According to the latest information released by the Drug Evaluation Center of the China National Medical Products Administration, the clinical trial application for GC301 adeno-associated virus injection, a gene therapy product independently developed by GeneCradle, has been accepted by the National Medical Products Administration. The product is also a Class 1 therapeutic biological product in terms of registration classification, and its clinical indication is glycogen storage disease type 2 (Pompe disease).

Based on the proof of concept of the Pompe disease gene therapy project (RDGT-003) completed by Ruixi Institute in 2019, GeneCradle took over the baton of preclinical research of Pompe disease gene drugs, and at the same time took over the expectations and attention of domestic Pompe disease patients. After nearly three years of research, the Jinlan team finally pushed the GC301 project to clinical registration acceptance. This is the first AAV gene therapy drug for Pompe disease accepted by CDE in China, hoping to bring patients hope of long-term effectiveness of one-shot treatment.