MyoDys45-55

MyoDys45-55 is a gene editing therapy in preclinical development for Duchenne muscular dystrophy (DMD), a severe and fatal muscle-wasting disease. Developed by MyoGene Bio, this therapy uses CRISPR/Cas9 gene editing to permanently delete exons 45 to 55 of the DMD gene, which is a mutation hotspot affecting approximately half of all DMD patients. By removing these exons, the therapy reframes the DMD gene and restores expression of a shortened but functional dystrophin protein—similar to that found in Becker muscular dystrophy, which has a much milder clinical course. The approach aims for permanent correction at the DNA level, potentially stabilizing or slowing disease progression and improving quality of life and lifespan for patients. Delivery is achieved via adeno-associated virus (AAV) vectors[1][3][5][6].

Other names

MyoDys