2022 Symposium in Genomic Medicine: Frontiers in Gene Editing
Event Information
The David Geffen School of Medicine and UCLA Institute for Precision Health hosted the first Annual Ginsburg Symposium in Genomic Medicine: Frontiers in Gene Editing on May 11, 2022 at UCLA's Carnesale Commons. This full-day symposium highlighted progress and challenges in the therapeutic applications of gene-editing technology and associated ethical and regulatory issues.
The symposium included a keynote speaker, panel discussion, posters and oral presentations. Students, postdocs, faculty, and staff from all campuses were invited to attend.
All graduate students and postdoctoral fellows were encouraged to submit an abstract for the poster session.
Agenda
| Time | Topic |
|---|---|
| 8:00am – 8:30am | BREAKFAST |
| 8:30am - 8:40am | WELCOME REMARKS Steven M Dubinett, MD Dr. Allen and Charlotte Ginsburg |
| 8:45am - 9:20am | Hematopoietic Stem Cell Gene Therapy-Add it or Edit? Donald Kohn, MD Distinguished Professor of Microbiology, Immunology and Molecular Genetics UCLA |
| 9:25am - 10:00am | Engineering Next-Generation CAR-T Cells for Cancer Immunotherapy Yvonne Chen, MD, PhD Associate Professor of Microbiology, Immunology, and Molecular Genetics UCLA |
| 10:00am - 10:15am | BREAK |
| 10:15am - 11:00am | PLENARY SPEAKER Base Editing and Prime Editing: Precision Gene Correction Without Double-Strand DNA Breaks David Liu, PhD Professor and Director of the Merkin Institute of Transformative Technologies in Healthcare Broad Institute |
| 11:00am - 12:00pm | KEYNOTE SPEAKER CRISPR Cures: Genome Editing as a New Biomedical Frontier Jennifer Doudna, PhD Li Ka Shing Chancellor's Chair in Biomedical and Health Sciences Professor, Depts. of Molecular & Cell Biology and Chemistry UC Berkeley |
| 12:00pm - 12:15pm | ANNOUNCEMENT: 2022 GINSBURG FELLOW Dr. Dan Geschwind, MD, PhD and Dr. Allen Ginsburg, MD |
| 12:15pm – 1:00pm | LUNCH |
| 1:00pm - 1:30pm | LIGHTNING ROUND: Trainee Presentations (3) |
| 1:35pm - 2:10pm | Fetal Therapy for Single-Gene Disorders Tippi C. Mackenzie, MD Professor and Director of the Eli and Edythe Broad Institute for Regenerative Medicine UCSF |
| 2:15pm - 2:50pm | Ethics and Governance of Human Genome Editing R. Alta Charo, JD Professor Emerita of Law & Bioethics University of Wisconsin – Madison |
| 2:50pm - 3:00pm | BREAK |
| 3:00pm - 3:20pm | Disrupting Duchenne: Advancing Precision Genetic Medicine through Science, Policy, and Practice Diane Berry, PhD Senior Vice President, Global Policy, Government & Patient Affairs Sarepta Therapeutics |
| 3:20pm - 3:40pm | Development of Treatments for Rare Diseases Emil D. Kakkis, MD, PhD Chief Executive Officer, President and Director Ultragenyx |
| 3:40pm - 4:00pm | FDA’s Efforts to Advance Gene Therapy Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration |
| 4:00pm - 4:10pm | BREAK |
| 4:10pm - 4:50pm | Meet the Experts: Frontiers in Ethical and Regulatory Perspectives Panelists: R. Alta Charo, JD, Diane Berry, PhD, Emil D. Kakkis, MD, PhD, Peter Marks, MD, PhD Moderator: Carrie Miceli, PhD, Professor of Microbiology Immunology and Molecular Genetics, UCLA |
| 4:50pm - 5:00pm | CLOSING REMARKS |
| 5:00pm - 6:30pm | WINE RECEPTION |
Jennifer Doudna, PhD
Dr. Jennifer A. Doudna is the Li Ka Shing Chancellor’s Chair and a Professor in the Departments of Chemistry and of Molecular and Cell Biology at the University of California, Berkeley. Her groundbreaking development of CRISPR-Cas9 as a genome-engineering technology, with collaborator Emmanuelle Charpentier, earned the two the 2020 Nobel Prize in Chemistry and forever changed the course of human and agricultural genomics research.
Symposium Speakers
David Liu, PhD
David R. Liu is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice chair of the faculty at the Broad Institute of Harvard and MIT, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator.
Donald B. Kohn, MD
Distinguished Professor of Microbiology, Immunology and Molecular Genetics
Pediatrics (Hematology/Oncology)
Molecular and Medical Pharmacology at the University of California, Los Angeles
Yvonne Chen, PhD
Dr. Yvonne Chen is an Associate Professor of Microbiology, Immunology, and Molecular Genetics at UCLA. She is the co-director of the Tumor Immunology program in the UCLA Health Jonsson Comprehensive Cancer Center at UCLA, and a member researcher of the Parker Institute for Cancer Immunotherapy.
Tippi MacKenzie, MD
Tippi MacKenzie is a Professor of Surgery at the University of California, San Francisco and the Director of the Eli and Edythe Broad Institute for Regeneration Medicine. She is a pediatric and fetal surgeon who is focused on developing better ways to diagnose and treat genetic diseases before birth.
R. Alta Charo, JD
Diane Berry , PhD
Senior Vice President, Global Policy, Government & Patient Affairs at Sarepta Therapeutics
Emil Kakkis, MD, PhD
A medical geneticist by training, Dr. Kakkis is a pioneer in the development and commercialization of treatments for rare diseases. He is founder and CEO at Ultragenyx, a biopharmaceutical company developing novel products for the treatment of rare and ultra-rare diseases.
Peter Marks, MD, PhD
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.