Webbluebird bio Announces FDA Approval of ZYNTEGLO®, the First Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions ZYNTEGLO … Web1 day ago · Gene therapy is safer and effective for curing beta-thalassemia than hematopoietic stem cell transplantation (HSCT), reveals a new study. Advertisement << …
Gene Therapy in a Patient with Sickle Cell Disease NEJM
WebFeb 3, 2024 · Efficacy and Safety of Gene Therapy for β-Thalassemia Efficacy and Safety of Gene Therapy for β-Thalassemia Efficacy and Safety of Gene Therapy for β … 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. th-issd
Efficacy and Safety of Gene Therapy for β-Thalassemia
WebAug 25, 2024 · Gene Therapy Offers Promise Although luspatercept-aamt has been a great advancement in the treatment of transfusion-dependent β-thalassemia, a need remains for therapies that can eliminate the need for transfusions and cure the disease. The known genetic origins of β-thalassemia make it an excellent candidate for gene therapy, and 2 … 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 competitor to CRISPR Therapeutics, the company is currently working on EDIT-301 for SCD and TDT as well. Unlike CRISPR’s product, EDIT-301 uses AsCas12a to edit the … WebApr 19, 2024 · LentiGlobin Gene Therapy for β-Thalassemia 02:03 The β-hemoglobinopathies, which include β-thalassemia and sickle cell disease, are among … th-jjl-0043h