Bioanalysis Zone

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


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 to highly diverse bioanalytical support of the AAV based therapeutics. This includes assessment of pre and post-treatment immunity, evaluation of post-treatment viral shedding and infectivity, as well as detection of transgene protein expression. An overview of the current regulatory recommendations as found in currently active and published draft FDA and EMA guidance or guideline documents is presented herein.

Keywords: Gene Therapy, immune response, neutralizing antibody, total antibody, infectivity, genome integration, viral vector shedding, transgene protein

In recent years a variety of Gene therapy (GTx) approaches using viral vectors to treat a variety of conditions have been quickly gaining momentum. Many of these gene therapies are aimed at treating rare genetic conditions by introducing a functional copy of a defective gene. Application of these approaches presents an opportunity for a transformative, potentially curative treatment able to correct the root condition caused by a genetic deletion or mutation in a critical gene. In addition to this increase in development of GTx for the treatment of various rare disease conditions, other applications have been increasingly explored, including treatment of cardiovascular diseases [1], Alzheimer, Parkinson’s and other chronic life threatening conditions [2, 3].

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