CASE STUDY

The high natural prevalence of AAV wild-type viruses has resulted in a high frequency of capsid-directed humoral immunity in human populations and many animal models, which may interfere with the effectiveness of AAV-based gene therapies if not identified and managed properly. This case study describes the development of an anti-AAV antibody assay for use in preclinical studies that involve cynomolgus monkeys. This will allow the ability to screen for pre-existing AAV immunity and identify potential low-responding individuals to increase the likelihood of successful transduction and to maximize the utility of the preclinical investigations.