Nanoscope Therapeutics’ clinical stage gene therapy focuses on restoring vision for people suffering from retinal degenerative diseases. Our gene therapy delivers light sensitive Multi-Characteristic Opsins (MCOs) into retinal cells with potential to detect ambient light and improve activities of daily living.
Nanoscope targets diseases for which there are no known cures:
Retinitis Pigmentosa
(RP) is a rare inherited retinal disease characterized by progressive peripheral vision loss and blindness. It is the most common genetic cause of blindness and affects about 150,000 people in the US. MCO-010 has been granted Orphan Drug and Fast Track designation for RP by the US Food and Drug Administration.
Dry Age-Related Macular Degeneration (Geographic Atrophy)
A common disorder leading to central vision loss in the elderly. Geographic atrophy is caused by gradual deterioration of the macula, the central retina.
Cone Rod Dystrophy
A rare group of inherited disorders caused by deterioration of cones and rods which are photoreceptor cells in the retina responsible for detecting light intensity levels.
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Usher Syndrome
A rare inherited disease that causes retinitis pigmentosa and hearing loss.
Stargardt Disease
One of the most common forms of inherited macular degeneration, affecting about 30,000 people in the U.S. The rare disease causes progressive degeneration of the macula, causing dark spots in the central field of vision. MCO-010 has been granted Orphan Drug and Fast Track designation for this disease.