Stage of Program: Translational Stage Programs (2.0 and relevant 1.0 projects)
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 hESC-derived protein therapy that positively regulates the regenerative capacity of post-natal muscle for treating DM1
Translational Candidate We engineered a human embryonic stem cell-secreted signaling protein into a biologic for treatment of skeletal muscle disorders. Area of Impact Skeletal muscle disorders (including DM1 and sarcopenia) remain major unmet needs that require treatments restoring muscle strength and function. Mechanism of Action Our animal data demonstrate an endocrine stimulation by our biologic […]
CAR-Tnm cell therapy for melanoma targeting TYRP-1
Translational Candidate Autologous naive/memory progenitor T cells genetically modified to express a chimeric antigen receptor targeting the Tyrosinase-related protein 1 Area of Impact Patient with melanoma, without response or with relapse after immune checkpoint blockade therapy and patients with rare melanoma subtypes. Mechanism of Action T cells genetically modified to express the 20D7SL CAR detect […]
HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma
Translational Candidate stem cell-based off-the-shelf CAR-iNKT cells Area of Impact multiple myeloma (MM) Mechanism of Action The proposed therapeutic candidate can directly kill MM tumor cells. Unmet Medical Need MM remains an incurable disease, with a high relapse rate. The proposed therapeutic candidate can offer a new treatment opportunity for a broad base of MM […]
Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma
Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately 3 out of 100,000 people/year in the USA with extremely poor prognosis. Mechanism of Action In our proposed system, the first antigen EGFRvIII, which is […]
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 […]
BCMA/CS1 Bispecific CAR-T Cell Therapy to Prevent Antigen Escape in Multiple Myeloma
Translational Candidate A single-chain bispecific chimeric antigen receptor (CAR) targeting BCMA and CS1 will be used to in autologous T-cell therapy for multiple myeloma. Area of Impact Translational candidate will enable treatment of patients with heterogeneous or BCMA– multiple myeloma and prevent cancer relapse due to antigen loss. Mechanism of Action BCMA and CS1 are […]
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 […]