AL01811

AL01811 is a preclinical, selective small molecule inhibitor of β-glucosidase 2 (GBA2) with first-in-class potential as an oral disease-modifying treatment for Parkinson’s disease. The drug is designed to address lysosomal dysfunction, which plays a key role in the pathology of neurodegenerative diseases such as Parkinson’s. By inhibiting GBA2, AL01811 reduces lysosomal pH and increases levels of the lysosomal proton pump vATPase, helping maintain the acidic environment required for proper lysosome function. This mechanism may help prevent the accumulation of misfolded proteins like alpha-synuclein in nerve cells—a hallmark feature of Parkinson’s disease. The compound is being developed through a collaboration between Alectos Therapeutics and Biogen, with Biogen responsible for development, manufacturing, and commercialization[1][2][3][4][5][7].