Bioanalysis of adeno-associated virus gene therapy therapeutics: regulatory expectations

In this perspective article, published by Bioanalysis, the authors present the current regulatory recommendations for bioanalytical support of adeno-associated virus-based gene therapy development.  This article was published as part of the special focus issue, ‘The Exciting World of Oligonucleotides.’  Keywords: gene therapy, genome integration, immune response, infectivity, neutralizing antibody, total antibody, transgene protein, viral vector shedding Abstract: The number of gene therapy (GTx) modality therapies in development has grown significantly in the last few years. Adeno-associated virus (AAV)-based delivery approach has become most prevalent among other virus-based GTx vectors. Several regulatory guidelines provide the industry with general considerations related to AAV GTx development including discussion and recommendations related...