Nanoscope Secures EMA, FDA Status for MCO-010

Nanoscope Therapeutics has announced key regulatory designations in both Europe and the United States for its lead gene therapy candidate, MCO-010 (sonpiretigene isteparvovec), targeting inherited retinal diseases.

The European Medicines Agency (EMA) has granted Orphan Drug Designation for MCO-010 across five categories of retinal dystrophies, including non-syndromic and syndromic rod-dominant dystrophies, non-syndromic and syndromic cone-dominant dystrophies, as well as macular dystrophies.

In parallel, the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for MCO-010 in Stargardt disease (SD).

Regulatory Designations and Therapeutic Scope

The EMA’s Orphan Drug designations provide MCO-010 with a potential regulatory pathway across a wide spectrum of retinal conditions, underscoring its promise as a mutation-independent, disease-agnostic therapy. In the European Union, Orphan Drug Designation offers benefits such as protocol assistance and up to 10 years of market exclusivity following approval.

In the United States, the newly granted RMAT designation builds on prior Orphan Drug and Fast Track designations for both Stargardt disease and retinitis pigmentosa (RP). These FDA programs offer advantages such as accelerated development timelines, flexibility in clinical trial design, and seven years of market exclusivity post-approval.

MCO Platform: An In-Office Gene Therapy Approach

MCO-010 is based on Nanoscope’s multi-characteristic opsin (MCO) platform, a one-time, intravitreal gene therapy designed for in-office administration. The therapy targets photoreceptor degeneration, including in conditions such as RP, Stargardt disease (SD), and geographic atrophy (GA). Unlike other gene therapy approaches, MCO-010 does not require genetic testing, surgical delivery, or repeat dosing.

MCO-010 works by making bipolar retinal cells, which remain abundant even in advanced retinal disease, light-sensitive, leveraging the eye’s existing visual circuitry to restore functional vision. This mechanism supports seamless integration into current retina clinic workflows.