A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

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.

• OpCT-001 (Biological): Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
• OpCT-001 (Biological): Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.

Primary outcomes

• Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit [From OpCT-001 administration through the Week 52 visit]

Secondary outcomes

• Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT) [From baseline through the Week 52 visit]

Sex: All, ages: 18 Years to any

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