ASO2

ASO2 is a designation used for preclinical antisense oligonucleotide (ASO) candidates, most notably a **VCP (valosin-containing protein) modulator** developed by **Ionis Pharmaceuticals**. VCP, also known as **transitional endoplasmic reticulum ATPase**, is a member of the AAA+ ATPase family that plays a critical role in protein quality control, cellular homeostasis, and the degradation of misfolded proteins via the ubiquitin-proteasome system. Mutations or dysfunction in VCP are linked to neurodegenerative and muscular disorders, including inclusion body myopathy with Paget disease and frontotemporal dementia (IBMPFD) and amyotrophic lateral sclerosis (ALS). Additionally, the term "ASO2" is frequently employed in research to identify specific lead sequences targeting the **SNCA** gene (encoding **alpha-synuclein**) for the treatment of **Parkinson's disease** and other synucleinopathies. These ASOs are designed to reduce the production of pathogenic proteins by promoting the RNase H-mediated degradation of target mRNA, thereby preventing the accumulation of toxic aggregates in the nervous system.

Other names

ASO 2ASO2ASO-2VCP-targeting ASOSNCA-targeting ASO