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Blood Disease Start-Up Lands $40.7M in Venture Funding

Blood bag (NIH)

(National Institutes of Health)

Global Blood Therapeutics Inc., a biotechnology company in San Francisco launched today with $40.7 million in funding from venture capital company Third Rock Ventures. The company plans to develop small molecule drugs to treat genetic blood disorders, such as sickle cell disease.

The company’s founders include three biomedical researchers from University of California in San Francisco — pharmaceutical chemist Matthew Jacobson, computational biologist Andrej Sali, and pharmacologist Jack Taunton — as well as biotech entrepreneurs Charles Homcy and Craig Muir, and Third Rock Ventures partner Charles Homcy. Another Third Rock Ventures partner, Mark Goldsmith, serves as the company’s CEO.

Global Blood Therapeutics aims to develop therapies to change the shape of blood proteins for treating chronic blood-based diseases and genetic disorders. The company’s technology platform combines computational biology and modeling of proteins and ligands (signaling molecules that bind to proteins) with medicinal chemistry and empiric screening.

The company plans to apply the technology to the development of oral drugs, with the lead product being a treatment that addresses the underlying causes of sickle cell disease. This disorder, also known as sickle cell anemia, is an inherited disorder in which the body produces abnormally shaped red blood cells that have a crescent or sickle shape. These cells do not last as long as normal, round, red blood cells, which leads to anemia, as well as the condition where sickle cells get stuck in blood vessels, blocking blood flow.

The Centers for Disease Control and Prevention says sickle cell disease affects 90,000 to 100,000 Americans, with the condition occuring in 1 of every 500 African-American births. CDC says sickle cell disease occurs in 1 of every 12 African-Americans.

Third Rock Ventures is a venture capital company in Boston and San Francisco that specializes in life science start-ups. In addition to providing financing, Third Rock says it also creates and manages of promising new companies.

Hat tip: Fortune/Term Sheet

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