On Ver­tex and CRISPR Ther­a­peu­tics’ heels, blue­bird sub­mits sick­le cell gene ther­a­py to FDA

Blue­bird bio said Mon­day morn­ing it has sub­mit­ted an ap­pli­ca­tion for its sick­le cell ther­a­py to the FDA for po­ten­tial ap­proval. The news comes weeks af­ter Ver­tex and CRISPR Ther­a­peu­tics sub­mit­ted their own sick­le cell ther­a­py ap­pli­ca­tion to the agency.

Blue­bird is ask­ing for ap­proval in pa­tients with sick­le cell dis­ease who are 12 or old­er and have ex­pe­ri­enced se­ri­ous at­tacks as­so­ci­at­ed with the dis­ease. In clin­i­cal tri­als, 28 of 29 pa­tients who got blue­bird’s gene ther­a­py, known as lo­vo-cel, had no se­vere at­tacks in the two years of the study com­pared to an av­er­age of 3.5 events in the two years pri­or to en­rolling in the study.

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