Bluebird is an important company in gene therapy’s reemergence. The company’s progress developing treatments for rare genetic diseases early last decade helped boost confidence in gene therapy at a time when the field was still recovering from setbacks. Now, gene therapy is a fast-growing field, with many publicly traded companies, a handful of approved products and dozens of startups raising record levels of investment from venture investors.
But Bluebird has had a bumpy ride since debuting as a public company in 2013. Shares swung wildly over the years amid various clinical delays and manufacturing setbacks, while competition from newer gene editing technologies dimmed the outlook for some of its treatments. At less than $30 per share, Bluebird’s stock currently trades at levels not recorded for eight years.
The approval of Skysona reflects Bluebird’s up-and-down story. It’s a scientific achievement, making Bluebird the first company with two marketed gene replacement therapies. (The company also successfully developed an genetically engineered cell therapy called Abecma for the blood cancer multiple myeloma. Others have multiple cell therapies approved.) READ MORE
By Ben Fidler
UCB (Euronext: UCB) and Zogenix (NASDAQ: ZGNX) announced that the companies have entered into a definitive agreement under which UCB would acquire Zogenix, Inc., a global biopharmaceutical company commercializing and developing therapies for rare diseases.
argenx SE, a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved VYVGART™ (efgartigimod alfa) intravenous infusion for the treatment of adult patients with generalized myasthenia gravis (gMG) who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs).
GSK has rejected three offers from Unilever to buy GSK’s consumer health unit, the company said Saturday. The latest offer from the fellow U.K. consumer goods giant, received Dec. 20 for a total value of 50 billion pounds ($68 billion), “fundamentally undervalued” the business and its prospects, GSK said.