Small molecules to control gene expression are an idea whose time has come--at least Third Rock Ventures thinks so. It’s been working for two years on incubating a startup to do just that in genetic disorders. Now, the firm is ready to launch the fruit of that labor, Fulcrum Therapeutics, with a rich Series A round of $55 million.
Up first, the Cambridge, MA-based startup will focus on a pair of severe monogenic diseases: fragile X syndrome (FXS) and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD). Eventually, it expects to address other indications including neurologic disorders, skeletal muscular disorders and cardiac muscular disease.
“Fulcrum is really based on the increased understanding and critical role that genetics plays in diseases, particularly in monogenic disorders that respond to an increase or decrease in proteins,” President and CEO of Fulcrum Robert Gould told FierceBiotech. “We are utilizing pluripotent stem cells derived from skin to mimic the cellular processes that have gone wrong in these diseases with altered gene expression. We use small molecules to modulate it, particularly in these two diseases.”
Gould was previously the president and CEO of Epizyme; he joined Fulcrum in January. Prior to that, he was the director of novel therapeutics at The Broad Institute of MIT and Harvard and also held the posts of VP of licensing and external research and VP of basic research at Merck Research Laboratories ($MRK).
The startup models gene regulation in disease using patient cells. These are either donated via tissue biopsy or induced pluripotent stem cells derived from skin cells. It uses CRISPR/CAS9 and chemical probe libraries to better understand gene regulatory mechanisms in these cellular models. Then, Fulcrum creates genome-wide maps of gene regulation to better identify drug targets for activation or repression of disease genes.
As with many Third Rock startups, Fulcrum started with a thesis that the firm then gathered talented scientists around. The exploration this time was to understand if rare diseases could be treated by altering disease expression.
Gould said Fulcrum needs such a large Series A to simultaneously advance its platform and get into the clinic.
“The $55 million will really enable us to build out our product engine and ongoing approaches to treating monogenic disorders. We’ll build out in Fragile X and muscular dystrophy, get those programs through to compounds that may be ready to go into human trials,” he summed up as the goal for this financing.
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