Dipti Gupta^1,2, Samuel Lessard, PhD^1*, Shujia Dai^3*, Yukio Nakamura, MD, PhD^4*, Alexandra Hicks, PhD^1*, Thomas K Harris, PhD^5* and Sriram Krishnamoorthy, PhD¹

¹Rare Blood Disorders, Sanofi, Waltham, MA
²Department of Biochemistry and Molecular Biology, University of Miami Miller School of Medicine, Miami, FL
³Biochemistry and Bioanalytics, Translational Science, Sanofi, Cambridge, MA
⁴Cell Engineering Division, RIKEN BioResource Research Center, Tsukuba, Japan
⁵Department of Biochemistry and Molecular Biology, University of Miami, Miller School of Medicine, Miami, FL

Thomas Gentinetta, PhD^1*, Gregory M. Vercellotti, MD², Chunsheng Chen^2*, Julia Nguyen^2*, Fuad Abdulla^2*, Ping Zhang, PhD^2*, Gregory J Kato, MD³, Nathan Brinkman, PhD^4*, Adrian Zuercher, PhD^1* and John D Belcher, PhD⁵

¹Research and Development, CSL Behring AG, Bern, Switzerland
²Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota Medical School, Minneapolis, MN
³Clinical Research & Development, CSL Behring, King of Prussia
⁴Research & Development, CSL Behring, Kankakee, IL
⁵Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, MN

Yuncheng Man^1*, Debnath Maji, PhD^1*, Ran An, PhD¹, Sanjay P Ahuja, MD², Jane A. Little, MD³, Pedram Mohseni, PhD^1*, Michael A Suster, PhD^1* and Umut A. Gurkan, PhD¹

¹Case Western Reserve University, Cleveland, OH
²University Hospitals Cleveland Medical Center, Rainbow Babies and Children's Hospital, Cleveland, OH
³University of North Carolina at Chapel Hill, Chapel Hill, NC

Maliha Maryam Ahmad, BS^1*, Laxminath Tumburu, PhD^2*, Chunyu Liu, PhD^3*, Mehdi Pirooznia, MD, PhD^4* and Swee Lay Thein, DSc, FRCP, MBBS, MRCP²

¹Sickle Cell Branch, NHLBI, National Institutes of Health, Alexandria, VA
²Sickle Cell Branch, NHLBI, National Institutes of Health, Bethesda, MD
³Department of Biostatistics, Boston University, Boston, MA
⁴Bioinformatics and Computational Biology Laboratory, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD

Billy Stuart^*, Peter Rahl^*, Kingsley Appiah^*, Ivan Efremov^*, Lorin Thompson^*, Owen Wallace^* and Christopher Moxham^*

Fulcrum Therapeutics, Cambridge, MA

Yankai Zhang, PhD¹, Celeste K. Kanne, BA^1*, Jennifer N. Tran, DO¹, Michael J. Lacagnina, PhD^2*, Peter M. Grace, PhD^2*, Mark J. Burish, MD, PhD^3* and Vivien A. Sheehan, MD, PhD¹

¹Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, TX
²The University of Texas MD Anderson Cancer Center, Houston, TX
³The University of Texas Health Science Center, Houston, TX

Jen Judy, PhD, MS^1*, Xunde Wang, PhD^2*, Fayaz Seifuddin, PhD^3*, Laxminath Tumburu, PhD^2*, Mehdi Pirooznia, MD, PhD^3* and Swee Lay Thein, DSc, FRCP, MBBS, MRCP²

¹National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Sickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
³Bioinformatics and Computational Biology Laboratory, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD

Christopher M Ventura, Ph.D.^1*, Stacy B Kiven, BS^2*, Nils Lambrecht, MD, PhD^3,4* and Kalpna Gupta, PhD⁵

¹Department of Medicine- Hematology/Oncology, University of California, Irvine, Irvine, CA
²Division of Hematology/Oncology, Southern California Institute for Research and Education, VA Medical Center, University of California Irvine, Long Beach, CA
³VA Long Beach Health Care System, Long Beach, CA
⁴VA Long Beach Medical Center, Long Beach, CA
⁵Division of Hematology/Oncology, Department of Medicine, University of California, Irvine, Irvine, CA

Madhav Vissa^1,2, Eric Soupene, PhD^1,2*, Sandra K. Larkin, MS^1,2*, Lynne D. Neumayr, MD^1,2, Elliott P. Vichinsky, MD^1,2 and Frans A. Kuypers, PhD^1,2*

¹UCSF Benioff Children's Hospital Oakland, Oakland, CA
²Children's Hospital Oakland Research Institute, Oakland, CA

Sharjeel Syed^1*, Jihad Aljabban, MMSc, MD², Jonathan Trujillo, MD, PhD³, Saad Syed, BS^4*, Robert Cameron, MD, PhD^5*, Maryam Panahiazar, PhD^6* and Dexter Hadley, MD, PhD^6*

¹Univeristy of Chicago Hospital, Chicago
²University of Wisconsin Hospital and Clinics, Madison
³Department of Medicine, Section of Hematology/Oncology, University of Chicago, Chicago, Chicago, IL
⁴Stanford University School of Medicine, Stanford, CA
⁵University of Chicago Hospitals, Chicago
⁶University of California San Francisco, San Francisco, CA

Ravi Vats, MSc¹, Enrico M Novelli, M.D., M.S.², Mark T. Gladwin, MD³, Prithu Sundd, PhD¹ and Tirthadipa Pradhan⁴

¹Pittsburgh Heart, Lung and Blood Vascular Medicine Institute, University of Pittsburgh School of Medicine, Pittsburgh, PA
²Heart, Lung and Blood Vascular Medicine Institute, University of Pittsburgh, Pittsburgh, PA
³University of Pittsburgh, Pittsburgh, PA
⁴University of Pittsburgh, Pittsburgh