Program Type: Discovery


Assessing the Functional, Immunologic and Microbiologic Characteristics of Human Livers Created in Chimeric Pigs

Research Objective Generating human liver using pig as a bioreactor Impact The livers produced will be used for transplantation into patients with end-stage liver disease, metabolic disorders, and for metastatic liver disease not amenable to resection Major Proposed Activities MILESTONE 1 TASKS 1) Procure liver from control pig 2) Place on XVIVO Perfusion Device MILESTONE […]

RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis

Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive immunity to limit inflammation/heart injury in myocarditis. Boosting regulatory T cells is not yet a viable option. Major Proposed Activities Investigate the mechanism(s) by which […]

Reprogramming Somatic Cells into iPSCs Engineered with an Anti-PSCA CAR to Develop Allogeneic Off-the-Shelf Cell Therapy to Treat Pancreatic Cancer

Research Objective Our candidate product PSCA-CAR_s15 uiNK is derived from transduction of iPSCs selected from the most ideal source and episomally reprogrammed from mature NK cells or CD34+ cells. Impact Cell Functionality and Quality; Scale up and Manufacture Major Proposed Activities Development and characterization of induced pluripotent stem cells (iPSCs) via somatic cell reprogramming of […]

Expanded Capacity AAV Retinal Gene Therapy Enabled by Efficient RNA-Joining Technology

Research Objective A novel AAV-based gene therapy candidate for use in treatment of inherited ocular disease. Impact AAV vectors have a limited cargo capacity (<5kb), preventing their use in the treatment of many untreatable genetic diseases caused by large genes. Major Proposed Activities In vitro optimization and selection of gene therapy candidates In vivo testing […]

Vax-CT to promote formation of cancer-specific T memory stem cell for personalized cancer immunotherapy

Research Objective A vaccine booster to induce antigen-specific T memory stem cells that will help enhance the long-term immunity against cancer recurrence Impact Cancer recurrence presents an unmet medical need. Cancer vaccines are promising, but often lack a long-term protection. We will induce T memory stem cells (TMSCs) to boost the long-term immunity. Major Proposed […]

Excitatory spinal interneurons from human pluripotent stem cells to treat spinal cord injury

Research Objective The primary objective of this research is to test whether excitatory human V2a spinal interneurons engineered from PSCs can repair the damaged spinal cord and restore motor function. Impact Currently no existing therapies are capable of repairing the injured spinal cord. Our therapeutic cell candidate – human ‘V2a’ spinal interneurons – could address […]

Reversal of dysregulated myelopoiesis in breast cancers and cancer stem cells to boost antitumor immunotherapy

Research Objective A new antiestrogen drug will be developed to stop breast cancer (BC) by direct effects on BC cells including stem cells and indirect action on specific procancer immune cells that surround the cancer. Impact Substantial numbers of patients with localized breast cancer (BC) and essentially all patients with advanced BC become resistant to […]

Drug Discovery for Duchenne Muscular Dystrophy Using Patient-Derived Human iPSCs

Research Objective We will utilize human induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) patients for drug testing and drug discovery for this rare genetic disease. Impact Diverse iPSC lines that recapitulate patient phenotypes will supplement preclinical studies to de-risk clinical trials while identifying a therapeutic target for DMD-associated cardiomyopathy. Major Proposed Activities […]

A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells

Research Objective A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells Impact We aim to develop a novel genome editing based therapy for Artemis-deficient severe combined immunodeficiency that would improve upon prior gene therapies in efficacy, safety, and scalability. Major Proposed Activities Discover optimal approach for nonviral […]

In Utero Treatment of Duchenne Muscular Dystrophy with Non-viral Gene Editing

Research Objective To develop a lipid nanoparticle/mRNA complex that can safely and efficiently edit muscle stem cells in utero, correct the dystrophin mutation, and develop a treatment for Duchenne muscular dystrophy Impact If successful, we will have developed an effective and low-cost treatment for Duchenne muscular dystrophy and a robust method to safely and efficiently […]