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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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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: Recruiting
Phase of Trial: Phase II

Latest Information Update: 13 Nov 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

    • 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.

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