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molecules that are well on their way to being in clinical trials,” Ogier said, “but we also have a very powerful discovery platform.”
For you science junkies, here’s why blocking the HDAC6 enzyme could be useful in treating diseases: in studies, the enzyme has shown to play a key role in functions in diseased cells that destroy and dispose of damaged proteins. But when the enzyme is inhibited, these so-called misfolding proteins pile up in diseased cells to a point where the cells self-destruct. This could be useful in treating certain illnesses.
The way Ogier tells it, the company grew out of a research collaboration between Dana-Farber physician and researcher Kenneth Anderson, an expert on multiple myeloma, and Harvard chemist Stuart Schreiber, who has co-founded such well-known Massachusetts biotechs as Vertex Pharmaceuticals (NASDAQ:VRTX), Ariad Pharmaceuticals (NASDAQ:ARIA), and Infinity Pharmaceuticals (NASDAQ:INFI), as well as biotech startup Forma Therapeutics. Anderson, a scientific founder of Acetylon, and Schreiber were among the lead researchers in a study that showed that a novel compound that inhibited HDAC6 could play a role in killing blood cancer cells. Other scientific co-founders of the biotech are Dana-Farber and Harvard Medical School researcher James Bradner and Harvard Medical School scientist Ralph Mazitschek, who is based at Massachusetts General Hospital in Boston. Bradner and Mazitschek developed the startup’s technology for identifying which HDAC enzymes are blocked by certain compounds. (Editor’s note: A previous version of this paragraph described Schreiber as a scientific founder of Acetylon, based on a press release from the company that erroneously described him as such. He is not a scientific co-founder of the startup.)
Acetylon, which is initially focused on multiple myeloma and rheumatoid arthritis, expects to zero in this year on its first compound to advance into human clinical trials, Ogier said. The firm, which now operates virtually, plans to soon move into lab and office space most likely in Cambridge, MA, and grow to about 10 employees over the next year and a half. He believes that the company’s technology could also lead to the discovery of drugs for psoriasis, Parkinson’s disease, and sickle cell anemia, to name a few.
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