Drugs / OPGx-BEST1
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT07185256 | Sep 2025 → Aug 2030 expected | BEST1-related dominant retinopathy, vitelliform macular dystrophy | Opus Genetics, Inc | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2026-09-09 | Ocuphire Pharma, Inc. | Results Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027 opusgtx.com ↗
OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no serious adverse events or dose-limiting toxicities observed |
| 2026-06-16 | Ocuphire Pharma, Inc. | Results Opus Genetics Highlights Five Gene Therapy Programs Targeting Significant Unmet Need in Patients with Inherited Retinal Diseases opusgtx.com ↗
visual acuity was improved and maintained in the adult cohort over 24 months and improved in the pediatric cohort over 6 months. |
| 2026-05-07 | Ocuphire Pharma, Inc. | Results Opus Genetics Presents Clinical and Preclinical Data at ARVO 2026 Demonstrating Continued Pipeline Advancement in Inherited Retinal Diseases opusgtx.com ↗
These findings provide early clinical evidence for BEST1 gene augmentation in a large inherited retinal disease population. |
| 2026-02-27 | Ocuphire Pharma, Inc. | Results Opus Genetics Announces Initial Clinical Data from Phase 1/2 OPGx-BEST1 Gene Therapy Study at the Macula Society Annual Meeting opusgtx.com ↗
The data demonstrated that OPGx-BEST1 was well tolerated with no ocular inflammation, no ocular or treatment-related adverse events, and no dose limiting toxicities. |
| 2025-12-09 | Ocuphire Pharma, Inc. | Results Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease opusgtx.com ↗
The IDMC overseeing the trial completed its pre-specified safety review of the one-month data from the sentinel participant and recommended advancing enrollment and dosing of additional participants in the trial, without modification. |
| 2025-08-18 | Ocuphire Pharma, Inc. | Regulatory Opus Genetics Announces FDA Clearance of IND Application for Gene Therapy Candidate OPGx-BEST1 opusgtx.com ↗
With this IND clearance, Opus Genetics plans to initiate a Phase 1/2 clinical trial in the second half of 2025. |
| 2025-05-12 | Ocuphire Pharma, Inc. | Results Opus Genetics Announces Presentations on Inherited Retinal Disease Programs at Medical Conferences in May opusgtx.com ↗
Restoration of the RPE-photoreceptor interface was demonstrated in the treated animals, including resolution of the retinal microdetachments. |
All press releases naming this drug 11 releases
Evidence & citations 1 cited value
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OPGx-BEST1 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07185256 ↗ |