Steven W. Pipe, MD, Faculty Chair
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases,
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program,
Director, Special Coagulation Laboratory,
University of Michigan
Ann Arbor, Michigan


Kollet Koulianos, MBA
Senior Director Payer Relations
National Bleeding Disorders Foundation  

Kayla Douglas, PharmD, CSP
Director of Pharmacy
Mississippi Center for Advanced Medicine
Madison, MS

Michael Wang, MD
Hemophilia Treatment Center Clinical Director
Hemophilia & Thrombosis Center
University of Colorado Denver

Rebecca Kruse-Jarres, MD, MPH
Director, Washington Center for Bleeding Disorders
BloodWorks NW
Associate Professor of Medicine
University of Washington
Seattle, WA

Radek Kaczmarek, PhD
Postdoctoral Research Associate
Gene and Cell Therapy Group
Herman B. Wells Ctr for Pediatric Research
Indiana University School of Medicine
Indianapolis, IN


Christopher E. Walsh, MD, PhD
Associate Professor of Medicine, Division of Hematology and Medical Oncology
Icahn School of Medicine at Mount Sinai
Director, Hemophilia Treatment Center, Mount Sinai Hospital
New York, NY   

By the end of this session, the participant should be better able to:

  • Describe the latest developments in hemophilia gene therapy and practical considerations needed to identify eligible patients and meet their ongoing therapeutic needs
  • Review the logistics, precautions and staffing infrastructure needs for hemophilia treatment centers to adequately deliver and implement gene therapy infusions to patients with hemophilia
  • Describe alternative and potential non-viral approaches for addressing more challenging patients who face eligibility challenges to hemophilia gene therapy
  • Discuss the pharmacy considerations needed in receiving, storing or reconstituting a novel gene therapy product for hemophilia
  • Recognize the clinical concerns raised by Covid-19 and its effects upon bleeding disorder patients with compromised health and/or in gene therapy trials with added immunomodulating therapies.