Human Stem Cell Use: Adult or Tissue Stem Cell
Living Synthetic Vascular Grafts with Renewable Endothelium
Translational Candidate LXW7 coated ePTFE vascular graft achieves rapid endothelization and improved graft patency by capturing endogenous endothelial progenitor cells Area of Impact This technology will produce long-lasting vascular grafts with self-renewable “living” endothelium and improve dialysis patients’ quality of life Mechanism of Action The arteriovenous ePTFE dialysis graft approach is the most common form […]
Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer and can be conducted earlier than the current methods of prenatal testing (e.g. chorionic villous sampling and amniocentesis) Mechanism of Action Our test uses next […]
Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions
Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer and can be conducted earlier than the current methods of prenatal testing (e.g. chorionic villous sampling and amniocentesis) Mechanism of Action Our test uses next […]
Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells
Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells undergo genome editing to restore the deficient enzyme. Reintroducing these edited cells replaces the patient's bone marrow, establishing a lasting enzyme reservoir. The bone marrow […]
Neurogenic hydrogel stimulation of stem cells to regenerate radiation-damaged salivary glands
Translational Candidate Ceviginate is a neuromimetic encapsulated in a hydrogel Area of Impact Dry mouth as a result of injury to the salivary glands by radiation therapy for head and neck cancer Mechanism of Action Regenerate damaged salivary gland tissue through neurogenic stimulation of stem cells Unmet Medical Need Current treatment options for dry mouth/xerostomia, […]
Adenine Base Editing for Autologous Hematopoietic Stem Cell Gene Therapy of CD3δ SCID
Translational Candidate The translational candidate is Autologous Hematopoietic Stem and Progenitor Cells from CD3δ SCID Patients Corrected by Adenine Base Editing Area of Impact The candidate will provide treatment for a fatal inborn error of immunity (CD3δ SCID) affecting a genetically-isolated population. Mechanism of Action Autologous Hematopoietic Stem and Progenitor cells from CD3δ SCID Patients […]
Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction
Translational Candidate Injectable biomaterial derived from the natural scaffolding of pig hearts Area of Impact Improving the quality of life of patients with heart attacks Mechanism of Action The proposed mechanism of action is through recruitment of the body's own stem cells and reducing inflammation to heal the heart. Unmet Medical Need The prevalence of […]
Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease
Translational Candidate The candidate is CD34+ HSPCs transduced ex vivo with a LAMP2 lentiviral vector. Area of Impact Danon Disease, Lysosomal Storage Diseases, Drug Development for Rare Disease Mechanism of Action Engrafted HSPC progeny will supply normal LAMP2B to the heart, liver, muscle, and brain via lysosomal cross-correction. Specifically, macrophages transfer lysosomes containing LAMP2B to […]
Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA)
Translational Candidate Autologous CD34+ hematopoietic stem and progenitor cells (HSPC) with BTK gene insertion for treatment of X-linked agammaglobulinemia (XLA). Area of Impact The candidate will provide improved outcomes for patients with XLA, by allowing autologous transplantation with reduced intensity conditioning Mechanism of Action The Drug Product has biological activity of hematopoietic stem cells (HSC) […]
Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody
Translational Candidate s15.BsAb.BCMA-CAR NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with multiple myeloma Mechanism of Action s15.BsAb.BCMA-CAR NK cells are umbilical cord blood-derived CD34+ hematopoietic stem cells that are engineered to include two components of the BCMA-CAR and the anti-NKG2D-anti-GPRC5D BsAb. s15.BsAb.BCMA-CAR NK cells can launch dual […]