MYO-201

MYO-201 is an investigational gene therapy developed for the treatment of limb girdle muscular dystrophy type 2B (LGMD2B, also known as dysferlinopathy). It is designed as a two-vector adeno-associated virus (AAV) gene therapy that enables muscle cells to produce functional dysferlin protein, which is deficient in LGMD2B. The two viral vectors each contain approximately one half of the DYSF gene, and after simultaneous intravenous administration, these gene fragments self-assemble intracellularly into a full-length, functional gene under the control of a muscle-specific promoter (MHCK7), ensuring expression only in muscle cells[1][15]. The therapy aims to address the underlying genetic cause of LGMD2B by restoring normal dysferlin function.

Other names

AAVrh-74-MHCK7-DYSFAAVrh74-MHCK7-DYSFAAVrh 74-MHCK7-DYSFAAVrh.74.MHCK7.DYSFMYO201MYO-201MYO 201SRP6004SRP-6004SRP 6004