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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: 17 Dec 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

    • 12 Dec 2024 According to an Editas Medicine media release, Company will work closely with the clinical trial sites, regulators, and other parties to determine the path forward for patients enrolled in the trial.
    • 22 Oct 2024 According to an Editas Medicine media release, the company is on-track to present additional clinical data from the EdiTHAL trial by year-end 2024, and continues to dose patients.
    • 07 Aug 2024 According to an Editas Medicine media release, status changed from recruiting to active, no longer recruiting.

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