Therapeutic/Technology: Gene Therapy (All)


AAV9-Cas13 gene therapy for Angelman syndrome

Research Objective AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons. Impact Angelman syndrome is a rare (1 in 15,000 births) neurogenetic disorder caused by loss of UBE3A in the brain, causing severe developmental delay, ataxia […]

Hypoxia-specific Production of Exosomes from iPSC-derivatives for Myocardial Repair

Research Objective A lead therapeutic candidate will be selected: 1) exosomes from hypoxia-injured iPSC-derived cardiomyocytes (iCMs), 2) exosomal miRNA cluster, and 3) siRNA inhibition of exosomal target gene, Notch3. Impact Effective targeted therapy to restore the injured and vulnerable myocardium is urgently needed to reduce the high mortality of HF patients. Promising discovery of iPSC […]

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 […]

Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS

Research Objective We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS). Impact ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients. Major Proposed Activities Selection of the lead drug by testing […]

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 […]

Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia

Research Objective The objectives of this study are to advance a stem cell gene therapy for the immunodeficiency XLA, defining the final therapeutic candidate and showing therapeutic activity in a relevant mouse model. Impact XLA can be treated with chronic immunoglobulin replacement, but may be sub-optimal due to infections and inflammatory complications. Stem cell gene […]

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. […]

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. […]