Project Objective: Proof of Concept
Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome
Research Objective We propose a platform to generate transplantable thymus organoids derived from human pluripotent stem cells designed to treat severe immunodeficiencies in children affected by 22q11 DS Impact Our product could impact 22q11DS and many other pathologies characterized by absence, degeneration or injury of the thymus and resulting in severe immunodeficiencies. Major Proposed Activities […]
Chimeric Antigen Receptor-Engineered Stem/Memory T Cells for the Treatment of Recurrent Ovarian Cancer
Research Objective We are developing a tumor-associated glycan-targeting CAR T cell with inducible cytokine production that drives T cell stem/memory phenotype and persistence for effective treatment of ovarian cancer. Impact 25% of ovarian cancer patients recur within 6 months. Targeting cancer stem cells with a persistent progenitor CAR T cell product offers a potent strategy […]
Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer
Research Objective We will 1) identify non-invasive bladder cancer patients with (pre)cancerous urothelium by single-cell RNA-seq and 2) replace this dangerous lesion with normal hESC-derived bladder progenitors. Impact Replacement of corrupted (pre)cancerous urothelium with pluripotent cell-derived normal bladder progenitors will provide a definitive treatment for bladder cancer, expected to eliminate recurrence. Major Proposed Activities To […]
Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs
Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum Disorder (ASD). Impact Our goal is to develop a small molecule to treat Autism Spectrum Disorder (ASD), which currently affects 1/68 children born in the […]
Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas
Research Objective We will develop a non-viral gene editing technology to replace the endogenous TCRα locus of stem memory T cells with transgene TCRs that are specific to brain cancer neoantigens. Impact Gliomas are lethal tumors often affecting children and young adults. Therapy using Tscm directed to attack truncal neoantigens in these tumors may provide […]
Universal Pluripotent Liver Failure Therapy (UPLiFT)
Research Objective Universal Pluripotent Liver Failure Therapy (UPLiFT) is composed of 2 lines- UPLiFT0 ( from LiPSC-GR1.1) and UPLiFT1 which will be derived from gene edited universal human pluripotent stem cells. Impact In some liver-based metabolic diseases, replacement of 5-10% of the liver mass may salvage the patient. Transplantation of hepatic progenitors from universal donor […]
Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases
Research Objective We will discover small molecule compounds that correct disease in eyecups (retinal organoids) differentiated from patient iPSCs with photoreceptor diseases. Impact Our small molecule agents will provide new treatments for achromatopsia and cone-rod dystrophy. These are rare hereditary blinding diseases with no cures Major Proposed Activities Transcriptomic and proteomic profiling of control and […]
Engineering Lifelong Cellular Immunity to HIV
Research Objective We aim to uncover a therapeutic approach to attempt to treat and potentially cure HIV infection using gene modified blood forming stem cells to enhance the immune response against HIV. Impact The study will allow a potentially curative treatment for HIV infection, which currently doesn’t exist. This will eliminate the need to administer […]
Targeting Cancer Stem Cells in Hematologic Malignancies
Research Objective We will develop a biotherapeutic/monoclonal antibody that blocks the growth of human AML cancer stem cells in vitro and in vivo. Impact Treatment of the cancer stem cell driven disease Acute Myelogenous Leukemia (AML) will be impacted. AML is the most common acute leukemia in adults and current treatments are largely ineffective. Major […]
Towards hepatocyte cell replacement therapy: developing a renewable source of human hepatocytes from pluripotent stem cells
Research Objective To develop a consistent and abundant source of transplantable human hepatocytes for transplantation. Impact Developing an abundant and consistent source of human hepatocytes that can be used to treat patients with liver failure. Major Proposed Activities To determine the degree by which human pluripotent stem cell (hPSC)-derived hepatocytes engraft and restore liver function […]