bluebird bio(美国)

bluebird bio

美国bluebirdbiowww.bluebirdbio.com单基因突变基因疗法bluebirdbio isdevelopinginnovativegenetherapiesforseveregeneticdisorders. Attheheartofbluebirdbio’sproductcreationeffortsisitsbroadlyapplicablegenetherap  yplatformforthedevelopmentofnoveltreatmentsfordiseaseswithfewornoclinicaloptions. Thecompany’snovelapproachusesstemcellsharvestedfromthepatient’sbonemarrowintowhichahealthyversionofthediseasecausinggeneisinserted. Afterbeinggrowninculture,thosecellsaregivenbacktothepatient. bluebirdbio’sapproach representsatrueparadigmshiftinthetreatmentofseveregeneticdiseasesbyeliminatingthepotentialcomplicationsassociatedwithdonorcelltransplantationandpotentiallypresentingaone-timetransformativetherapy.bluebirdbiohastwolaterstageclinicalproductsindevelopmentforchildhoodcerebraladrenoleukodystrophy(CCALD)