uniQure(荷兰)

uniQure

荷兰uniQure,www.uniqure.comuniQure是在人类基因疗法的研究和早期开发的地位。uniQureisaworldleaderinthedevelopmentofhumangenebasedtherapies.uniQurehasaproductpipelineofgenetherapyproductsindevelopmentforhemophiliaB,acuteinter mittentporphyria,Parkinson’sdiseaseandSanfilippoB.Usingadeno-associatedviral(AAV)derivedvectorsasthedeliveryvehicleofchoicefortherapeuticgenes,thecompanyhasbeenabletodesignandvalidateprobablytheworld’sfirststableandscalableAAVmanufacturingplatform.Thisproprietaryplatformcanbeappliedtoalargenumberofrare(orphan)diseasescausedbyonefaultygeneandallowsuniQuretopursueitsstrategyoffocusingonthissectoroftheindustry.uniQure'slargestshareholdersareForbionCapitalPartnersandGildeHealthcare,twooftheleadinglifesciencesventurecapitalfirmsintheNetherlands.