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Sub-Retinal Administration of OCU400-301 · 1 trial · 1 indication
Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
| Arm | Type | Description |
|---|---|---|
| RHO Arm | EXPERIMENTAL | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye |
| Gene Agnostic Arm | EXPERIMENTAL | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye |
| Control for RHO Arm | NO_INTERVENTION | Will not receive any active study intervention |
| Control for Gene Agnostic Arm | NO_INTERVENTION | Will not receive any active study intervention |
| Name | Type | Description |
|---|---|---|
| Sub-Retinal Administration of OCU400-301 | GENETIC | Sub-Retinal Administration of OCU400-301 |
Inclusion Criteria: 1. Males or females ≥ 3 years of age 2. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP 3. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2...
Sub-Retinal Administration of OCU400-301 is an investigational gene therapy being developed for the treatment of Retinitis Pigmentosa, a group of inherited retinal diseases that cause progressive vision loss. It is administered via sub-retinal injection and is currently in Phase 3 clinical development.
Sub-Retinal Administration of OCU400-301 targets the NR2E3 gene, which plays a role in retinal function. By delivering a functional copy of this gene, the therapy aims to address the underlying genetic cause of Retinitis Pigmentosa in affected patients.
Sub-Retinal Administration of OCU400-301 is being developed by Ocugen, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol OCGN. The company is conducting a Phase 3 clinical trial to evaluate the therapy in patients with Retinitis Pigmentosa.
Sub-Retinal Administration of OCU400-301 is currently in Phase 3 clinical development. It is an investigational therapy and has not yet been approved by regulatory authorities. The therapy has received a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.
Sub-Retinal Administration of OCU400-301 is being evaluated in a Phase 3 clinical trial with the identifier NCT06388200. This active, not recruiting study is enrolling approximately 140 participants with Retinitis Pigmentosa across the United States and Canada, with a minimum age of 3 years.