This is an open-label, multicenter, active treatment study in approximately 12 subjects aged 18 to 55 years (inclusive) with STGD1, genotyped with a minimum of two ABCA4 gene mutations. Treatment course of STG-001 at Dose 1 (Cohort 1) or Dose 2 (Cohort 2) testing doses will be administered once daily for 28 days. Cohorts will run in parallel. Subjects will receive their designated dose daily for 28 days and be monitored for safety measures during the dosing period and for an additional 28 days after dosing (through Day 56).
This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.