Shares of biotechnology company bluebird bio (NASDAQ:BLUE) slumped on Monday even as the company submitted its Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) gene therapy for the treatment of sickle cell disease patients with a history of vasoocclusive events.
The BLA submission is based on efficacy results in multiple patient groups and also includes safety data from 50 patients.
The application also includes a Priority Review request. If the priority review is granted, the review process for lovo-cel could be shortened to six months. Moreover, if approved, the therapy would become BLUE’s third ex-vivo gene therapy on the market for a rare genetic disease.
Sickle cell disease impacts nearly 100,000 people in the U.S. with half the people not living past 40 years of age. Overall, the Street has a $6.67 consensus price target on BLUE, pointing to a 94.5% potential upside in the stock. That’s after a nearly 51% slide in the share price so far in 2023.
Read full Disclosure