CDE accepts IND application for Reforgene's novel-target thalassemia gene editing medicine

On August 15, 2022, the IND application for RM001, Reforgene's independently developed beta-thalassemia ex vivo gene editing medicine, was accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (Acceptance No.: CXSL2200378). RM001 is China's first gene editing product with a novel HBG target, for the treatment of beta-thalassemia.

The RM001 product uses CRISPR/Cas gene editing technology to permanently modify the gamma-globin promoter, activating the body's natural fetal hemoglobin synthesis, restoring normal physiological function of red blood cells and freeing patients from blood transfusion, achieving the goal of a one-time administration to cure beta-thalassemia. In early clinical research, Reforgene used HBG novel-target gene editing therapy for the first time in the world to successfully cure 5 patients with transfusion-dependent beta-thalassemia, including one adult patient, achieving excellent treatment results. The preliminary clinical research data were reported at the 27th European Hematology Association Congress (27th EHA Congress) in 2022.

About thalassemia

Thalassemia is the monogenic genetic disease with the widest affected population worldwide. As an inherited blood disease, it is named after its high incidence in the countries along the Mediterranean coast. There remains a huge unmet need in thalassemia treatment, with a lack of universally applicable curative medicines. In China, thalassemia is concentrated in 10 provinces south of the Yangtze River, including Guangdong, Guangxi, Hainan, Fujian and Yunnan. The 2015 China Thalassemia Blue Book research report shows that there are more than 345 million thalassemia gene carriers worldwide, with 30 million carriers in China. The severe genetic burden of thalassemia affects the health and quality of life of the Chinese people. Among them, transfusion-dependent patients require long-term blood transfusion and iron chelation treatment, with long-term organ damage, shorter life expectancy and a significantly higher mortality rate than the normal population.