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A Phase 1/2 Study of 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 Thalassemia

Trial Profile

A Phase 1/2 Study of 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 Thalassemia

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 16 Nov 2018

At a glance

  • Drugs CTX 001 (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors CRISPR Therapeutics
  • Most Recent Events

    • 07 Nov 2018 According to a CRISPR Therapeutics media release, first patient has been enrolled.The first two patients in the trial will be dosed sequentially and, pending data from these initial two patients, the trial will be open for broader concurrent enrollment.
    • 05 Oct 2018 Planned number of patients changed from 30 to 45.
    • 19 Sep 2018 Planned number of patients changed from 45 to 30.
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