Nanoscope’s AAV Gene Therapy Gains Key EMA and FDA Designations for Retinal Diseases

DALLAS, Texas – September 2, 2025 – Nanoscope Therapeutics Inc., a biotechnology company focused on developing novel disease-agnostic therapies for vision impairment, today announced that its leading candidate, MCO-010 (sonpiretigene isteparvovec), has received multiple key regulatory designations in both the United States and Europe.

The European Medicines Agency (EMA) granted Orphan designations for MCO-010 across five broad categories of retinal dystrophies. Simultaneously, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for MCO-010 in Stargardt disease (SD).

These actions, which underscore regulatory recognition of MCO-010’s potential to address significant unmet needs, build upon prior FDA Orphan Drug and Fast Track designations for both retinitis pigmentosa (RP) and SD.

“Securing RMAT designation for Stargardt disease in addition to our prior FDA designations for SD and RP is a major validation for our therapies that warrant expedited development and review,” said Sulagna Bhattacharya, CEO of Nanoscope.

MCO-010: A Disease-Agnostic Optogenetic AAV Gene Therapy

MCO-010 is a one-time, in-office, intravitreal, disease-agnostic optogenetic gene therapy designed to restore vision regardless of the patient’s underlying genetic mutation. The therapy uses an adeno-associated virus (AAV) vector, specifically AAV2, to deliver a multi-characteristic opsin (MCO) transgene.

This AAV gene therapy targets remaining bipolar retinal cells, genetically modifying them to produce a photosensitive protein. This allows the bipolar cells to compensate for the death of typical photoreceptors and utilize the remaining visual circuitry.

• Mechanism: By activating these bipolar cells, the AAV allows patients to regain light sensitivity and potentially functional vision.
• Broad Applicability: The platform does not require genetic testing, surgical intervention, or repeat dosing, enabling broad patient applicability.
• Expedited Pathway: The EMA Orphan status provides up to 10 years of market exclusivity in the EU, while the FDA’s RMAT, Orphan, and Fast Track designations provide opportunities for accelerated development and review in the U.S.

Clinical Data Shows Durable Efficacy

The regulatory recognition follows positive clinical data from Nanoscope’s programs:

• RP (RESTORE Trial): Three-year follow-up data from the Phase 2b RESTORE trial, presented in September 2025, demonstrated durable efficacy and long-term tolerability. At 152 weeks, the low-dose group experienced a mean improvement in Best-Corrected Visual Acuity (BCVA) of 0.453 LogMAR from baseline.
• SD (STARLIGHT Trial): Encouraging findings have also emerged from the Phase 2 STARLIGHT trial in Stargardt disease.

Nanoscope is currently submitting a Biologics License Application (BLA) for MCO-010 to the FDA, with full filing expected in early 2026. Furthermore, a Phase 3 registrational trial in SD and a Phase 2 trial in Geographic Atrophy (GA) are both expected to begin by the end of 2025.