Therapeutic/Technology: Donor cell therapy
Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia
Translational Candidate Human induced pluripotent stem cell-derived glial enriched progenitors Area of Impact Vascular dementia and white matter stroke, addressing a current bottleneck of poor scale up for existing cell differentiation protocols. Mechanism of Action Preliminary in vivo efficacy studies indicate that the MOA is in the promotion of new connections in the brain after […]
Human Neural Stem Cells (hNSCs) for neuroprotection in perinatal hypoxic-ischemic brain injury (HII)-Pre-IND-enabling Studies
Translational Candidate An established stable human neural stem cell line unmanipulated genetically & propagated under defined conditions Area of Impact Perinatal asphyxia (also called hypoxic-ischemic injury), a major untreatable cause of cerebral palsy & cognitive disability Mechanism of Action hNSCs rescue the penumbra, the part of the brain lesion following perinatal asphyxia that still has […]
Development of a human stem cell-derived inhibitory neuron therapeutic for the treatment of chronic focal epilepsy
Translational Candidate A cellular therapeutic comprised of inhibitory nerve cells produced from human stem cells Area of Impact Drug-resistant chronic temporal lobe epilepsy Mechanism of Action The product candidate is intended to be delivered into the seizure focus, integrate, and secrete the inhibitory neurotransmitter GABA to rebalance neural electrical activity in the brain and eliminate/reduce […]
Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)
Translational Candidate H9 (WA09) embryonic stem cell-derived neural stem cells with a spinal cord identity (H9-NSCsc) Area of Impact Severe spinal cord injury Mechanism of Action Our candidate therapy for SCI uses human neural stem cells in a gel-like matrix containing growth factors. We aim to fill the injury site with replacement neural stem cells […]
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 […]
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 […]
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 […]
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 […]
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 […]
Pluripotent stem cell-derived chondrocytes for articular cartilage repair
Translational Candidate We propose to develop a universal, off-the-shelf treatment for articular cartilage repair based on pluripotent stem cell (PSC)-derived chondrospheres Area of Impact The proposed therapy could treat the major cartilage lesions present in more than 10% of people under 50; which often result in pain and arthritis Mechanism of Action Untreated cartilage defects […]