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out the links between the networks with bioinformatics, Licholai says. It’s also designing drug that won’t necessarily shut down, or completely block, the protein network activity, but rather will alter the pathways to make them more stable and preserve their healthy function, Licholai says.
Avid biotech readers will notice anytime you talk about diseases of aging that it sounds a lot like what Cambridge, MA-based Sirtris Pharmaceuticals has been pursuing for years. While Proteostasis doesn’t focus on the same exact targets, known as sirtuins, Licholai says there is some overlap between the companies because the sirtuins are elements of, and are related to, the protein networks that Proteostasis is focusing on.
Proteostasis’ lead program at the moment is being designed to treat Huntington’s disease, although it could be useful in a number of neurodegenerative diseases, Licholai says. It is being aimed at HSF1, a heat shock transcription factor. It’s a master switch regulator of a process cells undergo when they are under stress. This program climbed on the priority list after the study in C. elegans showed a “very dramatic” effect with a small molecule compound that doubled lifespan. The next step is to see if the finding can be confirmed in mice, Licholai says.
Heat shock proteins are familiar as cancer targets, and have long been subject of research and development at companies like Cambridge, MA-based Infinity Pharmaceuticals, Biogen Idec, and Hayward, CA-based Kosan Biosciences before that company was acquired by Bristol-Myers Squibb two years ago. One difference with Proteostasis is that it’s not focusing on cancer, Licholai says.
Other companies, like Cambridge, MA-based FoldRx Pharmaceuticals and Cranbury, NJ-based Amicus Therapeutics, are concentrating on how to stop proteins from causing diseases when they become misfolded. FoldRx is a close relative to Proteostasis, given that Scripps’ Kelly is one of the founders, and HealthCare Ventures invested.
What’s different at Proteostasis, Licholai says, is its focus on the entire network of proteins, and its insistence on marrying bioinformatics talent to map out the networks, along with a traditional drug discovery capability that comes from chemistry, biology, and pharmacology. “I don’t think anybody else is looking at it from a network biology point of view,” Licholai says.
Licholai is not a newbie in the field, he previously worked five years at Amicus, telling its story to doctors, business development pros, and investors. He said he’s seen protein folding and protein network biology rise from obscurity just five years ago, into the kind of thing that hundreds of labs around the world now pursue, and which draws conferences at places like Cold Spring Harbor Laboratory.
“How many times do you get a chance to catch the wave, in a new field of science?” Licholai says. “When I heard this company was being put together by HealthCare Ventures and Jeff Kelly, I thought that sounds great and I really want to get involved.”
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