Ali T. Taher, MD, PhD, FRCP¹, Vip Viprakasit^2*, Olivier Hermine^3,4, John B. Porter, MA, M.D., FRCP, FRCPath^5*, Antonio Piga, MD⁶, Kevin H.M. Kuo, MD, MSc, FRCPC⁷, Thomas D. Coates, MD^8,9, Ersi Voskaridou^10*, Abderrahim Khelif^11*, Antonis Kattamis¹², Jeevan K. Shetty^13*, George Zhang^14*, Yu (Olivia) Tian^14*, Dimana Miteva^13*, Tatiana Zinger^13*, Derek Tang^14*, Jay T. Backstrom^15* and Maria Domenica Cappellini¹⁶

¹Department of Internal Medicine, American University of Beirut - Medical Center Raid El Solh, Beirut, Lebanon
²Siriraj Hospital, Mahidol University, Bangkok, Thailand
³Imagine Institute, INSERM U1163, University of Paris, Paris, France
⁴Department of Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
⁵University College London, University College London Hospitals, London, United Kingdom
⁶Department of Clinical and Biological Sciences, University of Turin, Turin, Italy
⁷Division of Medical Oncology and Hematology, Department of Medicine, University Health Network, Division of Hematology, Department of Medicine, University of Toronto, Toronto, ON, Canada
⁸Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, CA
⁹USC Keck School of Medicine, Los Angeles, CA
¹⁰Thalassemia and Sickle Cell Center of Laiko General Hospital, Athens, Greece
¹¹Farhat Hached Teaching Hospital, Sousse University, Tunisia
¹²First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
¹³Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
¹⁴Bristol Myers Squibb, Princeton, NJ
¹⁵Acceleron Pharma, Cambridge, MA
¹⁶Fondazione IRCCS Ca’ Granda Policlinico Hospital, University of Milan, Milan, Italy

Matthew P Parker, BS^1*, Halyna Fedosyuk, MS^1*, Lesya V Novikova, PhD^1*, Chad Slawson, PhD^1* and Kenneth R Peterson, PhD²

¹Department of Biochemistry and Molecular Biology, University of Kansas Medical Center, Kansas City, KS
²Department of Biochemistry and Molecular Biology, Univ. of Kansas Medical Center, Kansas City, KS

Olivier Hermine, MD, PhD^1,2*, Maria Domenica Cappellini³, Ali T. Taher, MD, PhD, FRCP^4,5, Thomas D. Coates, MD^6,7, Vip Viprakasit^8*, Ersi Voskaridou^9*, Ashutosh Lal, MD¹⁰, Hong Keng Liew^11*, Silverio Perrotta^12*, Abderrahim Khelif^13*, Antonis Kattamis¹⁴, Jeevan K. Shetty^15*, George Zhang^16*, Yu (Olivia) Tian^16*, Dimana Miteva^15*, Tatiana Zinger^15*, Derek Tang^16*, Jay T. Backstrom^17* and John B. Porter, MA, M.D., FRCP, FRCPath^18*

¹Imagine Institute, INSERM U1163, University of Paris, Paris, France
²Department of Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
³Fondazione IRCCS Ca’ Granda Policlinico Hospital, University of Milan, Milan, Italy
⁴Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon
⁵Department of Internal Medicine, American University of Beirut - Medical Center Raid El Solh, Beirut, Lebanon
⁶Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, CA
⁷USC Keck School of Medicine, Los Angeles, CA
⁸Siriraj Hospital, Mahidol University, Bangkok, Thailand
⁹Thalassemia and Sickle Cell Disease Center of Laiko General Hospital, Athens, Greece
¹⁰University of California San Francisco Benioff Children’s Hospital, Oakland, CA
¹¹Hospital Sultanah Bahiyah, Alor Setar, Malaysia
¹²Università degli studi della Campania "Luigi Vanvitelli", Napoli, Italy
¹³Farhat Hached Teaching Hospital, Sousse University, Tunisia
¹⁴First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
¹⁵Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
¹⁶Bristol Myers Squibb, Princeton, NJ
¹⁷Acceleron Pharma, Cambridge, MA
¹⁸University College London, University College London Hospitals, London, United Kingdom

Tippi C. MacKenzie, MD¹, Michela Frascoli^2*, Renan Sper^3*, Billie Rachael Lianoglou, MS^4*, Juan Gonzalez Velez^3*, Christopher C. Dvorak, MD⁵, Sandhya Kharbanda, MD⁶ and Elliott Vichinsky, MD⁷

¹Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, UCSF, San Francisco, CA
²University of Massachusetts, Amherst, MA
³UCSF, SF, CA
⁴Department of Surgery, UCSF, San Francisco, CA
⁵Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California, San Francisco, San Francisco, CA
⁶Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation, University of California San Francisco, Benioff Children's Hospital, San Francisco, CA
⁷Hematology/Oncology, Children's Hosp. & Rsrch. Ctr. Oakland, Oakland, CA

Mark C. Walters, MD¹, David H.K. Chui, MD, FRCPC², John J Farrell, PhD^2*, Ashutosh Lal, MD¹, Franco Locatelli, MD, PhD³, Janet L. Kwiatkowski, MD, MSCE^4,5, John B. Porter, MA, M.D., FRCP, FRCPath^6*, Martin G. Sauer, MD⁷, Isabelle Thuret, MD^8*, Suradej Hongeng, MD⁹, Andreas E. Kulozik, MD, PhD^10*, Adrian J. Thrasher, MBBS, PhD, FMedSci^11*, Evangelia Yannaki, MD^12*, Julia Yang^13*, Dustin Whitney, PhD^13*, Alexandria Petrusich^13*, Richard A. Colvin, MD, PhD^13* and Alexis A. Thompson, MD^14,15

¹UCSF Benioff Children's Hospital Oakland, Oakland, CA
²Boston University School of Medicine, Boston, MA
³The Department of Pediatrics, Sapienza, University of Rome, and the Department of Pediatric Hematology–Oncology, IRCCS Bambino Gesù Children’s Hospital, Rome, Italy
⁴University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
⁵The Children's Hospital of Philadelphia, Philadelphia, PA
⁶Haematology Department, University College London Hospitals, London, United Kingdom
⁷Medizinische Hochschule Hannover, Hannover, Germany
⁸Pediatric Hematology, Hôpital de la Timone, Marseille, France
⁹Mahidol University, Ramathibodi Hospital, Bangkok, Thailand
¹⁰University of Heidelberg, Heidelberg, Germany
¹¹UCL Great Ormond Street Institute of Child Health, London, United Kingdom
¹²G. Papanikolaou Hospital, Thessaloniki, Greece
¹³bluebird bio, Inc., Cambridge, MA
¹⁴Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
¹⁵Northwestern University Feinberg School of Medicine, Chicago, IL

Claire Berger, MD^1*, Philippe Joly, PhD^2*, Serge Pissard, PhD^3*, Sandrine Baron-Joly, MD^4*, Yoann Huguenin, MD^5*, Aurélie Cantais, MD^6*, Sophie Brun, MD^7*, Cecile GED, MD^8*, Catherine Badens, PhD, PharmD^9*, Muriel Giansily-Blaizot, MD^10*, Aguilar-Martinez Patricia, MD, PhD¹¹ and Isabelle Thuret, MD^12*

¹Service d’hémato-oncologie pédiatrique, CHU Saint-Etienne, Saint-Etienne, France
²Centre de Biologie,"Biochimie des pathologies érythrocytaires",, Groupement Hospitalier Lyon Est, BRON, France
³Laboratoire de Biochimie Génétique, Hôpital Universitaire Henri Mondor, Créteil, France
⁴Department of Pediatrics, Nîmes University Hospital,, Nîmes, France
⁵CHU de Bordeaux, Bordeaux Cedex, FRA
⁶Neonatal Pediatric Care unit, University Hospital,, Saint-Etienne, France
⁷Department of Hematological biology, Nîmes University Hospital, Nîmes, France
⁸INSERM, Bordeaux, FRA
⁹INSERM, MMG, Aix Marseille Univ, Marseille, France
¹⁰Department of Hematological biology, CHU de Montpellier, Montpellier, France
¹¹CHU de Montpellier, Montpellier, France
¹²Pediatric Onco-Hematology, Thalassemia Reference Center, Timone Enfant Hospital, APHM, Marseille, France

Donald Lavelle, PhD¹, Vinzon Ibanez, B.S.^1*, Kestis Vaitkus^1*, Yogenthiran Saunthararajah, MD² and Robert E. Molokie, MD^3,4

¹Department of Medicine, University of Illinois at Chicago, Jesse Brown VA Medical Center, Chicago, IL
²Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
³Jesse Brown VA Medical Center, Chicago, IL
⁴Department of Medicine, Division of Hematology and Oncology, University of Illinois at Chicago, Chicago, IL

Antonis Kattamis¹, Ersi Voskaridou^2*, Sophia Delicou^3*, Evangelos Klironomos, MD^4*, Ioannis Lafiatis^5*, Fotini Petropoulou, MD^6*, Michael D. Diamantidis, MD, PhD, MSc⁷, Stylianos Lafioniatis, MD^8*, Loukia Evliati^9*, Eleni Kapsali, MD, PhD¹⁰, Kiki Karvounis-Marolachakis, BS^11*, Despoina Timotheatou^12* and Alexandra Kourakli, MD, PhD¹³

¹First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
²Thalassemia and Sickle Cell Disease Center of Laiko General Hospital, Athens, Greece
³Thalassemia and Sickle Cell Unit, Hippocrateion General Hospital, Athens, Greece, Athens, Greece
⁴Thalassemia and Sickle Cell Unit, "Venizelion" General Hospital, Herakleion, Greece, Herakleion, Greece
⁵Thalassemia and Sickle Cell Unit, "Vostanio" General Hospital of Mytilini, Mytilini, Greece, Mytilini, Greece
⁶General Hospital of Athens ‘Georgios Gennimatas’,, Thalassemia Unit,, Athens, Greece
⁷General Hospital of Larissa, Larissa, Greece
⁸General Hospital of Volos,, Thalassemia Unit,, Volos, Greece
⁹Thalassemia and Sickle Cell Unit, General Hospital of Athens "Evaggelismos", Athens, Greece, Athens, Greece
¹⁰Department of Hematology, University Hospital of Ioannina, Ioannina, Greece
¹¹Medical Department, Genesis pharma SA, Chalandri, Greece
¹²GENESIS PHARMA, Athens, ITA
¹³Hematology Division, Department of Internal Medicine, University of Patras Medical School, Patras, Greece