Editing the Future: Gene Editing Tools and Technology

19 Jan 2023
New & Enabling Technologies
Mission Bio

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


Chris Brown
Head of Discovery
David Yoder
Senior Manager of Product Strategy
Jian Irish
President and Chief Operating Officer
Marco Ruella
Assistant Professor of Medicine in the Division of Hematology/Oncology and the Center for Cellular Immunotherapies
University of Pennsylvania
Matthew Cato
Senior Director Cell & Gene Therapy
Mission Bio
Raisa Lowe
Vice President, Head of CMC
Arbor Biotechnologies