AAV9-midlength-dystrophin

AAV9-midlength-dystrophin (INSMid) is an investigational gene therapy being developed by Insmed for the treatment of Duchenne muscular dystrophy (DMD). Unlike traditional single-vector gene therapies that are limited by the packaging capacity of adeno-associated virus (AAV) vectors, this therapy utilizes a dual-vector system and a novel RNA end-joining (REJ) technology. By delivering two separate AAV9 vectors carrying complementary synthetic DNA molecules, the REJ technology enables the splicing and joining of two RNA fragments within the target cell. This process reconstitutes a larger, 248 kilodalton (kDa) midlength-dystrophin protein, which contains more functional domains than the standard ~140 kDa micro-dystrophin. Preclinical studies in mdx mouse models have demonstrated robust expression of the midlength-dystrophin in skeletal and cardiac muscles, leading to significant improvements in muscle strength and contractile torque.

Other names

AAV9-mid-dystrophinAAV-9-mid-dystrophinAAV 9-mid-dystrophindual AAV9-mid-dystrophin construct