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Nanoscope preps filing for retinitis pigmentosa gene therapy

March 26, 2024

The Dallas, Texas-based biotech said it plans to submit a dossier to the FDA later this year after its MCO-010 (sonpiretigene isteparvovec) gene therapy achieved almost all its primary and secondary efficacy objectives in the phase 2b RESTORE trial.

Approximately 100,000 people in the US and an estimated 2 million people worldwide suffer from RP, a condition that results in progressive damage to light-detecting cells in the retina and is the leading cause of inheritable blindness.

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