AVXS-201

AVXS-201 is an investigational gene therapy designed for the treatment of Rett syndrome. It utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the MECP2 gene (methyl-CpG-binding protein 2) to patient cells. The therapy aims to address the underlying genetic cause of Rett syndrome by replacing or supplementing defective MECP2 in affected individuals. Originally developed by AveXis and later acquired and further developed by Novartis Gene Therapies, AVXS-201 was intended as a one-time treatment delivered via intrathecal injection. Despite initial progress through preclinical studies and plans for clinical trials, development was ultimately discontinued after additional preclinical data did not support advancement into human trials[1][3][5][6].

Other names

AVXS201AVXS-201AVXS 201