Gene Editing and Gene Therapy: The Next Frontier in Human Health

Co-chairs: Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx; Stephen Squinto, PhD, Interim CEO, Passage Bio

11:50-11:55 Opening by Co-chairs

11:55-12:20 Adi Barzel, PhD, Senior Lecturer, Department of Biochemistry at Tel Aviv University; Co-Founder of LogicBio Therapeutics, Inc.; President of the Israeli Society of Gene and Cell Therapy: “Principles of Gene Editing; CRISPR in B Cells Fighting AIDS”

12:20-12:40 Ulrich Brinkmann, PhD, Scientific Director, Roche Pharma Research & Early Development, Roche Diagnostics GmbH: “Novel Approaches for Antibody-targeted Intracellular Delivery of Large Nucleic Acids incl. CRISPR Mediated Gene Editing Modules”

12:40-13:00 Prof. Nadav Ahituv, PhD, Department of Bioengineering and Therapeutic Science, University of California San Francisco: “CRISPRa Therapeutics for Haploinsufficient Disease”

13:00-13:20 Rachel Haurwitz, PhD, Co-founder, President and CEO, Caribou Biosciences

13:20-13:35 Kyle Chiang, PhD, VP Product Strategy, LogicBio Therapeutics: “In Vivo Genome-editing for Rare Pediatric Diseases”

13:35-13:50 David Baram, PhD, Chief Executive Officer, Emendo Bio Inc: “Emendo Biotherapeutics – High Precision Gene Editing with Next Generation CRISPR Nucleases”

13:50-15:20 Lunch Break

15:20-15:45 Ronald G. Crystal, MD, Founder, LEXEO Therapeutics and Chair, Department of Genetic Medicine, Weill Cornell Medical College: “Gene Therapy for CNS Disorders”

15:45-16:05 Stephen Squinto, PhD, Interim CEO, Passage Bio: “Passage Bio: Developing Transformative Genetic Medicines for Rare Monogenic CNS Diseases”

16:05-16:25 Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx; “Using Gene Therapy for the Treatment of Rare Genetic Liver Disease”

16:25-16:40 Trevor Martin, PhD, CEO and Co-Founder, Mammoth Biosciences: “The Next Generation of CRISPR Tools”

16:40-16:55 Dalia El-Ani, PhD, Chief Executive Officer, CRISPR Stem & Therapeutics: “CRISPR: A Novel Gene Therapy to Protect the Heart from Acute Ischemic Damage”

16:55-17:40 “Panel Discussion
Panelists:
Ronald G. Crystal, MD, Founder, LEXEO Therapeutics and Chair, Department of Genetic Medicine, Weill Cornell Medical College
Rachel Haurwitz, PhD, Co-founder, President and CEO, Caribou Biosciences
Prof. Nadav Ahituv, PhD, Department of Bioengineering and Therapeutic Science, University of California San Francisco
Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx
Stephen Squinto, PhD, Interim CEO, Passage Bio