Therapeutic/Technology: Gene-modified, personalized cell therapy
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 […]
Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome
Translational Candidate Human hematopoietic stem cells that have been modified to express a functional FOXP3 gene to treat patients with IPEX Syndrome Area of Impact These studies will bring stem cell gene therapy for IPEX closer to the clinic especially for those without an HLA match or disease too severe for HSCT Mechanism of Action […]
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 […]
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 […]
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 […]
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 […]
DEBCT: Genetically Corrected, Induced Pluripotent Cell-Derived Epithelial Sheets for Definitive Treatment of Dystrophic Epidermolysis Bullosa
Translational Candidate DEBCT is an autologous iPS-derived COL7A1-corrected keratinocyte graft indicated for the treatment of all chronic open wounds in patients with RDEB. Area of Impact RDEB patients lack type VII collagen and have chronic wounds that lack an abundance of keratinocyte stem cells. DEBCT skin grafts will close wounds. Mechanism of Action RDEB patient […]
Development of ROR1 CAR-T cells to target cancer stem cells in advanced malignancies
Translational Candidate Autologous ROR1 CAR-T cell transduced with a lentiviral vector containing scFv (cirmtuzumab) with CD28, CD137, CD3zeta signaling domains Area of Impact ROR1 expressing cancer stem cells in solid tumors and hematologic tumors Mechanism of Action ROR1 CAR is a 3rd generation chimeric construct with an internal endodomain that transmits a CD3 zeta signal […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]