OC 02 - Gene therapy - Clinical and pre-clinical

Moderator(s)

• 

  Robert Klamroth, MD

  Head of Department
  Vivantes Klinikum, Friedrichshain, Berlin, Germany
  Berlin, Berlin, Germany
• 

  Paul Batty, MBBS PhD

  Associate Professor
  Cancer Institute, University College London, London, UK
  London, England, United Kingdom

Presentations:

• 13:00 – 13:15 ICT

  Kinetic profiles of anti-AAV5 antibody generation and clearance in neonatal, infant, and adult hemophilia A dogs
  

  Presenting Author: Paul Batty, MBBS PhD – Cancer Institute, University College London, London, UK

  Co-Author: Britta Handyside – BioMarin Pharmaceutical, Novato, United States

  Co-Author: Laura Swystun – Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada

  Co-Author: Aomei Mo – Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada

  Co-Author: Lori Harpell – Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada

  Co-Author: Abbey Pender – Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada

  Co-Author: Andrew Winterborn – Animal Care Services, Queen's University, Kingston, Canada

  Co-Author: Sylvia Fong – BioMarin Pharmaceutical

  Co-Author: David Lillicrap, MD – Molecular Hemostasis Research Group, Department of Pathology and Molecular Medicine, Queen's University, Kingston, ON, Canada.
• 13:15 – 13:30 ICT

  Knock-in genome editing therapy with a single AAV vector harboring an engineered AsCas12f for hemophilia B in mice
  

  Presenting Author: Tomoki Togashi, Master – Department of Biochemistry, Jichi Medical University School of Medicine

  Co-Author: Tomohiro Hino – Department of Cardiovascular Medicine, Graduate School of Medical Science, Kyoto Prefectural University of Medicine

  Co-Author: Satoshi N. Omura – Department of Biological Sciences, Graduate School of Science, The University of Tokyo

  Co-Author: Ryoya Nakagawa – Department of Biological Sciences, Graduate School of Science, The University of Tokyo

  Co-Author: Atsushi Hoshino – Department of Cardiovascular Medicine, Graduate School of Medical Science, Kyoto Prefectural University of Medicine

  Co-Author: Osamu Nureki – Department of Biological Sciences, Graduate School of Science, The University of Tokyo

  Co-Author: Tsukasa Ohmori – Department of Biochemistry, School of Medicine, Jichi Medical University
• 13:30 – 13:45 ICT

  Etranacogene dezaparvovec hemophilia B gene therapy phase 2b trial final results: stable and durable FIX level expression over 5 years
  

  Co-Author: Annette von Drygalski, MD, PharmD, RMSK – University of California San Diego

  Co-Author: Adam Giermasz – Division of Hematology/Oncology, Hemophilia Treatment Center, University of California Davis

  Co-Author: Esteban Gomez – Center for Inherited Blood Disorders

  Co-Author: Paul E. Monahan – CSL Behring

  Presenting Author: Sandra Le Quellec, MD, PhD – CSL Behring

  Co-Author: Steven W. Pipe, MD – University of Michigan
• 13:45 – 14:00 ICT

  Long-Term Follow-Up of Participants in the Phase I/II Trial of Dirloctocogene Samoparvovec (SPK-8011): Durable FVIII Expression and Clinically Meaningful Reduction of Bleeding
  

  Co-Author: Lindsey A. George – Division of Hematology, Children's Hospital of Philadelphia

  Presenting Author: Stacy E. Croteau, MD, MMS – Boston Children’s Hospital

  Co-Author: Tiffany Chang – Spark Therapeutics, Inc.

  Co-Author: M. Elaine Eyster – Department of Medicine, Division of Blood and Vascular Disorders, Penn State Health Milton S. Hershey Medical Center

  Co-Author: Huyen Tran, MBBS (Hons), MClinEpi, FRACP, FRCPA – Ronald Sawyers Haemophilia Centre, The Alfred Hospital, Melbourne, Victoria, Australia

  Co-Author: Margaret V. Ragni – Department of Medicine, University of Pittsburgh

  Co-Author: Spencer Sullivan – Mississippi Center for Advanced Medicine

  Co-Author: John E. J. Rasko – Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital

  Co-Author: Jill Moormeier – Department of Medicine, UMKC School of Medicine

  Co-Author: Pantep Angchaisuksiri – Department of Medicine, Ramathibodi Hospital, Mahidol University

  Co-Author: Jerome Teitel – St Michael's Hospital Haemophilia Treatment Centre, University of Toronto

  Co-Author: Gili Kenet – National Hemophilia Center Sheba Medical center, Tel Hashomer & Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Israel,

  Co-Author: Tung Wynn, MD – Division of Hematology and Oncology, Department of Pediatrics, University of Florida

  Co-Author: Benjamin Samelson-Jones – Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia

  Co-Author: Savina Jaeger – Spark Therapeutics, Inc.

  Co-Author: Julia Ramos – Spark Therapeutics, Inc.

  Co-Author: Federico Mingozzi – Spark Therapeutics, Inc.

  Co-Author: Gallia Levy – Spark Therapeutics, Inc.