Project Objective: Pre-IND/IDE or Equivalent

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 […]

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 […]

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 […]