Translational Candidate A tri-specific chimeric antigen receptor (CAR) T cell product that will prevent relapse since targets 3 different tumor antigens Area of Impact Relapse associated with single or double…
Translational Candidate Autologous human CD34+ HSPC of patients with Friedreich’s ataxia, modified ex vivo using CRISPR/Cas9 to remove the GAA expansion mutation in frataxin Area of Impact Friedreich’s ataxia (FRDA)…
Translational Candidate Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells Area of Impact Pulmonary Arterial Hypertension (PAH), initially associated with Scleroderma (Systemic Sclerosis -SSC), and then applied to other causes…
Translational Candidate A next generation cell therapy product that targets prostate cancer cells Area of Impact Prostate cancer Mechanism of Action The therapeutic candidate when expressed on the surface of…
Translational Candidate CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies Area of Impact Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative…
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,…
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…
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…
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…
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…
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