-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

801. Gene Editing, Therapy and Transfer: Poster III

Program: Oral and Poster Abstracts
Type: Poster
Hematology Disease Topics & Pathways:
Adult, AML, Biological, HSCs, sickle cell disease, Diseases, Lymphoma (any), CAR-Ts, thalassemia, Therapies, Adverse Events, B-Cell Lymphoma, Biological Processes, DLBCL, Hemoglobinopathies, gene therapy, Technology and Procedures, Cell Lineage, Xenograft models, erythropoiesis, gene editing, Lymphoid Malignancies, Study Population, Clinically relevant, Myeloid Malignancies, flow cytometry, NGS
Monday, December 7, 2020: 7:00 AM-3:30 PM

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 Mayuranathan1*, Jonathan S. Yen, PhD1, Gregory A. Newby, PhD2,3,4*, Yu Yao, MD1*, Shaina N. Porter, PhD5*, Kaitly J. Woodard, BS1, Jingjing Zhang, MS1*, Kalin Mayberry, BS1*, Akshay Sharma, MBBS6, Shondra M. preutt-Miller, PhD5*, John F. Tisdale, MD7, Shengdar Q. Tsai, PhD1, David R. Liu, PhD2,3,4* 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 Li1*, Afrodite Georgakopoulou, PhD student2*, Sucheol Gil3* 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, MD1*, Mary K McKenna, PhD1*, Norihiro Watanabe, PhD1, Maksim Mamonkin, PhD2, Malcolm K. Brenner, MD, PhD2* and Erden Atilla, MD1*

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, PhD1*, Breanna DiAndreth, PhD1*, Mark E. Daris, BS1*, Melanie L. Munguia, BS1*, Kiran Deshmukh, MS1*, Jee-Young Mock, PhD1*, Grace E. Asuelime, MS1*, Emily D. Lim, BS1*, Michelle R. Kreke, PhD1*, Talar Tokatlian, PhD1*, David Maloney, MD, PhD2, William Y. Go, MD, PhD1 and Alexander Kamb, PhD1*

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

Ameet K. Mishra1*, Iris Kemler2* 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, PhD1*, Andrea Venuso, Masters1* and Ghulam J. Mufti, Professor2*

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, MD3*, Peter Kampmann, MD4*, Robert Klamroth, MD, PhD5*, Roger Schutgens, MD, PhD, MSc6*, Giancarlo Castaman, MD7*, Erhard Seifried, MD, PhD8, Joachim Schwaeble9*, Halvard Bönig1,10*, Eileen K K Sawyer, PhD11* and Wolfgang A. Miesbach, MD12*

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

*signifies non-member of ASH