Annie Clark, PhD, PharmD^*, Kevin Hammon^*, Krystal Sandza^*, Richard Torres^*, Elli Koziol^*, Jennifer Holcomb^*, Benjamin Kim, MD, Kala Jayaram, MD^*, Chris Russell, PhD^*, Christian Vettermann, PhD^* and Joshua Henshaw, PhD^*

BioMarin Pharmaceutical Inc., Novato, CA

Thiyagaraj Mayuranathan^1*, Jonathan S. Yen, PhD¹, Gregory A. Newby, PhD^2,3,4*, Yu Yao, MD^1*, Shaina N. Porter, PhD^5*, Kaitly J. Woodard, BS¹, Jingjing Zhang, MS^1*, Kalin Mayberry, BS^1*, Akshay Sharma, MBBS⁶, Shondra M. preutt-Miller, PhD^5*, John F. Tisdale, MD⁷, Shengdar Q. Tsai, PhD¹, David R. Liu, PhD^2,3,4* and Mitchell J. Weiss, MD, PhD¹

¹Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
²Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA
³Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA
⁴Howard Hughes Medical Institute, Harvard University, Cambridge, MA
⁵Center for Advanced Genome Engineering (CAGE), St. Jude Children's Research Hospital, Memphis, TN
⁶Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN
⁷Cellular and Molecular Therapeutics Branch, NHLBI, National Heart, Lung, and Blood Institute, Bethesda, MD

Chang Li^1*, Afrodite Georgakopoulou, PhD student^2*, Sucheol Gil^3* and Andre Lieber, MD, PhD³

¹Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, WA
²Gene and Cell Therapy Center, Hematology-HCT Unit, G. Papanikolaou Hospital, Thessaloniki, Greece
³University of Washington, Seattle, WA

Pinar Ataca Atilla, MD^1*, Mary K McKenna, PhD^1*, Norihiro Watanabe, PhD¹, Maksim Mamonkin, PhD², Malcolm K. Brenner, MD, PhD^2* and Erden Atilla, MD^1*

¹Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX
²Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston Methodist Hospital, Houston, TX

Agnes E. Hamburger, PhD^1*, Breanna DiAndreth, PhD^1*, Mark E. Daris, BS^1*, Melanie L. Munguia, BS^1*, Kiran Deshmukh, MS^1*, Jee-Young Mock, PhD^1*, Grace E. Asuelime, MS^1*, Emily D. Lim, BS^1*, Michelle R. Kreke, PhD^1*, Talar Tokatlian, PhD^1*, David Maloney, MD, PhD², William Y. Go, MD, PhD¹ and Alexander Kamb, PhD^1*

¹A2 Biotherapeutics, Agoura Hills, CA
²Fred Hutchinson Cancer Rsrch. Ctr., Seattle, WA

Ameet K. Mishra^1*, Iris Kemler^2* and David Dingli, MD, PhD³

¹Division of Hematology, Mayo Clinic, Rochester, MN
²Division of Hematology, Mayo Clinic, Rochester
³Mayo Clinic, Rochester, MN

Sneha Chitre, PhD¹, Joop Gaken, PhD^1*, Andrea Venuso, Masters^1* and Ghulam J. Mufti, Professor^2*

¹King's College London, London, United Kingdom
²Department of Haemato-Oncology, King's College London, London, United Kingdom

Frank W.G. Leebeek, MD, PhD¹, Karina Meijer, MD, PhD², Michiel Coppens, MD^3*, Peter Kampmann, MD^4*, Robert Klamroth, MD, PhD^5*, Roger Schutgens, MD, PhD, MSc^6*, Giancarlo Castaman, MD^7*, Erhard Seifried, MD, PhD⁸, Joachim Schwaeble^9*, Halvard Bönig^1,10*, Eileen K K Sawyer, PhD^11* and Wolfgang A. Miesbach, MD^12*

¹Erasmus University Medical Center, Rotterdam, Netherlands
²Dept. of Hematology, University of Groningen, University Medical Center Groningen, Groningen, Netherlands
³Amsterdam University Medical Center, University of Amsterdam, Amsterdam, Netherlands
⁴Rigshospitalet, Copenhagen, Denmark
⁵Department for Internal Medicine, Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin, Berlin, Germany
⁶Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
⁷Azienda Ospedaliera Universitaria Careggi, Florence, Italy
⁸Institute Frankfurt, German Red Cross Blood Service Baden Wurttemberg-Hessen, Frankfurt, Germany
⁹Institute Frankfurt, German Red Cross Blood Service Baden-Wurttemberg-Hessen, Frankfurt, Germany
¹⁰University of Washington School of Medicine, Seattle, WA
¹¹uniQure Inc., Lexington, MA
¹²Universitatsklinikum Frankfurt, Frankfurt, Germany