Stephan A. Grupp

About

Stephan A. Grupp, MD, PhD, is a Professor of Pediatrics (Oncology) at the University of Pennsylvania and a leading expert in CAR T cell therapy and pediatric transplant/cell therapy. He serves as an Attending Physician in Pediatrics at the Children's Hospital of Philadelphia, where he is also the Medical Director of the Cell and Gene Therapy Laboratory. Grupp's research focuses on the development and preclinical testing of engineered cell therapies and signal transduction inhibitors in leukemia, with extensive experience in pediatric trials utilizing these therapies. He has led most of the CTL019 (CD19 CAR) clinical trials at CHOP and globally, contributing to the field of highly active CAR T cell therapy, culminating in the FDA approval of CTL019/tisagenlecleucel as Kymriah, the first CAR T product and gene therapy approved in the US.

Research topics

• Medicine
• Internal medicine
• Immunology
• Biology
• Oncology
• Genetics
• Cancer research
• Pathology
• Cell biology
• Bioinformatics

Selected publications

• CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

  New England Journal of Medicine · 2020 · 1793 citations

  enhancer. More than a year later, both patients had high levels of allelic editing in bone marrow and blood, increases in fetal hemoglobin that were distributed pancellularly, transfusion independence, and (in the patient with SCD) elimination of vaso-occlusive episodes. (Funded by CRISPR Therapeutics and Vertex Pharmaceuticals; ClinicalTrials.gov numbers, NCT03655678 for CLIMB THAL-111 and NCT03745287 for CLIMB SCD-121.).

  DOI

• ‘Off-the-shelf’ allogeneic CAR T cells: development and challenges

  Nature Reviews Drug Discovery · 2020 · 1143 citations

  DOI

• Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma

  Blood Advances · 2020 · 428 citations

  Senior authorCorresponding

  Tisagenlecleucel is a CD19 chimeric antigen receptor (CAR) T-cell therapy approved for treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL) and adults with non-Hodgkin lymphoma (NHL). The initial experience with tisagenlecleucel in a real-world setting from a cellular therapy registry is presented here. As of January 2020, 511 patients were enrolled from 73 centers, and 410 patients had follow-up data reported (ALL, n = 255; NHL, n = 155), w…

  DOI

• In vivo hematopoietic stem cell modification by mRNA delivery

  Science · 2023 · 297 citations

  Hematopoietic stem cells (HSCs) are the source of all blood cells over an individual's lifetime. Diseased HSCs can be replaced with gene-engineered or healthy HSCs through HSC transplantation (HSCT). However, current protocols carry major side effects and have limited access. We developed CD117/LNP-messenger RNA (mRNA), a lipid nanoparticle (LNP) that encapsulates mRNA and is targeted to the stem cell factor receptor (CD117) on HSCs. Delivery of the anti-human CD117/LNP-based editing system yiel…

  PublisherOA PDFDOI

• Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immune effector cell-related adverse events

  Journal for ImmunoTherapy of Cancer · 2020 · 265 citations

  Senior authorCorresponding

  Immune effector cell (IEC) therapies offer durable and sustained remissions in significant numbers of patients with hematological cancers. While these unique immunotherapies have improved outcomes for pediatric and adult patients in a number of disease states, as 'living drugs,' their toxicity profiles, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), differ markedly from conventional cancer therapeutics. At the time of article prepara…

  DOI

Recent grants

• NIH Grant R29AI040111

  NIH · $612k · 2001

• Defining and overcoming intrinsic T cell dysfunction to enable pediatric immunotherapy

  NIH · $3.7M · 2019–2024

• Radiobiology and Imaging Program

  NIH · $93.0M · 1997–2027

Frequent coauthors

• David T. Teachey

  Children's Hospital of Philadelphia

  410 shared

• Shannon L. Maude

  University of Pennsylvania

  405 shared

• Carl H. June

  Parker Institute for Cancer Immunotherapy

  336 shared

• David M. Barrett

  Kite (United States)

  313 shared

• Michael A. Pulsipher

  282 shared

• Franco Locatelli

  Bambino Gesù Children's Hospital

  250 shared

• Donna A. Wall

  Children’s Hospital at TriStar Centennial

  239 shared

• Haydar Frangoul

  University College London

  201 shared

Education

• B.S.

  University of Cincinnati

  1981

• Ph.D.

  University of Cincinnati College of Medicine

  1985

• M.D.

  University of Cincinnati College of Medicine

  1987

Similar researchers at University of Pennsylvania

• John Q. Trojanowskih-296
• Craig B. Thompsonh-277
• Daniel J Raderh-236