Program Type: Translation
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. […]
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 […]
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 […]
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 […]
Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome
Translational Candidate Human hematopoietic stem cells that have been modified to express a functional WAS gene to treat patients with Wiskott Aldrich Syndrome (WAS) Area of Impact These studies will bring stem cell gene therapy for WAS closer to the clinic especially for those without an HLA match or disease too severe for HSCT Mechanism […]
AAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations
Translational Candidate Therapeutic candidate rAAV8-EF1α-hSLC4A11 is a recombinant AAV vector with single-stranded cDNA encoding the wild-type human SLC4A11 protein. Area of Impact The candidate is for treatment of congenital hereditary endothelial dystrophy, an orphan disease associated with congenital corneal opacification. Mechanism of Action Therapeutic candidate rAAV8-EF1α-hSLC4A11 introduces normal copies of human SLC4A11 gene into the […]