Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

Simplified Inclusion Criteria: * Male or female between 8 and 70 years old (inclusive), with any visual acuity * Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1) * Has provided a genetic report indicating at least two ABCA4 disease-causing mutations. When only one ABCA4 disease-causing mutation is reported, sponsor's permission will be required. * At least one eye (called the "primary study eye") must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF), have decreased retinal sensitivity as measured by microperimetry, or have maculopathy expected to progress over the duration of the study * Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging * Healthy as judged by investigator * Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study * Has signed and dated the informed consent forms (or assent where appropriate) to participate * Female of childbearing potential has signed the informed consent about birth defects or attestation on contraception requirements Main Exclusion Criteria: * Has taken disallowed items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days * Is lactating, pregnant, or has a pos…