Product
RP-L301
2 clinical trials
1 indication
Indication
Pyruvate Kinase DeficiencyClinical trial
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKDStatus: Active (not recruiting), Estimated PCD: 2025-05-01
Clinical trial
A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Subjects With Pyruvate Kinase DeficiencyStatus: Not yet recruiting, Estimated PCD: 2026-11-01