Disease Focus: Blood Cancer
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 […]
BCMA/CS1 Bispecific CAR-T Cell Therapy to Prevent Antigen Escape in Multiple Myeloma
Translational Candidate A single-chain bispecific chimeric antigen receptor (CAR) targeting BCMA and CS1 will be used to in autologous T-cell therapy for multiple myeloma. Area of Impact Translational candidate will enable treatment of patients with heterogeneous or BCMA– multiple myeloma and prevent cancer relapse due to antigen loss. Mechanism of Action BCMA and CS1 are […]
Developing engineered autologous leukemia vaccines to target residual leukemic stem cells
Translational Candidate A universally applicable, patient-specific leukemia vaccine engineered to express a novel immune stimulatory combination for post-remission therapy Area of Impact There is a critical and unmet need for new and safe treatment for older acute myelogenous leukemia (AML) patients whose current prognosis is poor Mechanism of Action In older patients with AML, treatment […]
Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment
Translational Candidate Human embryonic stem cell (hESC)-derived natural killer (NK) cells to target relapsed/refractory Acute Myelogenous Leukemia (AML) Area of Impact hESC-derived NK cells provide a novel and potent approach to treat relapsed or refractory AML that is resistant to current chemotherapy options. Mechanism of Action hESC-derived NK cells provide a standardized, homogeneous, off-the-shelf cellular […]
A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia
Translational Candidate 17S-FD-895 is a potent small molecule splicing modulator that inhibits aberrant splicing in CSCs that have deregulated SF3B1 expression. Area of Impact Development of 17S-FD-895 could address a major bottleneck to reducing AML mortality by targeting splicing deregulated-CSCs that fuel AML relapse. Mechanism of Action 17S-FD-895 will positively impact patients with AML by […]