China-based Guangzhou Reforgene Medicine Co., Ltd’s in-house developed β-thalassemia gene editing drug RM-001 (HBG gene modified autologous CD34+ hematopoietic stem cell injection) has obtained tacit clinical trial approval from the National Medical Products Administration (NMPA). This marks a significant milestone for the first thalassemia gene editing novel drug program in China with a new target, aiming to cure transfusion-dependent type β-thalassemia.
Thalassemia Overview
Thalassemia is a hereditary single-gene disease characterized by defective globin genes that cannot produce normal hemoglobin. According to the 2015 Blue Book of Thalassemia in China, over 345 million people worldwide carry the thalassemia gene, with approximately 30 million in China. Among them, around 300,000 patients are transfusion-dependent.
RM-001 Profile
RM-001 utilizes gene editing technology to permanently modify the γ-globin promoter in hematopoietic stem cells. This innovation activates the synthesis of natural fetal hemoglobin in the human body, restores the normal physiological function of red blood cells, and eliminates the need for blood transfusions, achieving a radical cure for β-thalassemia with a single administration. Early clinical studies have shown that RM-001 exhibits excellent clinical efficacy and safety.
Future Development
The tacit approval from the NMPA underscores Guangzhou Reforgene Medicine’s commitment to advancing innovative gene therapies. The company aims to further explore the potential of RM-001 in treating transfusion-dependent β-thalassemia, offering a new hope for patients with this genetic disorder.-Fineline Info & Tech
