Venture capital firm Third Rock Ventures has launched a drug development startup to treat genetic blood disorders, with sickle-cell anemia as its lead program target, providing $40.7 million in series A financing.
Global Blood Therapeutics‘ drug development pipeline will target severe, genetic blood diseases that currently have no effective cures and limited therapeutic options. It will develop orally available compounds that are potent, have a rapid onset and have a high therapeutic efficacy and decreased toxicity, according to a company statement.
About 100,000 people suffer from sickle-cell anemia in the United States, and more than 15 million outside the country have the disease. The company’s treatment will alter the crescent-shaped red blood cells, which is caused by a mutation in the protein controlling oxygen transport.
Among the company’s founders are three scientific researchers from University of California, San Francisco: Dr. Matthew Jacobson, Dr. Andrej Sali and Dr. Jack Taunton, and two Third Rock directors: Dr. Charles Homcy and Craig Muir. Dr. Mark Goldsmith is the CEO. Goldsmith previously led Constellation Pharmaceuticals, an epigenetics biopharmaceutical company and Third Rock portfolio company. Epigenetics focuses on changes in gene expression other than those caused by underlying DNA.
Global Blood Therapeutics is the fourth biotechnology company the Boston-based venture capital firm has served as the solo series A investor in just over one year and the first to be located at its San Francisco office, which opened two years ago, a spokeswoman said. In April, it launched Blueprint Medicines, a personalized cancer therapy firm, followed by Sage Therapeutics, developing treatments for central nervous system disorders including schizophrenia, depression, pain and traumatic brain injury, and Ember Therapeutics addressing metabolic diseases.