After leaving the helm of Epizyme last fall, Robert Gould took some time off, then took some more time to look over some biotech startups in the Third Rock pipeline. Taking them out for a spin, so to speak.
Then he got a closeup of Fulcrum Therapeutics, a new model platform company taking shape on Third Rock’s backyard in Cambridge, MA, looking to use small molecules to regulate genes, turning them on and off in pursuit of controlling a disease.
It had all the right elements:
— Great science board driving a cutting-edge approach? Check.
— Product engine tuned for a variety of therapeutic tracks? Check.
— Enough fuel to really get someplace? Check and double check.
Third Rock has now seen to that last part with a $55 million startup round.
“I just like building stuff,” Gould tells me, with the kind of enthusiasm you’d expect to hear in the voice of an entrepreneur drawn to biotech.
There’s nothing new about exploring the genetic drivers of disease. That work has been piling up new targets for years. The big idea at Fulcrum is that you can use new technologies, like CRISPR Cas9 gene editing, stem cell tech and more, to dissect the problem and find the best treatments that can most effectively regulate targets of gene expression.
Potentially, there are hundreds of targets to go after, but Gould’s new crew is going to start with two: Fragile X syndrome and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy. Both of these diseases are triggered by a single issue in gene regulation; the silencing of the FMR1 gene for Fragile X and the activation of a gene called DUX4 for FSHD.
“We’re about 10 people currently,” says Gould, who plans to gradually double that in the not distant future. “We expect that we’ll be in the clinic a few years from now,” he adds, “but we’re still at the early stages of drug discovery.”
Each of the two initial diseases arise from a single gene mutation that creates an error in gene regulation. Gould wants to use them as test cases to demonstrate how they can turn genes on and off. In the case of Fragile X, the silencing of the FMR1 gene eliminates the cell’s ability to make a protein needed for brain function. In FSHD, the precipitating cause is the activation of a gene called DUX4 that should be silent in adulthood. This activation leads to muscle wasting as cells die.
In classic Third Rock stye, Gould will be working with a scientific cabinet which includes:
- Michael Green, University of Massachusetts Medical School; The Howard Hughes Medical Institute; National Academy of Sciences; providing expertise in gene regulation
- Danny Reinberg, New York University School of Medicine; The Howard Hughes Medical Institute; providing expertise in chromatin structure and function
- Rudolf Jaenisch, Whitehead Institute for Biomedical Research; Massachusetts Institute of Technology; Institute of Medicine; National Academy of Sciences; providing expertise in stem cell biology
- Jeannie Lee, Harvard Medical School; The Howard Hughes Medical Institute; providing expertise in gene regulation and X chromosome genetics
- And Brad Bernstein, Harvard Medical School; Broad Institute of MIT and Harvard; providing expertise in genome-wide characterization of gene regulation
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