Stem Cell Gene Therapy for Cystinosis

NCTID	NCT03897361 (View at clinicaltrials.gov)
Description	This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST) (Show More)
Indication	Cystinosis
Compound Name	CTNS-RD-04 or CTNS-RD-04-LB
Sponsor	University of California, San Diego
Funder Type	Other
Status	Active not recruiting
Enrollment Count	6

Target Gene/Variant	CTNS
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
Dose 1	Min dose: 3 x 10^6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
Recent Updates	AVROBIO sold product to Novartis in May 2023