Therapeutic/Technology: Gene-modified, donor cell therapy
hNSC-mediated delivery of ApiCCT1 as a candidate therapeutic for Huntington’s disease
Research Objective The therapeutic candidate is a human Neural Stem Cell that secretes a protein, ApiCCT1, that aids in the prevention of disease phenotypes, for application in treatment of Huntington's disease (HD). Impact No treatment currently exists that can slow or prevent the unrelenting progression of Huntington’s disease, a devastating brain disease, therefore a completely […]
MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Research Objective Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease. Impact It could lead directly to a treatment for Angelman Syndrome, but the approach could be used to alter gene expression in almost any brain disorder. It could overcome the brain […]
Novel Rejuvenated T Cell Immunotherapy for Lung Cancer
Research Objective Through this project, we would like to evaluate how this T-iPSC-based immunotherapy that we have developed can eliminate lung cancer cells effectively in vivo using xenografted SCID mice. Impact This novel T-iPSC-based immunotherapy will provide another effective treatment for lung cancer and possible other malignancies by supplying unlimited number of young and active […]
Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody
Translational Candidate s15.BsAb.BCMA-CAR NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with multiple myeloma Mechanism of Action s15.BsAb.BCMA-CAR NK cells are umbilical cord blood-derived CD34+ hematopoietic stem cells that are engineered to include two components of the BCMA-CAR and the anti-NKG2D-anti-GPRC5D BsAb. s15.BsAb.BCMA-CAR NK cells can launch dual […]
Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1α
Translational Candidate EGFR-CAR_sIL15 NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with metastatic breast cancer, especially HER2-low breast cancer Mechanism of Action EGFR-CAR_sIL15 NK cells are umbilical cord blood-derived CD34+ HSCs that are engineered to target EGFR and express soluble IL-15, and then are differentiated into NK cells. […]
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 […]
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 […]
Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia
Therapeutic Candidate or Device Immune T cells from a patient’s transplant donor engineered to express chimeric antigen receptors for targeted leukemia killing Indication Relapsed or refractory acute myeloid leukemia Therapeutic Mechanism Upon adoptive transfer, patient specific immune T cells that express chimeric antigen receptors will specifically recognize and directly kill leukemia cells expressing CD33. Unmet […]
AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205
Therapeutic Candidate or Device AB-205 is an intravenously infused medicine containing genetically modified human vascular (endothelial) cells intended to repair damaged tissue Indication Treatment to reduce/prevent severe multi-organ complications from curative high-intensity cancer treatments involving stem cell transplantation Therapeutic Mechanism AB-205 acts through paracrine effects from expression of a rich mixture of reparative proteins called […]
A Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas
Therapeutic Candidate or Device Neural stem cells that are genetically engineered to express a cancer-killing virus that specifically targets brain tumor cells Indication Recurrent brain tumors in adults: high grade gliomas (HGG), such as glioblastoma (GBM) Therapeutic Mechanism The neural stem cells will act as carriers to deliver a cancer-killing virus to brain tumors (at […]