GC301 AAV Gene Therapy for Late-Onset Pompe Disease Enters Phase I/II Clinical Trial in China
Genecradle
Gene Therapy Drug Developer
Recently, the kick-off meeting for the "Single-arm, Multi-center, Open-label, Intravenous Injection Phase I/II Clinical Study on the Safety and Efficacy of GC301 Adeno-associated Virus Injection for the Treatment of Patients with Late-onset Pompe Disease" was successfully held at the First Medical Center of the Chinese People's Liberation Army General Hospital. This is the world’s first gene therapy drug for the treatment of late-onset Pompe disease to receive clinical trial approval, independently developed by Beijing Genecradle Therapeutics Co., Ltd. Director Yang Guang is leading the study as the principal investigator.
Professor Yang Guang, Director of the Pediatrics Department at the First Medical Center of the Chinese People's Liberation Army General Hospital, Deputy Chief Physician Hu Linyan, Dr. Dong Xiaoyan, Co-founder of Beijing Genecradle Therapeutics Co., Ltd., Dr. Yu Shuangqing and Dr. Wang Xiaodong from the Clinical Center, as well as the heads of collaborating clinical trial organizations, were invited to attend the project launch meeting.
Pompe disease, also known as glycogen storage disease type II, is caused by variations in the gene encoding acid alpha-glucosidase (GAA) located at 17q25.3, leading to a deficiency in GAA enzyme activity within lysosomes. This results in the accumulation of glycogen in skeletal muscles, cardiac muscles, and smooth muscles, causing lysosomal swelling and rupture, organ dysfunction, and a series of clinical manifestations. Based on the age of onset and the organs involved, it is divided into infantile-onset and late-onset types. Although late-onset Pompe disease progresses more slowly than the infantile-onset form, it severely impairs the respiratory and motor functions of patients, affecting their quality of life. GC301 injection is a gene therapy drug that uses recombinant AAV as a vector carrying the GAA gene expression sequence. Animal experimental results show that through a single intravenous infusion, GAA can be widely distributed in the heart, skeletal muscles, liver, nervous system, etc., achieving long-term improvement in vitality levels, continuous clearance of accumulated glycogen, and restoration of muscle strength. This drug has received a clinical trial approval notice from the Center for Drug Evaluation of the National Medical Products Administration.
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Disclaimer: The content is sourced from the medical and health services of the PLA General Hospital. This article aims to share knowledge, and all content is for reference only and does not constitute any advice.