SPARINGVISION | MedPath

Nanoscope's Optogenetic Gene Therapy Shows Promise in Retinitis Pigmentosa Clinical Trials

2 months ago

• Nanoscope Therapeutics' MCO-010 gene therapy demonstrated clinically meaningful vision improvements in 16 out of 18 patients with severe retinitis pigmentosa in a Phase 2 trial, compared to only four of nine in the placebo group. • The mutation-agnostic therapy uses a synthetic opsin (MCO protein) delivered via intravitreal injection to restore light-detecting function in damaged retinal cells, potentially benefiting patients regardless of their specific genetic mutation. • Following treatment, patients showed significant improvements in vision-guided mobility and object recognition, with some experiencing "transformative" results while maintaining a favorable safety profile with no serious adverse events reported.

Milestone Pharmaceuticals Gains New Patent for Etripamil, Prepares for Potential Launch

6 months ago

• Milestone Pharmaceuticals received a Notice of Allowance from the USPTO for a new patent covering the repeat dose regimen of etripamil nasal spray, extending potential IP protection to July 2042. • The patent supports the New Drug Application (NDA) for CARDAMYST™ (etripamil) currently under FDA review, with a PDUFA target date of March 27, 2025. • Milestone is actively preparing for a mid-2025 commercial launch of CARDAMYST for Paroxysmal Supraventricular Tachycardia (PSVT), pending FDA approval. • A Phase 3 trial of etripamil nasal spray for Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR) is planned to commence in the first half of 2025.