INS1202 / Insmed - LARVOL DELTA

Impact of intrathecal delivery of INS1202 AAV9-SOD1-shRNA on hallmarks of neurodegeneration in a murine disease model of ALS and patient-derived in vitro models (ALS-MND 2025) - "SOD1G93A mice treated with INS1202 (n¼ 7–12/sex/ group) (1) showed widespread biodistribution of INS1202 DNA(2) showed a 70% improvement in median survival (p<0.0001; median age of death 220 days) compared to vehicle-treated mice, (3) gained and sustained physiological weight (p<0.0001),(4) retained muscle function measured via the rotarod test (p<0.0001), and muscle force measured with the muscle physiology Aurora system (p<0.0001). Additionally, compared to vehicle-treated, SOD1G93A mice treated with INS1202 exhibited significantly lower plasma neurofilament levels presymptomatically (p50,p<0.01,n¼ 6/group), at p110 ± 10, when vehicle-treated mice show signs of paralysis (p<0.0001,n¼ 4–6/ group) and at p200 ± 5, which was the established end of study. INS1202 treatment of SOD1G93A mice resulted in rescue of lumbar MN numbers (n¼ 2/sex/group,p¼ 0.024), and glia marker staining intensity to levels observed in wildtype..."

Preclinical • CNS Disorders • SOD1

The Company's Investigational New Drug (IND) filing for INS1202, an intrathecally-delivered gene therapy for patients with Amyotrophic lateral sclerosis (ALS), has been cleared by the FDA. (PRNewswire)

IND • Amyotrophic Lateral Sclerosis

Intrathecal delivery of INS1202 AAV9-SOD1-shRNA rescues hallmarks of neurodegeneration in a disease model of ALS and targets glia and neurons in the spinal cord and brain of non-human primates (ASGCT 2025) - "A 3-month GLP NHP toxicology study is nearing completion to evaluate safety in a larger number of animals, which will provide additional safety and tolerability data. Disease Focus of Abstract:Neuromuscular Disorders"

Amyotrophic Lateral Sclerosis • CNS Disorders • CHAT • GFAP