When his company raised nearly $5 million in seed funding last year, Lysosomal Therapeutics founder, president, and CEO Kees Been said he had two goals: learn more about the underlying connection between the rare Gaucher disease and Parkinson’s disease, and sort through compounds licensed from the National Institutes of Health to find the best one to turn into a Parkinson’s drug.
The Cambridge, MA-based startup has reached the second goal. It has found its compound and is ready to move it ahead in preclinical studies. The work will be fueled by a Series A round that could close by the end of the month, Been (pictured) said during an interview at the J.P. Morgan conference in San Francisco.
Atlas Venture led LTI’s seed round last year, and former Genzyme CEO Henri Termeer with three other Genzyme alumni were cofounders. Genzyme pioneered enzyme replacement therapy that has extended the lives of patients with Gaucher, a hereditary disease caused by a deficiency in the enzyme glucocerebrosidase (GCase). As Gaucher patients began to live longer, their doctors started noticing that they had a greater than normal incidence of Parkinson’s.
That connection has been borne out by genetic studies at the National Institutes of Health and elsewhere. LTI was founded to exploit the link between levels of GCase and α-synuclein, the protein that clumps up in the brains of Parkinson’s patients.
The link is more like a playground seesaw. When one goes up, the other goes down. In Gaucher patients, a mutation in the gene for GCase prevents it from clearing a fatty substance from cells, which eventually die. Low levels of active GCase also correlate to high levels of damaging synuclein.
LTI is betting the reverse will be true: Boost healthy GCase with a drug, and Parkinson’s patients will benefit as synuclein clears out. LTI has competition. Amicus Therapeutics (NASDAQ: [[ticker:FOLD]]) of Cranbury, NJ, is working on the problem, and its research in the area is under license to Cambridge, MA-based Biogen Idec (NASDAQ: [[ticker:BIIB]]).
Researchers at the University of California, Los Angeles, recently found that an Amicus drug being tested against Gaucher has shown promise for Parkinson’s—albeit only in mice so far.
Part of LTI’s confidence in its own approach comes from work by its scientific founder Dmitri Krainc of the Feinberg School of Medicine at Northwestern University, who has studied its compounds in neurons derived from an extremely rare group of Parkinson’s patients. Those neurons had three copies of the synuclein gene, which meant they produced an overabundance of the damaging protein. But when treated with one of the test compounds, the cells’ synuclein production dropped.
There’s still a ways to go before human testing, however. First, chemical modifications are required to “optimize” the lead compound—make it behave more like a drug. While LTI is doing that, it will also run experiments to see if
