Biogen and Alectos Therapeutics have announced that they will collaborate on the development of AL01811, a novel β-glucosidase 2 (GBA2) inhibitor and first-in-class small molecule with the potential to become a disease-modifying treatment for Parkinson’s disease.
The accumulation of the misfolded protein alpha-synuclein is a key feature of Parkinson’s. Previous research has suggested that a reason alpha-synuclein builds up in nerve cells is because of malfunctioning lysosomes, which are responsible for ridding cells of waste.
Lysosomes need an acidic environment to work properly. AL01811 blocks GBA2 and both reduces lysosomal pH and increases levels of vATPase, a proton pump that helps maintain the acidity levels needed for proper lysosome function. Blocking GBA2 has demonstrated efficacy in a mouse model of the liposomal storage disorder, Batten disease.
“Through this collaboration with Alectos, we hope to improve the lives of people living with Parkinson’s disease by advancing the research and development of a potential-first-in-class oral treatment that may slow disease progression,” said Priya Singhal, Interim Head of R&D at Biogen.