OC 11 - Gene editing/Engineering

Moderator(s)

• 

  Margareth Ozelo, MD, PhD

  Professor
  Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas
  Campinas, Sao Paulo, Brazil
• 

  Alastair Barraclough, MSc

  PhD Candidate
  Department of Pediatric Hematology, Amsterdam UMC, University of Amsterdam, Emma Children’s Hospital, Amsterdam, The Netherlands
  Amsterdam, Noord-Holland, Netherlands

Presentations:

• 09:30 – 09:45 ICT

  CRISPR-based gene editing for hemophilia: comprehensive analysis of off-target and on-target effects
  

  Presenting Author: Thierry VandenDriessche, PhD – Vrije Universiteit Brussel

  Co-Author: Kshitiz Singh – Vrije Universiteit Brussel

  Co-Author: Raffaele Fronza – ProtaGene CGT GmbH

  Co-Author: Marinee Chuah – Vrije Universiteit Brussel
• 09:45 – 10:00 ICT

  In vivo correction of a severe Hemophilia A patient mutation using CRISPR base editing
  

  Presenting Author: Yuanyi Xu – Seattle Children's Research Institute

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

  Co-Author: Meng-Ni Fan – Seattle Children's Research Institute

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

  Co-Author: Barbara A. Konkle – Washington Center for Bleeding Disorders and the University of Washington, Seattle, WA, USA

  Co-Author: Carol H. Miao – Seattle Children's Research Institute
• 10:00 – 10:15 ICT

  Development of BE-101, an autologous ex vivo precision gene engineered B cell medicine that produces active and sustained levels of factor IX for the treatment of hemophilia B
  

  Presenting Author: Wing Yen Wong, MD – Be Biopharma

  Co-Author: Hanlan Liu – Be Biopharma

  Co-Author: Swati Singh – Be Biopharma

  Co-Author: Timothy Mullen – Be Biopharma

  Co-Author: Caroline Bullock – Be Biopharma

  Co-Author: Sean Keegan – Be Biopharma

  Co-Author: Troy Patterson – Be Biopharma

  Co-Author: Amy Lundberg – Be Biopharma

  Co-Author: Sakshisingh Thakur – Be Biopharma

  Co-Author: Charuta Yadav – Be Biopharma

  Co-Author: Shamael Dastagir – Be Biopharma

  Co-Author: Lily Li – Be Biopharma

  Co-Author: Wayne Bainter – Be Biopharma

  Co-Author: Shalini Chilakala – Be Biopharma

  Co-Author: Anja Hohmann – Be Biopharma

  Co-Author: Adam Lazorchak – Be Biopharma

  Co-Author: Chris Scull – Be Biopharma

  Co-Author: Richard Morgan – Be Biopharma
• 10:15 – 10:30 ICT

  Base and Prime Editors as new correction approaches for recurrent Hemophilia A Nonsense Mutations
  

  Presenting Author: Maria Francesca Testa, PhD – Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara 44121, Italy

  Co-Author: Francesco Bernardi – Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara 44121, Italy

  Co-Author: Alessio Branchini – Dipartimento di Scienze della Vita e Biotecnologie, Università di Ferrara, Ferrara, Italy

  Co-Author: Mirko Pinotti – Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara 44121, Italy

  Co-Author: Dario Balestra – Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara 44121, Italy
• 10:30 – 10:45 ICT

  Lipid nanoparticles encapsulating FVIII transgene as re-dosable and safe gene therapy in hemophilia A
  

  Presenting Author: Meng-Ni Fan – Seattle Children's Research Institute

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

  Co-Author: Ting-Yen Chao – Seattle Children's Research Institute

  Co-Author: Yuanyi Xu – Seattle Children's Research Institute

  Co-Author: Carol H. Miao – Seattle Children's Research Institute