Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCTID	NCT05477563 (View at clinicaltrials.gov)
Description	This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001. (Show More)
Development Status	Approved
Indication	Beta-Thalassemia, Sickle Cell Disease
Disease Ontology Term	DOID:0081445; DOID:0080771
Compound Name	CASGEVY
Compound Alias	exagamglogene autotemcel, CTX001
Sponsor	Vertex Pharmaceuticals Incorporated
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	26 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	BCL11A
Therapy Type	Gene editing
Therapy Route	Ex-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Electroporation
Vector Type	RNP
Editor Type	Cas9 mRNA
Dose 1	Minimum dose: 3E6 CD34+ cells/kg
Dose 2	Maximum dose: 20E6 CD34+ cells/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	Saudi Arabia,United States,Italy,Germany

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	FDA approved 12/8/23, price/treatment $2.2M, expanded indication to beta thalassemia 1/16/24