Human Stem Cell Use: Adult or Tissue Stem Cell
NeuBright, a purified allogeneic cell therapy product for treatment of Dry Age-related Macular Degeneration
Translational Candidate NeuBright is an allogeneic cryopreserved neural stem cell therapy product Area of Impact Dry Age-Related Macular Degeneration (AMD) Mechanism of Action Similar to RPE cells, NeuBright cells restore phagocytic function to the retina, secrete anti-inflammatory and trophic factors (VEGF and BDNF), maintain retinal integrity and prevent vision decline. Cells migrate radially from transplant […]
Therapeutic development of Oxy200, an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis
Translational Candidate A novel oxysterol with bone anabolic and anti-resorptive activity that will effectively and safely treat osteoporosis better than current options. Area of Impact Osteoporosis that results in bone fractures. Mechanism of Action The proposed candidate will target Mesenchymal Stem Cells in the skeleton to stimulate their differentiation into bone forming osteoblasts that will […]
Injectable pro-regenerative scaffold for treating symptomatic peripheral artery disease
Translational Candidate Injectable biomaterial derived from the natural scaffolding of porcine muscle Area of Impact Improving the quality of life of patients with symptomatic peripheral artery disease. Mechanism of Action The proposed mechanism of action is through recruitment of blood vessels and recruitment and differentiation of muscle stem cells. The injected material forms a new […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
An autologous somatic stem cell therapy for the treatment of osteonecrosis
Translational Candidate An autologous somatic stem cell therapy for the treatment of osteonecrosis. Area of Impact Osteonecrosis is a painful, progressive disease for which there is no treatment, save replacing the dead bone with a metal implant. Mechanism of Action Autografts contain skeletal stem cells. In young patients, these stem cells differentiate and give rise […]
Placental Derived Natural Killer Cells to Target Solid Tumor Cancer Stem Cells (CSC)
Translational Candidate Placental-derived stem cells becoming a unique NK-like cell Area of Impact solid tissue cancers Mechanism of Action These expanded cells share many characteristics as NK cells but appear to represent a unique stage in differentiation and a cell-type with advantageous properties. These cells have been demonstrated to persist for much longer periods of […]
Stem Cell-Based iNKT Cell Therapy for Cancer
Translational Candidate Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs) Area of Impact The targeted area of impact for the candidate is cancer therapy, in particular cancers that are lacking existing effective treatments. Mechanism of Action The proposed candidate will generate therapeutic levels of invariant natural killer T (iNKT) cells in cancer patients, helping […]
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 […]
RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration
Therapeutic Candidate or Device Retina pigment epithelial stem cell (RPESC)-derived RPE progeny at 4 weeks of differentiation (RPESC-RPE-4W) Indication Dry age-related macular degeneration (dry AMD). Therapeutic Mechanism Dry AMD involves loss of the RPE cells that support the overlying retina and vision. We transplant laboratory grown RPESC-RPE-4W cell to replace the RPE cells lost in […]