By Martin Lock, Director, Process Development, Gene Therapy Program — University of Pennsylvania
Recombinant adeno-associated virus (AAV) is rapidly becoming the vector of choice for human gene therapy applications due to the ability to achieve prolonged gene expression in a variety of tissues without a significant immune response or toxicity. Our laboratory has discovered a number of novel AAV serotypes with enhanced gene expression and alternate tissue tropisms and has pioneered the study of these vectors for gene delivery. Some of these novel serotypes have been tested as gene delivery vehicles in clinical trials with great success and the first AAV-based gene therapy product was recently approved in Europe for use in humans.
Large-scale manufacture of AAV vector for clinical trials requires a high degree of sophistication in both process design and lot release assays. Read how ddPCR represents a highly precise method to determine AAV vector genome titers and is not unduly influenced by inefficient amplification or standard curve issues.