Sickle cell disease (SCD) is a type of genetic disease that is expected to be treated with gene therapy. Bluebird bio's approach is to transfer the genes that encode beta-globin into the hematopoietic stem cell, promoting the patient to produce the mutant beta-globin, which has anti-sickle cell characteristics. In 2016, bluebird updated the process of gene therapy, increased the number of copies of the carrier, and improved the infusion process. At the annual meeting of ASH, we will see the efficacy of the updated gene therapy.
The study showed that the median value of the copy number of the old gene therapy in peripheral blood was about 0.1, the new gene therapy can be as high as 2.6. After three months of treatment, the number still reached the value of 1. Although the time that patients were assessed wascloser to treatment time, researchers thought it was still a positive result.
Bluebird will recruit more patients in future clinical trials to assess the effectiveness of the new gene therapy. This is a newr step in a positive direction, says Dr. Mohammed Asmal, bluebird's vice President for clinical development.
Wuxi PharmaTech: "quick delivery| the abstract of ASH's annual meeting is hot, what are the anti-cancer developments that deserve attention?"