创新生物医药科技
Search documents
正序生物完成首例新疗法治愈镰刀型细胞贫血病
Xin Hua Cai Jing· 2025-08-26 06:20
Core Insights - The innovative biopharmaceutical company Zhengxu Bio (Shanghai) announced the successful clinical trial of its base editing drug CS-101 for Sickle Cell Disease (SCD), marking the first successful case of curing SCD in China through base editing therapy [1] Group 1: Company Developments - Zhengxu Bio has partnered with the First Affiliated Hospital of Guangxi Medical University to conduct clinical research on CS-101, which has successfully cured its first patient [1] - The treated patient showed a significant and sustained increase in fetal hemoglobin (HbF) levels and a notable decrease in sickle hemoglobin (HbS) levels, stabilizing the HbF to HbS ratio at 6.5:3.5 after six months [1] Group 2: Industry Context - Hemoglobin disorders are the most common single-gene hereditary diseases globally, with approximately 7% of the population carrying abnormal hemoglobin genes, resulting in about 400,000 newborns affected each year [1] - Sickle Cell Disease, caused by mutations in the beta-globin gene, leads to chronic hemolytic anemia and can be life-threatening, with a global carrier rate of about 3.5% and an annual incidence of 300,000 newborns affected [2]