The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-BEST1. The main questions it aims to answer are:
Evaluate the safety and tolerability of drug OPGx-BEST1 in one eye (the treatment eye), for 5 years post-injection, in participants with BVMD or ARB.
A second question it aims to answer is identification of the most appropriate dose strength of OPGx-BEST1 for clinical development.
Evaluate the efficacy of single injection of OPGx-BEST1 in one eye for 5 years post-injection.
What medical problems do participants have when taking drug OPGx-BEST1?
Provide informed consent to study assessments.
Able and willing to comply with all study assessments for the duration of the study.
≥18 years old.
For the sentinel participant in each cohort, ≤20 letters (Snellen equivalent of 20/200 [1.30 logMAR] or worse)
Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration. See Section 13.3 (Appendix 3) for contraception guidelines.
Men who are unwilling to use adequate contraception from Screening through 180 days after IMP administration. See Section 13.3 (Appendix 3) for contraception guidelines.
Have a pre-existing eye condition or complicating systemic disease that could preclude the planned surgery (e.g., individuals who are immunocompromised, on continuous systemic immunosuppressive treatment or anticipate a need to initiate it for non-study reasons, or unable to take the concomitant immuno-suppressive regimen necessary for IMP administration).
Have a history of disease that may preclude the individual from study participation (e.g., other bestrophinopathy, such as AOFVD or ADVIRC) or that may interfere with or preclude outcome measure testing as described in the protocol.
Single group
OPGx-BEST1
GeneticExperimental Genetic Therapy
Number of dose-limiting toxicity (DLT) events at the dose tested
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 procedure-related adverse events
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 related to OPGx-BEST1
Number and severity of adverse events considered related to the study drug, OPGx-BEST1
Changes from baseline in retinal morphology assessed by spectral-domain optical coherence tomography
Changes from baseline in retinal morphology assessed by spectral-domain optical coherence tomography
Changes from baseline in retinal morphology assessed by 55 degree fundus autofluorescence (FAF)
Changes from baseline in retinal morphology assessed by 55 degree fundus autofluorescence (FAF)
Changes from baseline in retinal morphology assessed by ultra-wide angle autofluorescence (FAF)
Changes from baseline in retinal morphology assessed by ultra-wide angle autofluorescence (FAF)
Changes from baseline in neovascularization assessed by optical coherence tomography
Changes from baseline in neovascularization assessed by optical coherence tomography angiography
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