Nanoscope's Optogenetic Gene Therapy Shows Promise in Retinitis Pigmentosa Clinical Trials
• 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.
Global Diabetic Retinopathy Clinical Trials Landscape Analysis Reveals Key Research Trends for 2024
• A comprehensive analysis of global diabetic retinopathy clinical trials has been released, offering insights into trial distribution across G7 and E7 countries and various research phases.
• The report highlights significant trends in trial enrollment patterns over the past five years, providing valuable data for healthcare stakeholders and pharmaceutical companies.
• The analysis covers detailed assessment of trial statuses, sponsor types, and endpoint evaluations, helping identify key opportunities and challenges in diabetic retinopathy research.
Ocugen's OCU400 Shows Promise in Retinitis Pigmentosa Treatment with Positive Clinical Data and Regulatory Milestones
• Ocugen's OCU400 gene therapy demonstrated a statistically significant improvement in low-luminance visual acuity in patients with retinitis pigmentosa (RP).
• The European Medicines Agency (EMA) has granted OCU400 an Advanced Therapy Medicinal Product (ATMP) classification, expediting its regulatory review process.
• Phase 1/2 trial results showed that 100% of treated patients experienced improved or preserved visual function over two years, regardless of the underlying genetic mutation.
• Ocugen's Phase 3 liMeliGhT clinical trial is ongoing, with plans for BLA/MAA submissions in the U.S. and Europe anticipated in the first half of 2026.
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