The World Federation of Hemophilia Gene Therapy Registry

05 December 2020

Highlights From the 62nd ASH Annual Meeting and Exposition

The World Federation of Hemophilia Gene Therapy Registry

Barbara A. Konkle, MD^1,2, Donna Coffin, MSc³, Mayss Naccache¹, Robert Clark⁴, Lindsey George, MD⁵, Alfonso Iorio, MD, PhD⁶, Wolfgang A. Miesbach, MD⁷, Brian O'Mahony⁸, Flora Peyvandi^9,10, Steven W. Pipe, MD¹¹, Adrian Quartel¹², Eileen K Sawyer, PhD¹³, Mark W Skinner, JD¹⁴, Bartholomew J. Tortella, MD, MBA¹⁵, Crystal Watson, BS¹⁶, Leonard A. Valentino, MD¹⁷, Ian Winburn¹⁸, Johnny Mahlangu¹⁹, and Glenn F. Pierce, MD, PhD²⁰

¹World Federation of Hemophilia, Montreal, QC, Canada

²University of Washington, Washington Center for Bleeding Disorders, Seattle, WA

³World Federation of Hemophilia, Montréal, QC, Canada

⁴International Society on Thrombosis and Haemostasis, Inc, Carrboro

⁵The Children’s Hospital of Philadelphia, Philadelphia, PA

⁶McMaster University, Ontario, Canada

⁷Hemophilia Centre, University Hospital Frankfurt, Frankfurt, Germany

⁸Irish Haemophilia Society, Dublin, Ireland

⁹Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

¹⁰Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

¹¹Pediatrics and Pathology, University of Michigan, Ann Arbor, MI

¹²BioMarin Pharmaceutical Inc, Novato

¹³uniQure Inc, Lexington, MA

¹⁴Institute for Policy Advancement Ltd, Washington, DC

¹⁵Spark Therapeutics, Philadelphia, PA

¹⁶American Thrombosis and Hemostasis Network, Decatur, GA

¹⁷National Hemophilia Foundation (NHF), New York

¹⁸Pfizer Inc, New York

¹⁹Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg North, South Africa

²⁰World Federation of Hemophilia, La Jolla, CA

REVIEW PUBLICATION

REVIEW ABSTRACT

Key Data Points

WFH GTR CORE DATA SET

Following guidance of the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA), the WFH GTR SC has developed this core set of data to be collected on all patients with hemophilia, who receive gene therapy. Collection of data will be requested quarterly during the first year post-gene therapy infusion and annually thereafter, over the patient’s lifetime. Outreach to HTCs began in August 2020 with initiation visits beginning in October 2020. The registry will go live in January 2021.