Our Story
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
1998
uniQure founded as Amsterdam Molecular Therapeutics (AMT) Holding N.V.
2008
Licensed wild-type FIX gene cassette used by St. Jude in watershed first in-human hemophilia B clinical trial
2012
First approved gene therapy in the western world—Glybera in the EU
2014
Successfully completed initial public offering in the United States
2014
Constructed cGMP gene therapy manufacturing facility in Lexington, MA
2015
Initiated Phase 1-2 clinical trial of AMT-060 in hemophilia B with AAV5/wtFIX transgene
2017
Announced transition to AMT-061 in hemophilia B with AAV5/FIX Padua transgene
2018
Initiated Phase 2b dose-confirmation study and Phase 3 HOPE-B pivotal study of AMT-061
2019
Initiated Phase 1-2 clinical trial of AMT-130 in Huntington’s disease
2020
Completed dosing of 54 patients in Phase 3 HOPE-B pivotal study
2020
Announced license agreement with CSL Behring to commercialize hemophilia B gene therapy
2021
Announced top-line primary endpoint data in HOPE-B pivotal trial
2022
Regulatory filings submitted in U.S. and EU for hemophilia B gene therapy
2022
FDA approval of the world's first gene therapy for hemophilia B
2023
Announced interim data in Phase I/II clinical trials of AMT-130 in Huntington's disease
2023
FDA Clearance of Investigational New Drug Applications for AMT-260 in Temporal Lobe Epilepsy and AMT-191 in Fabry disease
