Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

NCTID	NCT04945772 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010). (Show More)
Development Status	Active
Indication	Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration
Disease Ontology Term	DOID:10584
Compound Name	MCO-010
Compound Alias	Sonpiretigene isteparvovec
Sponsor	Nanoscope Therapeutics Inc.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	27 (ACTUAL)
Results Posted	Not Available

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	none
Dose 1	0.9E11 gc/eye
Dose 2	1.2E11 gc/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	Puerto Rico,United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	First modules of BLA submitted in July 2025, full submission anticipated in early 2026