April 15, 2021

Progress in Halting Pompe Disease

Date and time

April 15, 2021 ~ April 15, 2021

Dear Colleagues and Friends,

In commemorating International Pompe Day on April 15, we are proud to host a special webinar, “Progress in Halting Pompe Disease,” to raise awareness of not only the disease but of ongoing efforts for better care and cure. This is the fifth complimentary webinar in the rare disease series, where we convene physicians, drug developers, patients, and advocates to critically examine the current state of rare diseases that impact more than 300 million patients worldwide.

You are welcome to watch the on-demand video on WuXi AppTec YouTube page.

Agenda

08:00 AM - 08:05 AM Welcome
Hui Cai, Program Chair, VP and Head of Content, WuXi AppTec
08:05 AM - 08:25 AM Racing against Time in Rare Diseases
John Crowley, Chairman & CEO, Amicus Therapeutics
Moderator: Simone Fishburn, VP & Editor in Chief, BioCentury
08:25 AM - 09:10 AM Closing the Gap in Pompe Disease: Where Are We Heading
Tiffany House, Chairman & Board Member, International Pompe Association
Priya Kishnani, C.L. and Su Chen Professor of Pediatrics, Division Chief, Medical Genetics, Duke University Medical Center
Brad Crittenden, Executive Director, Canadian Association of Pompe
Jordi Díaz-Manera, Professor of Neuromuscular Disorders, Translational Medicine and Genetics Honorary Consultant in Clinical Genetics, Newcastle University
Moderator: Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec
09:10 AM - 09:50 AM Realizing the Promise of Gene Therapy in Pompe
Barry Byrne, Professor and Associate Chair, Director, Powell Gene Therapy Center, Department of Pediatrics, University of Florida
Edward Conner, Site Lead and Division Head of Gene Therapy Medical & Development, Astellas Gene Therapies
Geoff MacKay, President & CEO, AVROBIO
Moderator: David Chang, CEO, WuXi Advanced Therapies, WuXi AppTec

Speakers

Hui Cai
Program Chair, VP and Head of Content, WuXi AppTec
John Crowley
Chairman & CEO, Amicus Therapeutics
Simone Fishburn
VP & Editor in Chief, BioCentury
Tiffany House
Chairman & Board Member, International Pompe Association
Priya Kishnani
C.L. and Su Chen Professor of Pediatrics, Division Chief, Medical Genetics, Duke University Medical Center
Brad Crittenden
Executive Director, Canadian Association of Pompe
Jordi Díaz-Manera
Professor of Neuromuscular Disorders, Translational Medicine and Genetics Honorary Consultant in Clinical Genetics, Newcastle University
Richard Soll
Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec
Barry Byrne
Professor and Associate Chair, Director, Powell Gene Therapy Center, Department of Pediatrics, University of Florida
Edward Conner
Site Lead and Division Head of Gene Therapy Medical & Development, Astellas Gene Therapies
Geoff MacKay
President & CEO, AVROBIO
David Chang
CEO, WuXi Advanced Therapies, WuXi AppTec