Product Description
OPGx-BEST1 leverages Opus Genetics' proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. (Sourced from: https://ir.opusgtx.com/press-releases/detail/495/opus-genetics-announces-fda-clearance-of-ind-application-for-gene-therapy-candidate-opgx-best1)
Mechanisms of Action: Gene Therapy, BEST1
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Ocuphire Pharma
Company Location:
Company Founding Year: None
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: United States
Active Clinical Trial Count: 1
Recent & Upcoming Milestones
- Clinical Outcomes Expected - Opus Genetics announced they will present P1 Vitelliform Macular Dystrophy results on 2026-02-27 for OPGx-BEST1
- Clinical Outcomes Expected - Opus Genetics announced they will present P1 Vitelliform Macular Dystrophy results in 3Q26 for OPGx-BEST1
Highest Development Phases
Phase 2: Vitelliform Macular Dystrophy
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT07185256 |
BIRD-1 | P2 |
Recruiting |
Vitelliform Macular Dystrophy |
2030-08-01 |
50% |
2025-09-23 |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
02/02/2026 |
News Article |
Opus Genetics to Participate in Upcoming Medical and Industry Conferences in February 2026 |
|
01/27/2026 |
News Article |
Opus Genetics Launches Gene Therapy Clinical Trial for MERTK-related Retinitis Pigmentosa |
|
01/08/2026 |
News Article |
Opus Genetics Highlights 2025 Progress and Upcoming 2026 Catalysts |
|
12/09/2025 |
News Article |
Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease |