WebMay 25, 2015 · Brief Summary: This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia. Condition or disease. Web20 hours ago · Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy. Bluebird already uses a lentiviral vector in its approved gene therapy for beta-thalassemia called Zynteglo.. However, Vertex and CRISPR’s exa-cel is designed to remove a part of the patient’s BC11A gene to increase foetal haemoglobin levels in red blood cells, using …
Thalassemia Clinical Trials Currently Available to U.S. Patients
WebApr 25, 2024 · Bluebird bio’s beta thalassemia gene therapy treatment Zynteglo might have some grounds to support an anticipated high price point, on top of a likely FDA approval, experts tell Pharmaceutical Technology.Potential cost savings regarding blood transfusions and improved quality of life (QOL) weigh in Zynteglo’s cost favour but due to … WebApr 10, 2024 · Editas Medicine has undergone some internal restructuring in Q1 2024, with strategic reprioritization focusing on hemoglobinopathies and in vivo gene editing. A … jonathan bhend
Vertex/CRISPR
WebJan 6, 2024 · Clinical Trial for Beta-Thalassemia Brings Important Insights for Treating Blood Disorders with Stem Cell Gene Therapy Thursday, January 6, 2024 A clinical trial led by physician-scientist Michel Sadelain showed that gene therapy targeting blood stem cells appeared safe and effective. WebNov 10, 2024 · Clinical Trials of Gene Editing in Transfusion-Dependent Beta Thalassemia A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta- thalassemia (TDT) A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent Beta-Thalassemia WebApr 18, 2024 · Gene therapy is showing promise for treating one of the most common genetic disorders. Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got... jonathan bicher ey