Lead Sponsor

Alkeus Pharmaceuticals, Inc.

Age

8 - 70 years

Sex

ALL

Healthy Volunteers

No

•Clinical diagnosis of Stargardt disease (STGD1)
•Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
•Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
•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 been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
•Female of childbearing potential has signed the attestation on contraception requirements

•Is lactating or pregnant
•Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
•Has abnormal laboratory result(s) at screening
•Has an ocular disorder that may confound ocular assessments
•Has a history of ocular intervention within 90 days of screening