Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

NCTID	NCT04443907 (View at clinicaltrials.gov)
Description	This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease. (Show More)
Development Status	Inactive
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	OTQ923
Sponsor	Novartis Pharmaceuticals
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	4
Results Posted	Not Available

Target Gene/Variant	HBG1/HBG2
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	Minimum dose: 3E6 CD34+ cells/kg
Dose 2	Dose range: 2.8-5.99E6 CD34+ cells/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Product was developed in collaboration with Intellia, Novartis opted to discontinue development in February 2023