Human Stem Cell Use: Adult or Tissue Stem Cell


Matrix Assisted Cell Transplantation of Promyogenic Fibroadipogenic Progenitor (FAP) Stem Cells

Research Objective We seek to develop a cell based-hydrogel therapy to improve outcomes in patients with muscle degeneration. The technology will improve muscle through sustained release of cell-based cytokines. Impact While designed for rotator cuff injuries based on the model, low back pain and spinal degeneration as well as traumatic muscle loss would be well […]

A Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste Grafting

Research Objective Articular paste graft containing MSCs and an adhesive hydrogel that support cartilage growth will be combined for an effective and functional stem cell based cartilage repair procedure. Impact The proposed biologic cartilage repair therapy results in accessibility of an effective, low cost, one-step and functional biologic solution to those with cartilage injuries and […]

New noncoding RNA chemical entity for heart failure with preserved ejection fraction.

Research Objective Modified synthetic noncoding RNA molecule Impact Heart failure with preserved ejection fraction Major Proposed Activities Lead optimization Perform extensive preclinical testing and select a therapeutic candidate. Develop and test preliminary potency assays based on mechanistic insights. Demonstration of injury-modifying bioactivity in a clinically-relevant human progenitor cell population. Optimize formulation and dosing for intravenous […]

Development of a new therapeutic for directing target specific stem cell migration and treatment

Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells Impact Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS Major Proposed Activities Complete […]

Small Molecules to inhibit Nemo-like Kinase for Treatment of Diamond Blackfan Anemia

Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan Anemia (DBA). Impact If small molecule NLK inhibitors are identified that are effective in improving the anemia of DBA and nontoxic, then treatment and transfusions […]

The First Orally Delivered Cell Therapy for the Treatment of Inflammatory Bowel Disease

Research Objective The goal of this project is to develop the first ORAL cell therapy as a breakthrough treatment for inflammatory bowel disease [IBD]. Impact We engineered a new way to deliver cells ORALLY instead of by injection. In doing so, we will better reach the inflamed tissues and provide a much-needed new treatment for […]

Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia

Research Objective The objectives of this study are to advance a stem cell gene therapy for the immunodeficiency XLA, defining the final therapeutic candidate and showing therapeutic activity in a relevant mouse model. Impact XLA can be treated with chronic immunoglobulin replacement, but may be sub-optimal due to infections and inflammatory complications. Stem cell gene […]

Modulation of the Wnt pathway to restore inner ear function

Research Objective We aim to identify drug regimens that stimulate endogenous progenitors in the inner to regenerate to restore hearing or balance functions. Impact Treatment for irreversible hearing loss and balance disorders is limited, a drug regimen to reverse is highly impactful. Major Proposed Activities Production of R-spondin proteins Drug testing in neonatal cochlear cultures […]

Mesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosis

Research Objective We propose to develop mesenchymal stem cell derived extracellular vesicles (MSC-EV) as treatment for lung fibrosis Impact MSC-EV are promising for several lung diseases, but we need to better understand how they work, where they go in the body, and whether there is a subset of MSC-EV with better efficacy Major Proposed Activities […]

Preclinical Development of An HSC-Engineered Off-The-Shelf iNKT Cell Therapy for Cancer

Research Objective The expected outcome is a therapeutic candidate, allogeneic HSC-engineered HLA-I/II-negative human iNKT cells, that can potentially be used as an off-the-shelf cellular therapy for treating cancer. Impact The proposed off-the-shelf HSC-engineered iNKT therapy has the potential to become a general cancer immunotherapy for treating multiple cancers and a large population of cancer patients. […]