Disease Focus: Neurological Disorders
An optimized human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI)
Translational Candidate Shef6.1 embryonic cells will be enriched for a neural stem cell marker, CD133. These human neural stem cells (hNSCs) are designated as S6.133.hNSCs. Area of Impact Shef6.1 human neural stem cells will be tested as a treatment for memory & behavioral deficits resulting from traumatic brain injury (TBI). Mechanism of Action Traumatic brain […]
Human iPSC-derived GABAergic Progenitors for Alzheimer’s Disease Treatment
Translational Candidate Human iPSC-derived GABAergic interneuron progenitors. Area of Impact Alzheimer's disease and related conditions. Mechanism of Action Transplantation of human iPSC-derived GABAergic progenitors, which will develop into mature GABAergic interneurons, to replace the lost GABAergic interneurons in the hippocampus of AD brains and related disorders. Unmet Medical Need As a complex disease that damages […]
Human Embryonic Stem Cell-Derived Neural Stem Cell Transplants in Amyotrophic Lateral Sclerosis
Translational Candidate Spinal cord injections of human embryonic stem cell (hESC)-derived allogeneic neural stem cells (heNSCs) for treatment of ALS Area of Impact Treatment of Amyotrophic Lateral Sclerosis (ALS) Mechanism of Action Although the exact molecular mechanism of action is unknown, extensive research supports the concept that the behavior of defective astrocytes is key to […]
Process development for establishing an iPSC-based therapeutic candidate for Canavan disease
Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This candidate has the potential to develop into a therapy for Canavan disease, a fatal neurological disease that has no cure or standard treatment. Mechanism of Action The proposed candidate is […]
Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease
Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs and Sandhoff disease patients who have no curative or ameliorating treatment. Mechanism of Action Wild type HexA and HexB will be delivered to affected neurons […]
ASCENT- Advanced Stem Cell Enteric Neuropathy Therapy
Translational Candidate ASCENT – Advanced Superdonor Cellular Enteric Neuropathy Therapy, is a donor progenitor cell population that replaces the enteric nervous system. Area of Impact ASCENT would treat enteric neuropathies including Hirschsprung disease and total intestinal aganglionosis which currently have no direct therapy Mechanism of Action Our goal is to develop an allogeneic “off the […]