On the morning of June 17, 2024, the launch meeting of the "Single-arm, multicenter, open, intravenous Phase I/II clinical study on the safety and efficacy of GC301 adeno-associated virus injection in the treatment of patients with late-onset Pompe disease" was successfully held at the First Medical Center of the General Hospital of the People's Liberation Army. This is a gene therapy drug independently developed by Beijing GeneCradle Technology Co., Ltd. and the world's first licensed clinical trial that can be used to treat late-onset Pompe disease. Director Yang Guang is the principal investigator leading the study.
GC301 injection is a gene therapy drug that uses recombinant AAV as a vector to carry the GAA gene expression sequence. Animal experimental results show that through a single intravenous infusion, GAA can be widely distributed in the heart, skeletal muscle, liver, nervous system, etc., achieving the purpose of long-term improvement of vitality levels, continuous removal of accumulated glycogen and restoration of muscle strength. This drug has obtained the drug clinical trial approval notice from the Drug Evaluation Center of the State Drug Administration. The project is currently recruiting subjects. Please scan the QR code below to obtain more information about enrollment.
Click on the picture below to read the original report from the PLA General Hospital Medical and Health Service WeChat Official Account.
About GeneCradle
Beijing GeneCradle Technology Co., Ltd. is a national high-tech enterprise with the development of gene therapy drugs mediated by AAV vector delivery technology as its core business. Its mission is to promote China's rare disease gene drugs from basic to clinical and market, benefiting patients and families. The company focuses on the development of gene therapy drugs in the fields of hereditary neuromuscular diseases, genetic metabolic diseases, lysosomal diseases and ophthalmic diseases. By promoting the development and clinical application of rare disease gene drugs, it has a deeper understanding of life and health, and has transitioned gene therapy technologies and products from rare diseases to the treatment and rehabilitation of chronic diseases and other major diseases.