A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants

NCTID	NCT06280378 (View at clinicaltrials.gov)
Description	This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major. (Show More)
Indication	Beta-Thalassemia, transfusion-dependent
Compound Name	KL003 (βA-T87Q-globin)
Sponsor	Kanglin Biotechnology (Hangzhou) Co., Ltd.
Funder Type	Industry
Status	Not yet recruiting
Enrollment Count	41

Target Gene/Variant	HBB
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	China

Has US IND	False
Recent Updates	Received NMPA approval on 1/3/24