Human Stem Cell Use: Adult or Tissue Stem Cell
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 […]
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.
Therapeutic Candidate or Device ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients. Indication Severe Sickle Cell Disease Therapeutic Mechanism Hematopoietic stem cell transplantation is the only cure for severe sickle cell disease. The ECT-001 expanded cord blood cells will replace the […]
Stimulating endogenous muscle stem cells to counter muscle wasting
Evaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8
Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application
Research Objective Genetically modified cells that help control immune reactions Impact Improved treatment for patients undergoing transplantation Major Proposed Activities To identify the optimal source of regulatory T cells for introduction of the novel IL2 receptor To test the impact of a novel IL2 protein on activating the regulatory T cells To determine the optimal […]
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 […]