Stage of Program: Translational Stage Programs (2.0 and relevant 1.0 projects)
T-Pure: Peripheral Blood Processing Tool for Point of Care CAR-T Manufacturing
Translational Candidate Tool kit added directly to peripheral blood allowing for purification of T cell enriched product suitable for (CAR)-T cell generation. Area of Impact The goal is to generate a tool that can address one of the most expensive and rate-limiting steps in the production of genetically engineered cells Mechanism of Action Our application […]
Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug Discovery
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical studies in human samples to derisk clinical trials and save time and costs. Mechanism of Action We will have several non-invasive human-derived stem cells collected […]
Human iPSC-derived micro-heart muscles for high-throughput cardiac drug discovery
Translational Candidate In vitro miniaturized array of heart muscle amenable for use in efficient high-throughput drug discovery and screening campaigns. Area of Impact Effective high-throughput screening of drugs on human heart muscles does not exist, hindering the discovery of therapeutics to treat heart failure. Mechanism of Action Current approaches for drug discovery often miss a […]
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 […]