Reuters logo
BlueBird's rare blood disorder drug gets breakthrough therapy status
February 2, 2015 / 1:11 PM / 3 years ago

BlueBird's rare blood disorder drug gets breakthrough therapy status

(Reuters) - The U.S. Food and Drug Administration has designated BlueBird Bio Inc’s blood disorder drug a breakthrough therapy, speeding up the treatment’s development process.

The company’s shares rose nearly 7 percent in premarket trading.

Breakthrough therapy designation is based on initial trial data and granted to drugs with the potential to treat serious diseases better than existing therapies.

BlueBird’s drug, LentiGlobin, aims to treat beta-thalassemia — a disease that results in lower levels of hemoglobin in the blood. The disease is caused by mutations in the human beta-globin gene.

Low levels of hemoglobin cause thalassemia patients to suffer from weakness, fatigue and other serious complications.

The therapy involves the insertion of a functional human beta-globin gene into the patient’s stem cells, which are then retransplanted into the patient.

The treatment is currently in early-stage trials.

Reporting by Amrutha Penumudi in Bengaluru; Editing by Simon Jennings

Our Standards:The Thomson Reuters Trust Principles.
0 : 0
  • narrow-browser-and-phone
  • medium-browser-and-portrait-tablet
  • landscape-tablet
  • medium-wide-browser
  • wide-browser-and-larger
  • medium-browser-and-landscape-tablet
  • medium-wide-browser-and-larger
  • above-phone
  • portrait-tablet-and-above
  • above-portrait-tablet
  • landscape-tablet-and-above
  • landscape-tablet-and-medium-wide-browser
  • portrait-tablet-and-below
  • landscape-tablet-and-below