Third
Rock Ventures, LLC today announced the formation of Global
Blood Therapeutics, Inc. and its $40.7 million Series A financing of
the company. The company's unique SHAPE Platform is enabling the
discovery and development of oral, small molecule drugs that change the
shapes of key blood proteins to modify their functions, enhance vital
biological pathways and drive therapeutic benefits. Global Blood
Therapeutics is building a pipeline of innovative medicines for severe,
genetic blood diseases for which there are currently no effective cures
and only extremely limited therapeutic options. Founders of the company
include Charles Homcy, M.D., and Craig Muir of Third Rock Ventures,
David Phillips, Ph.D., co-founder of COR Therapeutics and co-founder
with Dr. Homcy of Portola Pharmaceuticals, and three leading scientific
researchers at the University of California, San Francisco: Matthew
Jacobson, Ph.D., professor, pharmaceutical chemistry, Andrej Sali,
Ph.D., professor, bioengineering and therapeutic sciences, and Jack
Taunton, Ph.D., associate professor, cellular and molecular pharmacology.
"Blood is one of the largest and most accessible organs and is a rich
source of biologically and clinically validated targets," said Mark
A. Goldsmith, M.D., Ph.D., chief executive officer of Global Blood
Therapeutics and venture partner at Third Rock Ventures, LLC. "Global
Blood Therapeutics aims to provide game-changing treatments for serious,
genetic blood diseases that are well-understood but poorly served today,
by developing innovative medicines directed against clinically validated
targets. Through our SHAPE Platform and our world-class team with a
strong track record in science and drug discovery and development, we
are positioned to develop rapidly a new generation of safe and effective
oral drugs that could truly change the treatment paradigm on behalf of
patients."
Global Blood Therapeutics' lead program focuses on Sickle Cell Disease
(SCD), one of the earliest rare genetic diseases to be defined on a
molecular basis. It is caused by a single mutation that alters the
oxygen-transport protein hemoglobin and results in the "sickling" - or
change to a crescent shape - of red blood cells. With 100,000 patients
in the United States and more than 15 million worldwide, SCD is a global
health challenge with very limited treatment options and no therapies
that address the underlying cause of the disease.
To address such worldwide need, Global Blood Therapeutics is focused on
the discovery and development of a pipeline of new therapies that change
the shapes of key blood proteins to treat chronic blood-based diseases
and genetic disorders. Specifically, the SHAPE
Platform brings together the most advanced computational biology and
protein-ligand modeling with medicinal chemistry and empiric screening
capabilities focused on allosteric modulation, while leveraging the
Global Blood Therapeutics team's leading expertise in blood biology and
track record of success in drug discovery and development. This approach
will enable Global Blood Therapeutics in the development of orally
available compounds that are potent, have a rapid onset of action and
ultimately should translate into high therapeutic efficacy and decreased
toxicity.
"Our vision at Third Rock Ventures is to improve patients' lives
decisively through innovation, and the unique approach and stellar team
at Global Blood Therapeutics are positioned to revolutionize how some of
the most severe, blood-based genetic diseases are treated," said Charles
Homcy, M.D., venture partner at Third Rock Ventures. "The launch of
Global Blood Therapeutics, the first company fully conceived of and
formed out of our West Coast office, underscores Third Rock's commitment
to our goal and marks the ongoing success of our bi-coastal model and
innovative, hands-on company ideation process. Global Blood expands our
strong and growing portfolio of exciting companies on both coasts."
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