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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
NCTID
NCT05417126
(View at clinicaltrials.gov)
Description
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
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Development Status
Active
Indication
Stargardt Disease
Disease Ontology Term
DOID:0050817
Compound Name
MCO-010
Compound Alias
Sonpiretigene isteparvovec
Sponsor
Nanoscope Therapeutics Inc.
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
6
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
1.2E11 gc/eye
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase2
Submit Date
2022-06-09
Completion Date
2023-09-28
Last Update
2023-11-09
Participation Criteria
Eligible Age
>=16 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
2
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Fast Track, Orphan Drug Designation
Recent Updates
Phase III Study expected to begin Q1 2025
Resources/Links
News and Press Releases
Nanoscope Therapeutics Announces End-of-Phase 2 Meeting with U.S. FDA and Plan to Initiate a Phase 3 Clinical Trial of MCO-010 to Treat Stargardt Macular Degeneration
Preclinical Publications
Biodistribution of adeno-associated virus type 2 carrying multi-characteristic opsin in dogs following intravitreal injection