Basic Information


GTO ID GTC2725
Trial ID NCT04945772
Disease Dystrophies Primarily Involving the Retinal Pigment Epithelium | Retinal Degeneration | Retinal Disease | Retinitis | Retinitis Pigmentosa
Altered gene MCO
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment MCO-010
PhasePhase2
Recruitment statusActive, Not Recruiting
TitleA Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Year2021
CountryPuerto Rico|United States
Company sponsorNanoscope Therapeutics Inc.
Other ID(s)NTXMCO-002.
Vector information
Vectoradeno-associated virus
ConstructAAV2-MCO
Vector typeadeno-associated virus serotype 2 (AAV2) vector
Transgene/Inserted genemulti-characteristic opsin (MCO) gene

Clinical Result

Cohort1: dose level 1
Administration route intravitreal injection
Dosage 1.2E11 gc/eye
Age Adult, Older_Adult
References Efficacy and safety of MCO-010 optogenetic therapy for vision restoration in patients with severe vision loss due to retinitis pigmentosa: A phase 2b randomized, sham-controlled, multi-center, multi-dose, double-masked clinical trial (RESTORE)
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 0.9E11 gc/eye
Age Adult, Older_Adult
References Efficacy and safety of MCO-010 optogenetic therapy for vision restoration in patients with severe vision loss due to retinitis pigmentosa: A phase 2b randomized, sham-controlled, multi-center, multi-dose, double-masked clinical trial (RESTORE)

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