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A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion Dependent β-Thalassaemia

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Trial Profile

A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion Dependent β-Thalassaemia

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

Latest Information Update: 22 Feb 2023

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At a glance

  • Drugs ET 01 (Primary) ; Busulfan
  • Indications Beta-thalassaemia
  • Focus Adverse reactions
  • Sponsors EdiGene Inc

    • Most Recent Events

    • 18 Nov 2022 According to an EdiGene media release, the company had completed the last patient dosing in this trial.
    • 09 May 2022 Status changed from recruiting to active, no longer recruiting.
    • 08 Sep 2021 According to an EdiGene media release, Jun Shi, and Xuedong Wu, are the co-lead Principal Investigators for this trial.

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