GNE-495 is primarily investigated for its potential in treating neurodegenerative diseases, particularly those involving the accumulation of misfolded proteins. It functions as a modulator of the unfolded protein response (UPR) pathway, which is crucial for maintaining cellular homeostasis under stress conditions. By targeting this pathway, GNE-495 aims to reduce the burden of protein aggregates in neurons, thereby slowing disease progression. Its application is particularly relevant in conditions like Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS), where protein misfolding plays a central role. Additionally, its ability to enhance cellular stress responses makes it a promising candidate for further research in other protein aggregation-related disorders.