Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner.

Dose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.

Key Inclusion Criteria:

• Confirmed genetic diagnosis of primary photoreceptor (PR) disease
• Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.
• Retinal structure examination in the study eye demonstrating regions suitable for cell administration.

Key Exclusion Criteria:

• Clinically relevant, active ocular inflammation or infection
• Glaucoma or other significant optic neuropathy
• Diabetic macular edema or diabetic retinopathy
• Clinically significant cystoid macular edema
• In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia
• Ocular surgery ≤3 months before Screening
• Monocular vision (ie, no light perception in the fellow eye)
• Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.
• Any current and active infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression
• History of any cell therapy, gene therapy, or retinal implant at any time
• Previously received a bone marrow or solid organ transplant

Phase 1: None (Open Label) Phase 2: Investigators and study site personnel outside of the surgical team will be masked to dose level assignments