Genetic Modification of Stem Cells and T cells to Activate the Immune System to Target Solid Tumors

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Therapeutic Candidate or Device Autologous Peripheral Blood Stem Cells expressing the NY-ESO-1 TCR and a suicide/reporter gene combined with T cells expressing the same TCR Indication Locally advanced (unresectable stage…

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A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

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Therapeutic Candidate or Device ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia Indication Transfusion-dependent beta-thalassemia Therapeutic Mechanism ST-400 is intended to disrupt BCL11A erythroid enhancer in…

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Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant

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Therapeutic Candidate or Device Haploidentical (half-match) T cell depleted blood stem cell transplant with a low-toxic conditioning regimen Indication Adult patients with severe sickle cell disease who are excluded from…

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Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies

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Therapeutic Candidate or Device T cells genetically engineered to express as bispecific Chimeric Antigen Receptor (CAR) targeting CD19 and/or CD22 Indication Patients with relapsed and refractory B cell malignancies Therapeutic…

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Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells

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Therapeutic Candidate or Device Bone marrow stem cells that have been treated by inserting a normal Artemis gene into the DNA using a modified virus called a lentivirus. Indication Children…

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A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC

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Therapeutic Candidate or Device Combination therapy with adenoviral CCL21 gene-modified DC and pembrolizumab Indication Patients with confirmed and measurable stage IV NSCLC expressing PD-L1 in less than 50% of cells…

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Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients

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Therapeutic Candidate or Device MDR-101 is cellular therapy consisting of kidney donor-derived CD34+ HSCs and CD3+ T-cells. Indication Maintenance of kidney allograft function after withdrawal of post-transplant immunosuppressant (IS) drugs…

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Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma

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Therapeutic Candidate or Device Genetically engineered, Centyrin-based, stem cell memory CAR-T cells (CARTyrin T cells) Indication Multiple Myeloma Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that…

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Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)

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Therapeutic Candidate or Device Partially HLA-matched virus-specific T-cell therapy targeting cytomegalovirus, Epstein-Barr virus, and adenovirus. Indication This study will treat persistent viral infections with CMV, EBV, and/or adenovirus in patients…

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