LTI-291 is primarily investigated for its potential therapeutic applications in neurodegenerative diseases, particularly Parkinson’s disease. It functions as a selective and brain-penetrant GCase (glucocerebrosidase) activator, which is crucial for addressing the underlying pathology associated with GBA1 mutations. By enhancing GCase activity, LTI-291 aims to improve lysosomal function and reduce the accumulation of toxic substrates, which are key contributors to neuronal damage. This makes it a promising candidate for disease-modifying treatment strategies, offering hope for slowing disease progression and improving quality of life in patients with Parkinson’s and related disorders. Ongoing research focuses on its efficacy, safety, and potential to complement existing therapies.