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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

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Trial Profile

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

Status: Suspended
Phase of Trial: Phase II

Latest Information Update: 21 Apr 2026

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At a glance

  • Drugs RP L301 (Primary)
  • Indications Pyruvate kinase deficiency of red cells
  • Focus Registrational; Therapeutic Use
  • Sponsors Rocket Pharmaceuticals

Most Recent Events

  • 21 Apr 2026 Planned initiation date changed from 1 Aug 2024 to 1 Apr 2026.
  • 21 Apr 2026 Status changed from recruiting to suspended.
  • 07 Nov 2024 According to a Rocket Pharmaceuticals media release, Initiated global Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency.

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