-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.O2.6 801. Gene Therapies: Addressing Challenges & Opportunities in Pre-clinical Settings

Symposia: Gene Therapies Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Research, Bleeding and Clotting, Biological therapies, Acute Myeloid Malignancies, AML, Fundamental Science, apoptosis, hemophilia, Translational Research, Chimeric Antigen Receptor (CAR)-T Cell Therapies, Thalassemia, Combination therapy, Hemoglobinopathies, hematopoiesis, drug development, Diseases, Gene Therapy, Therapies, Immunotherapy, emerging technologies, Biological Processes, Myeloid Malignancies, Technology and Procedures, gene editing, molecular biology, Study Population, Transplantation, Animal model
Saturday, December 10, 2022: 9:30 AM-11:00 AM
Great Hall BC (Ernest N. Morial Convention Center)
Moderators:
Akshay Sharma, MBBS, St. Jude Children's Research Hospital and Catherine E. McGuinn, MD, Columbia University
Disclosures:
Sharma: Vertex Pharmaceuticals/CRISPR Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: Clinical Trial Site PI; Novartis: Other: Clinical Trial Site PI; Magenta Therapeutics: Other: Clinical Trial Site PI; Spotlight Therapeutics: Consultancy; CRISPR Therapeutics: Other: Clinical Trial Site PI, Research Funding; Vindico Medical Education: Honoraria; Medexus Inc: Consultancy. McGuinn: BPL: Consultancy; CSL Berhing: Membership on an entity's Board of Directors or advisory committees; Genentech/ Roche: Membership on an entity's Board of Directors or advisory committees, Research Funding; HemaBiologics: Membership on an entity's Board of Directors or advisory committees; Octapharma: Membership on an entity's Board of Directors or advisory committees; Bayer: Membership on an entity's Board of Directors or advisory committees; Pfizer: Research Funding; Spark Therapeutics: Research Funding; Sanofi: Research Funding; Takeda: Research Funding.
9:30 AM

Maxwell Chappell1*, Laura Breda, PhD1*, Danuta Jadwiga Jarocha, PhD1*, Michael Triebwasser, MD, PhD, MSc, BS1,2, Tyler E Papp3*, Valentina Ghiaccio, PhD1*, Megan T Fedorky1*, Amaliris Guerra, PhD1*, Kandace Gollomp1*, Osheiza Y. Abdulmalik, DVM4*, Nattiya Teawtrakul, MD, PhD5*, Stavros Glentis, PhD6*, Antonis Kattamis, MD6,7,8,9,10, Hamideh Parhiz, PhD, PharmD3* and Stefano Rivella, PhD11,12,13,14,15

1Department of Pediatrics, Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
2U of Michigan, Ann Arbor, MI
3Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
4Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
5Srinagarind Khonkaen Hospital, Khonkaen, THA
6Pediatric Hematology Oncology Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, Aghia Sophia Children’s Hospital, Athens, Greece
7Thalassemia Unit, First Department of Pediatrics, ‘Aghia Sophia’ Children’s Hospital, National and Kapodistrian University of Athens, ATHENS, Greece
8Division of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens, “Aghia Sophia” Children’s Hospital, Athens, Greece, ATHENS, Greece
9"Agia Sophia" Children Hospital, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece, Athens, Greece
10National and Kapodistrian University of Athens, Athens, Greece
11Department of Pediatrics, Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
12Cell and Molecular Biology affinity group (CAMB), University of Pennsylvania, Philadelphia, PA
13Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
14Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA
15Penn Center for Musculoskeletal Disorders, University of Pennsylvania, Philadelphia, PA

9:45 AM

Gabriele Casirati, MD1,2,3*, Andrea Cosentino, MD1,4,5*, Adele Mucci, PhD1,6*, Iraxte Ugarte Zabala, BSc1,7*, Mohammed S. Mahmoud, PhD1,8*, Christian Brendel, PhD9,10*, Alessandro Rambaldi, MD11,12, Jerome Ritz, MD, PhD13,14, Scott A. Armstrong, MD, PhD15 and Pietro Genovese, PhD1,16,17

1Dana Farber Cancer Institute, Boston, MA
2Universita' Milano Bicocca, milan, Italy
3Boston Children's Hospital, Boston
4Ospedale Papa Giovanni XXIII, Universita' di Studi di Milano, Bergamo, ITA
5Boston Children Hospital, Boston, MA
6Pediatric Oncology, Boston Children Hospital, Boston, MA
7German Cancer Research Center (DFKZ), Heidelberg, Germany
8Zoology department, Faculty of Science, Fayoum University, Fayoum, Egypt
9Hematology/Oncology, Harvard Medical School, Boston, MA
10Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
11Hematology, ASST Papa Giovanni XXIII, Bergamo, Italy
12Department of Oncology and Hematology, University of Milan, Milan, Italy
13Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
14Department of Medicine, Harvard Medical School, Boston, MA
15Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA
16Boston Children's Hospital, Boston, MA
17Harvard Medical School, Boston, MA

10:00 AM

Laura Breda, PhD1*, Tyler E Papp2*, Michael Triebwasser, MD, PhD, MSc, BS1,3, Amir Yadegari2*, Megan T Fedorky1*, Naoto Tanaka, PhD1*, Houping Ni2*, Drew Weissman, MD, PhD2*, Stefano Rivella, PhD4,5,6,7,8 and Hamideh Parhiz, PhD, PharmD2*

1Department of Pediatrics, Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
2Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
3U of Michigan, Ann Arbor, MI
4Department of Pediatrics, Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
5Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
6Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA
7Cell and Molecular Biology affinity group (CAMB), University of Pennsylvania, Philadelphia, PA
8Penn Center for Musculoskeletal Disorders, University of Pennsylvania, Philadelphia, PA

10:15 AM

Daisuke Araki, MD1, Vicky Chen2*, Neelam Redekar3*, Poching Li4*, Yan Luo4*, Yuesheng Li4* and Andre Larochelle, MD, PhD1

1Laboratory of Regenerative Therapies for Inherited Blood Disorders, Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD
2Integrated Data Science Services, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD
3NIAID Collaborative Bioinformatic Resource (NCBR), NIH, Bethesda, MD
4DNA Sequencing and Genomics Core, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD

10:30 AM

Byung-Chul Lee1*, Max Grice2*, Chuanfeng Wu, MD, PhD3*, Ryland D Mortlock4*, Taehoon Shin5*, Yifan Zhou5*, Uimook Choi, PhD6*, Suk See De Ravin, MD, PhD6*, So Gun Hong, DVM5* and Cynthia E. Dunbar, MD5

1NHLBI, NIH, BETHESDA, MD
2NIH, Bethesda, MD
3Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, NHLBI/NIH, Bethesda, MD
4Translational Stem Cell Biology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD
5Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
6Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD

10:45 AM

Jyoti Rana, PhD1*, Sandeep RP Kumar, PhD1*, Maite Munoz1*, Kentaro Yamada, PhD1*, Thais Bertollini, PhD1*, David M Markusic, PhD1, Manuela Corti, PhD2*, Barry J Byrne, MD2*, Roland W Herzog, PhD1 and Moanaro Biswas, PhD1*

1Department of Pediatrics, IUPUI, Indianapolis, IN
2Powell Center for Rare Disease Research and Therapy, University of Florida, Gainesville, FL

*signifies non-member of ASH