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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

1998

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

Timeline amsterdam office faccade

2008

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

Timeline license fix gene cassette

2012

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

Timeline glybera

2014

Successfully completed initial public offering in the United States

Timeline first public offering

2014

Constructed cGMP gene-therapy manufacturing facility in Lexington, MA

Timeline lexington facility facade

2015

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

Timeline amt060 clinical trial

2017

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

Timeline amt061 announcement

2018

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

Timeline amt061 phase2b 3 studies

2019

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

Timeline amt130 phase1 2 clinical trial

2020

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

Timeline phase3 hope b study dosing

2020

Announced License Agreement with CSL Behring to Commercialize Hemophilia B Gene Therapy

Timeline csl behring license agreement

2021

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

Timeline hope b top line data

2022

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

1 H7 A7811

2022

Announced 12-month data on low-dose patient cohort in Phase 1-2 trial of AMT-130 in Huntington's disease

Chomitz2221 JCP2288prelim
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