Stage of Program: Candidate Discovery (DISC2, some 1.0 projects)
Platform Technology for Pluripotent Stem Cell-Derived T cell Immunotherapy
Research Objective We will combine a novel method to produce T cells from stem cells with gene editing tools, to create pluripotent stem cells that can serve as a universal source of T cells for cancer immunotherapy. Impact We will address a major bottleneck for T cell immunotherapy: the complexity and therefore limited access to […]
Non-Toxic, Highly-Effective Bioinspired Cryoprotectants for On-Demand Stem Cell Therapies
Research Objective An advanced technology is sought to replace the toxic, ineffective and highly processed components in legacy cryopreservation media. This technology is a fundamentally novel non-toxic freezing media. Impact The transport and storage of stem cell therapies is crippled by freezing media with poor cell preservation. Significantly improved freezing media would directly increase therapy […]
Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome
Research Objective We are seeking to develop site-specific hematopoietic stem cell gene therapy with autologous transplant as a definitive treatment option for X-linked Hyper-IgM Syndrome. Impact These studies would bring stem cell gene therapy for X-HIGM closer to the clinic, as there are currently no options for those without an HLA match or with infections […]
Microenvironment for hiPSC-derived pacemaking cardiomyocytes
Research Objective This proposal investigates the effects of the microenvironment on the development and maintenance of pacemaking function in human induced pluripotent stem cell (hiPSC)-derived cardiomyocytes. Impact Pacemaking function of hiPSC-derived cardiomyocytes is lost over time. Sustainability of pacemaking function of these cells is critical for engineering an biopacemaker from the patient's own cells. Major […]
Multipotent Cardiovascular Progenitor Regeneration of the Myocardium after MI
Research Objective We developed technology to reproducibly prepare large numbers of bonafide cardiac progenitor cells from patient iPSCs. We propose the first test of these cells as a therapy for myocardial infarction. Impact Heart failure resulting from myocardial infarction is responsible for 13% of human mortality (WHO statistic). This proposed therapy is to restore the […]
A Novel Approach to Eradicate Cancer Stem Cells
Research Objective The outcome is a therapeutic candidate ready for Investigational New Drug (IND)-enabling studies to target a central hub of stemness pathways of cancer stem cells (CSC) maintenance and self-renewal Impact To date, the majority of metastatic cancers remain incurable, because CSCs that can grow new tumors evades current therapy. The proposed studies aim […]
A Novel Approach to Eradicate Cancer Stem Cells
Research Objective The outcome is a therapeutic candidate ready for Investigational New Drug (IND)-enabling studies to target a central hub of stemness pathways of cancer stem cells (CSC) maintenance and self-renewal Impact To date, the majority of metastatic cancers remain incurable, because CSCs that can grow new tumors evades current therapy. The proposed studies aim […]
Human Cardiac Chip for Assessment of Proarrhythmic Risk
Research Objective This proposal will develop patient specific ‘heart-on-a-chip’ devices that will significantly impact early screening of drugs to accurately predict drug-induced proarrhythmia and toxicity. Impact Patient specific ‘heart-on-a-chip’ device will significantly reduce the cost of bringing a new drug candidate to market while improving efficacy. Major Proposed Activities To improve the maturity of human […]
Preclinical development of AAV vector-mediated in vivo hepatic reprogramming of myofibroblasts as a therapy for liver fibrosis
Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver failure. Impact The proposed research will develop a new therapy for liver cirrhosis, which can be cured by liver transplantation, but there are not enough […]
A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs
Research Objective Develop a tool that facilitates rapid, cost effective development of optimized GMP-grade hPSC differentiation into functional DA neurons and apply this device to a cohort of PD patient-derived iPSCs. Impact Creating GMP-grade, functionally consistent phenotypes for DA neurons from each patient will significantly increase the likelihood of stem cell-derived DA neuron-based therapy for […]