Gene Therapy for Sickle Cell Disease Shows Promise in Early Trial

Gene Therapy for Sickle Cell Disease Shows Promise in Early Trial

Bluebird Bio, Inc. has announced that promising data from an ongoing phase I/II study of LentiGlobin BB305 will be presented in June at the 20th Congress of the European HematologyAssociation in Vienna, Austria.

The company also offered a “sneak peek” at the presentation abstract, noting that two patients with beta-thalassemia major have remained transfusion independent at 14 and 11 months post-transplant.

LentiGlobin BB305 extracts blood stem cells and infuses them with a working version of the malfunctioning gene that had caused the SCD. People with this disorder must undergo monthly blood transfusions to survive — but if the investigational gene therapy continues to be successful, they may be freed from that burden, Bluebird Bio says. Between 1,000 and 3,000 babies are born with SCD in the U.S. each year.

More info on the press release: http://www.pharmscope.com/news/2015-05-22-000000/gene-therapy-sickle-cell-disease-shows-promise-early-trial

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