bluebird outlines beta thalassemia regulatory plans

On Sunday, bluebird bio Inc. (NASDAQ:BLUE) said its initial U.S. regulatory strategy for LentiGlobin BB305 in beta-thalassemia will focus on patients with non-beta-0/beta-0 genotypes. CEO Nick Leschly has previously said non-beta-0/beta-0 patients represent about two-thirds of the beta-thalassemia major population in the U.S. and Europe.

In an abstract released last month ahead of the American Society of Hematology (ASH) meeting in Orlando, LentiGlobin BB305 did not lead to sustained transfusion independence in three beta-thalassemia patients with the beta-0/beta-0 genotype. The patients were enrolled in the Phase I/II Northstar (HGB-204) study (see BioCentury Extra, Nov. 5). ...