INS1201 is an investigational adeno-associated virus 9–micro-dystrophin gene transfer therapy under development as a potential treatment for Duchenne muscular dystrophy (DMD). Administered into the ce...

INS1201 is an investigational adeno-associated virus 9–micro-dystrophin gene transfer therapy under development as a potential treatment for Duchenne muscular dystrophy (DMD). Administered into the cerebrospinal fluid (CSF), this delivery approach efficiently targets skeletal and cardiac muscle. Here, we present preclinical findings that demonstrated improved muscle histopathology and functional efficacy in the mdx mouse model starting at 2.0E+11 vector genomes (vg)/mouse and safety in Good Laboratory Practice–compliant toxicology studies in mice and nonhuman primates (NHPs) following INS1201 treatment.

Insmed reports strong ARIKAYCE growth across all regions, exceeding 2024 guidance. Pipeline expands with Brensocatib NDA submission and FDA clearance for DMD gene therapy INS1201.