Gene therapy company bluebird bio (NASDAQ: BLUE) gave an update on the clinical trial of its treatment for sickle cell disease (SCD) showing that patients in the study have remained free of the most serious symptoms of the disease. The results suggest that Bluebird is on track to submit the treatment to the Food and Drug Administration for accelerated approval by next year.
The report provided updated results from the 25 patients in group C of the study designated HGB-206 given LentiGlobin, a which treats the genetic disease by adding functional copies of a gene into the patient's stem cells, thus enabling them to generate red blood cells capable of producing normal anti-sickling hemoglobin, resulting in a lower proportion of sickled cells.
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