OC 30 - Hemophilia Gene Therapy Basic Science

Moderator(s)

• 

  David Lillicrap, MD

  Professor
  Department of Pathology and Molecular Medicine, Queen's University
  Kingston, Ontario, Canada
• 

  Radek Kaczmarek, PhD

  Assistant Research Professor of Pediatrics
  Department of Pediatrics, Indiana University School of Medicine
  Indianapolis, Indiana, United States

Presentations:

• 10:45 – 11:00 ET

  A Novel Strategy for Platelet-Specific Gene Therapy for the Treatment of Hemophilia A via Intranasal Delivery Lentiviral Vectors Containing Factor VIII
  

  Presenting Author: Cameron W. Rementer, PhD – Seattle Children's Research Institute

  Co-Author: Chong Li – Seattle Children's Research Institute - Seattle, Washington

  Co-Author: Chun-Yu Chen, PhD – Seattle Children's Research Institute

  Co-Author: Carol Miao – Seattle Children's Research Institute
• 11:00 – 11:15 ET

  Stable Factor VIII Expression and Improvement in Bleeding Phenotype Following Early Childhood Treatment with Adeno-Associated Viral Gene Therapy in the Severe Hemophilia A Dog Model
  

  Presenting Author: Paul Batty, MBBS, PhD (he/him/his) – University College London

  Co-Author: Alexandre Menard – Kingston Health Sciences Center

  Co-Author: Christine Brown – Queen's University

  Co-Author: Lorianne Harpell – Queen's University

  Co-Author: Ashrafali M. Ismail – BioMarin Pharmaceutical

  Co-Author: Aomei M. Mo – Queen's University

  Co-Author: Abbey Pender – Queen's University

  Co-Author: Bridget Yates – BioMarin Pharmaceutical

  Co-Author: Andrew Winterborn – Queen's University

  Co-Author: Sylvia Fong, PhD – BioMarin Pharmaceutical

  Co-Author: David Lillicrap, MD – Department of Pathology and Molecular Medicine, Queen's University
• 11:15 – 11:30 ET

  In vivo gene editing of hemophilia A mice using CRISPR/Cas9 mRNA LNP shows long-term expression of endogenous FVIII
  

  Presenting Author: Chun-Yu Chen, PhD – Seattle Children's Research Institute

  Co-Author: Xiaohe Cai – Seattle Children's Research Institute

  Co-Author: Carol Miao – Seattle Children's Research Institute
• 11:30 – 11:45 ET

  Efficacy and Safety of AAV Delivery of a Novel Factor VIII Variant with Improved Secretion in a Non-Human Primate Model
  

  Co-Author: Maria C. Seleme – The Children's Hospital of Philadelphia

  Presenting Author: Jonathan R. Lindgren, B.S. (he/him/his) – The Children's Hospital of Philadelphia

  Co-Author: Giang N. Nguyen – The Children's Hospital of Philadelphia

  Co-Author: Allysen C. Henriksen – The Children's Hospital of Philadelphia

  Co-Author: Charles-Antoine Assenmacher – School of Veterinary Medicine, University of Pennsylvania

  Co-Author: Amy Muehlmatt – Comparative Medicine Services Core, The Children's Hospital of Philadelphia

  Co-Author: Geary R. Smith – Comparative Medicine Services Core, The Children's Hospital of Philadelphia

  Co-Author: Melanie McFadden – Comparative Medicine Services Core, The Children's Hospital of Philadelphia

  Co-Author: Denise E. Sabatino, PhD (she/her/hers) – The Children's Hospital of Philadelphia
• 11:45 – 12:00 ET

  Expression and Activity of Human Factor VIII (hFVIII) Following Single and Repeat Administration of an Integrating Non-viral Gene Therapy in Multiple Mouse Models of Severe Hemophilia A
  

  Co-Author: Brian Truong, PhD (he/him/his) – Poseida Therapeutics

  Co-Author: Khal Hajj – Poseida Therapeutics

  Co-Author: Giang Nguyen – Children's Hospital of Philadelphia

  Co-Author: Johannes Schwerk – Poseida Therapeutics

  Co-Author: Jonnatan Tamayo – Poseida Therapeutics

  Co-Author: Daniel Smith – Poseida Therapeutics

  Co-Author: Manjunath Goolyam – Takeda Pharmaceutical Company

  Co-Author: Daniel Abernathy – Takeda Pharmaceutical Company

  Co-Author: Fernando Lopez Espinoza – Poseida Therapeutics

  Co-Author: Michelle Burrascano – Poseida Therapeutics

  Co-Author: James Yen – Poseida Therapeutics

  Co-Author: Kevin Briseno – Poseida Therapeutics

  Co-Author: Joseph Lucas – Poseida Therapeutics

  Co-Author: Pavan Battiprolu – Poseida Therapeutics

  Co-Author: Mike Bennett – Poseida Therapeutics

  Co-Author: Blair Madison – Poseida Therapeutics

  Presenting Author: Denise E. Sabatino, PhD (she/her/hers) – The Children's Hospital of Philadelphia

  Co-Author: Brent Warner – Poseida Therapeutics

  Co-Author: Eric Ostertag – Poseida Therapeutics

  Co-Author: Joshua Rychak – Poseida Therapeutics, Inc