Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
OCU410ST · 1 trial · 1 indication
Change in the area of atrophy will be evaluated from the baseline measurements, using FAF to assess the loss of retinal layers.
Safety will be determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
| Arm | Type | Description |
|---|---|---|
| Phase 2/3 Randomized Treatment Arm | EXPERIMENTAL | Subjects will receive a single subretinal injection of 200uL OCU410ST in concentration of 1.5 x 10E11vg/mL |
| Phase 2/3 Randomized Control Arm | NO_INTERVENTION | Subjects will not receive any active study intervention |
| Name | Type | Description |
|---|---|---|
| OCU410ST | DRUG | Subretinal Administration of OCU410ST |
Phase 2/3 Inclusion Criteria (applicable for both adult and pediatric subjects): 1. Males or females aged ≥5 years at the time of consent. 2. Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies). 3. Adul...
OCU410ST is an investigational small molecule gene therapy being developed by Ocugen, Inc. for the treatment of Stargardt disease. It is designed to target the RORA gene and is currently in Phase 2 clinical development. It has received Rare Pediatric Disease and Orphan Drug designations from the FDA.
OCU410ST is being developed as a potential treatment for Stargardt disease, an inherited retinal disorder that causes progressive vision loss. It is intended to address the underlying genetic cause by targeting the RORA gene. The therapy is investigational and has not been approved for any indication.
OCU410ST targets the RORA gene, which encodes the retinoic acid receptor-related orphan receptor alpha. By modulating this target, the therapy aims to treat Stargardt disease. The small molecule is delivered as a gene therapy approach and is currently in Phase 2 testing.
OCU410ST is being developed by Ocugen, Inc., a biotechnology company traded on the Nasdaq under the ticker symbol OCGN. Ocugen is conducting a Phase 2/3 clinical trial of OCU410ST for Stargardt disease in the United States.
OCU410ST is in Phase 2 clinical development. A Phase 2/3 trial is currently active but not recruiting participants. The trial is evaluating the efficacy and safety of OCU410ST in patients with Stargardt disease.
OCU410ST is being evaluated in one active clinical trial, NCT05956626, titled 'A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease.' The trial is active but not recruiting, has enrolled 51 participants, and is conducted in the United States.