Lysosomal Therapeutics has raised $20 million in Series A financing from Roche, Lilly and Sanofi to develop glucocerebrosidase (GCase) activator for the treatment of Parkinson’s disease. In May last year, the startup gained a $4.8 million seed commitment from a syndicate led by Atlas Venture.
Lysosomal was co-founded by former Genzyme CEO Henri Termeer. In 2011, the company’s scientific co-founders Dimitri Krainc and Joseph Mazzulli revealed that functional loss of GCase in neurons causes accumulation of α-synuclein. The less active GCase is, the more α-synuclein builds up.
Alpha-synuclein is a protein of unknown function primarily found in neural tissue. The aggregation of α-synuclein plays a role in Parkinson’s disease. Reducing α-synuclein expression or block its aggregation may prevent or delay the onset of Parkinson’s disease.
Mutations in GCase gene cause Gaucher disease but also increase the risk for Parkinson’s disease. Lysosomal aims to develop drugs that could keep GCase active.
Lysosomal isn’t the only company to develop anti-synuclein therapy. In September 2013, Biogen Idec (NASDAQ: BIIB) teamed up with Amicus Therapeutics (NASDAQ: FOLD) to develop GCase activators for the treatment of Parkinson’s disease.
Just two months ago, former Onyx CEO Tony Coles started a new company called Yumanity Therapeutics to develop small molecules that could reverse phenotypes caused by α-synuclein. In January 2015, Neuropore Therapies and UCB entered into an agreement to develop NPT200-11 which stabilizes conformations of α-synuclein and slows the progression of Parkinson’s disease. Moreover, Austria-based AFFiRiS is testing α-synuclein vaccine PD01A in clinical trials.
 Cell. 2011, 146(1), 37-52.