Disease Focus: Brain Cancer
Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas
Research Objective We will develop a non-viral gene editing technology to replace the endogenous TCRα locus of stem memory T cells with transgene TCRs that are specific to brain cancer neoantigens. Impact Gliomas are lethal tumors often affecting children and young adults. Therapy using Tscm directed to attack truncal neoantigens in these tumors may provide […]
Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma
Translational Candidate SRN-101, an experimental AAV gene therapy for treating glioblastoma Area of Impact Cancer – solid tumors Mechanism of Action Following delivery with an AAV, engineered cytokines are expressed from within the tumor to kill the tumor from the inside out, they are then further secreted to stimulate local immune cells to kill remaining […]
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 […]
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 […]
2nd Generation Vaccine for the Treatment of Glioblastoma
Translational Candidate It is the peptide LEEKKYNYVVVTDHC conjugated to KLH and used as an anti-cancer vaccine. Area of Impact This is a better optimized, more robust vaccine that aspires to greatly improve glioblastoma patient survival over the current vaccine. Mechanism of Action The vaccine stimulates B cell and T cells. We have found this may […]
Phase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ Glioblastoma
Therapeutic Candidate or Device Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Indication Patients with MGMT unmethylated newly diagnosed GBM (Cohort 1), and patients for whom re-resection of recurrent GBM (Cohort 2). Therapeutic Mechanism In our proposed system, the first antigen EGFRvIII, which is expressed exclusively but heterogeneously […]
Phase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme
Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting B7-H3 (B7-H3CART) Indication Brain tumors in adults: Glioblastoma Multiforme (GBM) Therapeutic Mechanism Progenitor B7-H3CART cells will recognize GBM cancer cells expressing B7-H3, become activated, divide, and kill the cancer cells Unmet Medical Need Glioblastoma Multiforme (GBM) is the most […]
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 […]
Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor T Cells for Diffuse Intrinsic Pontine Gliomas and Spinal Diffuse Midline Glioma
Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting GD2 (GD2-CART) Indication Brain tumors in children and young adults: Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG) Therapeutic Mechanism Progenitor GD2-CART cells will recognize DIPG/DMG cancer cells expressing GD2, become activated, divide, and kill the […]
Phase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors
Therapeutic Candidate or Device Autologous chimeric antigen receptor T cells derived from naive/memory T cells and engineered to target IL13Rα2 on pediatric malignant brain tumors. Indication Recurrent/refractory malignant pediatric brain tumors that express the tumor-associated antigen IL13Rα2. Therapeutic Mechanism Naive and memory T cells are harvested from patients and reprogrammed to express chimeric antigen receptors […]