Nanoscope's MCO-010 Gene Therapy for Retinitis Pigmentosa Gets FDA Nod for BLA Approval

Nanoscope Therapeutics, a late-stage clinical biotech company focused on gene therapies for inherited retinal diseases and age-related macular degenerations, has shared positive updates from regulatory discussions with the U.S. FDA and the Icelandic Medicines Agency regarding its MCO-010 optogenetic gene therapy for treating advanced Retinitis Pigmentosa (RP). Following a productive Type B End-of-Phase 2 discussion, the FDA indicated that a change from baseline in visual acuity could serve as a suitable primary efficacy assessment in a well-controlled study for substantial evidence of benefit, supporting the Biologics License Application (BLA) approval. MCO-010 has received orphan drug and fast track designations from the FDA.