Novel methods to eliminate cancer stem cells
Research Objective Our goal is to develop and optimize novel drugs that can attack blood cancer stem cells. These drugs interfere with a target protein, and will prevent relapse of…
Generating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineering
Research Objective We will use integrated gene editing techniques to develop a new CAR-T cell therapy for multiple myeloma treatment Impact Develop an improved CAR-T cell therapy for patients with…
Targeting Cancer Stem Cells in Hematologic Malignancies
Research Objective We will develop a biotherapeutic/monoclonal antibody that blocks the growth of human AML cancer stem cells in vitro and in vivo. Impact Treatment of the cancer stem cell…
Lgr5-mediated self-renewal in B cell selection and leukemia-initiation
Research Objective LGR5-antibody drug conjugate to target LIC in B cell tumors that undergo self-renewal Impact LIC were only defined in myeloid leukemia, while LIC populations in B cell tumors…
Protein tyrosine phosphatase – sigma inhibitors for hematopoietic regeneration
Research Objective We propose to develop a lead small molecule inhibitor of PTPσ, a receptor expressed by human blood stem cells, for the purpose of promoting human hematopoietic regeneration. Impact…
Targeted off-the-shelf immunotherapy to treat refractory cancers
Research Objective This project will use human pluripotent stem cells to produce a standardized, off-the-shelf immunotherapy using novel immune cells that are specifically targeted to cure otherwise lethal cancers. Impact…
Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α-hydroxylase for Acute Myeloid Leukemia Therapy
Research Objective We propose a new approach to differentiation therapy for acute myeloid leukemia by producing local level of high-dose vitamin D in bone marrow via cell therapy with engineered…
Genome editing for causation and reversion of MPN-associated mutations in human hematopoietic stem cells
Research Objective Use gene editing to create tools for the study of mechanisms by which patient-observed mutations lead to myeloproliferative neoplasms. Impact Editing reagents will yield new insight into how…
Hypoimmunogenic iPSC-derived TCR-NK cells for oncology
Translational Candidate iPSC-derived NY-ESO-1 TCR/IL-15 NK cells Area of Impact NK drug product homogeneity and engraftment/response durability will be improved by this hypoimmunogenic iPSC-TCR-NK therapy in cancer patients Mechanism of…
