Therapeutic/Technology: Therapeutic Approach
Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models
Translational Candidate Retina organoid sheets (ROs) derived from CSC14 human embryonic stem cells (NIH registry line #0284) manufactured under GMP conditions Area of Impact Retinitis PIgmentosa (RP) (irreversible loss of photoreceptors) due to mutation of photoreceptors and/or other retinal genes Mechanism of Action Proposed mechanism of action is cell replacement, combined with trophic effects. Transplanted […]
Autologous iPSC-derived smooth muscle cell therapy for treatment of urinary incontinence
Translational Candidate Smooth muscle cell progenitors (pSMCs) differentiated from patient iPSCs which is injected into the urethral muscle to regenerate a a weak urethra. Area of Impact Surgery for urinary incontinence is effective in 80% of patients. Our target is those who failed surgery (20%), or those who cannot undergo surgery. Mechanism of Action Our […]
Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment
Translational Candidate Human embryonic stem cell (hESC)-derived natural killer (NK) cells to target relapsed/refractory Acute Myelogenous Leukemia (AML) Area of Impact hESC-derived NK cells provide a novel and potent approach to treat relapsed or refractory AML that is resistant to current chemotherapy options. Mechanism of Action hESC-derived NK cells provide a standardized, homogeneous, off-the-shelf cellular […]
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 […]
A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia
Translational Candidate 17S-FD-895 is a potent small molecule splicing modulator that inhibits aberrant splicing in CSCs that have deregulated SF3B1 expression. Area of Impact Development of 17S-FD-895 could address a major bottleneck to reducing AML mortality by targeting splicing deregulated-CSCs that fuel AML relapse. Mechanism of Action 17S-FD-895 will positively impact patients with AML by […]
DEBCT: Genetically Corrected, Induced Pluripotent Cell-Derived Epithelial Sheets for Definitive Treatment of Dystrophic Epidermolysis Bullosa
Translational Candidate DEBCT is an autologous iPS-derived COL7A1-corrected keratinocyte graft indicated for the treatment of all chronic open wounds in patients with RDEB. Area of Impact RDEB patients lack type VII collagen and have chronic wounds that lack an abundance of keratinocyte stem cells. DEBCT skin grafts will close wounds. Mechanism of Action RDEB patient […]
Development of ROR1 CAR-T cells to target cancer stem cells in advanced malignancies
Translational Candidate Autologous ROR1 CAR-T cell transduced with a lentiviral vector containing scFv (cirmtuzumab) with CD28, CD137, CD3zeta signaling domains Area of Impact ROR1 expressing cancer stem cells in solid tumors and hematologic tumors Mechanism of Action ROR1 CAR is a 3rd generation chimeric construct with an internal endodomain that transmits a CD3 zeta signal […]
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 […]
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 […]
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 […]