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.
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.
U.S. regulators approved expanded use of Roche’s drug Hemlibra to include almost all patients with hemophilia A.
Clinical data suggests that uniQure’s AAV5 gene therapy may be viable treatments for 97 percent of hemophilia B patients.
Fast Company recently published The World’s 50 Most Innovative Companies 2018, topping off its edition of the top 350 most innovative companies. Here is a look at the life science companies that made the list.
Paris-based Sanofi has acquired Waltham, Mass.-based Bioverativ for about $11.6 billion.
Philadelphia-based Spark Therapeutics, with its partner Pfizer, published interim data from its Phase I/II clinical trial of a gene therapy for hemophilia B in The New England Journal of Medicine.
Barely a week after announcing it was moving into a new corporate headquarters, Sangamo Therapeutics announced that the company has dosed the first patient in its Phase I/II gene therapy trial.
Alnylam Pharmaceuticals indicated that the company suspended its Phase II trial of fitusiran for hemophilia A after a patient died from a thrombotic event, pending further investigation.
Shares of beleaguered Dimension Therapeutics Inc. soared more than 160 percent in premarket trading after gene therapy company Regenxbio Inc. announced its acquisition via an all-stock deal.