3:30pm Chair Introduction

Jian Irish, President/Chief Operating Officer, Metagenomi

3:35pm Presentation: Growing the CRISPR Toolbox for Genetic Medicines

• Genomic editors discovered from machine learning/AI driven discovery platform
• Benefits of editing in ex vivo cell therapy

Raisa Lowe, Vice President, Head of CMC and Quality, Arbor Biotechnologies

3:50pm Presentation: Evolution of Gene Editing – Discovery to cGMP

• The serendipitous discovery of CRISPR has evolved into a cutting-edge gene & cell therapy tool over the span of less than three decades.  With therapeutic applications expanding to multiple modalities targeting both cancers and genetic disorders, a continued need for greater specificity is propelling the evolution of new technologies.
• The greatest challenges to moving these new technologies into the clinical space revolves around scale and quality required to move a program through late Phase and eventual commercialization
• Finding the right partner is critical to realizing the goal of delivering a therapy to a population in need in a timely manner

David Yoder, Senior Manager of Product Strategy, Aldevron

4:05pm Presentation: Expanding the Genome-Editing Toolbox

• Unlocking the potential of the world’s natural microbial environment for the development of genome editing tools
• Rapidly identifying and enhancing natural enzyme systems for translation into therapeutic applications
• Developing highly active and specific gene editing tools that are driving in vivo and ex vivo clinical development

Chris Brown, Director of Discovery, Metagenomi

4:20pm Presentation: Single-cell Precision Measurement of Cell and Gene Therapy Genome Editing Protocols

• Single-cell analysis on the Tapestri platform simultaneously identifies edits, zygosity, co-occurrence, and translocations, enabling validation of gene editing platforms for therapeutic development
• Optimize genome editing protocols during development for control during production manufacturing and release testing
• Replace multiple traditional bulk assays with one single-cell sequencing assay

Matthew Cato, Senior Director Cell & Gene Therapy, Mission Bio

4:35pm Closing Panel with Q&A

With all session participants, joined by: 

Marco Ruella, Assistant Professor of Medicine in the Division of Hematology/Oncology and the Center for Cellular Immunotherapies, University of Pennsylvania