Basic Information


GTO ID GTC2710
Trial ID NCT04919473
Disease Retinitis Pigmentosa | Retinal Disease | Retinal Degeneration
Altered gene MCO
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment vMCO-I|AAV2-MCO
PhasePhase1|Phase2
Recruitment statusCompleted
TitleA Phase I/IIa Open Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
Year2021
CountryItaly
Company sponsorNanoscope Therapeutics Inc.
Other ID(s)NSCT/CT/18/01
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 3.5E11vg/eye
Age Adult, Older_Adult
References PMID: 37132114
Cohort2: dose level 2
Administration route intravitreal injection
Dosage 1.75E11vg/eye
Age Adult, Older_Adult
References PMID: 37132114

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