Hacking the genetic operating system
CEO: Dr. Nancy Simonian
Based: Watertown, MA
Clinical focus: Oncology and immunology
The scoop: Scientists successfully sequenced the human genome back in 2001, illuminating countless avenues of study and effectively inventing the multibillion-dollar genomics industry. But that was hardly the end of history with respect to human disease, as DNA can only explain so much about cellular activity. Startup Syros Pharmaceuticals is among the many groups of researchers looking to push the field further, delving into the upstream processes of protein interactions in hopes of understanding "the operating system by which genes are regulated," CEO Nancy Simonian said, developing new therapies along the way.
What makes Syros Pharmaceuticals Fierce: The bedrock of the company's platform is an advanced understanding of superenhancers, regions of DNA that dictate a cell's identity by controlling the expression of various genes.
Building on the work of scientific founder and MIT professor Richard Young, Syros has amassed what it says is the world's largest collection of gene regulators, creating an annotated map of superenhancers and the processes they govern. That allows its investigators to rapidly spotlight new gene control targets and craft small-molecule therapies to treat them, Syros said, setting the stage for a pipeline of therapies.
And the potential of such an approach has resonated with some high-profile investors. Syros got off the ground in 2013 with a $30 million A round drawing from Flagship Ventures, Arch Venture Partners and the investment arm of Chinese CRO WuXi PharmaTech ($WX). Last year, Syros added Polaris Partners to that syndicate for a $53 million Series B fundraise, building its war chest to get a pair of candidates into the clinic.
Syros' most advanced work is in cancer, where the company is moving forward with a treatment that targets a kinase called CDK7, which plays a role in DNA repair. If all goes according to plan, the drug will be ready for human trials next year, Simonian said. Behind that, Syros has a second preclinical cancer treatment directed at an undisclosed target, and the company expects to file an IND application for it in 2016, as well.
And now, with its latest fundraise extending the R&D runway, Syros is expanding its focus to include immunology, delving into the role superenhancers play in inflammatory cells. The company has also done some early-stage work in genetic disorders of the central nervous system and kidney, Simonian said, kicking the tires on how its platform might come into play.
Underlying all of Syros' work is an end-around take on drug discovery, Simonian said. The traditional model of development often sees scientists take a sort of compound-normative approach to R&D, picking a molecule and then searching for patients who might benefit from it, she said. That often leads to dead ends in development when researchers can't figure why some volunteers respond and others don't.
But Syros, thanks to its understanding of "the core operating system of a cell," can start with disease and essentially reverse-engineer a molecule to modulate it, Simonian said.
"Very importantly, rather than say 'We're going to work on pathway X,' we flipped it on its head and said we're going to figure out what's wrong in the disease and let the informatics drive the biology," she said. "And then from there we'll determine the optimal way to target it."
Simonian took the reins at Syros after roughly 20 years in Big Biotech, including stints as chief medical officer at Millennium Pharmaceuticals and vice president of clinical research at Biogen ($BIIB). The overarching lesson from each of those companies--which were once as small as Syros--is that assembling the right team and maintaining a strong foundation in science can protect the entrepreneurial spirit so often endangered by growth and its attendant bureaucracy, she said.
"A big part of what I spend my time on is really on the aspect of building the culture, the people piece," she said. "At the end of the day, it's all about the environment, the culture that will set your success."
Investors: Arch Venture Partners, Flagship Ventures, Aisling Capital, Polaris Partners, Redmile Group and WuXi PharmaTech
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-- Damian Garde (email | Twitter)