Blueprint Medicines
Based: Cambridge, MA
Founded: 2011
Website: www.blueprintmedicines.com
CEO: Chris Varma

The Scoop: Major efforts such as The Cancer Genome Atlas have greatly expanded the list of genetic drivers of tumor activity over the past several years. However, there's still a lot of work to do in sorting the reams of data from such sources as sequencing studies and gene-expression profiling to make drugs that shut down the molecular roots of disease. Blueprint Medicines has assembled a crack team of cancer veterans to utilize this genomic data in its process for advancing drugs that can precisely home in on tumor targets, specifically mutated kinase proteins. 

What Makes It Fierce: Blueprint sprinted from the gate this year with a $40 million Series A round of financing from Boston-based Third Rock Ventures, where the upstart was born. Beyond the big first round, the company is banking on some key players. Co-founders include biochemist Nicholas Lydon and oncologist Brian Druker, who worked on Novartis' ($NVS) Gleevec, a kinase inhibitor and the first FDA-approved drug to precisely block a cancer-causing protein.

"Ultimately, our vision is to transform subsets of cancer from an acute, dire condition to a manageable condition and be able to change the course of the disease like Gleevec did for [chronic myelogenous leukemia]," said Chris Varma, the start-up's co-founder and CEO. 

These days, many cancer developers are focused on molecularly targeted drugs. Companies are setting their sights on treatments that can block specific misfit proteins to kill cancer cells without packing the same punch against healthy tissues in patients. Yet Blueprint has the brains and technology to pursue rapid development of brand new inhibitors of misfit kinases. 

"We will be at IND much quicker than the typical cancer program, so we're talking about less than a few years," Varma said. "That's because we're able to know exactly what is driving a subset of cancer in terms of an aberrant mechanism, and in this case it's an aberrant kinase. And we're able to create a drug to inhibit, with high selectivity, that mutant kinase."

While the group has selected an initial drug program, the company is keeping it under wraps for the time being, Varma said. Yet he did shed light on what the company calls its "Insights-to-Validation" platform.

To advance targeted treatments quickly, the firm takes an approach that integrates informatics and biological screens. Its novel algorithms enable the firm to analyze vast numbers of genes and identify the aberrant kinases that are likely the bad characters in cancer, Varma explains. The company also has its own stock of kinase inhibitors from which to select compounds to block the mutated members of the protein group. 

Varma worked on putting together the company in his ongoing role as an entrepreneur-in-residence at Third Rock, and one of the venture firm's founding partners, Mark Levin, is the young company's chairman and co-founder. Levin has been an active proponent of personalized medicine and previously served as CEO of standout cancer drug developer Millennium Pharmaceuticals. Lydon, a company founder and director, led the protein kinase program at Ciba-Geigy--which was combined with Sandoz to form Novartis in 1996--that yielded the blockbuster Gleevec.

Blueprint has the pedigree to do great things itself, and it'll be exciting to see what its combination of top-flight talent, strong venture backing and technology produces in the years to come.

Venture Backer: Third Rock Ventures