Stage of Program: Translational Stage Programs (2.0 and relevant 1.0 projects)
COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage
Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit to COVID-19 affected patients. Mechanism of Action Our drug candidate, Berzosertib, works as a treatment against COVID-19 by blocking a critical step in virus replication. […]
Global gene expression profiling of single inhibitory neurons derived from human stem cells
A Translating Center designed to expedite the development and delivery of high quality stem cell therapies to patients
A successful partnership, resulting in the creation of a Translating Center recognized as the leader in providing end-to-end preclinical development services (preclinical research, CMC, regulatory) to accelerate the availability of stem cell treatments to patients with unmet medical needs. The proposed Translating Center will benefit the lives of patients in CA with serious unmet medical […]
An Accelerating Center designed to expedite the development and delivery of high quality stem cell therapies to patients
A successful partnership, resulting in the creation of an Accelerating Center recognized as the leader in providing end-to-end clinical development services (regulatory, clinical, data management) to accelerate the availability of stem cell treatments to patients with unmet medical needs. The proposed collaborative research will benefit the lives of patients in CA with serious unmet medical […]
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 […]