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Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD

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Trial Profile

Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD

Status: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 20 Sep 2024

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At a glance

  • Drugs RP L301 (Primary)
  • Indications Pyruvate kinase deficiency of red cells
  • Focus Adverse reactions; First in man; Pharmacodynamics
  • Sponsors Rocket Pharmaceuticals

Most Recent Events

  • 10 May 2024 Results published in the Rocket Pharmaceuticals Media Release
  • 10 May 2024 According to a Rocket Pharmaceuticals media release, data from this trial presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting.
  • 06 May 2024 According to a Rocket Pharmaceuticals media release, data from this trial to be presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting.

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