Shares of bluebird bio were down more than 24 percent after the company announced that the U.S. Food and Drug Administration placed a clinical hold on studies of the gene therapy elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).
Cambridge-based Magenta Therapeutics entered a non-exclusive research and clinical collaboration agreement with Beam Therapeutics.
Novartis AG secured Medicaid coverage for a pricey new sickle cell disease therapy in two U.S. states just weeks after winning U.S. approval, following an early campaign to convince local officials of the medicine’s value, according to a company executive and a Reuters review of public filings.
The return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.
Biopharma executives were asked to predict what the next year might reveal.
Cambridge, Mass.-based bluebird bio’s LentiGlobin gene therapy is increasing hemoglobin levels in anemia patients battling sickle cell disease and beta thalassemia, which reduces the need for blood transfusions in those patients.
Investor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.
bluebird bio announced that it expected to file three applications for regulatory approval by the end of 2019.
FDA approved Endari oral powder for patients 5 years and older with sickle cell disease to reduce severe complications associated with the blood disorder.
bluebird bio’s interim data for its Phase III gene therapy LentiGlobin trial showed promising results in reducing a patient’s need for blood transfusions after one month after treatment, but the data also raised some concerns.