uniQure(荷蘭)

uniQure

荷蘭uniQure,www.uniqure.comuniQure是在人類基因療法的研究和早期開發(fā)的地位。uniQureisaworldleaderinthedevelopmentofhumangenebasedtherapies.uniQurehasaproductpipelineofgenetherapyproductsindevelopmentforhemophiliaB,acuteinter mittentporphyria,Parkinson’sdiseaseandSanfilippoB.Usingadeno-associatedviral(AAV)derivedvectorsasthedeliveryvehicleofchoicefortherapeuticgenes,thecompanyhasbeenabletodesignandvalidateprobablytheworld’sfirststableandscalableAAVmanufacturingplatform.Thisproprietaryplatformcanbeappliedtoalargenumberofrare(orphan)diseasescausedbyonefaultygeneandallowsuniQuretopursueitsstrategyoffocusingonthissectoroftheindustry.uniQure'slargestshareholdersareForbionCapitalPartnersandGildeHealthcare,twooftheleadinglifesciencesventurecapitalfirmsintheNetherlands.