This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium.
Participants will receive a single administration of C.001 delivered by subretinal injection. The study will evaluate three dose levels in a sequential manner.
The primary objective is to assess the safety of C.001 over a 3-month period following administration. Participants will be followed for up to 12 months to further evaluate safety and explore changes in visual function and retinal structure.
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
Active or prior choroidal neovascularization (CNV)
Recent myocardial infarction or significant uncontrolled cardiovascular disease
Uncontrolled glaucoma
Significant ocular inflammatory disease
Sequential
Injection
Biological/VaccineC.001 administered once via subretinal injection
Incidence of Ocular and Systemic Adverse Events
Change in visual function measured in EDTRS letters
Findings of anatomic change on optical coherence tomography (OCT)
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Cellio Therapeutics Inc
Lead sponsor
California Institute for Regenerative Medicine (CIRM)
Collaborator
