Disease Focus: Blood Cancer

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 driven disease Acute Myelogenous Leukemia (AML) will be impacted. AML is the most common acute leukemia in adults and current treatments are largely ineffective. Major […]

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 remain elusive. LICs give rise to drug-resistance and relapse and remain unsolved clinical problems in B cell tumors. Major Proposed Activities Proof of concept studies- […]

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 Systemic administration of a PTPσ inhibitor can accelerate hematologic recovery in thousands of patients who have received myelosuppressive chemo- or radiotherapy. Major Proposed Activities Generate […]

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 Unlike current immunotherapies produced on a patient-specific basis, iPSC-derived immune cells are targeted to tumors with high specificity, no off-target effects and without need for […]

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 hematopoietic stem cells Impact If proven successful, the proposed research can serve as a major breakthrough in the treatment of multiple subtypes of AML and […]

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 acquired MPN-associated mutations cause disease by overproduction of various cell types and pave the way for gene editing therapies to reverse MPNs. Major Proposed Activities […]

Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody

Translational Candidate s15.BsAb.BCMA-CAR NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with multiple myeloma Mechanism of Action s15.BsAb.BCMA-CAR NK cells are umbilical cord blood-derived CD34+ hematopoietic stem cells that are engineered to include two components of the BCMA-CAR and the anti-NKG2D-anti-GPRC5D BsAb. s15.BsAb.BCMA-CAR NK cells can launch dual […]

Overcoming resistance to standard CD19-targeted CAR T using a novel triple antigen targeted vector

Translational Candidate A tri-specific chimeric antigen receptor (CAR) T cell product that will prevent relapse since targets 3 different tumor antigens Area of Impact Relapse associated with single or double antigen-targeted CAR T cells Mechanism of Action By being able to target 3 different tumor antigens simultaneously on a single CAR product, there is much […]

CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia

Translational Candidate CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies Area of Impact Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative options Mechanism of Action The proposed candidate functions as Chimeric Antigen Receptor (CAR) T cell. When the CAR-T cell recognizes tumor cell expressing the designed […]

HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma

Translational Candidate stem cell-based off-the-shelf CAR-iNKT cells Area of Impact multiple myeloma (MM) Mechanism of Action The proposed therapeutic candidate can directly kill MM tumor cells. Unmet Medical Need MM remains an incurable disease, with a high relapse rate. The proposed therapeutic candidate can offer a new treatment opportunity for a broad base of MM […]