Therapeutic/Technology: Donor cell therapy
iPS-Interneuron Transplantation for Neural Repair after Stroke
Research Objective To determine if transplantation of iPS-interneurons cells (iPS-3i cells) enhances functional recovery in stroke. Impact Successful completion of the proposed studies will develop a brain repair therapy for stroke, an unmet clinical need with significant impact on society. Major Proposed Activities To determine the recovery effect of transplantation of iPS-3i cells in the […]
Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy
Translational Candidate 'Pseudoislets' derive from human islets, but compared to islets have superior survival, function, and diabetes reversal after transplantation. Area of Impact Pseudoislets could transform islet replacement strategies in diabetes by increasing the number and durable function of transplanted islet cells. Mechanism of Action Transplantation of replacement human islet cells is approved in type […]
A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury
Translational Candidate The therapeutic candidate is a central nervous system tissue-derived GMP line developed under DISC2-10753 with an established GMP qualified seed bank. Area of Impact The target is chronic cervical spinal cord injury, which represents approximately 59% of clinical spinal cord injury cases. Mechanism of Action Integration of transplanted human neural stem cells is […]
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 […]
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 […]