gc301

GC301 is an investigational gene therapy developed for the treatment of Pompe disease, specifically both infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD). It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a codon-optimized human acid alpha-glucosidase (GAA) gene systemically via intravenous injection. The goal is to compensate for GAA enzyme deficiency in affected tissues such as the liver, heart, skeletal muscles, and central nervous system. Preclinical and early clinical data indicate that a single dose can achieve long-term GAA expression with improvements in motor function and quality of life. GC301 has received Orphan Drug Designation from the US FDA for Pompe disease[2][3][4][5][6][7][8].

Brand names

GC301 Adeno-Associated Virus InjectionGC-301 Adeno-Associated Virus InjectionGC 301 Adeno-Associated Virus Injection

Other names

AAV9-SGSHAAV-9-SGSHAAV 9-SGSHadeno-associated viral vector serotype 9 delivered SGSH gene therapy (mucopolysaccharidosis IIIA)SGSH gene therapy (adeno-associated viral vector serotype 9, mucopolysaccharidosis IIIA)