FDA Accepts Nanoscope's BLA for Optogenetic Gene Therapy MCO-010 in Retinitis Pigmentosa
On September 9, 2026, Nanoscope Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted and filed its Biologics License Application (BLA) for MOGENRY (sonpiretigene isteparvovec, MCO-010), an intravitreally delivered optogenetic gene therapy. The BLA seeks approval for the restoration of vision in adult patients with retinitis pigmentosa (RP) and severe vision loss.
Clinical and Scientific Significance
Retinitis pigmentosa is a progressive, inherited retinal degenerative disease that causes photoreceptor loss and severe visual impairment. Current approved gene therapies (such as Luxturna) are mutation-specific, targeting only a tiny fraction of the RP patient population.
If approved, MOGENRY will be the world's first mutation-agnostic therapy designed to restore useful vision in RP patients regardless of the underlying genetic mutation. The therapy works by delivering light-sensitive multi-characteristic opsins (MCOs) to remaining retinal bipolar cells, essentially transforming them into alternative photoreceptors.
Pivotal Data and Regulatory Status
The BLA acceptance is supported by positive data from the Phase 2b/3 RESTORE trial (NCT04945772), a randomized, double-masked, sham-controlled study:
- Efficacy: The trial demonstrated sustained, statistically significant, and clinically meaningful improvements in best-corrected visual acuity (BCVA) at Week 52 and Week 76.
- Safety: The intravitreal injection was well tolerated, with no treatment-related serious adverse events reported.
MCO-010 has previously received FDA Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations in the United States, as well as multiple Orphan Drug designations1 from both the FDA and the European Medicines Agency (EMA).
"The FDA has accepted and filed the Company's BLA for MOGENRY... optogenetic gene therapy designed for vision restoration for patients with retinitis pigmentosa (RP) having severe vision loss... if approved, [MOGENRY] would be positioned as the first mutation-agnostic therapy intended to restore useful vision in patients with RP regardless of the causative gene." — Nanoscope Therapeutics Press Release & Modern Retina Report
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An instance of Rare disease pipelines only accelerate through early FDA alignment and breakthrough designations. — Nanoscope expedited the regulatory pathway for its optogenetic pipeline by securing multiple fast track and orphan status designations. ↩︎