Trial Profile
A Phase 1/2/3 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:
Active, no longer recruiting
Phase of Trial:
Phase II/III
Latest Information Update: 03 Apr 2024
Price :
$35
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At a glance
- Drugs Exagamglogene autotemcel (Primary)
- Indications Beta-thalassaemia
- Focus Adverse reactions; First in man; Proof of concept; Registrational; Therapeutic Use
- Acronyms CLIMB TDT-111; CLIMB-111
- Sponsors CRISPR Therapeutics; Vertex Pharmaceuticals
- 01 Apr 2024 According to a Vertex Pharmaceuticals media release, New Drug Submission (NDS) for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for the treatment of patients aged 12 years and older with transfusion-dependent beta thalassemia (TDT).
- 13 Feb 2024 According to a Vertex Pharmaceuticals media release, company that the European Commission has granted conditional marketing authorization to CASGEVY (exagamglogene autotemcel) for the treatment of patients who are 12 years of age and older with severe sickle cell disease and transfusion-dependent beta thalassemia.
- 16 Jan 2024 According to a Vertex Pharmaceuticals media release, company announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY (exagamglogene autotemcel) for the treatment of transfusion-dependent beta thalassemia in patients 12 years and older.