for people ages 18 years and up (full criteria)
at La Jolla, California and other locations
study started
estimated completion



This is a multicenter, open-label, dose ranging study to assess the safety of unilateral subretinal administration of OCU400 in subjects with NR2E3-related retinitis pigmentosa.

Official Title

A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 (Nuclear Receptor Subfamily 2 Group E Member 3) and RHO (Rhodopsin) Mutations


This study is designed to evaluate the safety of ascending doses of OCU400 in subjects with retinitis pigmentosa associated with NR2E3 and RHO mutations.


Retinitis Pigmentosa NR2E3 Rhodopsin Enhanced S-cone syndrome Retinitis OCU400 Low Dose OCU400 Mid Dose OCU400 High Dose


For people ages 18 years and up

Diagnosis and main criteria for inclusion:

Subjects meeting all inclusion criteria and none of the exclusion criteria are eligible for study participation.

Inclusion Criteria:

Males or females ≥18 years of age at the time of informed consent.

Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations for Subgroup 1, autosomal dominant NR2E3 mutation for Subgroup 2 or autosomal dominant RHO mutations for Subgroup 3.

For the sentinel subject of Cohort 1-3, BCVA ≤ 20/160 in study eye or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.

For non-sentinel subject, BCVA ≤ 20/50 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.

Able to perform a multi-luminance mobility testing (MLMT) using study eye, but unable to pass the MLMT at 1 lux, the lowest luminance level tested.

Exclusion Criteria:

Subjects lacks evidence of outer nuclear layer, i.e., containing the nuclei of the retinal photoreceptors as determined by spectral-domain optical coherence tomography (SD-OCT).

Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator, i.e., inability to fixate, high myopia ≥10 diopters, glaucoma, medium haze, other retinal pathologies, and > 3-fold elevation of liver enzymes or > 2-fold elevation of serum creatinine, etc.

Previous treatment with a gene-therapy or cell therapy product.

Previous treatment with any investigational drug or device within one year.

Any contraindications for subretinal injection.

Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.

Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.

Any medical condition with life expectancy < 6 years.


  • Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute not yet accepting patients
    La Jolla California 92093 United States
  • Ocugen Site 2 - Casey Eye Institute - OHSU accepting new patients
    Portland Oregon 97239 United States


accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Phase 1/2 Retinitis Pigmentosa Research Study
Study Type
Expecting 18 study participants
Last Updated