Bluebird Bio Inc., a Cambridge, Mass., drug company whose market value has more than doubled to $5.94 billion this year, said Saturday that its experimental gene therapy helped a French teenager with sickle-cell disease go three months without a blood transfusion.<\/p>\n
Doctors said the result was an encouraging early sign that gene therapy could work in the disease, but that a one-patient study of short duration made it impossible to draw firm conclusions. Study data on the patient was presented Saturday at a meeting of the European Hematology Association in Vienna.<\/p>\n
\u201cIt\u2019s a promising start, but it\u2019s not definitive,\u201d\u009d said Michael DeBaun, a physician at Vanderbilt University School of Medicine, who wasn\u2019t involved in the study. \u201cThree months for patients with sickle cell doesn\u2019t tell us enough about the [treatment\u2019s] potential benefits and risks.\u201d\u009d<\/p>\n