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Delivering on the promise of gene therapy.

Decades of gene therapy leadership

uniQure innovation milestones in gene therapy

  • First approved gene therapy in the Western world
  • First commercially licensed gene therapy manufacturing facility
  • First AAV-delivered gene silencing therapy for Huntington’s Disease to enter clinic
  • First AAV vector clinically demonstrated to be effective in patients with pre-existing neutralizing antibodies

uniQure innovation timeline


uniQure founded as Amsterdam Molecular Therapeutics (AMT) Holding N.V.

Timeline amsterdam office faccade


Licensed wild-type FIX gene cassette used by St. Jude in watershed first in-human hemophilia B clinical trial

Timeline license fix gene cassette


First approved gene therapy in the western world—Glybera in the EU

Timeline glybera


Successfully completed initial public offering in the United States

Timeline first public offering


Constructed cGMP gene therapy manufacturing facility in Lexington, MA

Timeline lexington facility facade


Initiated Phase 1-2 clinical trial of AMT-060 in hemophilia B with AAV5/wtFIX transgene

IMG 3396 cropped


Announced transition to AMT-061 in hemophilia B with AAV5/FIX Padua transgene

Timeline amt061 announcement


Initiated Phase 2b dose-confirmation study and Phase 3 HOPE-B pivotal study of AMT-061

Timeline amt061 phase2b 3 studies


Initiated Phase 1-2 clinical trial of AMT-130 in Huntington’s disease

Timeline amt130 phase1 2 clinical trial


Completed dosing of 54 patients in Phase 3 HOPE-B pivotal study

Timeline phase3 hope b study dosing


Announced license agreement with CSL Behring to commercialize hemophilia B gene therapy

Timeline csl behring license agreement


Announced top-line primary endpoint data in HOPE-B pivotal trial

Timeline hope b top line data


Regulatory filings submitted in U.S. and EU for hemophilia B gene therapy

1 H7 A7811


FDA approval of the world's first gene therapy for hemophilia B

Ron timeline3


Announced interim data in Phase I/II clinical trials of AMT-130 in Huntington's disease

Chomitz2221 JCP2288prelim


FDA Clearance of Investigational New Drug Applications for AMT-260 in Temporal Lobe Epilepsy and AMT-191 in Fabry disease

Chomitz2221 JCP1193 webcrop
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