AAV Vector Integration

All times below listed in ET

Session 1 – Introduction and Overview

1:00 – 2:20 p.m.

Overview: Knowns and Unknowns of AAV Integration Risk
Mark Kay, MD, PhD, Stanford University

The Impact of CRISPR Editing in Rodent CNS
Beverly Davidson, PhD, Children’s Hospital of Philadelphia

Session 2 – Nonclinical Models for Assessing Clinical Risk

2:50 – 3:50 p.m.

Humanized Mouse Liver Systems
Markus Grompe, MD, Oregon Health & Science University

Sequence-Based Methods for Quality Control and Assessing Outcome in Gene Therapy
Frederic Bushman, PhD, University of Pennsylvania

Break: 2:20 – 2:50 p.m.

Session 3 – Clinical and Regulatory Considerations for Assessing AAV Integration Risk

2:50 – 4:50 p.m.

AAV Vector Integration Behavior in Primary Human Hepatocytes in the FRG Mouse Liver
Ian Alexander, PhD, University of Sydney

Pathogenesis of HCC and Implications for Clinical Monitoring
Dean Felsher, MD, PhD, Stanford University

Genomics of Hepatocellular Carcinoma: Role of Viral Insertional Mutagenesis
Jessica Zucman-Rossi, MD, PhD, University of Paris Descartes

Considerations for Preclinical Development of AAV-Based Gene Therapy Products: A Regulatory Perspective
Gaya Hettiarachchi, PhD, FDA

Industry Panel – Considerations for Assessing Risk in Clinical Trials
Ricardo Dolmetsch, PhD, Uniqure
Kevin Eggan, PhD, BioMarin
Sam Wadsworth, PhD, Ultragenyx
Laurence Whiteley, DVM, Pfizer

Moderated by Federico Mingozzi, PhD, Spark Therapeutics