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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: Not yet recruiting
Phase of Trial: Phase II

Latest Information Update: 24 May 2024

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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

    • 15 May 2024 Status changed from planning to not yet recruiting.
    • 06 May 2024 According to a Rocket Pharmaceuticals media release, company is working towards initiation of the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD) in 2024.
    • 06 Nov 2023 According to a Rocket Pharmaceuticals media release, the company has reached the on the study design of this trial and this trial will support accelerated approval.

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