Gene Therapy for Pyruvate Kinase Deficiency (PKD)

NCTID	NCT04105166 (View at clinicaltrials.gov)
Description	This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD). (Show More)
Indication	Pyruvate Kinase Deficiency
Compound Name	RP-L301
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	5

Target Gene/Variant	PKLR
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	PGK-coPRK-WPRE
Editor Type
Dose 1	Dose range: 2.3 x 10^6 - 6.5 x 10^6 transduced CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,Spain

Has US IND	True
Recent Updates	Phase I data presented at ASGCT 2024; Global Phase II planned late 2024/early 2025