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…

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

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…

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

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…

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

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…

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

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…

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

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…

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

Phase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma

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Therapeutic Candidate or Device A promising immunotherapy utilizing a patient’s memory T cells engineered to express chimeric antigen receptors for targeted tumor killing. Indication Malignant glioma (WHO III and IV),…

Continue ReadingPhase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma

Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning

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Therapeutic Candidate or Device Bone marrow stem cells will be transduced with a lentiviral vector to deliver a normal copy of the gamma-chain gene to treat X-linked SCID. Indication x-linked…

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Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase - Severe Combined Immunodeficiency (or…

Continue ReadingEfficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase - Severe Combined Immunodeficiency (or…

Continue ReadingEfficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects