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
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	BLA submission planned in Q1 2025