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With rising FDA approvals, gene therapy has made great strides in human clinical trials over the past decade, thanks largely to naturally occurring adeno-associated virus (AAV) vectors. However, given that the parent viruses of these vectors were not evolved by nature for application in medicine, they face several delivery barriers that limit their efficacy. Due to the complex mechanisms of virus-host interactions, there is currently insufficient mechanistic knowledge to enable the rational design of new vectors. As an alternative, David Schaffer’s lab at UC Berkeley has developed directed evolution to engineer highly optimized AAV variants for a broad range of cell and tissue targets.

In this keynote webinar, Professor Schaffer will describe how his team has engineered AAV variants with greatly improved delivery efficiency to multiple organs, as well as targeting delivery to specific cell types and building the capacity to evade immune responses. He will also provide an update on how these novel AAV variants are being deployed in eight clinical trials involving delivery to the retina, heart, and lung.

With the advent of CRISPR and other precision genome editing technologies offering the possibility of using gene delivery for repair or knockout of endogenous genes, Professor Schaffer will present data on combining engineered AAVs with CRISPR-Cas9 for a range of applications. The integration of these new technologies—AAV delivery or genome editing machinery—enables a broad range of exciting basic and therapeutic applications.

A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelist.