The Galien Forum USA 2018

PANEL
5

Chair
Mikael Dolsten, MD, PhD
President Worldwide Research and Development
Pfizer Inc.

Panelists
Philip D. Gregory, PhD
Chief Scientific Officer
Bluebird bio

Katherine A. High
President and head of Research and Development
Spark Therapeutics

Brian Kaspar, PhD
Senior Vice President Scientific Founder and Chief Scientific Officer
AveXis, a Novartis Company

Peter Marks, MD, PhD
Director
Center for Biologics Evaluation and Research, FDA

Gaurav Shah, MD
President and Chief
Executive Officer
Rocket Pharmaceuticals Inc.

Panel Intro

The Promise of Gene Therapy


Mikael Dolsten

Mikael Dolsten, MD, PhD
President Worldwide Research and Development
Pfizer Inc.

Mikael Dolsten, MD, PhD, is President of Pfizer Worldwide Research and Development and Executive Vice President of Pfizer Inc., where he co-chairs the company’s Portfolio Strategy and Investment Committee. Previously, he was President of Wyeth Research and Executive Vice President at Boehringer Ingelheim. He has also held positions with AstraZeneca, Pharmacia, and Upjohn.

He is a Fellow of the New York Academy of Medicine and is a Member of the Board of Karyopharm Pharmaceuticals. He serves on the PhRMA Foundation Board of Directors, and is a member of the Government University Industry Research Roundtable. He is a named inventor on several patents and has published approximately 150 articles in international journals.


Philip D. Gregory, PhD
Chief Scientific Officer
Bluebird bio

Philip D. Gregory, D. Phil., has served as Chief Scientific Officer of bluebird bio since June 2015.  Prior to joining bluebird Philip was at Sangamo BioSciences, Inc., where he held multiple leadership positions, most recently serving as Chief Scientific Officer and Senior Vice President, Research. Philip has led pioneering research in gene therapy and genome editing and was named by Thompson Reuters as one of “The World’s Most Influential Scientific Minds 2014” and “Most Cited Researchers 2015, 2016 & 2017”.  He served as a member of the Scientific Advisory Board of the Keystone Symposia from 2009 to 2015, and on the 2016 ASGCT Advisory Council. Before joining the biotechnology industry Philip was at the University of Munich, Germany, where he studied the role of chromatin structure in gene regulation. Philip earned a D. Phil. in biochemistry from the University of Oxford (Keble College) and holds a B.Sc. in microbiology from the University of Sheffield.


Katherine A. High

Katherine A. High
President and head of Research and Development
Spark Therapeutics

Dr. Katherine A. High is a physician-scientist with a longstanding interest in gene therapy for genetic disease. As an Investigator of the Howard Hughes Medical Institute at the Children’s Hospital of Philadelphia (CHOP) and a faculty member of the University of Pennsylvania School of Medicine, she carried out pioneering basic and clinical studies in gene therapy for hemophilia and for a rare form of retinal dystrophy caused by mutations in the RPE65 gene. As Co-founder, President and Head of R&D at Spark Therapeutics, she led the development and regulatory approval of the first gene therapy for a genetic disease in the United States. Spark has shown human proof-of-concept of its gene therapy platform in both the retina and the liver, and has received breakthrough therapy designations for three different therapeutics, including for retinal dystrophy due to RPE65 mutations, and for hemophilia B and hemophilia A.

Dr. High is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, and is a Fellow of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London). She received her A.B. in chemistry from Harvard University, and her M.D. from the University of North Carolina School of Medicine. She completed training in internal medicine and hematology. In addition to her operational role, Dr. High serves as a member of the Board of Directors of Spark.


Brian Kaspar

Brian Kaspar, PhD
Senior Vice President Scientific Founder and Chief Scientific Officer
AveXis, a Novartis Company

Dr. Kaspar is Scientific Founder and Chief Scientific Officer at AveXis, Inc., a clinical-stage gene therapy company focused on the treatment of neurological diseases. He is a former Endowed Chair and Professor of Pediatrics at The Ohio State University and principal investigator at Nationwide Children’s Hospital. His work on gene therapy for spinal muscular atrophy was Science’s People’s Choice Award for 2017 Breakthrough of the Year. Dr. Kaspar. He is a Fellow of the American Association for the Advancement of Science (FAAAS) with over 100 peer reviewed manuscripts and book chapters. He serves on NIH Review Panels, a number of ASGCT committees, as well as serves on the editorial board of Molecular Therapy.


Peter Marks

Peter Marks, MD, PhD
Director
Center for Biologics Evaluation and Research, FDA

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.


Gaurav Shah

Gaurav Shah, MD
President and Chief Executive Officer
Rocket Pharmaceuticals Inc.

Gaurav is CEO & President of Rocket Pharmaceuticals, Inc. Previously, he was a Global Program Head in the Cell & Gene Therapies Unit at Novartis, where he had strategic oversight of 12 functions and helped spearhead pivotal trials with CART-19 for patients with leukemia and lymphoma. Earlier roles at Novartis included Global Clinical Program Head for CART-19 and for Biosimilars, and lead physician for Afinitor in several global oncology submissions. Gaurav began his career in industry at ImClone/Eli Lilly as a Medical Director overseeing oncology trials focused on monoclonal antibodies.

Gaurav is a graduate of Harvard College with a degree in Behavioral Neuroscience. He received his MD from Columbia (AOA), completed his internal medicine residency at Brigham and Women’s Hospital, and hematology/oncology fellowship training at Memorial-Sloan Kettering. After receiving board certification in medical oncology, he served as an Adjunct Assistant Professor of Oncology at Columbia.