Cambridge, Mass.-based bluebird bio and Bagsvaerd, Denmark-based Novo Nordisk agreed to develop next-generation genome editing therapies for genetic diseases, including hemophilia.
The U.S. Food and Drug Administration approved Octapharma USA’s Wilate for the treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control of bleeding episodes.
Pfizer Inc. is investing $500 million to expand a manufacturing facility in Sanford, North Carolina, that plays a central role in the company’s efforts to become a major player in gene therapy.
San Rafael, Calif.-based BioMarin Pharmaceutical announced positive Phase III clinical trial results for the company’s gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
Bayer plans to build a Cell Culture Technology Center in Berkeley, Calif., investing $150 million in the project. The center will focus on developing biologics, an area where Bayer lags behind the company’s competitors.
Bayer is looking into future options for the established drugs business, partly because of price pressure in China, the German company’s head of pharmaceuticals said.
Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
Denmark-based Novo Nordisk snagged approval from the U.S. FDA for a new treatment for hemophilia A, though Esperoct will not be available in the United States until 2020.
Switzerland’s Roche will donate the company’s new hemophilia A drug Hemlibra to a World Hemophilia Federation program, joining rivals who also back the effort to help patients in developing countries who face treatment hurdles.