Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease

NCTID	NCT06399107 (View at clinicaltrials.gov)
Description	This study is an open-label, non-randomized, single-dose Phase 1/2 trial involving around 85 adult and pediatric participants aged between 2 and 50 years with sickle cell disease (SCD). It aims to assess the effectiveness of hematopoietic stem cell transplantation (HSCT) using BAH243 for SCD. (Show More)
Indication	Sickle Cell Disease
Compound Name	BAH243
Sponsor	Essen Biotech
Funder Type	Other
Status	Recruiting
Enrollment Count	85

Target Gene/Variant	HBB
Therapy Type	Gene transfer
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	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
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