What happens when you put a scientific discovery in the lab together with some experienced biopharma executives and a group of high-profile investors focused on new ideas in Cambridge, MA? You get Lysosomal Therapeutics, a newly hatched biotech venture which plans to follow the genetic underpinnings of Gaucher disease into the clinic with an experimental therapy that can also address Parkinson's disease.
Lysosomal Therapeutics is getting started with Kees Been--most recently the head exec at EnVivo during the ramp-up stage--at the helm, with advice coming from Bob Carpenter, Peter Wirth and the legendary Henri Termeer, all formerly at Genzyme, where they built a reputation for treating Gaucher. They were lured in by the prospect of developing drugs based on the observations of Dimitri Krainc and a postdoctoral fellow named Joseph Mazzulli when they were working together at Krainc's lab at Massachusetts General Hospital.
Gaucher disease has a well-recognized link to Parkinson's. Gaucher is triggered by mutations in the gene for glucocerebrosidase (Gcase). And the investigators found that increasing GCase activity in human neurons of both Gaucher's disease and Parkinson's patients can "normalize" lysosomal function, improving neuronal survival.
The strategy is pretty simple, Been tells FierceBiotech. First you find the right molecule that can go into the brains of Gaucher's patients--who are at a high risk of developing Parkinson's--and then follow that trail to the neurodegenerative disease. Termeer, who's a director at Mass General, quickly understood the potential when Krainc explained it to him.
"It's a reflection of the science," says Been. "There really is something there. We know that by replacing the deficient enzyme you have an effect" on the disease. "Do this another way, with a molecule that goes into the brain, should have the same effect."
This is also just the first such "pairing" of Gaucher with a neurodegenerative condition, adds Been. More programs will follow, but he's a bit reluctant to say which diseases are on the radar screen right now.
Termeer brought in his old Genzyme colleagues Carpenter, the new company's chairman, and Wirth and recruited Been to head what is now a 6-person staff, soon to swell to 10 as they get the full research team together.
They're working with a $4.8 million bankroll from some well known investors. Atlas Venture's Bruce Booth is taking one of the lead roles in this startup, with some financial help from Lilly Ventures, Hatteras Venture Partners, Sanofi-Genzyme BioVentures and Roche Venture Fund.
In many ways the new biotech represents the reason why Boston has emerged as one of the top global hubs for drug research and biotech startups. You see here the confluence of capital, experience and new ideas that are at the heart of every new company in the industry. Few places around the world can rival this kind of infrastructure.
- here's the release (PDF)
Special Report: The top 15 cities for biotech venture funding