Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)

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Therapeutic Candidate or Device MZ-9138 is an AAV9 gene therapy Indication WWOX-related epileptic encephalopathy Therapeutic Mechanism MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and…

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Ex vivo Engineering of Autologous Hematopoietic Stem Cells for the Treatment of Hypophosphatasia

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Therapeutic Candidate or Device Hematopoietic stem/progenitor cells collected from patients with hypophosphatasia and genetically modified with a lentiviral vector to release TNALP Indication Hypophosphatasia (HPP) Therapeutic Mechanism We are proposing…

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Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome)

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Therapeutic Candidate or Device Autologous blood stem cells edited to restore iduronidase expression Indication Severe Mucopolysaccharidosis Type 1 (MPS1/ Hurler's syndrome) Therapeutic Mechanism Autologous blood stem cells undergo genome editing…

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Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells

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Therapeutic Candidate or Device TriLeukeVax, an autologous AML vaccine designed to stimulate induction of anti-leukemic cytolytic activity and improve relapse free survival (RFS). Indication Older leukemia patients who achieve remission…

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Late Stage Pre-Clinical Development of a Cirmtuzumab Based CAR T-cell for the Treatment of ROR1+ Hematological Malignancies

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Therapeutic Candidate or Device We are developing ROR1 Chimeric Antigen Receptor (CAR) modified T-cells for the treatment of hematological and solid tumor cancers. Indication The target for our therapy are…

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IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome

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Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy…

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