Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

NCTID	NCT05417126 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease (Show More)
Indication	Stargardt Disease
Compound Name	Sonpiretigene isteparvovec (MCO-010)
Sponsor	Nanoscope Therapeutics Inc.
Funder Type	Industry
Status	Completed
Enrollment Count	6

Target Gene/Variant	MCO
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell	Bipolar cells
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	1.2 x 10^11 gc/eye
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
Recent Updates	Phase III Study expected to begin 2025