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Programs & Pipeline | uniQure
<!doctype html> <html lang="en"> <head> <title>Programs & Pipeline | uniQure</title> <meta charset="utf-8"> <meta http-equiv="x-ua-compatible" content="ie=edge"> <meta name="viewport" content="width=device-width, initial-scale=1"> <meta name="author" content="uniQure"> <meta name="description" content="Reimagining the future of healthcare through the power of gene therapy."> <meta property="og:site_name" content="uniQure"/> <meta property="og:type" content="website"/> <meta property="og:title" content="Programs & Pipeline"/> <meta property="og:description" content="Reimagining the future of healthcare through the power of gene therapy."/> <meta property="og:url" content="https://www.uniqure.com/programs-pipeline"/> <meta property="og:image" content="https://www.uniqure.com/assets/uploads/uniqure-sharing-logo.png" /> <meta property="og:image:width" content="1200" /> <meta property="og:image:height" content="630" /> <link rel="apple-touch-icon" sizes="120x120" 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})(window,document,'script','https://www.google-analytics.com/analytics.js','ga'); ga('create', 'UA-87370286-1', 'auto'); ga('send', 'pageview'); </script> </head> <body id="programs" class="loading landing"> <div class="site-wrapper"> <header> <a class="logo" href="https://www.uniqure.com/"> <svg viewBox="0 0 160 35"><use href="https://www.uniqure.com/assets/img/icons.svg#logo" /></svg> <span>uniQure</span> </a> <div class="menu-panel"> <nav> <ul class="main-nav"> <li class="nav-section"> <a href="https://www.uniqure.com/pioneers-leaders" >Pioneers & Leaders</a> <ul class="nav-sublinks"> <li><a href="https://www.uniqure.com/pioneers-leaders/our-story" >Our Story</a></li> <li><a href="https://www.uniqure.com/pioneers-leaders/leadership" >Our Leadership</a></li> <li><a href="https://www.uniqure.com/pioneers-leaders/partnerships" >Our Partners</a></li> </ul> <div class="section-toggle"></div> </li> <li class="nav-section"> <a href="https://www.uniqure.com/patients-families" >Patients & Families</a> <ul class="nav-sublinks"> <li><a href="https://www.uniqure.com/patients-families/huntingtons-disease" >Huntington's Disease</a></li> <li><a href="https://www.uniqure.com/patients-families/als" >ALS (SOD1)</a></li> <li><a href="https://www.uniqure.com/patients-families/patient-community-stories" >Patient Community Stories</a></li> <li><a href="https://www.uniqure.com/patients-families/community-resources" >Community Resources</a></li> <li><a href="https://www.uniqure.com/expanded-access-policy" >Expanded Access Policy</a></li> </ul> <div class="section-toggle"></div> </li> <li class="nav-section"> <a href="https://www.uniqure.com/science-innovation" >Science & Innovation</a> <ul class="nav-sublinks"> <li><a href="https://www.uniqure.com/science-innovation/aav-platform" >Our Platform</a></li> <li><a href="https://www.uniqure.com/science-innovation/enabling-technologies" >Enabling Technologies</a></li> <li><a href="https://www.uniqure.com/science-innovation/publications" 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Careers</a> <ul class="nav-sublinks"> <li><a href="https://www.uniqure.com/culture-careers/working-at-uniqure" >Working at uniQure</a></li> <li><a href="https://www.uniqure.com/culture-careers/social-responsibility" >Social Responsibility</a></li> <li><a href="https://www.uniqure.com/culture-careers/employee-stories" >Our People</a></li> <li><a href="https://www.uniqure.com/culture-careers/job-opportunities" >Job Opportunities</a></li> </ul> <div class="section-toggle"></div> </li> <li class="nav-section nav-single contact"> <a href="https://www.uniqure.com/contact" >Contact Us</a> </li> </ul> </nav> </div> <button class="mobile-menu-trigger">Menu<span></span></button> </header> <main> <section id="top" class="hero"> <div class="container"> <h1>Programs <span class="amp">&</span> Pipeline</h1> <p>Translating leading science into life-changing solutions.</p> </div> <div class="img-wrapper img-fit"> <img srcset="https://www.uniqure.com/assets/uploads/hero/_heroImgMbl/hero-programs-pipeline.jpg 800w, https://www.uniqure.com/assets/uploads/hero/_heroImg/hero-programs-pipeline.jpg 1800w" alt="Hero programs pipeline" data-object-fit> </div> </section> <section class="intro"> <div class="container"> <p>We’re building a promising pipeline of potentially transformative programs focused on leveraging our platforms for maximum impact.<br></p> </div> </section> <section class="banners"> <div class="container"> <div class="banner img-text img-left"> <div class="content-wrapper animate-on-scroll animate-slide-left"> <h2>Huntington’s disease</h2> <p>Our AAV5 gene therapy platform shows promise for Huntington’s disease, a devastating and fatal neurodegenerative disorder, through uniQure’s proprietary miQURE™ gene-silencing technology.</p> <a href="https://www.uniqure.com/programs-pipeline/huntingtons-disease" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-right"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/programs-pipeline-huntingtons-disease-thumb.jpg" alt="Programs pipeline huntingtons disease thumb" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-right"> <div class="content-wrapper animate-on-scroll animate-slide-right"> <h2>Temporal Lobe Epilepsy (TLE)</h2> <p>We are excited to begin the clinical investigation of AMT-260, our one-time administered gene therapy for refractory mesial temporal lobe epilepsy.</p> <a href="https://www.uniqure.com/programs-pipeline/temporal-lobe-epilepsy-tle" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-left"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/IMG_3740_web.jpg" alt="IMG 3740 web" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-left"> <div class="content-wrapper animate-on-scroll animate-slide-left"> <h2>Fabry disease</h2> <p>AMT-191 represents a novel potential one-time administered approach to treating Fabry disease.</p> <a href="https://www.uniqure.com/programs-pipeline/fabry-disease" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-right"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/webcrop-fabry.jpg" alt="Webcrop fabry" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-right"> <div class="content-wrapper animate-on-scroll animate-slide-right"> <h2>ALS (SOD1)</h2> <p>The clinical development of AMT-162 is based on nearly 30 years of research demonstrating the link between the SOD1 gene mutation and ALS.</p> <a href="https://www.uniqure.com/programs-pipeline/als-sod1" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-left"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/webcrop-ALS.jpg" alt="Webcrop ALS" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-left"> <div class="content-wrapper animate-on-scroll animate-slide-left"> <h2>Hemophilia Type B</h2> <p>In 2022 our gene therapy for hemophilia B became the world’s first approved gene therapy for hemophilia.</p> <a href="https://www.uniqure.com/programs-pipeline/hemophilia" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-right"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/programs-pipeline-hemophilia-thumb.jpg" alt="Programs pipeline hemophilia thumb" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-right"> <div class="content-wrapper animate-on-scroll animate-slide-right"> <h2>Clinical trials</h2> <p>We continue to make progress in advancing AMT-130 for Huntington’s disease, currently in multiple-cohort Phase 1/2 clinical studies, and AMT-260 for refractory mesial temporal lobe epilepsy.</p> <a href="https://www.uniqure.com/programs-pipeline/clinical-trials" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-left"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/programs-pipeline-clinical-trials-thumb.jpg" alt="Programs pipeline clinical trials thumb" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-left"> <div class="content-wrapper animate-on-scroll animate-slide-left"> <h2>Research programs</h2> <p>We are looking farther out on the horizon for new, life-changing applications of our expertise and technologies as we focus on advancing our first two potentially transformative gene therapies.</p> <a href="https://www.uniqure.com/programs-pipeline/research-programs" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-right"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/programs-pipeline-research-programs-thumb.jpg" alt="Programs pipeline research programs thumb" loading="lazy" data-object-fit> </div> </div> <div class="banner img-text img-right"> <div class="content-wrapper animate-on-scroll animate-slide-right"> <h2>Pipeline</h2> <p>We are executing an ambitious strategy to expand our pipeline and advance a new portfolio of next-generation gene therapies into clinical-stage development.</p> <a href="https://www.uniqure.com/programs-pipeline/pipeline" class="btn">Learn More</a> </div> <div class="img-wrapper img-fit animate-on-scroll animate-slide-left"> <img src="https://www.uniqure.com/assets/uploads/thumbnail/_thumbImg/programs-pipeline-thumb.jpg" alt="Programs pipeline thumb" loading="lazy" data-object-fit> </div> </div> </div> </section> <section class="banner cta-banner"> <div class="container animate-on-scroll animate-slide-up"> <h3>We want to hear from you.</h3> <p>Contact us for more information.</p> <div class="btn-grp"> <div class="btn-wrapper"> <a href="https://www.uniqure.com/contact" class="btn">Contact</a> </div> </div> </div> </section> <section class="related-links"> <div class="container"> <h4>Explore uniQure</h4> <ul class="animate-on-scroll animate-children"> <li> <a href="https://www.uniqure.com/pioneers-leaders" class="btn btn-rounded"> About uniQure <svg viewBox="0 0 76 18"> <use href="#arrow" /> </svg> </a> </li> <li> <a href="https://www.uniqure.com/science-innovation" class="btn btn-rounded"> Science & Innovation <svg viewBox="0 0 76 18"> <use href="#arrow" /> </svg> </a> </li> <li> <a href="https://www.uniqure.com/patients-families" class="btn btn-rounded"> Patients & Families <svg viewBox="0 0 76 18"> <use href="#arrow" /> </svg> </a> </li> </ul> </div> </section> </main> <footer> <div class="footer-primary"> <div class="container"> <div class="branding"> <a class="logo" href="/"> <svg viewBox="0 0 160 35"><use href="#logo" /></svg> 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