Bluebird Bio Posts Updated Data From Sickle Cell Gene Therapy Program

Bluebird bio Inc BLUE announced updated results from its Phase 1/2 HGB-206 study of lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin for SCD, bb1111) gene therapy for sickle cell disease.

• The updated data included further analyses from its pivotal cohort, HGB-206 Group C. 
• Patients in Group C achieved near-normal key hemolysis markers and experienced sustained improvements in patient-reported quality of life following treatment. 
• Following engraftment, median total hemoglobin increased from 8.5 g/dL at baseline to less than 11 g/dL from six through up to 36 months post-infusion in all patients.
• Notably, in all patients, sickle hemoglobin (HbS) was less than 60% of total hemoglobin, and gene therapy-derived anti-sickling hemoglobin, HbAT87Q, contributed at least 40% of total hemoglobin.
• All evaluable patients (n=25) continued to experience complete resolution of severe vaso-occlusive events (VOEs) through up to 36 months of follow-up, compared with a median of 3.5 per year in the 24 months before enrollment.
• From six months post-infusion through the last visit, several indicators of the health of red blood cells suggest that treatment with lovo-cel improved biological markers for SCD to near-normal levels. 
• Related Link: FDA Gives Signal to Halted Bluebird Bio's Gene Therapy Trials In Sickle Cell Disease, Thalassemia.
• Price Action: BLUE shares are up 9.82% at $9.84 during the premarket session on the last check Monday.
• Check out our coverage of the American Society of Hematology (ASH) Annual Meeting 2021 here.