Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has received clinical trial approval from the National Medical Products Administration (NMPA) for its therapy BD211. This gene-modified autologous hematopoietic stem cell therapy is designed for the treatment of thalassemia.
In a preliminary study, BD211 demonstrated safety and efficacy in two severe thalassemia β 0/β patients over a two-year follow-up period. BDgene, in collaboration with Ruijin Hospital, initiated an investigator-led trial for the drug in thalassemia last September under the guidance of the National Health Commission.
β Thalassemia is a type of hereditary blood disorder caused by mutations in the globin gene. Approximately 1.5% of the global population carries the β thalassemia gene, and around 60,000 to 70,000 children with severe β thalassemia are born each year. In China, there are an estimated 300,000 individuals with moderate-to-severe forms of the disease who require long-term blood transfusions and iron removal treatments. BDGene aims to develop a one-time, lifelong treatment option for this condition.- Flcube.com
