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

Gene therapy using adeno-associated virus (AAV) as a vector represents a novel therapeutic modality to treat ocular diseases by transferring engineered genetic material into target cells for the purpose of preventing, halting or reversing pathological processes.

Despite the enormous advantages of AAV-based gene therapy, there are still concerns about long-term efficacy and safety issues. Given the rising of this groundbreaking approach, we summarized a dataset of ocular AAV gene therapy studies (trials/publications).

Data source: ClinicalTrials.gov, Embase and PubMed.

Search strategy: Strategies are provided in the folder " Extraction process".

Data dictionary: Unifying each variable, a normalized list of data items is provided in the folder " Extraction process".

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.