bluebird bio Inc (NASDAQ:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, announced that data from the ongoing Phase 1/2 HGB-205 study of LentiGlobin BB305 Drug Product will be presented in an oral presentation on June 13, 2015 at the 20thCongress of the European Hematology Association (EHA) in Vienna, Austria.

“The early data included in our abstract provide further validation for our approach and important insights into the safety and mechanism of action of LentiGlobin in both beta-thalassemia and sickle cell disease,” said David Davidson, chief medical officer, bluebird bio. “As noted in the abstract, we are pleased to report that the two patients with beta-thalassemia major, on whom we first reported last year at EHA, remained transfusion independent at 14 and 11 months post-transplant. In addition, it is very encouraging that the patient with sickle cell disease is increasing production of HbAT87Q, which has anti-sickling properties, and has not had a post-treatment hospitalization for a sickle cell disease-related event. At EHA we will present further follow up data on all three subjects.”

Abstract Highlights (Data as of February 2015):

  • Beta-thalassemia: Beta-thalassemia major subjects (1201 and 1202) remained transfusion independent at 14 months and 11 months, respectively
  • Sickle Cell Disease: This subject (1204) entered the trial receiving chronic transfusions and began the process of being weaned from transfusions after day 37, receiving the last transfusion on day 88
    • Increasing production of HbAT87Q; the first-ever SCD patient treated with gene therapy (subject 1204) had a HbAT87Q level of 24% at 4.5 months follow up, compared to an HbAT87Q level of 9.6% at three months post-transplant
      • Note that this subject did not engraft until after month one, so their level of HbAT87Q production at months three and 4.5 are actually months two and 3.5, after engraftment
    • At 4.5 months follow up, total anti-sickling hemoglobin (HbAT87Q + HbF) was 31.6%
    • Subject 1204 has not had any hospitalizations for SCD-related complications post-transplant
  • Safety: No subject has experienced a drug product-related adverse event, and integration site analyses demonstrate highly polyclonal reconstitution without clonal dominance

Based on historical clinical observations in patients with SCD, bluebird bio believes that individuals who achieve ≥ 30 percent of anti-sickling hemoglobin (HbAT87Q + HbF) have the potential to reduce or eliminate the serious and life-threatening events associated with SCD. (Original Source)

Shares of bluebird bio closed yesterday at $165.71 . BLUE has a 1-year high of $174.85 and a 1-year low of $20.01. The stock’s 50-day moving average is $139.59 and its 200-day moving average is $99.00.

On the ratings front, bluebird bio has been the subject of a number of recent research reports. In a report issued on May 19, J.P. Morgan analyst Cory Kasimov reiterated a Buy rating on BLUE, with a price target of $117, which implies a downside of 29.4% from current levels. Separately, on the same day, Piper Jaffray’s Joshua Schimmer reiterated a Buy rating on the stock and has a price target of $186.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Cory Kasimov and Joshua Schimmer have a total average return of 12.8% and 14.8% respectively. Kasimov has a success rate of 65.0% and is ranked #269 out of 3607 analysts, while Schimmer has a success rate of 67.9% and is ranked #171.

The street is mostly Bullish on BLUE stock. Out of 5 analysts who cover the stock, 5 suggest a Buy rating . The 12-month average price target assigned to the stock is $184.40, which implies an upside of 11.3% from current levels.

bluebird bio Inc is a clinical-stage biotechnology company. It is engaged in developing potentially transformative gene therapies for severe genetic and rare diseases and in the field of T cell-based immunotherapy.