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A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)

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Trial Profile

A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)

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

Latest Information Update: 14 Aug 2024

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At a glance

  • Drugs Renizgamglogene autogedtemcel (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms EDITHAL
  • Sponsors Editas Medicine

    • Most Recent Events

    • 07 Aug 2024 According to an Editas Medicine media release, status changed from recruiting to active, no longer recruiting.
    • 07 Aug 2024 According to an Editas Medicine media release, the company has completed enrollment of the adult cohort in this trial and continues to dose patients.
    • 14 Jun 2024 Results published in the Editas Medicine Media Release

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