David Schaffer
Hubbard Howe Jr. Distinguished Professor, Chemical and Biomolecular Engineering, Bioengineering, Molecular and Cell Biology, and the Helen Wills Neuroscience Institute University of California at Berkeley
Each year, the previous year's Andreas Acrivos Award for Professional Progress in Chemical Engineering recipient is invited to deliver a special plenary. The plenary session is typically chaired by the previous lecturer (winner from two years previous).
The 2021 Andreas Acrivos Award Lecture will be delivered by 2020 award recipient David V. Schaffer, Hubbard Howe Professor of Chemical and Biomolecular Engineering, University of California at Berkeley. The topic of the lecture is "Directed Evolution of New Adeno-Associated Viral Vectors for Clinical Gene Therapy".
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About the Andreas Acrivos Award for Professional Progress in Chemical Engineering
In honor of one of the chemical engineering profession’s most influential leaders and one of the great fluid dynamacists of the 20th century, the American Institute of Chemical Engineers (AIChE) has renamed the Professional Progress Award the Andreas Acrivos Award for Professional Progress in Chemical Engineering.
The award recognizes outstanding progress in the field of chemical engineering. The awardee will have made a significant contribution to the science of chemical engineering through one of the following means:
- A theoretical discovery or development of a new principle in the chemical engineering field.
- Development of a new process or product in the chemical engineering field.
- An invention or development of new equipment in the chemical engineering field.
- Distinguished service rendered to the field or profession of chemical engineering.
The recipient is invited to deliver an address at the next Annual Meeting.
Directed Evolution of New Adeno-Associated Viral Vectors for Clinical Gene Therapy
David V. Schaffer, Hubbard Howe Professor of Chemical and Biomolecular Engineering, University of California at Berkeley
Gene therapy – the delivery of genetic material to the cells of a patient for therapeutic benefit – has been increasingly successful in human clinical trials over the past decade, and there are currently five FDA-approved gene therapies. The most successful gene delivery vehicles, or vectors, are based on adeno-associated viruses (AAV); however, vectors based on natural versions of AAV face a number of delivery barriers that limit their efficacy and will thus preclude the extension of these successes to the majority of human diseases. These delivery limitations arise since the parent viruses upon which these vectors are based were not evolved by nature for our convenience to use as human medicine. Unfortunately, due to the highly complex mechanisms of virus-host interactions, there is currently insufficient mechanistic knowledge to enable rational design to be sufficiently successful in creating new vectors. As an alternative, however, we developed the concept of using directed evolution to engineer highly optimized variants of AAV for a broad range of cell and tissue targets. Directed evolution involves the iterative genetic diversification of a biomolecule to create a gene pool and functional selection to isolate variants with optimal properties. Using this approach, we have engineered AAV variants with greatly improved delivery efficiency to multiple organs including the retina; lung, and muscle; targeted delivery to specific cell types; and the capacity to evade immune responses. Our novel AAV variants are currently used in 5 human clinical trials involving delivery to the retina and heart, and additional products will enter into the clinic this year.
In parallel, the advent of genome editing technologies such as the CRISPR/Cas9 system raise the possibility of using gene delivery not only for gene replacement but for repair or knockout of endogenous genes. We have thus been combining engineered AAVs with CRISPR/Cas9 for a range of applications. The integration of these new technologies – AAV delivery or genome editing machinery – can enable a broad range of basic and therapeutic applications.