Zocaglusagene Nuzaparvovec

AT-845 AT845 DNA (recombinant adeno-associated virus vector AT845 mouse muscle-type creatine kinase enhancer plus mouse muscle-type creatine kinase promoter plus human lysosomal α-glucosidase precursor codon-optimized transgene plus simian virus 40 polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying) Recombinant, non-replicating adeno-associated virus serotype 8 (rAAV8) vector encoding codon-optimized human acid alpha-glucosidase (GAA), also called lysosomal α-glucosidase or acid maltase, under control of a mouse muscle creatine kinase (MCK) enhancer/promoter followed by a simian virus 40 (SV40) intron, terminated by the SV40 late polyadenylation sequence, and flanked by AAV2 inverted terminal repeats (ITRs) zocaglusagene nuzaparvovec [INN] ZOCAGLUSAGENE NUZAPARVOVEC [USAN]