Drug intelligence / Profile preview

OPGx-RHO

Development stage
Preclinical
Lead developer
Opus Genetics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Ophthalmic, Subretinal
01

Overview

OPGx-RHO is an AAV-based gene therapy being developed by Opus Genetics for autosomal dominant retinitis pigmentosa caused by mutations in the rhodopsin gene. It is designed as a single, mutation-independent rhodopsin replacement strategy to preserve rod photoreceptors by delivering a functional copy of RHO to retinal photoreceptors, with a first-in-human trial targeted to begin in 2026.[7][2][10] AdisInsight also lists IC 100 as an alternative designation associated with rhodopsin-mediated autosomal dominant retinitis pigmentosa.[8]

Other names
IC 100IC100IC-100
02

Targets

RHO (Rhodopsin)

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