The fast and the curious – optimized development of high performing AAV vectors

Thursday 08:00 PST / 11:00 EST / 16:00 GMT / 17:00 CET

Sponsor

The fast and the curious – optimized development of high performing AAV vectors

Adeno-associated virus (AAV) forms the basis of one of the most promising and versatile vector systems for therapeutic human gene transfer. The genetic simplicity and low immunogenicity, paired with high plasticity and amenability to engineer and repurpose the viral capsid and genome, make it a popular choice for gene therapy.

In this webinar, our distinguished guests, Dr. Dirk Grimm and Dr. David Schaffer will

Discuss the concept of using directed evolution to engineer highly optimized variants of AAV for a broad range of cell and tissue targets.

Introduce key technologies for targeted modification and directed evolution of critical AAV components

Address vector delivery barriers using directed evolution approaches

Attendees will discover novel approaches to:

Configure viral vectors to mitigate toxicity from dosage and immunogenicity
Prevent off-target viral delivery in healthy tissues
Leverage directed evolution strategies based on two fundamental steps of natural evolution: creation of a gene pool and selection of the fittest serotype
Select, create, and develop a top performing AAV viral vector to enable gene therapy

Dirk Grimm

Professor for Viral Vector Technologies, Medical Faculty, University of Heidelberg

Prof. Grimm is an expert in AAV vector design and engineering who pioneered the use of molecular evolution technologies for generation of AAV capsids with altered cell specificities and other improved properties. Located in the BioQuant building in the center of the Heidelberg University life sciences campus, his large team of scientists and technicians exploits these technologies to create novel AAV vectors for human gene therapies of diseases such as infection with viral or parasital pathogens, or of monogenic disorders affecting major organs such as skeletal muscle, heart or lung. Documented in over 80 peer-reviewed publications. Prof. Grimm is an integral member of several comprehensive and international research consortia such as the EU H2020 program MYOCURE, in which he and his team essentially contribute to the preclinical and clinical development of novel AAV-based human gene therapies.

David Schaffer

Chemical and Biomolecular Engineering, Bioengineering, Molecular and Cell Biology, and the Helen Wills Neuroscience Institute Director

Dr. Schaffer is a Professor of Chemical and Biomolecular Engineering, Bioengineering, and Neuroscience at University of California, Berkeley, where he also serves as the Director of the Berkeley Stem Cell Center and the Director of QB3-Berkeley.

Dr. Schaffer applies engineering principles to enhance stem cell and gene therapy approaches for neuroregeneration. This work includes mechanistic investigation of stem cell control, as well as molecular evolution and engineering of viral gene delivery vehicles. David Schaffer has received an NSF CAREER Award, Office of Naval Research Young Investigator Award, Whitaker Foundation Young Investigator Award, and was named a Technology Review Top 100 Innovator. He was also awarded the American Chemical Society BIOT Division Young Investigator Award in 2006, the Biomedical Engineering Society Rita Shaffer Young Investigator Award in 2000 and was elected to the College of Fellows of the American Institute of Medical and Biological Engineering in 2010.