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OPGx-BEST1
- Development stage
- Unknown
- Lead developer
- Opus Genetics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Ophthalmic, Subretinal
01
Overview
OPGx-BEST1 is an investigational adeno-associated virus (AAV)-based gene therapy developed for the treatment of inherited retinal diseases associated with mutations in the BEST1 gene, also known as bestrophinopathies or Best disease. The BEST1 gene encodes bestrophin-1, a calcium-activated chloride channel protein expressed in retinal pigment epithelial (RPE) cells. Mutations in this gene disrupt ion homeostasis and lead to progressive macular degeneration characterized by vitelliform lesions and vision loss. OPGx-BEST1 delivers a functional copy of the human BEST1 cDNA via AAV vector directly to the retina, aiming to restore normal chloride channel function and preserve or improve visual function. The therapy is currently being evaluated in Phase 1/2 clinical trials for safety and preliminary efficacy[2][5][9].
- Other names
- AAV-based bestrophin 1 gene therapy (NightstaRx)
02
Targets
BEST1 (Bestrophin-1)
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