Efficacy and Safety of Exagamglogene Autotemcel for Sickle Cell Disease

Speaker(s)

• 

  William Hobbs

  VP, Clinical Development
  Vertex Pharmaceuticals, Inc
  Lincoln, Massachusetts, United States

  Slides

Author(s)

• HF

  Haydar Frangoul, MD

  Medical Director, Pediatric Hematology/Oncology
  Sarah Cannon Center for Blood Cancer at The Children’s Hospital at TriStar Centennial, Tennessee, United States
• FL

  Franco Locatelli, MD

  Head of the Department of Paediatric Haematology and Oncology
  IRCCS, Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Lazio, Italy
• AS

  Akshay Sharma, MD

  Assistant Member, St. Jude Faculty
  Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Tennessee, United States
• MB

  Monica Bhatia, MD

  Department of Pediatrics
  Columbia University Irving Medical Center, New York – Presbyterian-Morgan Stanley Children’s Hospital, New York, United States
• MM

  Markus Y. Mapara, MD

  Department of Medicine, Division of Hematology/Oncology
  Columbia University, New York, United States
• LM

  Lyndsay Molinari, MD

  Sarah Cannon Pediatric Transplant and Cellular Therapy Program
  Methodist Children’s Hospital, Texas, United States
• DW

  Donna Wall, MD

  Section Head, Blood and Marrow Transplant/Cellular Therapy, Division of Haematology/Oncology
  The Hospital for Sick Children/University of Toronto, Ontario, Canada
• RL

  Robert Liem, MD

  Section Head, Hematology; Director, Comprehensive Sickle Cell Program
  Ann & Robert H. Lurie Children's Hospital of Chicago, Illinois, United States
• PT

  Paul Telfer, MD

  Royal London Hospital
  Barts Health NHS Trust
  London, England, United Kingdom
• AS

  Ami Shah, MD

  Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine
  Stanford University, California, United States
• MC

  Marina Cavazzana, MD

  Head of the Biological Therapy Department and the Biotherapy Clinical Investigation Center
  Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris, Ile-de-France, France
• SC

  Selim Corbacioglu, MD

  Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation
  University of Regensburg, Bayern, Germany
• DR

  Damiano Rondelli, MD

  Chief of the Division of Hematology/Oncology
  University of Illinois at Chicago, Illinois, United States
• RM

  Roland Meisel, MD

  Head of Division of Pediatric Stem Cell Therapy
  Heinrich-Heine-University, Nordrhein-Westfalen, Germany
• LD

  Laurence Dedeken, MD

  Haemato-oncology
  Hopital Universitaire des Enfants Reine Fabiola, Brussels Hoofdstedelijk Gewest, Belgium
• SL

  Stephan Lobitz, MD

  Head of Pediatric Hematology and Oncology Department
  Gemeinschaftsklinikum Mittelrhein, Rheinland-Pfalz, Germany
• Md

  Mariane de Montalembert, MD, PhD

  Medical Doctor
  Sickle Cell Center, Necker-Enfants Malades Hospital
  Paris, Ile-de-France, France
• MS

  Martin Steinberg, MD

  Professor, Pathology & Laboratory Medicine, Pediatrics, BU Chobanian & Avedisian School of Med
  Boston University, Massachusetts, United States
• MW

  Mark C. Walters, MD

  Director of the Pediatric Blood and Marrow Transplant Program
  UCSF Benioff Children's Hospital Oakland, California, United States
• SI

  Suzan Imren, MD

  Clinical Development Medical Director
  Vertex Pharmaceuticals, Inc, Massachusetts, United States
• DS

  Daoyuan Shi, n/a

  Biostatistics Associate Director
  Vertex Pharmaceuticals, Inc, Massachusetts, United States
• LB

  Laura Bower, n/a

  Patient Safety Senior Medical Director
  Vertex Pharmaceuticals, Massachusetts, United States
• CS

  Christopher Simard, n/a

  Vice President, Patient Safety, Medical Safety & Risk Mgmt
  Vertex Pharmaceuticals, Inc, Massachusetts, United States
• LZ

  Lanju Zhang, n/a

  Senior Director, Biostatistics
  Vertex, Massachusetts, United States
• PM

  Phuong Khanh Morrow, MD

  Chief Medical Officer
  CRISPR Therapeutics, California, United States

Corresponding Author(s)

• 

  William Hobbs

  VP, Clinical Development
  Vertex Pharmaceuticals, Inc
  Lincoln, Massachusetts, United States

  Slides

Author(s)

• 

  William Hobbs

  VP, Clinical Development
  Vertex Pharmaceuticals, Inc
  Lincoln, Massachusetts, United States

  Slides
• SG

  Stephan Grupp, MD

  Medical Director of the Cell and Gene Therapy Laboratory and Children's Hospital of Philadelphia
  Children's Hospital of Philadelphia, Pennsylvania, United States