Core Insights - Hangzhou Rongze Biotechnology Group Co., Ltd. has developed the world's first First-in-Class gene therapy product RZ-g001BE, which has received Orphan Drug Designation from the FDA for the treatment of Long QT Syndrome (LQTS) [1][3] - This marks a significant breakthrough in the field of gene therapy for hereditary cardiomyopathy and establishes a foundation for clinical research related to LQTS [1][3] Company Developments - The FDA's recognition of RZ-g001BE as an orphan drug is a milestone in Rongze's international strategy in gene therapy, accelerating the industrialization process of gene therapies for hereditary cardiomyopathy [3] - The company will benefit from U.S. orphan drug policies, including tax credits for clinical costs, waiver of new drug application fees, and seven years of market exclusivity post-approval [3] Industry Context - Long QT Syndrome is a serious condition with a 10-year mortality rate of up to 50% if untreated, and currently, there are no effective curative drugs available [4] - RZ-g001BE utilizes base editing technology to correct genetic mutations associated with hereditary LQTS, potentially offering a cure for the disease [4][9] - The therapy employs a single-base editing tool, achieving an in vivo gene correction rate of 99.20%, effectively eliminating the pathogenic mRNA with a single treatment [7]
荣泽生物集团全球首款遗传性心肌病体内基因疗法获批FDA孤儿药认定
Sou Hu Wang·2025-04-28 08:15