Program Type: Translation
PRPE-SF, polarized hESC-derived RPE Soluble Factors, as a Therapy for Early Stage Dry Age-related Macular Degeneration
Translational Candidate PRPE-SF is a preparation of soluble factors from polarized retinal pigment epithelial cells, to support survival of photoreceptors in dry AMD (dAMD). Area of Impact dAMD with early geographic atrophy (RPE dysfunction/photoreceptor degeneration) that does not involve the fovea, with visual acuity better than 20/80. Mechanism of Action PRPE-SF is composed of multiple […]
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 […]
Clinical Translation of Autologous Regenerative Cell Therapy for Blindness
Translational Candidate We are studying autologous induced pluripotent stem cell-derived retinal pigment epithelium (AiPSC-RPE) cells for the treatment of maculopathies. Area of Impact Maculopathies (including AMD, SMD, & MMD) may be treated with AiPSC-RPE cells to replace RPE and support photoreceptors to improve vision. Mechanism of Action AiPSC-RPE cells replace RPE lost to disease, and […]
Developing engineered autologous leukemia vaccines to target residual leukemic stem cells
Translational Candidate A universally applicable, patient-specific leukemia vaccine engineered to express a novel immune stimulatory combination for post-remission therapy Area of Impact There is a critical and unmet need for new and safe treatment for older acute myelogenous leukemia (AML) patients whose current prognosis is poor Mechanism of Action In older patients with AML, treatment […]
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 […]
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 […]
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 […]
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 […]