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- In preclinical studies presented today at the RNA Leaders USA Congress 2025, investigational ARO-MAPT demonstrated potent and long-lasting MAPT mRNA and Tau protein suppression with uniform distribution throughout the CNS after subcutaneous administration in non-human primates
Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) today announced that it has filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-MAPT, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies including Alzheimer's disease, a progressive neurodegenerative disease characterized by cognitive and functional decline. Alzheimer's disease is the most common cause of dementia and is estimated to affect 32 million people worldwide and is part of a group of neurodegenerative diseases called tauopathies that are marked by the abnormal accumulation and formation of tau tangles in neurons.
ARO-MAPT is Arrowhead's first investigational RNAi-based therapy to utilize a new proprietary delivery system which, in preclinical studies, has achieved blood-brain-barrier penetration and deep knockdown of target genes across the central nervous system (CNS), including deep brain regions, after subcutaneous injections. This underscores Arrowhead's leadership in the delivery of siRNA to multiple tissues and cell types throughout the body utilizing its proprietary and differentiated Targeted RNAi Molecule (TRiM™) platform.
Posted In: ARWR
