved Mozafancogene Autotemcel Phase 2 RP-A501 Phase 1 RP-L401 Phase 1

Sources

ClinicalTrials.gov NCT06422351

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