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.

: Time Topic
8:00am – 8:30am BREAKFAST
8:30am - 8:40am
WELCOME REMARKS
Daniel Geschwind, MD, PhD
Distinguished Professor and Associate Vice Chancellor, Precision Health
UCLA
Steven M Dubinett, MD
Interim Dean, David Geffen School of Medicine
UCLA
Dr. Allen and Charlotte Ginsburg
Honored Guests
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

Time	Topic
8:00am – 8:30am	BREAKFAST
8:30am - 8:40am	WELCOME REMARKS Daniel Geschwind, MD, PhD Distinguished Professor and Associate Vice Chancellor, Precision Health UCLA Steven M Dubinett, MD Interim Dean, David Geffen School of Medicine UCLA Dr. Allen and Charlotte Ginsburg Honored Guests
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

Symposium Photo Gallery

Ginsburg Symposium 2022

11 Gallery Items

Keynote Speaker

Jennifer Doudna, PhD

Professor, Depts. of Molecular & Cell Biology and Chemistry, UC Berkeley Founder, Innovative Genomics Institute

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

Broad Institute, Harvard University, and HHMI.

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

Pediatric Hematologic Malignancy

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

Associate Professor of Microbiology, Immunology, and Molecular Genetics at the University of California, Los Angeles

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

Professor of Surgery of University of California San Francisco Division of Pediatric Surgery and the Fetal Treatment Center

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

Principal, Alta Charo Consulting LLC and Knowles Professor Emerita of Law & Bioethics, University of Wisconsin – Madison

Diane Berry , PhD

Senior Vice President, Global Policy, Government & Patient Affairs at Sarepta Therapeutics

Emil Kakkis, MD, PhD

Chief Executive Officer, President and Director of Ultragenyx

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

Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration

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.