CHOP Research Institute Scientific Symposium

9:00 – 9:15 am
Welcome

Bryan Wolf, MD, PhD
Executive Vice President and Chief Scientific Officer
Chair, Department of Biomedical and Health Informatics
Edmond F. Notebaert Endowed Chair in Pediatric Research
Children’s Hospital of Philadelphia
Professor of Pathology and Laboratory Medicine
Perelman School of Medicine at the University of Pennsylvania

Susan Furth, MD, PhD
Division Chief, Division of Nephrology
Vice Chair, Department of Pediatrics
Laffey-Connolly Endowed Chair in Pediatric Nephrology
Associate Chair, Academic Affairs, Department of Pediatrics
Children’s Hospital of Philadelphia
Professor of Pediatrics and Epidemiology
Perelman School of Medicine at the University of Pennsylvania

Session 1: Gene Therapy - Chaired by Lindsey A. George, MD

9:15 – 9:35 am
“Drug-Regulated Splicing Switch for Gene Expression Control”
Alejandro Mas Monteys, PhD
Research Scientist, Davidson Laboratory
Raymond G. Perelman Center for Cellular and Molecular Therapeutics
Children’s Hospital of Philadelphia
Research Assistant Professor of Pathology and Laboratory Medicine
Perelman School of Medicine at the University of Pennsylvania

9:35 – 9:45 am
“A New Gene Therapy Approach for the Cure of Alpha Thalassemia”
Maxwell Chappell, PhD Student
Rivella Laboratory, Division of Hematology
Children’s Hospital of Philadelphia

9:45 – 9:55 am
“Translating Gene Therapy for Spinocerebellar Ataxia Type 1: Optimization of Efficacious Therapeutic Vectors in Nonhuman Primates”
Megan Keiser, PhD
Research Scientist, Davidson Laboratory, Pathology and Laboratory Medicine
Children’s Hospital of Philadelphia

9:55 – 10:00 am
Break

Session 2: Cell Therapy for Cancer - Chaired by Stephan A. Grupp, MD, PhD

10:00 – 10:20 am
“Lessons Learned from Generating and Tracking the Fate of Chimeric Antigen Receptor T-Cells”
Jan Joseph Melenhorst, PhD
Research Professor of Pathology and Laboratory Medicine
Perelman School of Medicine at the University of Pennsylvania

10:20 – 10:30 am
“Integrative Bulk and Single-cell Profiling of Pre-manufacture T-Cell Populations Reveals Factors Mediating Long-term Persistence of CAR T-Cell Therapy”
Gregory Chen, MD/PhD Student
Tan Laboratory, Division of Oncology
Children’s Hospital of Philadelphia

10:30 – 10:40 am
“Development of mRNA CAR T Cells for Malignant Pediatric Brain Tumors”
Jessica Foster, MD
Attending Physician, Division of Oncology
Children’s Hospital of Philadelphia
Instructor
Perelman School of Medicine at the University of Pennsylvania

10:40 – 10:50 am
“Dual Targeting of Pediatric Solid Tumors Through CAR T-cells Secreting Bispecific
Innate Immune Cell Engagers”
Guillem Pascual-Pasto, PhD
Research Postdoctoral Fellow, Bosse Laboratory, Division of Oncology
Children’s Hospital of Philadelphia

10:50 – 11:00 am
Break

Session 3: Emerging Approaches for Cell & Gene Therapies - Chaired by Beverly L. Davidson, PhD

11:00 – 11:20 am
"Prenatal Gene Editing: Curing Disease Before It Happens"
William H. Peranteau, MD
Adzick-McCausland Distinguished Chair in Fetal and Pediatric Surgery
Attending Surgeon, Division of Pediatric General, Thoracic and Fetal Surgery
Children's Hospital of Philadelphia
Assistant Professor of Surgery
Perelman School of Medicine at the University of Pennsylvania

11:20 – 11:30 am
“Enhancing CD20-targeted Immunotherapies by Modulating Splicing with Antisense
Oligonucleotides”
Zhiwei Ang, PhD
Research Postdoctoral Fellow, Thomas-Tikhonenko Laboratory, Pathology and Laboratory Medicine
Children’s Hospital of Philadelphia

11:30 – 11:40 am
“Use of a Factor VIII Variant with Enhanced Hemostatic Function for Hemophilia A Gene Therapy”
Anna Sternberg, PhD
Research Postdoctoral Fellow (T32), George Laboratory, Division of Hematology
Children’s Hospital of Philadelphia

11:40 – 11:50 am
“Improved Gene Therapy for Metachromatic Leukodystrophy”
Lucas Tricoli, PhD
Research Associate, Rivella Laboratory, Division of Hematology
Children’s Hospital of Philadelphia

11:50 am – 12:00 pm
“Therapeutic Targeting of Intracellular Oncoproteins with Peptide -Centric CAR T Cells”
Mark Yarmarkovich, PhD
Research Postdoctoral Fellow, Maris Laboratory, Division of Oncology
Children’s Hospital of Philadelphia

12:00 – 12:15 pm
Lunch Break

Session 4: Translating Gene Therapies - Chaired by Stefano Rivella, PhD

12:15 – 12:35 pm
“LentiGlobin (beti-cel) Gene Therapy for Beta Thalassemia”
Janet Kwiatkowski, MD, MSCE
Director, Thalassemia Center
Attending Physician, Division of Hematology
Children's Hospital of Philadelphia
Professor of Pediatrics
Perelman School of Medicine at the University of Pennsylvania

12:35 – 12:45 pm
“Combined Transgene and Intron-derived miRNA Therapy Reverses Motor Phenotypes in SCA1 Mice”
Ellie Carrell, PhD
Research Postdoctoral Fellow, Davidson Laboratory, Pathology and Laboratory Medicine
Children’s Hospital of Philadelphia

12:45 – 12:55 pm
“AAV Integration Analysis After Long Term Follow Up in Hemophilia A Dogs Reveals the Genetic Consequences of AAV-Mediated Gene Correction”
Denise Sabatino, PhD
Researcher, Division of Hematology
Children’s Hospital of Philadelphia
Research Associate Professor of Pediatrics
Perelman School of Medicine at the University of Pennsylvania

12:55 – 1:00 pm
Break

1:00 – 1:45 pm
Keynote Address
Keynote Speaker Introduction by Lindsey A. George, MD
"Gene Editing for Blood Disorders"
Daniel Bauer, MD, PhD
Associate Professor of Pediatrics
Harvard Medical School
Director of the Gene Therapy Program and Hematology Staff
Physician
Boston Children’s Hospital

1:45 – 2:00 pm
Closing Remarks

Daniel E. Bauer, MD, PhD

Daniel E. Bauer, MD, PhD
Associate Professor of Pediatrics, Harvard Medical School
Director of the Gene Therapy Program and Hematology Staff Physician, Boston Children’s Hospital

Daniel Bauer is a physician-scientist whose research focuses on functional genomics to dissect determinants of blood cell development and disease and develop innovative therapies. His clinical work is in pediatric hematology. He identified the BCL11A erythroid enhancer as a favorable target for therapeutic genome editing for the β-hemoglobinopathies, now being investigated in several clinical trials. His research has elucidated molecular mechanisms of hemoglobin switching; advanced high-throughput gene editing approaches to associate genetic variants with phenotypes; and developed methods for highly efficient and specific gene editing in human hematopoietic stem cells. He is a Principal Investigator, Staff Physician, and Director of the Gene Therapy Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Associate Professor of Pediatrics at Harvard Medical School, Principal Faculty at the Harvard Stem Cell Institute, and Associate Member of the Broad Institute of MIT and Harvard. He was recipient of the NIH Director’s New Innovator Award (National Institutes of Health 2016), Young Investigator Award (Society for Pediatric Research 2017), Outstanding New Investigator Award (American Society of Gene and Cell Therapy 2020), and elected member of the American Society of Clinical Investigation (2021).