Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors

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Most normal tissues are maintained by a small number of stem cells that can both self-renew to maintain stem cell numbers, and also give rise to progenitors that make mature…

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A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells

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β-thalassemia is a genetic disease caused by diverse mutations of the β-globin gene that lead to profoundly reduced red blood cell (RBC) development. The unmet medical need in transfusion-dependent β-thalassemia…

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Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetes

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Diabetes exacts a tremendous toll on patients, their families, and society. Autoimmune Type 1 diabetes, often called juvenile-onset diabetes, is caused by a person’s own immune system mistakenly destroying their…

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A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia by Transplantation of Autologous Hematopoietic Stem Cells Transduced with the Lentiviral Vector LentiGlobin® Encoding the Human β-A-T87Q-…

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[REDACTED] plans to carry out a Phase 1/2 study to evaluate the safety and efficacy of [REDACTED] for the treatment of β-Thalassemia Major(BTM). [REDACTED] consists of autologous patient hematopoietic stem…

Continue ReadingA Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia by Transplantation of Autologous Hematopoietic Stem Cells Transduced with the Lentiviral Vector LentiGlobin® Encoding the Human β-A-T87Q-…