GEN6050X

GEN6050X is a first-in-class, intravenously administered cytosine base editing gene therapy developed for Duchenne muscular dystrophy (DMD) patients amenable to exon 50 skipping. It utilizes GenAssist’s proprietary RNA editing-free Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology and is delivered via dual single-stranded adeno-associated virus serotype 9 (AAV9) vectors. The therapy aims to permanently restore dystrophin expression by directly correcting the mutated DMD gene through a one-time systemic administration. In addition to mediating exon skipping, one vector encodes the oTAM base editor under a muscle-specific promoter, while the other delivers three copies of single-guide RNA and human ACTG1 gene, also under a muscle-specific promoter; ACTG1 overexpression further enhances cytoskeletal stability and muscle function. Early clinical data show favorable safety and tolerability with clinically meaningful improvements in motor function and no significant adverse events[1][3][8].

Brand names

GEN6050XGEN-6050XGEN 6050X

Other names

GEN6050XGEN-6050XGEN 6050X