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)
Development Status	Active
Indication	Cystinosis
Disease Ontology Term	DOID:1064
Compound Name	CTNS-RD-04 or CTNS-RD-04-LB
Compound Alias	AVR-RD-04
Sponsor	University of California, San Diego
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	6
Results Posted	Not Available

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	none
Dose 1	Min dose: 3E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	AVROBIO sold product to Novartis in May 2023, Novartis will sponsor any subsequent trials