Wentian Wang, PhD^1*, Yuanyuan Tuo^1*, Yuchen Gao^1*, Qianyu Huo^1*, Jiaying Liu^1*, Lei Zhang¹, Ying Chi^1*, Huiyuan Li^1*, Xiaolei Pei^1* and Zhixu He^2*

¹State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²Stem Cell and Tissue Engineering Research Center, Department of Immunology, School of Basic Medical Sciences, Guizhou Medical University; Key Laboratory of Adult Stem Cell Translational Research, Chinese Academy of Medical Sciences, Guiyang, China

John B. Porter^1*, Maria Domenica Cappellini, MD², Kevin H.M. Kuo, MD, MSc, FRCPC^3,4, Maria Dimopoulou^5*, Marta Reverte^6*, Loyse Felber Medlin^6*, Shweta Sharma^7*, Kefeng Wang^8*, Jennie Zhang^8* and Ali T. Taher, MD, FRCP⁹

¹University College London, University College London Hospitals, London, United Kingdom
²Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, SC Medicina ad Indirizzo Metabolico, Milan, Italy
³Division of Medical Oncology and Hematology, Department of Medicine, University Health Network, Toronto, ON, Canada
⁴Division of Hematology, Department of Medicine, University of Toronto, Toronto, ON, Canada
⁵Thalassemia Unit Centre of Expertise in Hemoglobinopathies, Laiko General Hospital of Athens, Athens, Greece
⁶Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
⁷Bristol Myers Squibb, New Delhi, India
⁸Bristol Myers Squibb, Princeton, NJ
⁹Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon

Nivedhitha Devaraju^1,2*, Harish Prakash^3,4*, Anila George^4*, Nithinsam Ravi^4,5*, Saravanabhavan Thangavel^6*, Merlin Crossley^7*, Alok Srivastava^4,8,9,10 and Mohankumar K Murugesan^11*

¹Centre for Stem cell research, VELLORE, India
²Department of Haematology, Christian Medical College, Vellore, India
³Tata Institute for Genetics and Society, bangalore, India
⁴Centre for Stem cell research, Vellore, India
⁵University of Pennsylvania, Philadelphia
⁶Centre For Stem Cell Research (a Unit of Instem, Bengaluru), Vellore, IND
⁷School of Biotechnology and Biomolecular Sciences, University of NSW, NSW, AUS
⁸Unit of inStem Bengaluru, Centre for Stem Cell Research, Vellore, India
⁹Department of Haematology, Christian Medical College Vellore, Vellore, IND
¹⁰Christian Medical College, Vellore, India
¹¹Centre for Stem cell research, Bagayam, Vellore, Tamil Nadu, India

Dong Liu, PhD^*, Min Xiong^* and Shaojiang Deng, PhD^*

Kind Pharmaceuticals, LLC, Redwood City, CA

Georgios E. Christakopoulos, MD¹, Alfonso G. Fernandez, PhD^2*, Yu Yao, MD^3*, Rahul Telange, MSc, BS^4*, Dudley W. Michael^5*, Tomas Ganz, MD, PhD⁶, Elizabeta Nemeth, PhD⁶ and Mitchell J Weiss, MD, PhD³

¹Department of Hematology, St Jude Children's Research Hospital, Memphis, TN
²Dept. of Hematology, St Jude Children's Research Hospital, Memphis, TN
³Department of Hematology, St. Jude Childrens Research Hospital, Memphis, TN
⁴Dept. of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁵Hematology, St Jude Children's Research Hospital, MEMPHIS, TN
⁶Center for Iron Disorders, University of California, Los Angeles, Los Angeles, CA

Thidarat Suksangpleng, PhD¹, Kalaya Tachavanich, M.Sc.^2* and Vip Viprakasit, MD, DPhil(Oxon)^2*

¹Faculty of Medicine Siriraj Hospital, Siriraj Thalassemia Center, Mahidol University, Bangkok, Thailand
²Faculty of Medicine Siriraj Hospital, Department of Pediatrics, Mahidol University, Bangkok, Thailand