TRANSLATIONAL CENTER OF EXCELLENCE FOR HEMATOPOIETIC STEM CELL ENGINEERING

The University of Pennsylvania Perelman School of Medicine (PSOM), in collaboration with the Division of Hematology-Oncology in the Department of Medicine and the Abramson Cancer Center (ACC) are thrilled to house a new Translational Center of Excellence (TCE) in Genetically Engineered Hematopoietic Stem Cell Transplantation.

The mission of this TCE is to catalyze translational research in gene-engineered hematopoietic stem cell therapy and to drive first-in-human testing through the ACC's Cell Therapy Trials unit.

A new era of genetically engineered hematopoietic stem cell products has emerged to treat sickle cell disease, thalassemia and other non-malignant indications. In cancer therapeutics, genetic engineering has been used for modification of immune effector cells such as T cells, including chimeric antigen receptor (CAR) T cells. While CAR T cells have made a major impact in lymphoma and myeloma, their impact in other cancers has been limited owing to lower than expected antitumor efficacy and high rates of toxicity arising from the lack of cancer-specific targets. This TCE will establish new approaches to gene editing of hematopoietic stem cells in order to shift the paradigms and boundaries of cutting-edge cell therapy.

Exciting News

  • Our Team at the ASGCT 28th Annual Meeting May 13-17, 2025 | New Orleans Thursday, May 15, 2025

    Oral Presentation:

    Nils Wellhausen, PhD

    “Selection for an HIV-Resistant Immune System by Multiplex Base-Edited CD45 CAR-T Cell Therapy”

  • Funding Awarded to Saar Gill, MD, PhD Thursday, February 13, 2025

    NEW YORK, Feb. 13, 2025 –The DeGregorio Family Foundation with support from the Torrey Coast Foundation Gastro Esophageal Investigator Network Initiative (GEMINI) has awarded $450,000 to Saar Gill, MD, PhD, of the University of Pennsylvania, to complete his 2022 grant for $1 million.

    Click here for Full Press Release

  • Our Team Members at Tandem Transplantation & Cellular Therapy Meeting of ASTCT and CIBMTR February 12-15, 2025 | Hawai'i Convention Center Thursday, February 13, 2025

    Oral Presentations:

    Saar Gill, MD, PhD

    "ACT To Sustain: Adoptive Cell Therapy to Sustain Access to Non-Commercialized Genetically Modified Cell Therapies" 

     

    Nils Wellhausen, PhD 

    'Selection for an HIV-Resistant Immune System by Multiplex Epitope Base-Edited CD45 CAR-T Cell Therapy" 

     

    Daniel Brown

    “Cytokine-Mediated Expansion of Clonal Hematopoiesis after CART Cell Therapy” 

     

    Poster Presentation:

    Joanne Baek

    “Base-Editing Human Hematopoietic Stem Cells to Enable Venetoclax Post-Transplant Maintenance Therapy for AML”