Stage of Program: Translational Tool/Bottleneck


Identifying roadblocks to neural stem cell transplantation into human tissues.

Research Objective We will generate a comprehensive map of human neural stem cell differentiation profiles that will serve as a reference for enhancing neural stem cell-based therapies. Impact Our project will develop improved protocols for human neural stem cells differentiation, enhancing the fidelity, safety and robustness of future cell therapies. Major Proposed Activities Establish quantitative […]

Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies

Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]

Autologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cells

Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury

Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human disease. Impact OPCs have therapeutic potential for spinal cord injury, restoration of cognitive function after cancer radiation therapy, inherited demyelinating disease, and potentially multiple sclerosis. […]

Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing

Translational Candidate HiDef-B8, an iPS cell maintenance medium designed to reduce stem cell GMP manufacturing costs and risk. Area of Impact HiDef-B8 addresses scale-up manufacturing, by being lower-cost ($300/L) and requiring fewer passages per week (1-2 as opposed to 3-5). Mechanism of Action HiDef-B8 underwent extensive empirical optimization and alternative component screening, focused on reducing […]

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 […]

Generation of human universal donor iPS cells

Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection by host immune cells. Mechanism of Action The universal donor cell mitigates immune rejection by host immune cells and is equipped with a suicide gene […]

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 […]

Embryonic stem cell-based generation of rat models for assessing human cellular therapies

Heart failure, diabetes and neurodegenerative diseases are among the leading causes of death and disability worldwide. These diseases are characterized by the loss of specific cell types and can be treated and potentially cured with stem cell-based therapies. Before human stem cells can be used in clinical trials, however, their safety and efficacy need to […]

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