A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

NCTID	NCT06291961 (View at clinicaltrials.gov)
Description	The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia. (Show More)
Indication	Beta-Thalassemia Major
Compound Name	CS-101
Sponsor	CorrectSequence Therapeutics Co., Ltd
Funder Type	Industry
Status	Recruiting
Enrollment Count	8

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	none
Editor Type	transformer BE RNP
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	China

Has US IND	False
Recent Updates	Information on tBE system, IIT study given at ASGCT 2024