Sep 9, 2026 · 5 min · 5 segments
The FDA accepts a biologics license application (BLA) for a first-of-its-kind optogenetic gene therapy for RP; a software upgrade from Heru combines several pretesting assessments into a single guided…
Briana ClarkHost
In our top story, the FDA has accepted Nanoscope Therapeutics biologics license application for optogenetic gene therapy, mogenri.
The investigational therapy is designed for vision restoration in patients with retinitis pigmentosa who have severe vision loss.
Mogenri, also known as MCO101, is a one-time, in-office, intravitreal gene therapy built on Nanoscope's MCO platform.
By delivering a multi-characteristic opsin gene to the highly dense bipolar retinal cells, Mogenri is designed to make surviving cells directly light sensitive, enabling them to utilize the remaining visual circuitry following photoreceptor loss.
The BLA is supported by positive efficacy and safety data from the Phase 1/2A trial and the Restor Phase 2b/3 trial.
Restor met its primary and key secondary endpoints, demonstrating improvements in visual acuity at weeks 52 and 76.
Mogenri was well-tolerated with no treatment related serious adverse events reported.
Most patients dosed in Restor have continued into the Remain study, which provides long-term follow-up data in the BLA.
If approved, Mogenri could become the first gene agnostic therapy to improve vision in patients with RP and severe vision loss.
In our top story, the FDA has accepted Nanoscope Therapeutics biologics license application for optogenetic gene therapy, mogenri.
The investigational therapy is designed for vision restoration in patients with retinitis pigmentosa who have severe vision loss.
Mogenri, also known as MCO101, is a one-time, in-office, intravitreal gene therapy built on Nanoscope's MCO platform.
By delivering a multi-characteristic opsin gene to the highly dense bipolar retinal cells, Mogenri is designed to make surviving cells directly light sensitive, enabling them to utilize the remaining visual circuitry following photoreceptor loss.
The BLA is supported by positive efficacy and safety data from the Phase 1/2A trial and the Restor Phase 2b/3 trial.
Restor met its primary and key secondary endpoints, demonstrating improvements in visual acuity at weeks 52 and 76.
Mogenri was well-tolerated with no treatment related serious adverse events reported.
Most patients dosed in Restor have continued into the Remain study, which provides long-term follow-up data in the BLA.
If approved, Mogenri could become the first gene agnostic therapy to improve vision in patients with RP and severe vision loss.
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