2023 ARCHIVES

PLENARY KEYNOTE SESSIONS

Monday, May 15 | 4:10 – 4:55pm

Carl-June Advances in CAR T Therapy
Carl June, MD, Richard W. Vague Professor in Immunotherapy; Professor of Medicine; Director, Center for Cellular Immunotherapies; Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine

Advances in the understanding of basic immunology have ushered in two major approaches for cancer therapy over the past 10 years. The first is checkpoint therapy to augment the function of the natural immune system. The second uses the emerging discipline of synthetic biology and the tools of molecular biology and genome engineering to create new forms of engineered cells with enhanced functionalities. The emergence of synthetic biology approaches for cellular engineering provides a broadly expanded set of tools for programming immune cells for enhanced function. Barriers to therapy of solid tumors will be discussed.

Monday, May 15 | 4:55 - 5:40pm

The Next Frontier in Machine Learning and Biologics: "Lab in a Loop" Large Molecule Drug Discovery, From Optimization to de novo Discovery
John C. Marioni, PhD, Senior Vice President and Head of Computation, Research and Early Development, Genentech

A key opportunity in applying machine learning to augment biologic drug discovery and development is through constant iteration – a process we call "lab in a loop." By developing integrated methods for optimizing affinity and multiple developability parameters, as well as a close integration of antibody engineering, machine learning, and structural biology, we have the potential to more rapidly identify and test novel candidate molecules. Sophisticated machine learning frameworks allow us to integrate later stages of optimization into the earliest stages of discovery, while high-throughput experimental systems allow rapid improvement of all methods and molecules. This process starts with the integration of people and scientific culture and ends with tightly integrated computational and experimental systems.

Wednesday, May 17 | 11:30am – 12:15pm

Advancing Innovative Biologics Modalities from Research to Clinical Application – Novel Platforms, Automation, and Computation
Rebecca A. Sendak, PhD, Head, Global Large Molecules Research Platform, Sanofi

Addressing disease biology in the clinic with protein therapeutics has become increasingly complex. Turning to innovative and novel scaffolds offers opportunities to tailor therapeutics not previously possible due to advances in host cell engineering and protein design approaches. Designing and developing these modalities requires a next-generation approach as we exploit increased potential design space and also growing data sources to leverage as we invent the next wave of therapeutics.

Wednesday, May 17 | 12:15 – 1:00pm

Andrew V. Anzalone, PhD PEGS 2023 YOUNG SCIENTIST KEYNOTE HONOREE
Engineering Prime Editor Proteins for Therapeutic Applications
Andrew V. Anzalone, MD, PhD, Director & Head, Prime Editing Platform, Scientific Co-Founder, Prime Medicine, Inc.

Precision gene editing technologies have the potential to address a wide range of genetic diseases. Prime Editing is a recently developed “search-and-replace” gene editing approach that can precisely perform a wide variety of DNA sequence edits at programmed target sites in human genomes without requiring double-strand DNA breaks or donor DNA templates. I will describe advances to prime editing technology that improve its efficiency, specificity, and capabilities for therapeutic applications.

PLENARY KEYNOTE BIOGRAPHIES

Carl June, MD, Richard W. Vague Professor in Immunotherapy; Professor of Medicine; Director, Center for Cellular Immunotherapies; Director, Parker Institute for Cancer Immunotherapy, University of Pennsylvania Perelman School of Medicine
Dr. June is the Richard W. Vague Professor in Immunotherapy in the Department of Pathology and Laboratory Medicine and is currently Director of the Center for Cellular Immunotherapies at the Perelman School of Medicine, and Director of the Parker Institute for Cancer Immunotherapy at the University of Pennsylvania. The CAR T cells invented in the June laboratory were awarded “Breakthrough Therapy” status by the FDA for acute leukemia in children and adults in 2014 and were approved by the FDA for acute leukemia in 2017 and afterwards, for diffuse large B cell lymphoma. These accomplishments have been recognized by the White House on several occasions. He has published more than 500 manuscripts and is the recipient of numerous honors, including election to the National Academy of Medicine, the National Academy of Sciences, the American Academy of Arts and Sciences and the American Philosophical Society.

Rebecca A. Sendak, PhD, Head, Global Large Molecules Research Platform, Sanofi
As Global Head of the Large Molecules Research Platform at Sanofi, Rebecca is responsible for the progression of the Large Molecules portfolio, which includes discovery, design, and generation of novel biologics from target validation to IND-filing. Sanofi’s innovative biologics portfolio includes antibodies, NANOBODY proteins, SYNTHORIN proteins, fusion proteins, multi-specific antibodies, and antibody-drug conjugates. Previously, Rebecca spent 20 years working in the area of Biologics Development, starting with Genzyme in 2000, and as part of Sanofi starting in 2011. During this time, she contributed to the successful development, commercialization, or post-marketing commitments for several biologics. From 2015 to 2018, she was responsible for the end-to-end Sanofi US CMC (Chemistry, Manufacturing and Controls) organization spanning cell line development through GMP (Good Manufacturing Practices) manufacture of Drug Product for clinical studies, including gene therapy applications. Rebecca holds a PhD in Biophysical Chemistry from Cornell University and earned her undergraduate degree in Biology and Chemistry from the University of Vermont.

John C. Marioni, PhD, Senior Vice President and Head of Computation, Genentech Research and Early Development, Genentech
John Marioni, PhD, is Senior Vice President and Head of Computation at Genentech Research and Early Development (gRED). In this role, Dr. Marioni oversees the development and application of computational approaches across gRED in the biological, chemical and clinical sciences. Previously, Dr. Marioni was Head of Research at the European Bioinformatics Institute (EMBL-EBI), an Associate Faculty at the Wellcome Sanger Institute and a Senior Group Leader at the CRUK Cambridge Institute within the University of Cambridge. John's personal research has focused on using computational approaches to study cell fate decisions in normal development and in disease.

YOUNG SCIENTIST KEYNOTE BIOGRAPHY

Andrew V. Anzalone, PhD, Director & Head, Prime Editing Platform, Scientific Co-Founder, Prime Medicine, Inc.
Andrew Anzalone is head of the prime editing platform at Prime Medicine, where he currently leads efforts to advance prime editing technologies for human therapeutic applications. Prior to joining Prime Medicine, Dr. Anzalone was a Jane Coffin Childs Memorial Fund Postdoctoral Fellow in the laboratory of David R. Liu at the Broad Institute of Harvard and MIT. In the Liu laboratory, Dr. Anzalone pioneered the development of prime editing, a novel search-and-replace gene editing technology that has the potential to correct the large fraction of known human genetic variants associated with disease. He completed his M.D. and Ph.D. degrees at Columbia University, where his thesis research in Virginia W. Cornish’s laboratory focused on chemical biology and synthetic biology. He received his Sc.B. degree in chemistry from Brown University, where he performed undergraduate research in synthetic organic chemistry.