Disease Focus: Infectious Disease


Development of Anti-COVID RNAi Therapeutics Using Human iPSC-Derived Alveolar Epithelial Cells

Research Objective To optimize a new approach to deliver Anti-COVID siRNAs into human iPSC-derived lung cells that can selectively kill the COVID virus Impact Our proposal, if successful, will solve the siRNA delivery problem and rapidly open the door to Anti-COVID siRNA therapeutics. Major Proposed Activities Complete synthesis of a new delivery device called a […]

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 […]

Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)

Therapeutic Candidate or Device Novel testing of a cell based therapy (Mesenchymal Stromal Cells) for respiratory failure from ARDS. Indication COVID-19 positive or negative ARDS patients Therapeutic Mechanism It will hopefully improve oxygenation in ARDS patients and potentially decrease mortality. Unmet Medical Need There is an unmet need for more effective treatments for ARDS both […]

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 […]

COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage

Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit to COVID-19 affected patients. Mechanism of Action Our drug candidate, Berzosertib, works as a treatment against COVID-19 by blocking a critical step in virus replication. […]

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 […]

Battling COVID-19 Using Off-The-Shelf HSC-Engineered iNKT Cells

Research Objective allogeneic HSC-engineered iNKT (HSC-iNKT) cells Impact treatment for COVID-19 Major Proposed Activities Milestone 1. Production of AlloHSC-iNKT and UHSC-iNKT cells Milestone 2. Characterization of the AlloHSC-iNKT and UHSC-iNKT cells Milestone 3. Delivery of the new therapeutic candidate The novel SARS-CoV-2 is the cause of the coronavirus disease 19 (COVID-19) pandemic, which is responsible […]

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 […]

Persistent Off-the-Shelf meACE2-CAR-IL-15 NK Cells Derived from CD34(+) Cord Blood Stem Cells to Prevent and Treat COVID-19

Research Objective To develop and characterize meACE-2-CAR-IL15 NK cells expressing a mutated ACE2 and IL-15, allowing specific killing of SARS-CoV-2-infected cells and long in vivo persistence of the engineered cells. Impact To provide a timely, novel, and effective cell therapy for COVID-19, which has no FDA-approved vaccines and only remdesivir has received an emergency-use approval. […]

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