Summary of ocular adeno-associated virus (AAV) gene therapy studies

A trial was eligible for inclusion if the following criteria was met: (1) the study focused on patients with ocular diseases who received rAAV-based gene therapy; for publications, additional criteria were (2) study eyes for treatment are unilateral; (3) there was a clearly disclosed drug information and route of administration (ROA); and (4) the article reported the safety outcomes following gene therapy. If results from one trial were reported in several publications, data included in the ‘Dataset of ocular AAV gene therapy clinical studies.xlsx’ were extracted from the publication that was considered the most appropriate to avoid using duplicate data.

If a trial consisted of several phases and was reported separately in the publication, we included the publication from different phases separately. For trial with multiple publications, we list our reason for selection in the folder " Extraction process". For each study, extracted data included: (1) study characteristics (condition, age, sex, start date, region, phase, status, funder type, study design and enrollment); (2) interventions (study eye, dose, ROA, drug information, and prophylactic immunosuppression regimen); and (3) details of adverse event (AE) and/or a specific safety outcome and/or immune response.