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A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Trial Profile

A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 28 Dec 2018

At a glance

  • Drugs CTX 001 (Primary)
  • Indications Sickle cell anaemia
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors CRISPR Therapeutics
  • Most Recent Events

    • 26 Nov 2018 Status changed from not yet recruiting to recruiting.
    • 21 Nov 2018 Planned number of patients changed to 45.
    • 20 Nov 2018 Planned initiation date changed from 31 Dec 2018 to 1 Nov 2018.
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