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)
Indication	Retinitis Pigmentosa
Compound Name	Sonpiretigene isteparvovec (MCO-010)
Sponsor	Nanoscope Therapeutics Inc.
Funder Type	Industry
Status	Completed
Enrollment Count	27

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	0.9 x 10^11 gc/eye
Dose 2	1.2 x 10^11 gc/eye
Dose 3
Dose 4
Dose 5

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

Has US IND	True
Recent Updates	Anticipated BLA filing Q1 2025

PMID:34418301
https://abstracts.euretina.org/2024/ca24-2740-4044/r/rec9aVeQGG85rFV4N
Ho A. Longitudinal BCVA analysis of low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: 12-month results from a Phase 2b/3 randomized, sham-controlled, patient- and assessor-masked clinical trial (RESTORE). Presented at: Association for Research in Vision and Ophthalmology meeting
May 5-9, 2024
Seattle