Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

NCTID	NCT07185256 (View at clinicaltrials.gov)
Description	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? (Show More)
Development Status	Active
Indication	ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Disease Ontology Term	DOID:0050662
Compound Name	OPGx-BEST1
Compound Description	AAV2.VMD2.hBEST1
Sponsor	Opus Genetics, Inc
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	10 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	BEST1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell	Retinal pigment epithelial (RPE) cells
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	1.5E9 vg/eye
Dose 2	4.5E9 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	First participant dosed Q4 2025, initial data expected Q1 2026