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.
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.
“It’s a promising start, but it’s not definitive,” said Michael DeBaun, a physician at Vanderbilt University School of Medicine, who wasn’t involved in the study. “Three months for patients with sickle cell doesn’t tell us enough about the [treatment’s] potential benefits and risks.”
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(WSJ) Bluebird Bio Early Study of Sickle-Cell Gene Therapy Appears Encouraging
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.
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.
“It’s a promising start, but it’s not definitive,” said Michael DeBaun, a physician at Vanderbilt University School of Medicine, who wasn’t involved in the study. “Three months for patients with sickle cell doesn’t tell us enough about the [treatment’s] potential benefits and risks.”
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