The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease

This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.

Inclusion Criteria:

• Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.
• Subjects must have a defined aggregate atrophic lesion in 1 or both eyes.
• Minimum BCVA is required in the study eye

Exclusion Criteria:

• Any ocular disease other than STGD1 that, in the opinion of the investigator, would complicate assessment of a treatment effect.
• History of ocular surgery in the study eye in the last 3 months.
• Any prior gene therapy.