Disease Focus: Respiratory Disorders
Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis
Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the barriers to accessing airway basal stem cells for gene correction for Cystic Fibrosis (CF) and use a new gene correction strategy to correct >99% of […]
Modulation of human alveolar stem cells to promote lung regeneration and avoid pulmonary fibrosis
Research Objective Understanding regulators of human alveolar lung stem cell function will promote more normal lung regeneration after injury and avoid the nearly untreatable problem of advanced pulmonary fibrosis. Impact Idiopathic Pulmonary Fibrosis (IPF), Adult Respiratory Distress Syndrome (ARDS), and other chronic fibrotic lung disorders. Major Proposed Activities Modify human fibroblasts to create a supporting […]
A phase I/II study of human placental hematopoietic stem cell derived natural killer cells (CYNK-001) for the treatment of adults with COVID-19
Therapeutic Candidate or Device Human placental hematopoietic stem cell derived natural killer cells (CYNK-001) Indication SARS-CoV-2 positive patients requiring hospital admission and have any 2 out of 3 symptoms: fever, cough, or positive disease-related chest x-ray. Therapeutic Mechanism CYNK-001 is allogenic, human placental hematopoietic stem cell-derived NK cells that express the dominant NK cells marker […]
Evaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic Use
Therapeutic Candidate or Device The therapeutic candidate is COVID-19 convalescent plasma (CCP) Indication The target indication is treatment of severe COVID-19 infection Therapeutic Mechanism Neutralizing antibodies are part of the humoral response of the adaptive immune system against viruses, and can be detected in plasma of convalescent individuals. Transfusion of COVID-19 convalescent plasma can provide […]
Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy
Research Objective Using authentic in vitro models of the human lung, complete with inflammatory cells & vessels, we will validate drugs that might be rapidly repurposed for use in patients with COVID-19. Impact The impact will be the avoidance of an animal model once an approved medication hit has been verified by our model. The […]
Chimeric Antigen Receptor Targeting Spike Glycoprotein of SARS-cov2
Research Objective We expect to generate iPSC derived NK cells expressing a CAR against SARS-cov2 that could be used as an off-the-shelf therapy for COVID-19 Impact The proposed studies will provide a novel therapeutic approach to boost the cellular immunity against SARS-cov2, especially for high risk populations. Major Proposed Activities Construction of SARS-cov2 CAR constructs […]
Pro-healing biomaterial for treating lung inflammation associated with COVID-19
Research Objective Pro-healing biomaterial to treat lung inflammation and promote recovery. Impact COVID-19 associated acute respiratory distress syndrome Major Proposed Activities Evaluate biomaterial for ability to reduce lung inflammation in rodent model Evaluate biomaterial for ability to recruit stem cells in the lung Evaluate biomaterial for ability to improve lung function in rodent model Evaluate […]
Stem cell-based rapid identification of SARS-CoV-2 T cell epitopes and T cell receptors for therapeutic use
Research Objective We will identify SAR-CoV-2 T cell epitopes for vaccine development and specific TCRs for adoptive T cell therapy using a stem cell-based platform to generate specialized dendritic cells in vitro. Impact New methods to rapidly identify T cell epitopes would greatly accelerate development of vaccines and TCR-based therapeutics, and in the setting of […]
Identifying a lead compound for COVID19 using high throughput screening with lung stem cell organoids
Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be repurposed for COVID-19 infection. Impact If successful, we will find a therapy to treat COVID-19 infection and prevent the lung complications that are so deadly. […]