Phase 1 Study of C.001 in Retinal Degeneration

This is a Phase 1, single-center, open-label, dose-escalation study designed to evaluate the safety and tolerability of C.001 administered via subretinal injection.

Participants will be enrolled into three sequential dose cohorts. A sentinel dosing strategy will be used, with safety review by an independent Data Safety Monitoring Board (DSMB) prior to escalation to higher dose levels. A minimum observation period of 4 weeks will be required between dose escalations.

Participants will be followed for up to 12 months after treatment for safety assessments and exploratory evaluation of visual and anatomical outcomes.

Inclusion criteria

• Age ≥18 years
• Diagnosis of retinal disease associated with retinal pigment epithelium degeneration (e.g., geographic atrophy or Stargardt disease)
• Visual acuity within protocol-defined limits
• Stable ocular condition prior to enrollment
• Medically suitable for ophthalmic surgery and anesthesia
• Willingness to comply with study procedures and follow-up

Exclusion criteria

• Active or prior choroidal neovascularization (CNV)
• Recent myocardial infarction or significant uncontrolled cardiovascular disease
• Uncontrolled glaucoma
• Significant ocular inflammatory disease
• Recent intraocular surgery within protocol-defined timeframe
• Active systemic infection
• Poorly controlled diabetes (e.g., HbA1c >8.5%)
• Participation in another investigational study within 3 months prior to screening