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
Sponsor	Vertex Pharmaceuticals Incorporated
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	26
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