Human Stem Cell Use: Adult or Tissue Stem Cell
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. […]
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 […]
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 […]
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 […]
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 […]
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle globin and induction of anti-sickling fetal hemoglobin Unmet Medical Need Sickle cell is a severe disease with protean manifestations. The only curative therapy is an […]
Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle cell disease (adults and adolescents) with repeated, severe painful and lung events are eligible for the clinical trial Therapeutic Mechanism Blood stem cells are harvested […]