Clinical – Page 14

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

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Post published:May 9, 2025
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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…

Hematopoetic stem cell gene therapy for the treatment of Tay-Sachs disease

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Post published:May 9, 2025
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Therapeutic Candidate or Device Autologous hematopoietic stem cells transduced with a HexA/HexB expressing lentiviral vector Indication Tay-Sachs disease Therapeutic Mechanism The transplanted gene modified autologous hematopoietic stem cells will engraft…

Hematopoietic Stem Cell Gene Therapy for XCGD

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Post published:May 9, 2025
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Therapeutic Candidate or Device Hematopoietic stem and progenitor cells collected from X-CGD patients modified with a highly regulated lentiviral vector Indication X-linked Chronic Granulomatous Disease Therapeutic Mechanism Lentiviral vector (LV)…

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

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Post published:May 9, 2025
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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…

Plurocart: a novel stem cell-based implant for articular cartilage restoration

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Post published:May 9, 2025
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Therapeutic Candidate or Device Plurocart consists of pluripotent stem cell-derived chondrocytes, seeded onto a scaffold; it is intended to treat damaged cartilage in the knee joint. Indication Plurocart is intended…

Skin regeneration and wound healing with a topical BRAF inhibitor

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Post published:May 9, 2025
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Therapeutic Candidate or Device LUT017 gel is a small molecule inhibitor of BRAF Indication Venus leg non-healing ulcerous wounds Therapeutic Mechanism The administration LUT017 gel will regenerate cutaneous stem cells…

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

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Post published:May 9, 2025
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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…

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

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Post published:May 9, 2025
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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…

Curing Sickle cell Disease with CRISPR-Cas9 genome editing

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Post published:May 9, 2025
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Therapeutic Candidate or Device Blood stem cells collected from individuals with sickle cell disease will have the sickle gene corrected and then given back to the same individual. Indication Sickle…

Placental Mesenchymal Stem Cell Augmentation of Fetal Myelomeningocele Repair

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Post published:May 9, 2025
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Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenataly Therapeutic Mechanism Placenta-derived mesenchymal…

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