Third Rock funds $40M launch of San Francisco blood disease biotech

Third Rock Ventures is committing $40.7 million to launch a new biotech company on the West Coast which is devoted to finding new oral therapies for severe, genetic blood diseases. 

San Francisco-based Global Blood Therapeutics will focus on the development of a pipeline of oral drugs, with the lead program targeting sickle cell disease. Its investigators will use a tech platform dubbed SHAPE to tweak the shapes of key blood proteins in order to treat chronic blood-based diseases and genetic disorders. And they plan to bring together expertise in computational biology, protein-ligand modeling with medicinal chemistry and "empiric screening capabilities focused on allosteric modulation."

Unlike most other VCs, Third Rock doesn't just invest in early-stage companies through syndicates. Sometimes it creates them from the ground up, tapping an experienced pool of biotech players to take key positions. Third Rock partner Mark Goldsmith, who headed the startup team at Constellation, will now helm the new company. And he'll be joined by an experienced cast of drug developers, including Charles Homcy, who helped found Portola.

Homcy and Craig Muir of Third Rock Ventures are founding the company along with David Phillips, who co-founded Portola--a 2009 Fierce 15 company--with Homcy. And there are three top scientists at UC San Francisco adding to the brain trust: pharma chemistry professor Matthew Jacobson, Andrej Sali, an expert in bioengineering and therapeutic sciences, and Jack Taunton, Ph.D., associate professor, cellular and molecular pharmacology.

This is the first such company launch Third Rock has done on the West Coast, says Goldsmith, which follows two years of building the company in stealth mode. Now they have assembled the technology platform that can develop pills that can mend, or at least partially fix mutated proteins that are triggering some terrible diseases like sickle cell anemia. 

"In each of these cases," Goldsmith tells FierceBiotech, "a mutation in the protein changes the function of the protein. It's associated with an abnormal protein shape. Our approach is to use small molecules that bind to the target, contributing to a more favorable protein shape that can restore the activity of the protein."

In sickle cell disease "the hemoglobin assumes a shape that is abnormal." Then it aggregates in cells, warping them into the sickle shape that creates a rigid structure which has trouble passing through small blood vessels and triggering a litany of crises that lead to organ failure and death. By returning the cell shape back to a healthier shape, a regularly taken pill can ameliorate if not eliminate symptoms of the disease.

For now, Global Blood Therapeutics is an 8-person group which will grow and move into new digs this fall. In a few weeks Goldsmith will move out to the San Francisco hub, with plans to take the red eye back to Boston to keep in touch with his colleagues on the East Coast. And, eventually, he expects to see a business development strategy set up which will focus on deals for the company. For now, though, the focus is on preclinical work with an eye to eventually making their way into the clinic.

- here's the press release