Recent Updates
Recently added Catalysts

OPGx-BEST1

Phase 1

ARB | Gene therapy | Rare Disease |Opus Genetics, Inc.|Last Updated: Mar 24, 2026

Success Probability
Subscribe to view
Market & Valuation
Subscribe to view
Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment10
FDA Designations
No designations recorded
Clinical trial landscape

OPGx-BEST1 · 1 trial · 4 indications

Phase 1 1
NCT07185256Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)ARB
RECRUITING10 Analytics
PHASE1RECRUITING
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
ARB
NCT0718525610
Unlock trial analytics
Study Endpoints
Primary Endpoints
Number of dose-limiting toxicity (DLT) events at the dose tested
5 years

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
5 years

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
5 years

Number and severity of adverse events considered related to the study drug, OPGx-BEST1

Secondary Endpoints
Changes from baseline in retinal morphology assessed by spectral-domain optical coherence tomography
5 years
Changes from baseline in retinal morphology assessed by 55 degree fundus autofluorescence (FAF)
5 years
Changes from baseline in retinal morphology assessed by ultra-wide angle autofluorescence (FAF)
5 years
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
OPGx-BEST1EXPERIMENTALOPGx-BEST1, 1.5E9 vg/eye injected one time subretinally
Interventions
NameTypeDescription
OPGx-BEST1GENETICExperimental Genetic Therapy
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

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 ...

Countries:United States
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07185256primaryCompletionDate: changed
LOWMay 24, 2026NCT07185256studyFirstPostDate: changed