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May 12, 2026
Recent studies in retinitis pigmentosa showed mixed results for gene and optogenetic therapies. UGX-201 and MCO-010 were associated with visual acuity improvements, while AAV8.hPDE6A showed no significant functional gains at 1 year.
May 01, 2026
Ray Therapeutics received EMA PRIME designation for its RTx-015 optogenetic gene therapy for retinitis pigmentosa, following recent FDA RMAT status. The company also appointed Michael Murtagh as Chief Regulatory Officer to support global development.
Apr 04, 2026
An observational study in Documenta Ophthalmologica found the MLSDT showed reliability above 0.50 in advanced retinitis pigmentosa and correlated with visual acuity, visual fields and patient-reported outcomes. The test is being used as a secondary endpoint in the phase 2b/3 RESTORE trial of MCO-010.
Mar 04, 2026
Ocugen will host a conference call on March 4, 2026 at 8:30 a.m. ET to discuss fourth quarter and full year 2025 financial results and provide a business update, with a pre-market earnings announcement the same day.
Mar 03, 2026
Ocugen has completed enrollment of 140 patients in its Phase 3 liMeliGhT trial for OCU400, a gene therapy for retinitis pigmentosa. Topline data expected in Q1 2027 could support potential approval later that year.
Feb 28, 2026
International Rare Disease Day on February 28 underscores challenges in diagnosing and treating rare genetic conditions, with patients facing years-long diagnostic delays and geographic barriers to accessing gene therapies.
