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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

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 05 Dec 2019

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At a glance

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

    • 19 Nov 2019 According to a CRISPR Therapeutics media release, several additional patients have been enrolled in the study. All patients will be followed for approximately two years.
    • 19 Nov 2019 Interim results (n=1) are presented in a CRISPR Therapeutics media release.
    • 28 Oct 2019 According to a CRISPR Therapeutics media release, enrollment is.ongoing at six clinical trial sites in the U.S., Canada and Europe
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