Therapeutic/Technology: Gene-modified, donor cell therapy
Extending Immune-Evasive Human Islet-Like Organoids (HILOs) Survival and Function as a Cure for T1D
Research Objective Determine optimal islet transplant conditions and systemic treatments that promote graft survival upon transplantation into immune-competent diabetic subjects. Impact Our proposal will optimize the generation and viability of an unlimited, reproducible source of human engineered islets for transplantation. Major Proposed Activities Demonstrate improved HILO graft survival with FGF1 coating Prolong grafted HILO survival […]
Human iPSC-derived chimeric antigen receptor expressing macrophages for improved cancer treatment.
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in macrophage production and enable these cells to be engineered and manufactured in a standardized, off-the-shelf manner, rather than on a patient-specific basis. Major Proposed Activities […]
Combating COVID-19 using human PSC-derived NK cells
Research Objective We propose to generate NK cells with enhanced immunity from gene-edited human PSCs and use the resultant NK cells to kill SARS-CoV-2-infected cells to combat against COVID-19. Impact The use of gene-edited hPSCs as a source for genetically engineered NK cells will allow us to generate effective immunotherapy for COVID-19 that has no […]
Dual angiogenic and immunomodulating nanotechnology for subcutaneous stem cell derived islet transplantation for the treatment of diabetes
Research Objective Functional human islet like organoids differentiated from human pluripotent stem cells. Impact Providing the immediate cell therapeutic candidate for clinical trial of diabetic patients. Major Proposed Activities Fabrication and characterization of the injectable immunomodulating and pro-angiogenic material components: HA hydrogel, heparin nanoparticles and VEGF clusters. Generation of human islet like organoids from pluripotent […]
Human iPSC-derived chimeric antigen receptor-expressing macrophages for cancer treatment
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in macrophage production and enable these cells to be engineered and manufactured in a standardized, off-the-shelf manner, rather than on a patient-specific basis. Major Proposed Activities […]
Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes
Research Objective Development of immune tolerant human islet-like organoids for transplantation into diabetic patients. Impact Our proposal will progress the development of an unlimited, reproducible source of immune tolerant engineered islets for transplantation into Type I diabetics. Major Proposed Activities Demonstrate efficacy of immune tolerant HILOs in humanized diabetic mice Demonstrate safety of immune tolerant […]
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. […]
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 […]
Preclinical development of an immune evasive islet cell replacement therapy for type 1 diabetes
Research Objective We will produce a universal donor cell (UDC) line by gene editing an embryonic stem cell line. Cell therapies produced from the UDC line will not be rejected by a patient’s immune system. Impact The UDC line will address the bottleneck of patient immunity that is currently slowing development of many potential cell […]
Development of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCs
Research Objective Development of hESC-derived pancreatic beta cells that are protected from allogeneic and autoimmune attack into a cell therapy for type 1 diabetes (T1D) Impact Cell therapy of T1D is challenged by immune rejection. Therefore, we will develop pancreatic progenitors derived from genetically modified hESCs that can evade allogeneic and autoimmune responses. Major Proposed […]