OC 52 - Hemophilia Gene Therapy for Hemophilia B

Moderator(s)

• 

  Lindsey George, MD

  Assistant Professor of Pediatrics
  University of Pennsylvania
  Philadelphia, Pennsylvania, United States
• 

  Johnny Mahlangu, MBBCH, MMed

  Professor and Head of Department
  University of the Witwatersradn

Presentations:

• 10:45 – 11:00 ET

  Assessing the safety profile of AMT-060 and etranacogene dezaparvovec gene therapies across clinical trials in people with severe/moderately severe hemophilia B
  

  Presenting Author: Wolfgang Miesbach, MD – University Hospital Frankfurt, Frankfurt, Germany

  Co-Author: Michael Recht, MD, PhD, MBA (he/him/his) – National Hemophilia Foundation

  Co-Author: Nigel S. Key – Division of Hematology, University of North Carolina, Chapel Hill, NC, USA

  Co-Author: Roberto Guillen-Gonzalez – CSL Behring, King of Prussia, PA, USA;

  Co-Author: Krupa Sivamurthy – CSL Behring, King of Prussia, PA, USA

  Co-Author: Paul E. Monahan – CSL Behring, King of Prussia, PA, USA

  Co-Author: Steven W. Pipe, MD – University of Michigan, Ann Arbor, MI, USA
• 11:00 – 11:15 ET

  Residual burden of liver disease after HCV-clearance in hemophilia: a word of caution in the era of gene therapy.
  

  Presenting Author: Vincenzo La Mura, MD PhD – Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

  Co-Author: Niccolò Bitto – Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi and Thrombosis center, Milan, Italy

  Co-Author: Cecilia Capelli – Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi and Thrombosis center, Milan, Italy

  Co-Author: Camilla Caputo – Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi and Thrombosis center, Milan, Italy

  Co-Author: Simona Siboni – Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi

  Co-Author: Sara Arcudi, MD – Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi

  Co-Author: Alessandro Ciavarella, MD (he/him/his) – Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi

  Co-Author: Roberta Gualtierotti, MD, PhD – Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi

  Co-Author: Anna Ludovica Fracanzani – Department of Pathophysiology and Traplantation, University of Milan, Milan, Italy.

  Co-Author: Angelo Sangiovanni – Foundation I.R.C.C.S. Ca’ Granda, Ospedale Maggiore Policlinico, Gastroenterology and Hepatology, Milan, Italy.

  Co-Author: Flora Peyvandi, MD, PhD (she/her/hers) – IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy
• 11:15 – 11:30 ET

  Efficacy and Safety of Fidanacogene Elaparvovec in Adults with Moderately Severe or Severe Hemophilia B: Results from the Phase 3 BENEGENE-2 Gene Therapy Trial
  

  Presenting Author: Adam Cuker, MD, MS (he/him/his) – Department of Medicine and Department of Pathology & Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

  Co-Author: Hazzaa Alzahrani – Adult Haematology, King Faisal Specialist Hospital and Research Centre Riyadh, Saudi Arabia

  Co-Author: Jan Astermark – Department of Hematology Oncology and Radiation Physics, Skane University Hospital, and Institution of Translational Medicine, Lund University, Malmö, Sweden

  Co-Author: Laurent Frenzel – Department of Haematology, Institut Necker

  Co-Author: Olga Katsarou – Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Laiko General Hospital

  Co-Author: Kaan Kavakli – Department of Hematology, Ege University Children’s Hospital, Izmir, Turkey

  Co-Author: Robert Klamroth, MD, PhD (he/him/his) – Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology

  Co-Author: Davide Matino, MD (he/him/his) – Department of Medicine, Division of Hematology, McMaster University, Hamilton, ON

  Co-Author: Amy Shapiro – Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA

  Co-Author: Jiaan-Der Wang – Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital

  Co-Author: Joanne Fuiman – Pfizer Inc.

  Co-Author: John McKay – Pfizer Inc.

  Co-Author: Pengling Sun – Pfizer Inc.

  Co-Author: Jeremy Rupon, MD,PhD – Pfizer Inc.
• 11:30 – 11:45 ET

  The impact of steroids following gene therapy for haemophilia: results from the Exigency study
  

  Presenting Author: Simon Fletcher, MA, BA (Hons), Dip N, RGN (he/him/his) – Haemnet

  Co-Author: Kathryn Jenner – Haemnet

  Co-Author: Michael Holland – Haemnet

  Co-Author: Luke Pembroke – Haemnet, London, United Kingdom

  Co-Author: Kate Khair, RN, PhD – Haemnet, London, United Kingdom
• 11:45 – 12:00 ET

  Phase 3 HOPE-B trial of etranacogene dezaparvovec in severe/moderately severe hemophilia B: A post hoc responder analysis of participants who received full dose and responded to treatment
  

  Presenting Author: Steven W. Pipe, MD – University of Michigan, Ann Arbor, MI, USA

  Co-Author: Frank W.G. Leebeek, MD, PhD – Erasmus MC, University Medical Center Rotterdam, The Netherlands

  Co-Author: Michael Recht, MD, PhD, MBA (he/him/his) – National Hemophilia Foundation

  Co-Author: Nigel S. Key – Division of Hematology, University of North Carolina, Chapel Hill, NC, USA

  Co-Author: Susan Lattimore – Hemophilia Center, Oregon Health & Science University, Portland, OR, USA

  Co-Author: Giancarlo Castaman, MD – Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy

  Co-Author: Michiel Coppens, MD, PhD (he/him/his) – Amsterdam University Medical Centers, Amsterdam, The Netherlands

  Co-Author: Yanyan Li – CSL Behring, King of Prussia, PA, USA

  Co-Author: Paul E. Monahan – CSL Behring, King of Prussia, PA, USA

  Co-Author: Wolfgang Miesbach, MD – University Hospital Frankfurt, Frankfurt, Germany