Orchard Therapeutics announced that the European Medicines Agency granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia.
Bluebird bio announced interim data from two different two-year clinical trials of LentiGlobin gene therapy for transfusion-dependent beta-thalassemia (TDT).
Investor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.
Paris-based Sanofi has acquired Waltham, Mass.-based Bioverativ for about $11.6 billion.
bluebird bio announced that it expected to file three applications for regulatory approval by the end of 2019.
Genetics company 23andMe announced the launch of a new consumer genetic test service on Wednesday that will show whether an individual carries genes associated with 36 different disorders, such as cystic fibrosis. The launch is a major step for the company, which in 2013 was ordered by the Food and Drug Administration to stop selling […]
CAMBRIDGE, Mass. — Stocks for bluebird bio (BLUE) slid 6.5 percent Monday after the company revealed a patient who underwent an early gene study treatment for beta-thalassemia seven years ago required new treatments following a relapse. Marina Cavazzana, a professor of hematology and lead investigator for bluebird’s HGB-205 clinical study, said the patient she […]