RP-L301

BIOLOGICAL2 trials

Sponsors

Rocket Pharmaceuticals Inc.

Conditions

Pyruvate Kinase Deficiency

Phase 1

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

CompletedNCT04105166

Rocket Pharmaceuticals Inc.Pyruvate Kinase Deficiency

Start: 2020-07-06End: 2025-06-09Updated: 2025-09-05

Phase 2

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

SuspendedNCT06422351

Rocket Pharmaceuticals Inc.Pyruvate Kinase Deficiency

Start: 2026-04-01End: 2029-01-01Target: 10Updated: 2026-04-16

Related Papers

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Blood2024-11-05

The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design

BMJ Open2023-03-015 citations

Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Blood2022-11-157 citations

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Blood2021-11-054 citations

Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells

Blood2020-11-05

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Blood2020-11-049 citations