
Texas, USA-based Nanoscope Therapeutics has recently submitted a rolling biologics license application (BLA) to the Food and Drug Administration (FDA) for MCO-010 (sonpiretigene isteparvovec), a novel gene therapy candidate targeting retinitis pigmentosa (RP).
Currently, the RP treatment landscape includes only one FDA-approved therapy, Luxturna (voretigene neparvovec), from Roche (ROG: SIX) subsidiary Spark Therapeutics, which is indicated for a small subset of patients with biallelic RPE65 mutations. In contrast, MCO-010 is poised to expand therapeutic options with a distinct mechanism of action and potentially broader applicability, says pharma analytics company GlobalData.
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