Human Stem Cell Use: Adult or Tissue Stem Cell
Neural Stem cell-mediated oncolytic immunotherapy for ovarian cancer
Translational Candidate A clinically tested tumor-tropic neural stem cell (NSC) platform for effective distribution of oncolytic virotherapy to ovarian cancer metastases Area of Impact This NSC-delivered virotherapy approach will lead to a more efficacious, less toxic treatment for metastatic ovarian cancer and chemoresistent cells. Mechanism of Action CRAd-S-pk7 is a tumor specific replication-competent adenovirus driven […]
Ex Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome
Translational Candidate Human hematopoietic stem cells that have been gene-corrected at the CD40L gene to treat patients with X-Linked Hyper-IgM Syndrome Area of Impact These studies will bring stem cell gene therapy for XHIM closer to the clinic especially those without an HLA match or infections too severe for HSCT. Mechanism of Action The CRISPR/Cas9 […]
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 […]