Dean's Workshop

Join us for a dean’s workshop on advanced genetic therapies, titled “End-to-End Precision Genetic Therapies: From Emerging Technologies to Patient Delivery” on Monday, Nov. 2, from 1 - 4:30 p.m.

This workshop brings together leaders from across Yale and the broader precision genetics community to discuss the latest advances in the field. Our scientists and clinicians, together with representatives from the NIH, regulatory and biopharmaceutical sectors, and the patient advocacy community, will discuss next-generation gene-editing and delivery platforms and their application in the clinic across rare genetic disorders.

The event will feature two keynote addresses:

P.J. Brooks, PhD, acting director of the Division of Rare Diseases Research Innovation at the NIH’s National Center for Advancing Translational Sciences, and co-chair of the NIH’s Bespoke Gene Therapy Consortium and Somatic Cell Genome Editing Programs, will open the workshop on the future of gene editing.

Peter Marks, MD, PhD, our former colleague and a former director of the FDA’s Center for Biologics Evaluation and Research, now senior vice president, Molecule Discovery and head of infectious disease at Eli Lilly, will deliver the closing keynote on the evolving regulatory landscape.

The afternoon concludes with a panel discussion, “Precision Genetic Medicine: From Promise to Practice,” convening regulatory, clinical, and patient-advocacy leaders to explore the infrastructure, regulatory, and health-system challenges of bringing these therapies to patients.

This hybrid event, hosted by the School of Medicine dean’s office, will take place in person in Brady Auditorium, B131 (310 Cedar Street), and via Zoom.

WELCOME, 1 – 1:05 PM
Nancy J. Brown, MD, Jean and David W. Wallace Dean of Medicine and C.N.H. Long Professor of Internal Medicine, Yale School of Medicine

GENE EDITING DELIVERY TECHNOLOGIES, 1:05 – 2:25 PM
Moderator: Monkol Lek, PhD, Associate Professor of Genetics, Yale School of Medicine

OPENING KEYNOTE: THE NIH SOMATIC CELL GENOME EDITING PROGRAM AND THE FUTURE OF GENE EDITING AS A THERAPEUTIC PLATFORM, 1:05 – 1:45 PM
P.J. Brooks, PhD, Acting Director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences, NIH; and Co-Chair of the NIH’s Bespoke Gene Therapy Consortium and Somatic Cell Genome Editing Programs

NON-VIRAL DELIVERY OF GENE THERAPY: POLYMERIC NANOPARTICLES, 1:45 - 2:05 PM
Mark W. Saltzman, PhD, Sterling Professor of Biomedical Engineering; Faculty Director, Center for Engineering Innovation and Design; and Deputy Dean for Academic Affairs, Yale Engineering; and Head, Jonathan Edwards College, Yale University

NON-VIRAL DELIVERY OF GENE THERAPY: LIPID NANOPARTICLES AND STEP, 2:05 - 2:25 PM
Jiangbing Zhou, PhD, Nixdorff-German Professor of Neurosurgery, Yale School of Medicine; and Professor of Biomedical Engineering, Yale Engineering

NETWORKING BREAK, 2:25 - 2:40 PM

GENE EDITING: PATH TO THE CLINIC, 2:40 - 4:00 PM
Moderator: Peter Glazer, MD, PhD, Robert E. Hunter Professor and Chair of Therapeutic Radiology, and Professor of Genetics, Yale School of Medicine

INNOVATIVE GENOME AND EPIGENOME EDITING–BASED THERAPIES FOR NEUROGENETIC DISORDERS, 2:40 - 3:00 PM
Yong-Hui Jiang, MD, PhD, Professor of Genetics, of Pediatrics, and of Obstetrics, Gynecology, and Reproductive Sciences, and Dorys McConnell Duberg Professor of Neuroscience, Yale School of Medicine; and Chief of Medical Genetics, Yale New Haven Health System

GENE-EDITING PLATFORMS TO ADDRESS CYSTIC FIBROSIS, 3:00 - 3:20 PM
Marie Egan, MD, Professor of Pediatrics and of Cellular and Molecular Physiology; Director, Cystic Fibrosis Center; Vice Chair of Research, Department of Pediatrics, Yale School of Medicine; and Chief, Pediatric Pulmonary, Allergy, and Sleep Medicine; Yale New Haven Health System

CLOSING KEYNOTE: A POTENTIAL REGULATORY FRAMEWORK FOR GENE-EDITING THERAPIES, 3:20 - 4:00 PM
Peter Marks, MD, PhD, Senior Vice President, Molecule Discovery and Head of Infectious Disease, Eli Lilly and Company

PANEL: PRECISION GENETIC MEDICINE: FROM PROMISE TO PRACTICE, 4:00 - 4:25 PM
Moderator: Marie Egan, MD, Professor of Pediatrics and of Cellular and Molecular Physiology; Director, Cystic Fibrosis Center; Vice Chair of Research, Department of Pediatrics, Yale School of Medicine; and Chief, Pediatric Pulmonary, Allergy, and Sleep Medicine; Yale New Haven Health System

A DISCUSSION OF INFRASTRUCTURE, REGULATION, AND CLINICAL SYSTEMS
Panelists:
Allyson Berent, DVM, Dipl. ACVIM, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics; and Chief Development Officer, AS²Bio
Beth Heinz, MHA, MSW, Senior Vice President, Women’s & Children’s Services, Yale New Haven Hospital
Lakshmanan Krishnamurti, MD, Professor of Pediatrics, Yale School of Medicine and Chief, Section of Pediatric Hematology/Oncology/BMT, Yale New Haven Health System
Peter Marks, MD, PhD, Senior Vice President, Molecule Discovery and Head of Infectious Disease, Eli Lilly and Company

CLOSING REMARKS, 4:25 – 4:30 PM
Nancy J. Brown, MD


The workshop is being recorded and will be available online after the event at https://medicine.yale.edu/about/deanoffice/workshop