Project Objective: Development Candidate
Develop iPSC-derived microglia to treat progranulin-deficient Frontotemporal Dementia
Research Objective Develop stem cell-based therapy to treat dementia Impact There are no treatments for dementia. If successfully achieved, this study will lead to a cure of a familial form of dementia in the elderly population. Major Proposed Activities Develop a robust human stem cell-derived microglial platform for cell-based therapy Determine short-term safety and efficacy […]
Preclinical Development of An HSC-Engineered Off-The-Shelf iNKT Cell Therapy for Cancer
Research Objective The expected outcome is a therapeutic candidate, allogeneic HSC-engineered HLA-I/II-negative human iNKT cells, that can potentially be used as an off-the-shelf cellular therapy for treating cancer. Impact The proposed off-the-shelf HSC-engineered iNKT therapy has the potential to become a general cancer immunotherapy for treating multiple cancers and a large population of cancer patients. […]
Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease
Research Objective We propose to discover a novel, genetically modified hematopoietic stem cell based treatment for Danon disease, a rare lysosomal storage disease that affects the heart. Impact As the only existing treatment for Danon disease is cardiac transplant, this therapy would significantly meet an unmet need. It also may help many other similar diseases. […]
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 […]