Belite Bio has begun a rolling NDA submission for tinlarebant, an oral therapy for Stargardt disease type 1. The drug has Breakthrough Therapy Designation, and the company expects to complete the submission in Q2 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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
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First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration |
RECRUITING | EARLY_PHASE1 |
| NCT06789445 |
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO) |
RECRUITING | PHASE1/PHASE2 |
| NCT06167642 |
Retinal Atrophy and Neurofilament Light Chain in People With Multiple Sclerosis Taking Ofatumumab |
ENROLLING_BY_INVITATION | |
| NCT04545736 |
Oral Metformin for Treatment of ABCA4 Retinopathy |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT02617966 |
Rod and Cone Mediated Function in Retinal Disease |
RECRUITING | |
| NCT01736293 |
Natural History of Eye Diseases Related to ABCA4 Mutations |
ACTIVE_NOT_RECRUITING | |
| NCT01432847 |
Cell Collection to Study Eye Diseases |
RECRUITING |
