Gene Cradle, a Beijing-based gene therapy specialist, has achieved a milestone by administering its GC301 to the first patient with late-onset Pompe disease (LOPD) in a regulatory study in China. This marks the nation’s first patient dosing of an adeno-associated virus (AAV) gene therapy for LOPD.
GC301 is an innovative AAV gene therapy developed to address Pompe disease, employing a one-time intravenous injection strategy aimed at systemic expression. This approach is designed to directly compensate for the genetic defects in the GAA enzyme gene across various tissues, including the liver, myocardium, skeletal muscle, and central nervous system. Prior clinical trials have shown promising results, with infantile-onset Pompe disease (IOPD) patients discontinuing enzyme replacement therapy post-treatment and experiencing improvements in exercise capacity.
Pompe disease, formally known as glycogen storage disease type II, is a rare autosomal recessive genetic disorder stemming from a mutation in the gene encoding the acid alpha glucosidase (GAA) enzyme within lysosomes. It has been recognized as part of the first batch of rare diseases in China.
The ongoing study, conducted at the Chinese PLA General Hospital, continues to enroll eligible subjects, specifically LOPD patients who are six years of age or older at the time of enrollment.- Flcube.com
