Day 2 - Genome Editing Consortium Public Workshop

9:00 – 9:10 am

Welcome & Opening Remarks 
Workshop Overview

9:10 – 10:15 am

Update from Federal Partners with Related Efforts 
(10 min each + Q&A) 

Updates on the CBER Regulatory Approach for Gene Therapies Incorporating Human Somatic Genome Editing
            • Anna Kwilas, Branch Chief, Gene Therapy Branch 4
              U.S. Food and Drug Administration, Center for Biologics Evaluation and Research (FDA CBER) 

FDA’s Oversight of Intentional Genomic Alterations in Genome-Edited Animals
            • Stella Lee, Biologist
              U.S. Food and Drug Administration, Center for Veterinary Medicine (FDA CVM)

The NIH’s Somatic Cell Genome Editing Program
            • Timothy LaVaute, Program Director
              National Institutes of Health, Somatic Cell Genome Editing (NIH SCGE)

Safety by Design – Predictable and Reliable Biological Engineering
            • Dr. Anne Cheever, Program Manager
              Defense Advanced Research Projects Agency (DARPA), Biological Technologies Office

ARPA-H Mission Overview
            • Amy Jenkins, PhD, Director, Health Science Futures Mission Office
              The Advanced Research Projects Agency for Health (ARPA-H) 

10:15 – 10:30 am

Break

10:30 am – 12:00 pm                     

Current NIST Genome Editing Consortium Activities 
Update and Discussion of Next Steps  

WORKING GROUP 1: SPECIFICITY MEASUREMENTS (50 min)
            Overview and Current Results of the First Consortium Interlab Study         
                        • Samantha Maragh, Leader
                        • Simona Patange, Biologist
                        • Ayah Shevchenko, Bioinformaticist
                          NIST Genome Editing Program

            Introduction to Single-Cell DNA+Protein Analysis for GE 
                        • Alex Li, Staff Scientist
                          Mission Bio, Inc.

            Introduction to Engineered Cell Lines Project
                        • Samantha Maragh, Leader
                          NIST Genome Editing Program

                       • Shu-Yu (Simon) Wu, Ph.D, Associate Director, Technical Operations & Project Management
                          Applied Stem Cell

WORKING GROUP 2: DATA AND METADATA  (30 min)
            Unlocking the Power of Genome Editing: The Critical Role of Data and Metadata
                        • Kendell Clement, Assistant Professor
                          University of Utah Department of Biomedical Informatic

                        • Sierra D. Miller, Bioinformaticist
                          NIST Genome Editing Program

                        • Arlin Stoltzfus, Biologist
                          NIST Data Sciences Group

WORKING GROUP 3: LEXICON  (10 min)
            ISO 5058 Genome Editing Part 1 Vocabulary and Next Steps
                        • Samantha Maragh, Leader
                          NIST Genome Editing Program 

12:00 – 1:00 pm

Lunch Break

1:00 – 2:40 pm 

Discussion of New Areas of Work for the Consortium Proposed by Members 
Current Approaches, Challenges, and Standards Needs 
(guided interactive discussions) 

QUALIFICATION OF OFF-TARGET ASSAYS  (40 min)
                       • Samantha Maragh, Leader
                         NIST Genome Editing Program

QUALITY OF GENOME EDITING COMPONENTS  (40 min)
                      • Samantha Maragh, Leader
                         NIST Genome Editing Program

            A Composite Approach to Guide-RNA Purity
                       • Alex Campbell, Scientist, Translational Biology 
                       • Lina Jamis, Assoc. Director, Regulatory Affairs 
                       • Puzhou Wang, Manager, Chemistry Data Sciences
                         Synthego Corporation

            Quality Considerations of Therapeutic Oligonucleotides
                       • Ashley Jacobi, Director, Applications & Market Development
                       • Gavin Kurgan, Bioinformatics Applications Manager 
                       • Ashleigh Dawley, Director, Regulatory Affairs & Compliance
                         Integrated DNA Technologies, Inc. (IDT)

EPIGENOME ENGINEERING MEASUREMENTS  (20 min)
                       • Samantha Maragh, Leader
                         NIST Genome Editing Program

            On the (Non) Cutting Edge: the Case for Standardization in the Field of Epigenetic Editing
                        • Ben Demaree, Senior Scientist
                          Scribe Therapeutics Inc.

2:40 – 3:00 pm

Break

3:00 – 4:50 pm 

Technology Talks
(10 min each)

Detection and Characterization of Low Frequency Mutational Events Induced During Genome Editing Using Duplex Sequencing
            • Jesse Salk MD, PhD, Founder of TwinStrand / Assistant Professor, Division of Oncology
              University of Washington

Indel Detection by Amplicon Analysis (IDAA) for Fast and Accurate Profiling of Programmable Nuclease Induced Genome Editing Outcomes
            • Eric Paul Bennett, M.Sc., Dr.med., Scientific Advisor / Co-founder of 
              COBO Technologies 

Robust Transgene Integration Site Analysis and Comprehensive Genetic QC of Genetically Modified Cells
            • Ellen Stelloo, Head of Assay and Product Development
              Cergentis BV

The FluidFM® OMNIUM: A Tool to Systematically Improve Genome Engineering
            • Dr. Tobias Beyer, CSO / Application Specialist
              Cytosurge AG

INDUCE-seq: Ensuring the Safe Development of Cell and Gene Therapies Created by Gene Editing
            • Simon Reed, Professor
              Cardiff University/Broken String Biosciences

Off-Target Quantification at Single Cell Resolution
            • Stephanie Scherer, PhD, Manager of Scientific Engagement
              Bioskryb Genomics, Inc.

How Your Bioinformatics Solutions Impact Results
            • Kuan-Fu Ding, Chief Science Officer
              Catalytic Data Science 

Population-Scale Off-Target Profiling of Therapeutic Gene Editors using ONE-seq
            • Douglas R. Smith, Ph.D., Sr. VP, Research & Development
              SeQure Dx, Inc.

Quality Assessment for Cell Bioprocessing by Optical Genome Mapping
            • Andy Wing Chun Pang, Senior Staff Scientist, Bioinformatics
              Bionano Genomics 

In-Vitro Characterization of Gene Editing Reagent Performance
            • Ross Bundy, President & CEO
              CRISPR/QC 

4:50 – 5:00 pm 

Re-cap of workshop & adjourn