Scientists suspect there’s a connection between many common neurodegenerative diseases and malfunctioning lysosomes, or processing centers in human cells that break down proteins and eliminate debris. Now a new company called Lysosomal Therapeutics is tapping into the relationship between the two to discover and develop new treatments for rare diseases and common neurodegenerative diseases, starting with Parkinson’s disease.
Lysosomal Therapeutics debuted today with $4.8 million in seed funding from an impressive group of healthcare venture capitalists led by Atlas Venture. Three pharma corporate investment funds – Lilly Ventures, Sanofi-Genzyme BioVentures and Roche Venture Fund – also got in on the round, as did Hatteras Venture Partners and Partners Innovation Fund.
What the new company is proposing to do is use its molecular platform to discover and develop a new class of small molecules that cross the blood-brain barrier, normalize lysosomal function and improve the ability of neurons to survive.
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Its lead program is focused on a link between Parkinson’s disease and the most common lysosomal storage disease called Gaucher’s disease.
“This inventive approach to treating Parkinson’s disease was really facilitated by the success of current treatment strategies, such as enzyme replacement therapy, that have enabled the Gaucher disease patient population to live longer,” President and CEO Kees Been said in a statement. “However, an unfortunate consequence of this longevity is the finding that Gaucher patients experience a 20-fold increased risk of developing Parkinson’s disease.”
When there are genetic mutations in lysosomal enzymes, proteins build up in cells and cause cellular damage. In the case of Gaucher’s, the buildup is caused by a mutation in the glucocerebrosidase (GCase) gene.
Interestingly, as Dr. Dimitri Krainc and Joseph Mazzuli at Massachusetts General Hospital discovered, the toxic deposits that are characteristic of Gaucher’s are also found in Parkinson’s disease. They damage neurons and eventually cause neurons to die, which leads to a loss in cognitive, sensory and motor function.
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The idea, then, is that increasing GCase activity in these patients could not only slow disease progression but potentially reduce their risk of Parkinson’s disease.
Krainc and Mazzuli, both now working in the neurology department at Northwestern University, are co-founders in Lysosomal Therapeutics along with four former leaders at Genzyme: Bob Carpenter, Peter Wirth, Ted Sybertz and former CEO Henri Termeer.
President and CEO Been is a former SVP at Biogen Idec’s oncology business and has led EnVivo Pharmaceuticals and Bionaut Pharmaceuticals. In a statement, he said the new funding will allow the company to move through the lead-generation process and prepare for preclinical development.
Lysosomal Therapeutics is based in Cambridge.
[Image credit: Medline Plus]
