Recently, the first patient was successfully administered intravenously in a clinical study of GC301 adeno-associated virus injection for infantile Pompe disease ("A single-arm, multicenter, open, dose-escalation clinical trial of safety, tolerability and efficacy of GC301 adeno-associated virus injection in the treatment of infantile Pompe disease", registration number ChiCTR2200063229). The clinical study was hosted by Feng Zhichun, president of the Bayi Children's Hospital, and conducted by Ma Xiuwei, director of the hospital. It is the first clinical study of intravenous adeno-associated virus injection for the treatment of infantile Pompe disease in the world.

Pompe disease (also known as glycogen storage disease type II or acid maltase deficiency) is an autosomal recessive genetic disease caused by acid alpha-glucosidase (GAA) deficiency, with an incidence of approximately 1/8700~1/15000, and can be divided into two types: infantile and late-onset. Infantile Pompe disease develops within 1 year of age, and usually develops severe symptoms within a few months of birth, manifested as hypertrophic cardiomyopathy and severe systemic hypotonia. The disease progresses rapidly, and most patients die of heart failure and respiratory failure within 1 year of age. Due to the limited clinical treatment options, there is still a huge unmet clinical need for the treatment of infantile Pompe disease.

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