INS1201

INS1201 is an investigational adeno-associated virus (AAV)-based gene therapy being developed by Insmed for the treatment of Duchenne muscular dystrophy (DMD). It is designed as a micro-dystrophin gene replacement therapy delivered via a targeted intrathecal (IT) route, which directly targets the central nervous system. This delivery approach aims to address both skeletal and cardiac muscle at lower, safer doses compared to systemic intravenous gene therapies, by concentrating the required therapy at the site of need and thus greatly reducing the potential for systemic toxicity and off-target effects. The therapy's mechanism involves functional gene replacement to restore dystrophin protein function in affected tissues. The program is currently in Phase 1 clinical trials in ambulatory male children with DMD due to dystrophin gene mutations.

Other names

INS1201INS-1201INS 1201