Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
OPGx-BEST1 · 1 trial · 4 indications
Number of dose-limiting toxicity (DLT) events, defined as any ≥Grade 3 toxicity that occurs within 90 days after IMP administration based on the National Cancer Institute common toxicity criteria
Number and severity of adverse events related to the administration of OPGx-BEST1 involving pars plana vitrectomy and subretinal injection under general anesthesia
Number and severity of adverse events considered related to the study drug, OPGx-BEST1
| Arm | Type | Description |
|---|---|---|
| OPGx-BEST1 | EXPERIMENTAL | OPGx-BEST1, 1.5E9 vg/eye injected one time subretinally |
| Name | Type | Description |
|---|---|---|
| OPGx-BEST1 | GENETIC | Experimental Genetic Therapy |
Inclusion Criteria: Individuals who meet all the following criteria will be eligible to participate in the study: 1. Provide informed consent to study assessments. 2. Able and willing to comply with all study assessments for the duration of the study. 3. ≥18 years old. 4. ETDRS BCVA measured with ...
OPGx-BEST1 is an investigational gene therapy being developed for autosomal-recessive bestrophinopathy (ARB), a rare inherited retinal disease. It is also being studied in patients with Best vitelliform macular dystrophy (BVMD) and autosomal-dominant bestrophinopathy. The drug is administered by subretinal injection.
OPGx-BEST1 targets the BEST1 gene. It is designed to deliver a functional copy of the BEST1 gene to retinal cells to address the underlying genetic cause of BEST1-related retinal diseases, including autosomal-recessive bestrophinopathy (ARB).
OPGx-BEST1 is being developed by Opus Genetics, Inc., a biopharmaceutical company. Opus Genetics trades on the stock market under the ticker symbol IRD.
OPGx-BEST1 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The drug is currently being evaluated in an early-stage clinical trial to assess its safety and tolerability.
OPGx-BEST1 is being studied in a Phase 1 clinical trial with the identifier NCT07185256. This trial is recruiting participants in the United States and is evaluating the safety and tolerability of subretinally injected OPGx-BEST1 in patients with Best vitelliform macular dystrophy (BVMD) or autosomal-recessive bestrophinopathy (ARB). The trial plans to enroll 10 participants aged 18 years and older.
OPGx-BEST1 is being studied for use in Best vitelliform macular dystrophy (BVMD) as well as autosomal-recessive bestrophinopathy (ARB). The ongoing Phase 1 clinical trial includes patients with either condition, so OPGx-BEST1 is intended to address both BEST1-related retinal diseases.