John will discuss his views of the accomplishments, promises and challenges with using AAV as a delivery platform for gene therapy.
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The Biopharmaceutical Engineering Research Interest Group will welcome John Kerwin, PhD, Head of Technical Development at Ascidian Therapeutics, who will provide insights to the question: Are We There Yet: AAV beyond Gene Replacement. John will discuss his views of the accomplishments, promises and challenges with using AAV as a delivery platform for gene therapy. Bring your questions and engage in the discussion! Refreshments will be served.
Talk Summary:
Adeno-associated virus (AAV) is an established delivery vehicle for gene therapy in the treatment of a variety of human diseases. AAV has become a prominent viral vector due to its minimal pathogenicity and ability to establish long-term gene expression in different tissues and cell types. With several approved AAV-based gene therapies targeting gene replacement, developers and innovators seek to expand the application of the vector to the fields of gene editing, modulation, and modification. However, these fields warrant next-generation AAV vectors with enhanced sensitivity and specificity, while aiming to significantly reduce the cost of manufacturing. This talk will review the current technology and identify the critical challenges of AAV as a delivery vehicle for expanded applications beyond gene replacement.