SNUG01

SNUG01 is a first-in-class targeted gene therapy designed to treat amyotrophic lateral sclerosis (ALS). It uses a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver the human TRIM72 (Tripartite Motif Protein 72) gene via intrathecal administration. TRIM72 is a protein with neuroprotective properties that was identified through research at Tsinghua University in China. The therapy aims to counteract ALS pathogenesis through multiple synergistic mechanisms including reducing oxidative stress by scavenging reactive oxygen species (ROS), restoring mitochondrial homeostasis, suppressing stress granule dysregulation, inhibiting neuroinflammatory cascades, and enhancing neuronal membrane repair capacity. Unlike mutation-targeting gene therapies, SNUG01 leverages multiple neuroprotective pathways, potentially benefiting over 90% of ALS patients who suffer from sporadic (non-familial) disease.