MPS II: Plasma cell delivery of iduronate sulfatase
Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II,…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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