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

Prior treatment

• Previous treatment with a gene therapy or cell therapy product.
• Previous treatment with any investigational drug or device within one year.
• Cataract Surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
• Any intraocular surgery within 6 months.
• Previous treatment with a gene therapy or cell therapy product.
• Previous treatment with any investigational drug or device within one year.
• Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
• Any Intraocular surgery within 6 months.

Other illness

• Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function.
• Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
• Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).
• Any symptom of central nervous system involvement/disease that would impact the ability to measure visual function.
• Active ocular/intraocular infection (e.g., conjunctivitis, keratitis, scleritis, endophthalmitis).

Lab values

• 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.
• Considered unsuitable for any reason that may either place the patient at increased risk during participation or interfere with the interpretation of the study safety and efficacy outcomes by the Investigator, after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.
• 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.
• Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study safety and efficacy outcomes by the Investigator, after reviewing medical, ocular, and psychiatric history, clinical examination, and laboratory evaluation, as determined by the Investigator.

Age

• Any medical condition with life expectancy \< 6 years.
• Any medical condition with life expectancy \< 6 years.

Other

• Subject lacks evidence of outer nuclear layer.
• Any contraindications for subretinal injection.
• Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.
• Any contraindications for subretinal injection.
• Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception within the Treatment Observation Period.
• Subject lacks evidence of outer nuclear layer as determined by spectral-domain optical coherence tomography (SD-OCT).
• Any contraindications for subretinal injection.
• Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.
• Any contraindications for subretinal injection.
• Breast-feeding, pregnancy, or inability to practice strict contraception within the Treatment Observation Period for subjects of childbearing potential.

• Males or females ≥ 18 years of age at the time of informed consent.
• 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.
• 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.
• Males or females at least 18 years of age at the time of informed consent.
• Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
• Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.8 (light perception) in the study eye.
• Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).
• Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
• 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.
• BCVA ≤ 20/32 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.
• Males or females 6 - 17 years of age (inclusive) at the time of parental permission and/or assent, whichever is applicable.
• Clinical diagnosis of LCA and confirmed genetic diagnosis of CEP290 mutation.
• Best corrected visual acuity (BCVA) equal to or worse than LogMAR +0.7 but equal to or better than LogMAR 3.8 (light perception) in the study eye.
• Detectable outer nuclear layer in the macular region as determined by spectral-domain optical coherence tomography (SD-OCT).

Criteria the registry did not label

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