Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

NCTID	NCT06422351 (View at clinicaltrials.gov)
Description	This is an open-label Phase II trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD). (Show More)
Development Status	Active
Indication	Pyruvate Kinase Deficiency
Disease Ontology Term	DOID:0111077
Compound Name	RP-L301
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Recruitment Status	Not yet recruiting
Enrollment Count	10
Results Posted	Not Available

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	none
Dose 1	Dose range: 2.3E6 - 6.5E6 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
FDA Designations	Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
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