NI-112

NI-112 (also referred to as SB_NI_112) is an investigational, brain-penetrant RNA-targeting Nanoligomer™ therapeutic developed by Sachi Bioworks. It is designed to regulate gene expression and combat chronic neuroinflammation, a key driver of neurodegenerative diseases. NI-112 consists of modified antisense nucleic acid oligomers conjugated to gold nanoparticles, which facilitate effective crossing of the blood-brain barrier. The therapeutic targets the mRNA of nuclear factor kappa B (NF-κB) and the NLRP3 inflammasome, downregulating their expression to inhibit neuroinflammatory cascades. NI-112 is currently in Phase 1 clinical development for the treatment of amyotrophic lateral sclerosis (ALS) and is also being evaluated preclinically for Alzheimer's disease, multiple sclerosis, frontotemporal dementia, and inflammatory bowel disease.