Our screening service includes optimised, robust, and quantitative bioassays for the preclinical screening of small molecules, AAV and viral vectors, siRNA and oligo therapeutic candidates and base editing approaches.

The novel platform can generate custom liver disease models and supporting bioassays that can accurately evaluate compound potency, efficacy, and delivery.

DefiniGEN’s disease modelled alpha-1 antitrypsin deficiency hepatocytes demonstrate dose-dependent drug response across a broad range of concentrations.