Participate in Research at CU Anschutz

This is a prospective, observational, non-interventional, multi-center, global clinical study of subjects with early to advanced stage STGD1 caused by bi-allelic likely pathogenic or pathogenic variants in the ABCA4 gene, confirmed genotypically by an accredited genotyping laboratory. The study will consist of 6 visits over a 96-week study period: Visit 1 (Screening Visit), Visit 2 (Week 16), Visit 3 (Week 32) Visit 4 (Week 48), Visit 5 (Week 72), and Visit 6 (Week 96). Subjects who discontinue the study early will complete an Early Termination Visit. After Week 96, ongoing observation may be continued with approval of the subject and Sponsor. Subjects must have at least one eligible eye to participate in the study, defined as an eye that meets all relevant inclusion/exclusion criteria. All ophthalmology assessments will be conducted in both eyes, regardless of eligibility. Subjects participating in this observational study will be able to participate in future interventional studies conducted by the Sponsor with SB-007 gene therapy as long as they meet the entry criteria for the future relevant study. SB-007 is a novel dual-AAV gene therapy investigational medicinal product under development for the treatment of STGD1.