The Galien Forum USA 2019

PANEL
6

Chair
Kathy High, MD
President and Head of R&D
Spark Therapeutics

Panelists
Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research (CBER)
U.S. FDA

Maria Grazia Roncarolo, MD
Director
Center for Definitive and Curative Medicine (invited)

Robin L. Smith, MD, MBA
Founder
Neostem and Cura Foundation

 

Panel Intro

Delivering on the patient promise of gene therapy


Kathy High, MD
President and Head of R&D
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.  She also serves on the Board of Directors of CRISPR Therapeutics.


Peter Marks

Peter Marks, MD, PhD
Director, Center for Biologics Evaluation and Research (CBER)
U.S. 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.


Maria Grazia Roncarolo

Maria Grazia Roncarolo, MD
Director
Center for Definitive and Curative Medicine (invited)

Dr. Maria Grazia Roncarolo is George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine at Stanford School of Medicine. She co-directs the Institute for Stem Cell Biology and Regenerative Medicine and directs the Center for Definitive and Curative Medicine (CDCM) that aims to bridge the divide between laboratory discovery and translation to patients in stem cell and gene therapy.

She is an internationally renowned pediatric immunologist who has made seminal contributions to translational research in immunology and gene therapy. Her research studies have led to a greater understanding of the mechanisms underlying immune tolerance and have been of paramount importance to the development of novel cell and gene therapies for patients with genetic and acquired diseases of the hematopoietic and immune systems. Dr. Roncarolo has been the principal investigator for several stem cell and gene therapy trials, including the trial for Severe Combined Immunodeficiency (SCID) patients lacking adenosine deaminase (ADA), which led to European market authorization of Strimvelis.

In 2010 Dr. Roncarolo received the “Outstanding Achievement Award” from the European Society of Gence and Cell Therapy (ESGCT) for outstanding career and pioneering contributions to the field and in 2017 the “Outstanding Achievement Award” from the American Society of Gene & Cell Therapy (ASGCT) for a lifetime of significant scientific contributions to the field of gene and cell therapy.


Dr. Robin L. SmithRobin L. Smith, MD, MBA
Founder
Neostem and Cura Foundation

Dr. Robin L. Smith, age 54, is a business leader, entrepreneur, medical doctor and philanthropist and is uniquely positioned to assist public companies and health care systems successfully advance in the rapidly evolving health care industry. She received her BA from Yale, MD from the Yale School of Medicine and an MBA from the Wharton School of Business and completed the Stanford University Directors Program. Dr. Smith is a trailblazer in the fields of regenerative medicine and predictive analytics and has focused on turnarounds, M&A and disruptive innovations driving interest and growth.

In 2005, she was recruited to turn around Phase 3 Medical a bulletin board company with a non-sustainable plan. She became chairman of the board and chief executive officer in 2006 and created the NeoStem family of companies (NASDAQ: NBS) where she pioneered the company’s innovative business model: combining proprietary cell therapy development with successful contract development and manufacturing organization

In 2007, Dr. Smith founded the Stem for Life Foundation (SFLF), a nonpartisan 501(c)3 educational organization devoted to fostering global awareness of the potential for regenerative medicine to treat and cure a range of deadly diseases and debilitating medical conditions, as opposed to merely treating their symptoms. In 2016, under Dr. Smith’s leadership the foundation launched Unite To Cure, a global health movement that builds bridges among doctors, health systems, patients, business leaders, regulators, investors, politicians, philanthropists, and leaders of faith to collaborate and speed the development of cures, impact human health and save lives. The April 2018 global Unite To Cure event held at the Vatican, reached over 2.5 billion people standing alongside the World Economic Forum in Davos, Switzerland

Dr. Smith has coauthored two books: Cells Are the New Cure (2017) and The Healing Cell: How the Greatest Revolution in Medical History Is Changing Your Life (2013). She maintained a regular column for the Huffington Post for six years.