AAVantgarde Bio Srl
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
Single subretinal administration