Program Type: Translation
A Novel, Robust and Comprehensive Predictive Tool Using Human Disease-Specific Induced Pluripotent Stem Cells for Preclinical Drug Screening
Translational Candidate A library of induced pluripotent stem cell-derived cardiomyocytes from healthy subjects as well as patients with common hereditary cardiac disorders Area of Impact Preclinical toxicity screening and drug discovery Mechanism of Action Patients with pre-existing cardiac conditions are more susceptible to drug-induced cardiotoxicity than general population. Including iPSCs derived from this subset of […]
Living Synthetic Vascular Grafts with Renewable Endothelium
Translational Candidate LXW7 coated ePTFE vascular graft achieves rapid endothelization and improved graft patency by capturing endogenous endothelial progenitor cells Area of Impact This technology will produce long-lasting vascular grafts with self-renewable “living” endothelium and improve dialysis patients’ quality of life Mechanism of Action The arteriovenous ePTFE dialysis graft approach is the most common form […]
A Rapid Clinical Digital Patient-derived Organoid to Guide Breast Cancer Treatment Decision
Translational Candidate The digital patient organoid (DPO) assay is a microfluidic miniature organoid technology that allows high-throughput drug testing. Area of Impact Our functional DPO assay will guide personalized therapy in patients with MBC and fulfill a gap of huge unmet need Mechanism of Action DPO is a microfluidic miniature organoid technology that enables breast […]
Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer and can be conducted earlier than the current methods of prenatal testing (e.g. chorionic villous sampling and amniocentesis) Mechanism of Action Our test uses next […]
Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer and can be conducted earlier than the current methods of prenatal testing (e.g. chorionic villous sampling and amniocentesis) Mechanism of Action Our test uses next […]
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility Mechanism of Action The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a […]
Targeting pancreatic cancer with Allogeneic Off-the-Shelf PSCA-CAR NK cells
Translational Candidate PSCA-CAR_sIL15 NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with metastatic pancreatic cancer or other cancers that also highly express PSCA Mechanism of Action PSCA-CAR_sIL15 NK cells are umbilical cord blood-derived CD34+ HSCs that are engineered to target PSCA and express soluble IL-15, and then are […]
Combating Ovarian Cancer with Stem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy
Translational Candidate stem cell-based off-the-shelf CAR-NKT cells Area of Impact ovarian cancer (OC) Mechanism of Action The proposed cell therapy candidate, AlloMCAR-NKT cells, can directly kill OC tumor cells through CAR/NKR dual-targeting mechanisms, and can also modulate OC tumor microenvironment (TME) by depleting immunosuppressive tumor-associated macrophages (TAMs) and myeloid-derived suppressive cells (MDSCs) via iNKT TCR-mediated […]
Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells
Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells undergo genome editing to restore the deficient enzyme. Reintroducing these edited cells replaces the patient's bone marrow, establishing a lasting enzyme reservoir. The bone marrow […]
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 Action The MoA of the candidate involves engagement of NY-ESO-1-peptide-presented HLA-A*02 molecules on the surface of tumor cells in tissues by the NY-ESO-1-specific TCR on […]