Retinitis pigmentosa is a group of rare inherited retinal diseases that progressively damage photoreceptor cells and lead to vision loss. Symptoms often begin with night blindness and then progressive peripheral field loss. Over time, vision can narrow substantially and may progress to severe impairment.
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.
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.
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.
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.
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.
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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07509229 |
9-cis Beta-Carotene-Rich Extract of Dunaliella Alga in Retinitis Pigmentosa Patients |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07502664 |
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) |
RECRUITING | |
| NCT07408232 |
A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT07228793 |
Natural History Study of Patients With EYS-Associated RP |
RECRUITING | |
| NCT07082855 |
A Multicenter, Randomized, Double-Blind, Controlled Clinical Study of Minocycline for the Treatment of Retinitis Pigmentosa |
NOT_YET_RECRUITING | PHASE3 |
| NCT07056738 |
Prospective Exploratory Cohort Study on Ganglion Cell Degeneration in Retinitis Pigmentosa Patients |
ENROLLING_BY_INVITATION | |
| NCT06952842 |
Safety and Efficacy of ZVS203e in the Treatment of Retinitis Pigmentosa Caused by RHO Gene Mutation |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT06936787 |
An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa |
RECRUITING | PHASE1 |
| NCT06912633 |
Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP) |
RECRUITING | PHASE2 |
| NCT06891885 |
A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa |
RECRUITING | PHASE1/PHASE2 |
