Syros Pharmaceuticals raised $30 million in start-up funding for drug development targeting master regulators in the genome, with the aim of developing therapies for cancer and other diseases.

It's not unusual for venture capitalists to yawn at biomedical breakthroughs that garner headlines. Not so in the case of Syros Pharmaceuticals, a Watertown, MA-based company that recently snapped up $30 million in start-up funding for drug development targeting master regulators in the genome.

“It was all about the timing,” explains Nancy Simonian, MD, the company's chief executive officer. “Investors need to see a path from the breakthrough to potential therapeutics.”

Pioneered by researchers in the lab of Richard Young, PhD, at Whitehead Institute for Biomedical Research and Massachusetts Institute of Technology in Cambridge, MA, and published in Cell, the science behind Syros shows that gene regulators cluster into “super-enhancers” that control the fate of both normal and diseased cells (Cell 2013;153:307–19).

That just a few hundred super-enhancers may control the genes that dictate a cell's identity and its disease status is important scientifically, Simonian says. What set the stage for business investment, she adds, was evidence that small-molecule probes can manipulate super-enhancers in cancer cells while leaving healthy cells alone.

A second paper (Cell 2013;153:320–34) demonstrated that targeting a central super-enhancer component, the transcriptional coactivator BRD4, could selectively reduce the expression of the MYC oncogene in multiple myeloma, notes Christian Fritz, PhD, Syros head of biology. For this work, the White-head scientists teamed up with James Bradner, MD, of Dana-Farber Cancer Institute and Harvard Medical School in Boston, MA, and Christopher Vakoc, MD, PhD, of Cold Spring Harbor Laboratory in Cold Spring Harbor, NY.

“The combination of these discoveries is what made the start-up timing right,” Simonian says.

Syros's initial focus is on cancer, but the company hopes eventually to expand its clinical base to other diseases—another major plus for investors.

“It's too early to talk about clinical development partners,” Simonian says. “But we're investigating opportunities for further investments in the platform, which we think has potential applications for all human diseases.”

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