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
Echosens, a high-technology company offering liver diagnostic solutions, and Novo Nordisk A/S, a leading global healthcare company, announced a partnership to advance early diagnosis of non-alcoholic steatohepatitis (NASH) and increase awareness of the disease among patients, healthcare providers and other stakeholders.
Positive opinion based on Phase 3 ADAPT trial showing efgartigimod provided clinically meaningful improvements in strength and quality of life measures. If approved, efgartigimod will be the first neonatal Fc receptor (FcRn) blocker for the treatment of adults in Europe living with rare neuromuscular disease generalized myasthenia gravis (gMG).
Galapagos CEO Paul Stoffels, M.D., has finally taken the plunge on M&A. The newly minted chief executive has signed not one but two deals in an attempt to right the ship, bringing two small biotechs aboard for a combined 239 million euros ($251.4 million).