Adult or Tissue Stem Cell – Page 13

Stem Cell-Based iNKT Cell Therapy for Cancer

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Post published:July 3, 2025
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Translational Candidate Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs) Area of Impact The targeted area of impact for the candidate is cancer therapy, in particular cancers that are…

Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

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Post published:July 3, 2025
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Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs…

Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

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Post published:July 3, 2025
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Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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Post published:July 3, 2025
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RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration

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Post published:July 3, 2025
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Therapeutic Candidate or Device Retina pigment epithelial stem cell (RPESC)-derived RPE progeny at 4 weeks of differentiation (RPESC-RPE-4W) Indication Dry age-related macular degeneration (dry AMD). Therapeutic Mechanism Dry AMD involves…

The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

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Post published:July 3, 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 prenatally Therapeutic Mechanism Placenta-derived mesenchymal…

Phase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme

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Post published:July 3, 2025
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Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting B7-H3 (B7-H3CART) Indication Brain tumors in adults: Glioblastoma Multiforme (GBM) Therapeutic Mechanism Progenitor B7-H3CART…

The UCLA Delayed Immunological Tolerance after Kidney Transplantation Program

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Post published:July 3, 2025
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Therapeutic Candidate or Device CD34+ hematopoietic stem/progenitor cells (HSPC) and CD3+ cells Indication Phase I/II trial evaluating the safety/efficacy of infusion of donor CD34+ HSPCs and CD3+ T cells into…

Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect

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Post published:July 3, 2025
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Therapeutic Candidate or Device KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic Indication Treatment of persistent corneal epithelial defect, a cornea defect…

Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

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Post published:July 3, 2025
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Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias…

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