Stargardt disease is an inherited retinal dystrophy and the most common cause of juvenile macular dystrophy. It is usually associated with ABCA4 mutations and leads to progressive central vision loss.
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.
Ocugen has completed enrollment and dosing in its phase II/III GARDian3 study for OCU410ST gene therapy targeting Stargardt disease. The study includes 63 patients and aims to address over 1,200 ABCA4 gene mutations with a single treatment. Interim data is expected in Q3 2026, with a BLA submission targeted for mid-2027.
Two gene therapies for inherited eye diseases have progressed to clinical trials, with the FDA clearing Sanaregen's SVT-001 for Familial Drusen and SpliceBio treating its first patient in a Stargardt disease trial in Oxford.
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.
Insmed reported total revenues of $606.4 million for 2025, with ARIKAYCE growing 19% annually. The FDA granted orphan drug designation to treprostinil palmitil for pulmonary arterial hypertension treatment in January 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07594236 |
Phase 1 Study of C.001 in Retinal Degeneration |
RECRUITING | PHASE1 |
| NCT07502664 |
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) |
RECRUITING | |
| NCT07439887 |
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease |
RECRUITING | PHASE1/PHASE2 |
| NCT07425574 |
A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults |
RECRUITING | |
| NCT07419334 |
Study of ALK-001 on the Progression of Stargardt Disease |
RECRUITING | PHASE3 |
| NCT07417566 |
A Study of DC6001 Tablet in Healthy Chinese Adult Subjects |
RECRUITING | PHASE1 |
| NCT07298174 |
Wide Field OCTA in Ocular Diseases |
NOT_YET_RECRUITING | |
| NCT07266584 |
Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration |
RECRUITING | NA |
| NCT07265895 |
Inherited Retinal Diseases: Natural History and Genotype-Phenotype Correlations |
NOT_YET_RECRUITING | |
| NCT07161544 |
A Study of AAVB-039 in Participants With Stargardt Disease (STGD1) |
RECRUITING | PHASE1/PHASE2 |
