ALS News Today: MDA 2025: INS1202 Gene Therapy Shows Promise In SOD1-ALS Mice

A single dose of Insmed’s investigational gene therapy INS1202 preserved motor function and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations.

The therapy was also found to protect against nerve cell loss and had other beneficial effects on disease-related biomarkers.

“These preclinical data show very high efficacy that translates not only in improvement in survival and motor performance, but also in all of the biomarkers that we have measured,” Laura Ferraiuolo, PhD, an executive director in research and development at Insmed, said in a presentation at this week’s Muscular Dystrophy Association’s (MDA) Clinical & Scientific Conference in Dallas. The talk was titled, “CSF Delivery of INS1202 AAV9-SOD1-shRNA rescues muscle function and hallmarks of neurodegeneration in a disease model of ALS.”