Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

For people ages 6 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 for Adult RP

1. Males or females ≥18 years of age at the time of informed consent.
2. Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
3. For subjects in Cohort 1-3, BCVA ≤ 20/50 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
4. Unable to perform a Multi-Luminance Mobility Testing (MLMT) using study eye at 1 lux, the lowest luminance level tested.

Exclusion Criteria for Adult RP

1. Subject lacks evidence of outer nuclear layer
2. 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
3. Previous treatment with a gene-therapy or cell therapy product.
4. Previous treatment with any investigational drug or device within one year.
5. Any contraindications for subretinal injection.

Inclusion Criteria for Adult LCA

1. Males or females at least 18 years of age at the time of informed consent.
2. Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
3. Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.5 (light perception) in the study eye.
4. Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).

Exclusion Criteria for Adult LCA

1. Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function
2. Any contraindications for subretinal injection.
3. Any intraocular surgery within 6 months.
4. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis)

Inclusion Criteria for Pediatric RP

1. Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
2. Confirmed genetic diagnosis of biallelic autosomal recessive NR2E3 mutations or autosomal dominant NR2E3 mutation for Subgroup 1 or autosomal dominant RHO mutations for Subgroup 2.
3. BCVA ≤ 20/32 or visual field less than 20° in any meridian, as measured by a III4e isopter or equivalent in study eye.
4. 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 for Pediatric RP

1. Subject lacks evidence of outer nuclear layer as determined by spectral-domain optical coherence tomography (SD-OCT).
2. Previous treatment with a gene-therapy or cell therapy product.
3. Previous treatment with any investigational drug or device within one year.
4. Any contraindications for subretinal injection.
5. Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.
6. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis)

Inclusion Criteria for Pediatric LCA

1. Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
2. Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
3. Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.5 (light perception) in the study eye.
4. Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).

Exclusion Criteria for Pediatric LCA

1. Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function
2. Any contraindications for subretinal injection.
3. Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis)
4. Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.