Opus Genetics has dosed the first subject in its Phase I/II clinical trial of OPGx-Best1 gene therapy for the treatment of Best disease (BEST1).
Best disease, also known as vitelliform macular dystrophy, is a rare retinal condition that causes progressive vision loss due to BEST1 gene mutations.
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Delivered as a one-time subretinal injection, the therapy aims to restore function to the retinal pigment epithelium (RPE) cells that are affected by these mutations.
OPGx-Best1 employs the company’s adeno-associated viral (AAV) platform, delivering a functional BEST1 gene copy directly to RPE cells.
The programme builds on preclinical studies showing restored BEST1 protein expression and enhanced function of retina in disease models.
The open-label, multi-centre, adaptive dose-exploring trial, BIRD-1, will assess early efficacy, safety, and tolerability of OPGx-Best1 in subjects with best vitelliform macular dystrophy or autosomal-recessive bestrophinopathy.
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By GlobalDataSubjects will receive a single subretinal injection in one eye, with two dosing cohorts. Biological activity will be measured through anatomical and functional endpoints, including changes in retinal structure and visual function.
Initial data are anticipated in the first quarter of 2026.
Opus Genetics CEO George Magrath said: “Dosing the first participant in our OPGx-Best1 programme is a historic moment for the BEST disease community and for our team at Opus. This milestone reinforces our mission to develop one-time gene therapies for inherited retinal diseases that previously had no treatment options.
“We are deeply thankful to the Retina Foundation of the Southwest, Retina Consultants of Texas, and most importantly, the patients and families who place their trust in us. Together, we are striving to build a future where no one loses their sight to inherited retinal disease.”
The trial is being carried out by Dr Mark Pennesi from the Retina Foundation of the Southwest, in collaboration with the surgical team of Dr Charles Wykoff and Dr Kenneth Fan at Retina Consultants of Texas.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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