Clinical Study of BRL-101 in Severe SCD

NCTID	NCT06300723 (View at clinicaltrials.gov)
Description	This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101). (Show More)
Development Status	Active
Indication	Sickle Cell Disease, Beta Thalassemia
Disease Ontology Term	DOID:0081445; DOID:0080771
Compound Name	BRL-101
Sponsor	Bioray Laboratories
Funder Type	Industry
Recruitment Status	Enrolling by invitation
Enrollment Count	3 (ESTIMATED)
Results Posted	Not Available

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
Editor Type	Cas9 RNP
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China