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 Deficiency
Latest Information Update: 17 Feb 2025
At a glance
- Drugs RP L301 (Primary)
- Indications Pyruvate kinase deficiency of red cells
- Focus Registrational; Therapeutic Use
- Sponsors Rocket Pharmaceuticals
Most Recent Events
- 07 Nov 2024 According to a Rocket Pharmaceuticals media release, Initiated global Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency.
- 07 Nov 2024 Status changed to recruiting , according to a Rocket Pharmaceuticals media release
- 15 May 2024 Status changed from planning to not yet recruiting.