Being a highly innovative company isn't a sufficient condition for success in the biotech industry. Bluebird Bio (NASDAQ: BLUE), a small-cap gene-editing specialist, is a good example of this. Though the company has developed groundbreaking therapies, the stock has significantly lagged the market in recent years. Still, if Bluebird Bio can turn things around, the company could deliver explosive returns to investors who initiate a position today. Is Bluebird Bio worth the risk?
Bluebird Bio has three approved gene-editing therapies: Zynteglo, Skysona, and Lyfgenia. The most important of this trio, and the latest to earn approval (it did so in December), is Lyfgenia, which treats a rare blood disorder called sickle-cell disease (SCD).
What makes it the most important? The number of potential patients. Zynteglo, which targets another rare blood disease called transfusion-dependent beta-thalassemia (TDT), can target a maximum of 1,500 patients in the U.S. Skysona -- a therapy for a rare, progressive neurogenerative disease called cerebral adrenoleukodystrophy -- has a minuscule target market of just 40 patients. Meanwhile, Lyfgenia is looking at 20,000 potential takers.
Source Fool.com