Our Platform | Science & Innovation

<!doctype html> <html lang="en"> <head> <title>Our Platform | Science & Innovation | 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="Our Platform | Science & Innovation"/> <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/science-innovation/aav-platform"/> <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 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https://www.uniqure.com/assets/uploads/hero/_heroImg/hero-aav-gene-therapy-platform.jpg 1800w" alt="Hero aav gene therapy platform" data-object-fit> </div> </section> <section class="intro"> <div class="container"> <p>An industry-leading platform focused on the liver and CNS<br></p> </div> </section> <section class="body-blocks"> <div class="container"> <div class="body-block body-text wysiwyg standard-spacing"> <p>uniQure has built an industry-leading gene therapy platform focused on liver-directed and central nervous system (CNS) disorders.</p> <p><a href="https://www.uniqure.com/assets/uploads/doc/Gene-Therapy-Information.pdf">Gene therapy</a> offers the prospect of long-term treatments and potential cures to patients with genetic or acquired diseases. Research studies show our AAV platform can restore the expression of a missing protein or block the expression of a gene, after a single administration.</p> </div> <div class="body-block heading standard-spacing"> <h3 class="blue">Genes: Blueprints for Protein Production</h3> </div> <div class="body-block body-text wysiwyg standard-spacing"> <p>Genes provide the blueprint used by cells to make proteins. A defect or mutation in a gene can prevent the production of a protein, change its function, or make a protein toxic.</p> <p>For example, when the gene associated with the production of factor IX, a protein required for blood clotting, is missing or mutated in people with hemophilia B, they bleed even after minor trauma or surgery. Introducing a copy of the missing gene into the liver restores the levels of factor IX in the blood and reduces bleeding. Similarly, mutations in the huntingtin gene result in the production of a mutant protein that causes degeneration of neurons in the brain in patients with Huntington’s disease. Introducing a gene therapy into the brain that may prevent the expression of the huntingtin gene. A clinical program is ongoing to assess the potential to prevent brain degeneration in these patients.<br></p> </div> <div class="body-block heading standard-spacing"> <h3 class="blue">See below for an animated demonstration of how gene transfer technology functions within the body:</h3> </div> <div class="body-block video animate-on-scroll animate-fade no-space"> <figure> <div class="vid-wrapper"> <iframe id="ytplayer" type="text/html" width="720" height="405" src="https://www.youtube-nocookie.com/embed/SUOskEqLpyY?rel=0&modestbranding=1&color=white" frameborder="0"></iframe> </div> </figure> </div> <div class="body-block heading more-space"> <h3 class="blue">A Modular Platform with Potential for Expansion</h3> </div> <div class="body-block body-text wysiwyg standard-spacing"> <p>uniQure develops its gene therapies using an innovative and modular technology platform, that leverages a proprietary manufacturing process.</p> <p>The platform is modular in that it allows us to develop, manufacture and clinically test gene therapies for multiple diseases using the same set of components, including capsids, promoters, and manufacturing methods. In many cases, the disease-specific gene cassette is the only component that needs to be changed to target a new disease. We believe we will be able to reduce the time, cost, and risk of developing treatments while offering medicines that are effective and convenient alternatives to the current standard of care.</p> </div> <div class="body-block callout-modules grid-wrapper animate-on-scroll animate-children col-1 more-space"> <div class="module bg-orange"> <h3 class="caps-rule">Our gene therapy technology consists of three key elements:</h3> </div> </div> <div class="body-block accordion infographicNumbered no-space"> <ol class="accordions-wrapper"> <li class="accordion-wrapper animate-on-scroll animate-slide-up"> <div class="accordion-header"> <h3>AAV-Based Vector Delivery System</h3> </div> <div class="accordion-drawer"> <h4>Best-in-Class AAV-based Vector Delivery System</h4> <div class="wysiwyg"> <p>We deliver the genetic cargo to the target tissues using an engineered, non-replicating viral vector delivery system based on adeno-associated virus (AAV). Researchers have used AAV-based vectors in pre-clinical research and in more than a hundred clinical trials demonstrating efficacy and safety. In both preclinical and clinical studies, AAVs have also demonstrated lasting therapeutic effects extending for many years following a single administration.</p> <p>We use the AAV5 variant, or serotype, in our product candidates for hemophilia B and Huntington’s disease, and have exclusive, worldwide rights to its use in therapeutic products delivered to the brain or the liver.</p> <p>AAV5 has been demonstrated to be safe and well-tolerated in many clinical trials, including five uniQure trials that included more than 100 patients. Some of these patients were dosed more than 5 years ago showing that safety and tolerability are long-lasting.</p> <p>One of the major challenges in AAV-based gene therapy is the presence of circulating anti-AAV neutralizing antibodies that result from exposure to naturally occurring AAVs in the environment. These neutralizing antibodies can prevent successful gene transfer by AAV gene therapies and can lead to safety concerns. We believe that AAV5 gene therapy can overcome this limitation. Our data suggest that AAV5 gene therapies have the potential to treat nearly all patients, regardless of exposure to AAV5 or other AAV serotypes.<br></p> </div> <h4>Titration and Re-Administration</h4> <div class="wysiwyg"> <p>Delivering the right dose to individual patients is another significant challenge in gene therapy. In preclinical studies, we have made significant progress in re-dosing animal models. We believe this has the potential to revolutionize the way that gene therapies are applied by enabling us to potentially re-dose if necessary to deliver the right AAV dose for each patient. In addition, we have published data demonstrating successful cross-administration of gene therapies in NHPs using sequential administration of AAV5 and AAV1 vector serotypes, suggesting that cross-administration of AAV5 gene therapies with other vectors may be possible in humans.</p> </div> </div> </li> <li class="accordion-wrapper animate-on-scroll animate-slide-up"> <div class="accordion-header"> <h3>Therapeutic Gene in a <span class="push-double"></span><span class="pull-double">“</span>Gene Cassette”</h3> </div> <div class="accordion-drawer"> <div class="wysiwyg"> <p>A therapeutic gene in a <span class="push-double"></span><span class="pull-double">“</span>gene cassette” that delivers the missing protein or silences a disease-causing gene. We utilize our proprietary <a href="https://www.uniqure.com/science-innovation/enabling-technologies">enabling technologies</a> that include potent liver and brain-specific promoters, micro-RNA scaffolds and methods to effectively deliver transgenes and biologics to the liver and brain.</p> <p>uniQure has long been a leader in developing technologies for advancing gene therapy. Our AAV technology engine, built with more than 20 years of experience, drives our gene therapies, and we remain focused on developing innovations designed to address some of the major challenges in AAV gene therapy.</p> </div> </div> </li> <li class="accordion-wrapper animate-on-scroll animate-slide-up"> <div class="accordion-header"> <h3>State-of-the-Art Manufacturing Process</h3> </div> <div class="accordion-drawer"> <div class="wysiwyg"> <p>Our state-of-the-art manufacturing process brings together all key elements for a successful gene therapy product.</p> <p>We produce AAV-based gene therapies using a proprietary manufacturing process, which uses insect cells and baculoviruses, a common family of harmless viruses found in invertebrates and applies more than two decades of gene therapy manufacturing expertise. </p> </div> </div> </li> </ol> </div> </div> </section> <section class="related-links"> <div class="container"> <h4>More on Science & Innovation</h4> <ul class="animate-on-scroll animate-children"> <li> <a href="https://www.uniqure.com/science-innovation/enabling-technologies" class="btn btn-rounded"> Enabling Technologies <svg viewBox="0 0 76 18"> <use href="#arrow" /> </svg> </a> </li> <li> <a href="https://www.uniqure.com/science-innovation/publications" class="btn btn-rounded"> Publications <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> <span>uniQure</span> </a> </div> <div class="grid-wrapper"> <div class="col-group contact"> <address>uniQure N.V.<br /> Paasheuvelweg 25a, 1105 BP<br /> Amsterdam<br /> Chamber of Commerce<br /> 34275365</address> </div> <div class="col-group footer-nav"> <nav class="footer-nav-primary"> <ul> <li><a href="https://www.uniqure.com/pioneers-leaders" >Pioneers & Leaders</a></li> <li><a href="https://www.uniqure.com/patients-families" >Patients & Families</a></li> <li><a href="https://www.uniqure.com/science-innovation" >Science & Innovation</a></li> <li><a href="https://www.uniqure.com/programs-pipeline" >Programs & Pipeline</a></li> <li><a href="https://www.uniqure.com/investors-media" >Investors & Media</a></li> <li><a href="https://www.uniqure.com/culture-careers" >Culture & Careers</a></li> <li><a href="https://www.uniqure.com/contact" >Contact</a></li> </ul> </nav> </div> <div class="col-group social"> <p>Follow us on social</p> <ul> <li> <a href="https://twitter.com/uniQure_NV" target="_blank" title="Twitter"> <svg viewBox="0 0 18 18"><use href="#icon-twitter-sq" /></svg> </a> </li> <li> <a href="https://www.linkedin.com/company/uniqure" target="_blank" title="LinkedIn"> <svg viewBox="0 0 18 18"><use href="#icon-linkedin" /></svg> </a> </li> <li> <a href="https://www.youtube.com/channel/UCyFSY1dSni8hNFuOxsA7hMA" target="_blank" title="YoutTube"> <svg viewBox="0 0 22 16"><use href="#icon-youtube" /></svg> </a> </li> </ul> </div> </div> </div> </div> <div class="footer-secondary"> <div class="container"> <div class="copyright"> <p>©2024 uniQure NV. 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