Stage of Program: Candidate Discovery (DISC2, some 1.0 projects)


Modeling Retinitis Pigmentosa using patient-derived human iPSC organoids

Research Objective The objective of this proposal is to develop a human retinal organoid model of adRP to gain insights in pathogenesis and assess clinically relevant approaches to restore RHO protein function. Impact Upon successful completion of this study, we will have established a disease-in-a-dish model and a novel therapeutic approach towards management of the […]

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. […]

Identifying HLA Class I Restricted Peptides That Induce CD8+ T Cells Against SARS-CoV-2

Research Objective A vaccine to help prevent COVID-19 Impact There is a clear need for a vaccine to prevent the spread of the COVID-19 coronavirus that is effective, can be rapidly produced and can be scaled for worldwide demand. Major Proposed Activities Identify structural regions of SARS-Cov-2 that can inhibit viral entry Identify potential regions […]

Application of PGE2/BPV, a muscle stem cell targeting therapeutic, to the treatment of COVID-19 associated diaphragm atrophy

Research Objective Intramuscular delivery of 2 repurposed FDA approved drugs to activate diaphragm stem cells to augment regeneration and restore strength to COVID-19 patients with diaphragm atrophy from ventilation. Impact Currently effective treatments are lacking for diaphragm atrophy due to ventilation to treat COVID-19. Our treatment will promote full recovery of such patients. Major Proposed […]

Development of a novel PIKFYVE kinase inhibitor for the treatment of COVID-19

Research Objective Alveolar type II cells are the stem cells of the lung, and they are killed by SARS-CoV-2. We will determine if a novel PIKFYVE kinase inhibitor prevents SARS-CoV-2 infection of type II cells. Impact Although PIKFYVE inhibition blocks SARS-CoV-2 infection, no known PIKFYVE inhibitors have suitable drug-like properties. We would validate a novel […]

Biomaterial vaccine to enhance the formation of SARS-CoV-2-specific T memory stem cells

Research Objective The objective of this project is to develop an injectable biomaterial platform that can induce TMSCs and boost immunoactivation to vaccines against SARS-CoV-2, which will help protect elderly people. Impact This approach will boost T memory stem cell production to enhance immunization, and address the low/weak immunoresponses to vaccines, especially in the elderly […]

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