Therapeutic/Technology: Therapeutic Approach
CAR T cells targeting abnormal N-glycans for the treatment of refractory/metastatic solid cancers
Research Objective Develop genetically modified chimeric antigen receptor T cells to kill incurable solid cancers by targeting a previously un-targetable tumor associated carbohydrate antigen. Impact Refractory/metastatic solid cancers are almost always incurable and have limited therapeutic options. Directing the immune system to kill cancer cells provides an unprecedented new approach. Major Proposed Activities Engineer and […]
Combating Ovarian Cancer Using Stem Cell-Engineered Off-The-Shelf CAR-iNKT Cells
Research Objective HSC-engineered allogeneic mesothelin-targeting CAR-iNKT (AlloMCAR-iNKT) cells Impact treatment of ovarian cancer Major Proposed Activities Milestone 1. Production of the AlloMCAR-iNKT cells Milestone 2. Characterization of the AlloMCAR-iNKT cells Milestone 3. Delivery of the new therapeutic candidate Ovarian cancer (OC) is the leading cause of death among women with gynecological malignancies. In the USA, […]
Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients
Research Objective We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1 diabetes. Impact Our product will replace donor islets for cell replacement therapy in patients with type 1 diabetes, and will provide a readout of cell […]
Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease models
Research Objective We will develop a gene therapy for a major inherited optic nerve disease and test the effectiveness of the treatment by analyzing healthy and patient stem cell-derived mini human retinas. Impact The research will use stem cell-based methods to overcome the shortage of human retinal cells and establish disease models, thus allow testing […]
Novel antisense therapy to treat genetic forms of neurodevelopmental disease.
Research Objective We propose to discovery and evaluate antisense gene therapy for specific mutations underlying debilitating or life-threatening neurodevelopmental diseases including epilepsy and autism syndromes. Impact The conditions are four specific neurodevelopmental syndromes where mutations are well suited to ASO therapy. The bottlenecks are current lack of cellular evidence for ASOs to impact disease course. […]
Drug Development of Inhibitors of Inflammation Using Human iPSC-Derived Microglia (hiMG)
Research Objective We will screen for modifiers of the response to misfolded αSyn and Aβ, and their cognate antibodies. Development of drugs to combat this inflammation is important in neurodegenerative diseases. Impact Inhibiting the immune response to minimize NLRP3 inflammasome activation may prevent the neurotoxic effect of activated microglia, and attenuate disease progression in neurodegenerative […]
Novel methods to eliminate cancer stem cells
Research Objective Our goal is to develop and optimize novel drugs that can attack blood cancer stem cells. These drugs interfere with a target protein, and will prevent relapse of disease. Impact By targeting blood cancer stem cells, these compounds can be used to treat and prevent recurrence of cancer in patients. In the future, […]
Targeting pancreatic cancer stem cells with DDR1 antibodies.
Research Objective A therapeutic antibody to DDR1 for targeting pancreatic cancer stem cells to overcome resistance to chemotherapy and potentiate the treatment of advanced cancer. Impact PDAC is lethal cancer that poorly responds chemotherapy to which it becomes resistant. DDR1 antagonistic Abs should improve chemotherapy responsiveness and may cause tumor regression as a monotherapy. Major […]
Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)
Research Objective This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells. Impact This application will enable development of a therapeutic candidate for the treatment of Myelodysplastic Syndromes, a preneoplastic hematological condition of HSCs. Major Proposed Activities Determine the […]
AAV-dCas9 Epigenetic Editing for CDKL5 Deficiency Disorder
Research Objective We propose a gene therapy for the treatment of a severe infantile epilepsy called CDKL5 Deficiency Disorder using CRISPR-mediated epigenetic editing Impact A transformative treatment for females affected by CDKL5 Deficiency Disorder in addition a platform for the approximately 38 other X-linked intellectual disabilities that predominately affect females Major Proposed Activities Validation of […]