bluebird bio(美國)

bluebird bio

美國bluebirdbiowww.bluebirdbio.com單基因突變基因療法bluebirdbio isdevelopinginnovativegenetherapiesforseveregeneticdisorders. Attheheartofbluebirdbio’sproductcreationeffortsisitsbroadlyapplicablegenetherap yplatformforthedevelopmentofnoveltreatmentsfordiseaseswithfewornoclinicaloptions. Thecompany’snovelapproachusesstemcellsharvestedfromthepatient’sbonemarrowintowhichahealthyversionofthediseasecausinggeneisinserted. Afterbeinggrowninculture,thosecellsaregivenbacktothepatient. bluebirdbio’sapproach representsatrueparadigmshiftinthetreatmentofseveregeneticdiseasesbyeliminatingthepotentialcomplicationsassociatedwithdonorcelltransplantationandpotentiallypresentingaone-timetransformativetherapy.bluebirdbiohastwolaterstageclinicalproductsindevelopmentforchildhoodcerebraladrenoleukodystrophy(CCALD)