Foundation for Angelman Syndrome Therapeutics presents "Collaborative efforts toward an AAV gene replacement therapy for the treatment of
Angelman syndrome." 2019 FAST Science Summit - Dr Jim Wilson and John Crowley. Information presented at the 2019 FAST Summit and Gala, December 6, 2019.
John Crowley, the father of two children with a rare genetic disorder known as Pompe Disease,
will share his personal journey from drug discovery to the creation of Amicus Therapeutics, a
company focused on treating rare diseases. Amicus Therapeutics has recently partnered
with the University of Pennsylvania for various CNS gene therapy programs. The FAST-funded
Angelman syndrome program will be discussed as part of this collaboration.
BIOGRAPHIES
Jim Wilson, M.D., Ph.D., University of Pennsylvania
Rose H. Weiss Professor and Director, Orphan Disease Center
; Professor of Medicine and Pediatrics
; Director, Gene Therapy Program
; Perelman School of Medicine, University of Pennsylvania
; Advisor and Co-Founder of Scout Bio and Passage Bio; Co-Founder of Surmount Bio
James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he
has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare
diseases and ways to treat them by gene therapy. The Wilson lab identified a new type of vector based on novel isolates
of adeno-associated viruses which have become best in class for gene therapy being used by over 29 companies. More
recently Dr. Wilson’s laboratory has focused on improved vectors for gene therapy and clinical applications of genome
editing and mRNA therapy.
John F. Crowley, Chairman, Chief Executive Officer, Amicus Therapeutics
John F. Crowley is the Chairman, President and CEO of Amicus Therapeutics, Inc., a biotechnology company focused on
the development of novel treatments for human genetic diseases. John’s involvement with biotech stems from the 1998
diagnosis of two of his children with Pompe disease-a severe and often fatal neuromuscular disorder. In his drive to find a
cure for them, he left his position at Bristol-Myers Squibb and became an entrepreneur as the co-founder, president and
CEO of Novazyme Pharmaceuticals, a biotech start-up conducting research on a new experimental treatment for Pompe
disease (which he credits as ultimately saving his children’s lives.) In 2001, Novazyme merged into Genzyme Corporation
and John continued to play a lead role in the development of a drug for Pompe disease as Senior Vice President,
Genzyme Therapeutics. John and his family have been profiled in The Wall Street Journal and are the subjects of a book
by Pulitzer prize-winning journalist Geeta Anand, “The Cure: How a Father Raised $100 Million-And Bucked the Medical
Establishment- In a Quest to Save His Children.” The major motion picture, Extraordinary Measures, starring Brendan Fraser
and Harrison Ford, is inspired by the Crowley family journey. John is the author of a personal memoir: Chasing Miracles: The
Crowley Family Journey of Strength, Hope and Joy. John is also a commissioned officer in the U.S. Navy Reserve, assigned
to the United States Special Operations Command and is a veteran of the global war on terrorism, with service in
Afghanistan. He graduated with a B.S. in Foreign Service from Georgetown University, and earned a J.D. from the University
of Notre Dame Law School and an M.B.A. from Harvard. John is a Henry Crown Fellow at the Aspen Institute. He lives in
Princeton, NJ with his wife, Aileen and their three children, John, Megan and Patrick. John serves on the board of directors
of the National Make A Wish Foundation of America.
