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Biotech Bluebird Bio ( BLUE ) stock rose Friday after the company released positive data on its gene-therapy treatment for a rare disease. Late Thursday, a number of abstracts were released for presentations at next month’s meeting of the American Academy of Neurology (AAN). Bluebird highlighted its Starbeam study, which treated 17 boys with cerebral adrenoleukodystrophy, a rare genetic disease that leads to a breakdown of the nervous system. Bluebird used Lenti-D, which modifies the patient’s own stem cells to correct the gene mutation and re-implants them. Bluebird said that six months after treatment, subjects showed no worsening in neurological function and were expressing the protein needed to prevent the neurodegeneration. The company said that one serious adverse event possibly related to the drug had occurred, as a patient was infected with the BK virus, which is associated with transplants. However, Bluebird said the case had been resolved. Lenti-D is one treatment Bluebird is developing using its lentivirus platform for delivering gene therapy to patients. The better-known treatment LentiGlobin is being studied as a treatment for severe anemia, and it has driven the stock up and down over the last year as early trial results have trickled in. “While Lenti-D represents only a very modest commercial opportunity, we believe positive interim data at AAN should provide investors with more comfort that Bluebird’s technology can be applied effectively and consistently as a potential curative approach for severe genetic diseases,” Leerink analyst Michael Schmidt wrote in a research note. Bluebird will report full results at the AAN meeting in Vancouver on April 20. Bluebird stock was up 6.5% in midday trading on the stock market today , near 57. The stock is still more than 70% off last May’s record high above 197. Scalper1 News
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