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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Development of OSSM-007, cryopreserved interferon-gamma primed allogeneic MSCs, for treatment of steroid refractory acute graft versus host disease

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Therapeutic Candidate or Device OSSM007: cryopreserved, interferon-gamma-primed bone marrow mesenchymal stem cells (BM-MSC) Indication acute Graft versus host disease (aGVHD) resulting from hematopoietic cell transplantation Therapeutic Mechanism Immunomodulation of host-reactive…

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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…

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