A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)

The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.

Inclusion Criteria:

• Molecular diagnosis of Stargardt disease due to ABCA4 mutation
• Willingness to adhere to protocol per informed consent

Exclusion Criteria:

• Unwillingness to meet the requirements of the study
• Participation in a clinical study with another Investigation Medicinal Product
• Previous participation in another gene or cell therapy trial
• Any condition that would preclude subretinal surgery
• Complicating ocular and/or systemic diseases