bidridistrogene xeboparvovec + glucocorticoid

Bidridistrogene xeboparvovec + glucocorticoid is a combination therapy consisting of bidridistrogene xeboparvovec, an investigational gene therapy, co-administered with a glucocorticoid, typically oral prednisone or equivalent, as an immunosuppressive adjunct. Bidridistrogene xeboparvovec is a self-complementary, recombinant adeno-associated virus (AAVrh.74)-based gene therapy encoding a codon-optimized, full-length human beta-sarcoglycan (SGCB) gene. It is being developed as a single-dose intravenous treatment to restore SGCB expression in skeletal muscle cells of patients with limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4), who have mutations in the SGCB gene resulting in loss of functional beta-sarcoglycan protein and consequent progressive muscle wasting[1][2][3][4][5]. Glucocorticoids are used to mitigate immune responses to the AAV vector and to manage liver transaminase elevations or immune-related adverse events observed post-infusion[1][2]. Preclinical and clinical results show restoration of SGCB protein, reduced muscle damage markers (CK), and improved muscle function in treated patients. Bidridistrogene xeboparvovec was originally developed by Nationwide Children’s Hospital and Myonexus Therapeutics as MYO-101, and later acquired and further developed by Sarepta Therapeutics[3]. The therapy is in Phase 3 clinical development[5].

Other names

bidridistrogene xeboparvovec + glucocorticoid