A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major

NCTID	NCT06465550 (View at clinicaltrials.gov)
Description	This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia. (Show More)
Development Status	Active
Indication	Beta-Thalassemia Major
Disease Ontology Term	DOID:0080771
Compound Name	BD211
Compound Description	βA-T87Q-globin
Sponsor	Shanghai BDgene Co., Ltd.
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	9
Results Posted	Not Available

Target Gene/Variant	HBB
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	Transduced CD34+ cells (> 5E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	China