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 therapy is delivered by subretinal injection and is currently in Phase 1 clinical development.
OPGx-BEST1 is being developed by Opus Genetics, Inc., a biopharmaceutical company. The company is conducting a Phase 1 clinical trial of the therapy in the United States. Opus Genetics is focused on developing gene therapies for rare inherited retinal diseases.
OPGx-BEST1 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by the FDA. The ongoing Phase 1 trial is currently recruiting participants and is designed to evaluate the safety and tolerability of the therapy.
OPGx-BEST1 is being studied in a single Phase 1 clinical trial with the identifier NCT07185256. The trial is titled 'Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)' and is currently recruiting in the United States.
OPGx-BEST1 is administered by subretinal injection, a procedure in which the therapy is delivered directly beneath the retina. This route of administration is commonly used for gene therapies targeting retinal diseases. The ongoing Phase 1 trial is evaluating this delivery method in patients with ARB and related conditions.