-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.P1.45 801. Gene Therapies: Poster I

Symposia: Gene Therapies Program: Oral and Poster Abstracts
Type: Poster
Saturday, December 10, 2022: 5:30 PM-7:30 PM
Hall D (Ernest N. Morial Convention Center)

Franco Locatelli, Prof., MD, PhD1, Peter Lang, Dr.2*, Amanda Li, MD3*, Selim Corbacioglu, MD4, Josu de la Fuente, PhD5, Donna A. Wall6, Robert Liem, MD7, Roland Meisel, MD8, Markus Y. Mapara, MD, PhD9, Ami J Shah, MD10, Maria Domenica Domenica Cappellini, MD11, Antonis Kattamis, MD12, Sujit Sheth, MD13, Yael Bobruff, PhD14*, Laura Bower, MD14*, Lanju Zhang, PhD14*, Anjali Sharma, MD15*, Yang Song, PhD14*, William Hobbs, MD, PhD14* and Haydar Frangoul, MD16

1Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù Rome, Catholic University of the Sacred Heart, Rome, Italy
2University of Tübingen, Tuebingen, Germany
3BC Children’s Hospital, University of British Columbia, Vancouver, BC, Canada
4University of Regensburg, Regensburg, Germany
5Imperial College Healthcare NHS Trust, St Mary's Hospital, London, United Kingdom
6The Hospital for Sick Children, Toronto, ON, Canada
7Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
8Heinrich-Heine-University, Duesseldorf, Germany
9Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY
10Stanford University School of Medicine, Stanford, CA
11University of Milan, Milan, Italy
12National and Kapodistrian University of Athens, Athens, Greece
13Joan and Sanford I Weill Medical College of Cornell University, New York, NY
14Vertex Pharmaceuticals Incorporated, Boston, MA
15CRISPR Therapeutics, Cambridge, MA
16Sarah Cannon Center Research Institute and The Children’s Hospital at TriStar Centennial, Nashville, TN

Ami J Shah, MD1,2,3, José Luis López Lorenzo, MD4,5*, Julián Sevilla, MD, PhD6,7*, Susana Navarro, PhD5,7,8*, Lucía Llanos, MD, PhD4,5*, Begoña Pérez de Camino Gaisse, MD4,5*, Sol Sanchez, MD4,5*, Josune Zubicaray, PhD6*, Bert Glader, MD, PhD1,3, May Chien, MD1,3, Oscar Quintana Bustamante, PhD5,7,8*, Miriam Zeini, PhD9*, Grace Choi9*, Eileen Nicoletti, MD9*, Gayatri R. Rao, MD, JD9*, Maria Grazia Roncarolo, MD, PhD1,2,10, Juan A. Bueren, PhD5,7,8*, Jonathan D. Schwartz, MD9 and José C Segovia, PhD5,7,8*

1Lucile Packard Children’s Hospital, Palo Alto, CA
2Center for Definitive and Curative Medicine, Stanford University, Stanford, CA
3Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation, and Regenerative Medicine, Stanford University, Stanford, CA
4Instituto de Investigación Sanitaria Fundación Jiménez Díaz, Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
5Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain
6Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
7Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
8Unidad de Innovación Biomédica, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain
9Rocket Pharmaceuticals, Inc., Cranbury, NJ
10Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University, Stanford, CA

Steven W. Pipe, MD1, Frank W.G. Leebeek2, Michael Recht, MD, PhD, MBA3, Nigel S. Key4, Susan Lattimore3*, Giancarlo Castaman5*, David Cooper6,7*, Stephanie Verweij6,7*, Ricardo Dolmetsch6,7*, Jacqueline Tarrant8*, Yanyan Li8*, Paul E. Monahan8* and Wolfgang A. Miesbach9*

1University of Michigan, Ann Arbor, MI
2Erasmus Medical Center, University Medical Center, Rotterdam, Netherlands
3Oregon Health & Science University, Portland, OR
4University of North Carolina, Chapel Hill, NC
5Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
6uniQure BV, Amsterdam, Netherlands
7uniQure Inc., Lexington, MA
8CSL Behring, King of Prussia, PA
9University Hospital Frankfurt, Frankfurt, Germany

Asif Alavi, MD1, Mehrdad Abedi, MD2, Suhag Parikh, MD3*, Richard Boismenu, PhD4*, Michael Chen, MSc4*, Ben L Hsu4*, Bettina M. Cockroft, MD, MBA4*, Isobelle Galeon, MD, MS, MPH5*, Pablo Rendo, MD5 and Mark C. Walters, MD6

1Karmanos Cancer Institute, Wayne State University, Detroit, MI
2Department of Internal Medicine, Division of Cellular Therapy, Bone Marrow Transplantation and Malignant Hematology, University of California Davis School of Medicine, Sacramento, CA
3Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
4Sangamo Therapeutics, Inc., Brisbane, CA
5Sanofi, Cambridge, MA
6UCSF Benioff Children's Hospital Oakland, Oakland, CA

Steven W. Pipe, MD1, Frank W.G. Leebeek2, Michael Recht, MD, PhD, MBA3, Nigel S. Key4, Susan Lattimore3*, Giancarlo Castaman5*, Michiel Coppens6,7*, David Cooper8,9*, Robert Gut8,9*, Sergio Slawka8,9*, Stephanie Verweij8,9*, Ricardo Dolmetsch8,9*, Yanyan Li10*, Paul E. Monahan10* and Wolfgang A. Miesbach11*

1University of Michigan, Ann Arbor, MI
2Erasmus Medical Center, University Medical Center, Rotterdam, Netherlands
3Oregon Health & Science University, Portland, OR
4University of North Carolina, Chapel Hill, NC
5Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy
6Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands
7Amsterdam UMC location, University of Amsterdam, Vascular Medicine, Amsterdam, Netherlands
8uniQure BV, Amsterdam, Netherlands
9uniQure Inc., Lexington, MA
10CSL Behring, King of Prussia, PA
11University Hospital Frankfurt, Frankfurt, Germany

Wolfgang A. Miesbach1*, Michael Recht, MD, PhD, MBA2, Nigel S. Key3, Krupa Sivamurthy4*, Paul E. Monahan4* and Steven W. Pipe, MD5

1University Hospital Frankfurt, Frankfurt, Germany
2Oregon Health & Science University, Portland, OR
3University of North Carolina, Chapel Hill, NC
4CSL Behring, King of Prussia, PA
5University of Michigan, Ann Arbor, MI

Rongrong Liu, PhD, MD1*, Hui Xu, PhD2*, Junbin Liang, PhD2*, Wenqiang Xie1*, Gaohui Yang, PhD, MD1*, Lei Shi, MD2*, Lingling Shi, PhD, MD1*, Jianpei Fang, MD3*, Chunfu Li, MD4, Zeyan Shi, MD1*, Xuemei Zhou1*, Junjiu Huang, PhD5* and Yongrong Lai, PhD, MD1*

1Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, China
2Reforgene Medicine, Guangzhou, China
3Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Guangzhou, China
4Nanfang-Chunfu Children's Institute of Hematology & Oncology, Taixin Hospital, Dongguan, China
5School of Life Sciences, Sun Yat-sen University, Guangzhou, China

Socheatraksmey Ung, PharmB1,2*, Pongsakorn Choochuen, MD1,3*, Wannakorn Khopanlert, MD1,2*, Kajornkiat Maneechai, BSc1,2*, Surasak Sangkhathat, MD, PhD3*, Seitaro Terakura, MD, PhD4* and Jakrawadee Julamanee, MD, PhD2*

1Department of Biomedical Sciences and Biomedical Engineering, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
2Stem Cell Laboratory, Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
3Translational Medicine Research Center, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
4Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan

Lucas Van Gorder, BS1,2,3*, Bhavya S Doshi, MD2,3,4, Elinor Willis, VMD5*, Matthew Lanza, VMD6*, Valder R Arruda, MD, PhD1,2,3, Mary Beth Callan, VMD7* and Ben J Samelson-Jones, MD, PhD1,2,3

1Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
2Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA
3Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
4Department of Pediatrics, University of Pennsylvania, Philadelphia, PA
5Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA
6Department of Pathobiology,, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA
7Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA

Haokun Yuan, MSc1*, Hongwei Liu, MSc1*, Xiaomei Wu, BSc1*, Yuchen Liu, MSc2*, Rui Zhang, MSc2* and Lung-Ji Chang, PhD1,2

1School of Medicine, University of Electronic Science and Technology of China, Chengdu, China
2Geno-Immune Medical Institute, Shenzhen, China

Omar Banda, PhD1, Mohamad-Gabriel Alameh, PhD2*, Seul Jung, BS3*, Drew Weissman, MD, PhD4*, Stefano Rivella, PhD5 and Peter Kurre, MD6

1Children's Hospital of Philadelphia, Philadelphia, PA
2Department of Medicine, University of Pennsylvania, Philadelphia, PA
3Comprehensive Bone Marrow Failure Center, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
4University of Pennsylvania Medical Center, PHILADELPHIA, PA
5Department of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
6Comprehensive Bone Marrow Failure Center, Department of Pediatrics, Oregon Health & Science University (OHSU), Philadelphia, PA

Wei Liu1*, Feng Xue, MD1*, Ting Sun, MD1*, Bingqi Xu, PhD1*, Xiaolei Pei, PhD1*, Tenghui Yu, PhD2*, Fenglan Wu, PhD2*, Lijun Shen2*, Renchi Yang, MD1 and Lei Zhang, MD1*

1State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
2Gritgen Therapeutics, Suzhou, China, Suzhou, China

Diana M. Abraham, BA1*, Yifan Zhou1,2,3*, Richard Lozano, BS1*, Gregory A. Newby, PhD4,5,6*, David R. Liu, PhD5,6,7*, Chuanfeng Wu, MD, PhD1* and Cynthia E. Dunbar, MD1

1Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
2Wellcome Trust Sanger Institute, University of Cambridge, Cambridge, United Kingdom
3Wellcome-MRC Cambridge Stem Cell Institute, University of Cambridge, Cambridge, United Kingdom
4Broad Institute of Harvard and MIT, Harvard University, Cambridge, MA
5Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA
6Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA
7Howard Hughes Medical Institute, Harvard University, Cambridge, MA

Sandeep RP Kumar, PhD1*, Moanaro Biswas, PhD2, Ype P De Jong, MD3* and Roland W Herzog, PhD1

1Department of Pediatrics, IUPUI, Indianapolis, IN
2Pediatrics, Indiana University School of Medicine, Indianapolis, IN
3Division of Gastroenterology and Hepatology, Weill Cornell Medicine, New York, NY

Lucila Kerbauy, MD, PhD1, Larissa Leggieri Coa1*, Micheli Severo Sielski2*, Juliana Aparecida Preto Godoy, PhD1*, Andrea Tiemi Kondo, MD3*, Julia Teixeira Cottas Azevedo2*, Augusto Barbosa Jr.2*, Oswaldo Keith Okamoto, PhD4*, Jose Mauro Kutner, MD, PhD5, Nelson Hamerschlak, MD, PhD6 and Raquel de Melo Alves Paiva, PhD7*

1Hospital Israelita Albert Einstein, Sao Paulo, Brazil
2Cell Therapy Department, Hospital Israelita Albert Einstein, Sao Paulo, Brazil
3Hospital Israelita Albert Einstein, Sao Paulo, SE, Brazil
4Human Genome and Stem Cell Research Center, Department of Genetics and Evolutionary Biology, Biosciences Institute, Universidade de Sao Paulo, Sao Paulo, Brazil
5Hospital Israelita Albert Einstein, Sao Paulo, BRA
6Hospital Israelita Albert Einstein, Sao Paolo, Brazil
7Hospital Israelita Albert Einstein, São Paulo, AL, Brazil

*signifies non-member of ASH