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Nanoscope's Gene Therapy Gains FDA Designation, Promises Vision Restoration

9/6/2025

Summary

Story summary

Nanoscope Therapeutics gained FDA RMAT designation for MCO-010, a gene therapy targeting Stargardt disease.
The European Medicines Agency awarded MCO-010 five orphan drug designations for various retinal diseases.
MCO-010 aims to restore vision by converting retinal cells into light-sensitive cells with a single injection.
Positive phase 2b RESTORE trial results support the therapy's efficacy.
Nanoscope plans to start a phase 3 trial for Stargardt disease and a phase 2 trial for geographic atrophy by late 2025, with a full Biologics License Application submission targeted for early 2026.

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Story summary

Nanoscope Therapeutics gained FDA RMAT designation for MCO-010, a gene therapy targeting Stargardt disease.
The European Medicines Agency awarded MCO-010 five orphan drug designations for various retinal diseases.
MCO-010 aims to restore vision by converting retinal cells into light-sensitive cells with a single injection.
Positive phase 2b RESTORE trial results support the therapy's efficacy.
Nanoscope plans to start a phase 3 trial for Stargardt disease and a phase 2 trial for geographic atrophy by late 2025, with a full Biologics License Application submission targeted for early 2026.