A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]

NCTID	NCT05762510 (View at clinicaltrials.gov)
Description	This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced with LentiRed Lentivrial vector. (Show More)
Development Status	Active
Indication	Beta-Thalassemia Major
Disease Ontology Term	DOID:0080771
Compound Name	GMCN-508B
Sponsor	First Affiliated Hospital of Guangxi Medical University
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	5
Results Posted	Not Available

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	none
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

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