Contact SCGE




Gene Therapy Trial Report

Summary

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


NCTID NCT04945772 (View at clinicaltrials.gov)
Description
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

Therapy Information


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

Study Record Dates


Current Stage Phase2
Submit Date 2021-06-15
Completion Date 2024-01-18
Last Update 2024-03-22

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Age ≥ 18 years 2. Able to comprehend and give informed consent. 3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing. 4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening. Exclusion Criteria: Subjects are excluded from the study if any of the following criteria apply: 1. Prior participation in gene therapy program 2. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities). 3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function. 4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis. 5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


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

Regulatory Information


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

Resources/Links