Our Platform

Hero aav gene therapy platform

An industry-leading platform focused on the liver and CNS.

uniQure has built an industry-leading gene therapy platform focused on liver-directed and central nervous system (CNS) disorders.

Gene therapy 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.

Genes: Blueprints for Protein Production

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.

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.

See below for an animated demonstration of how gene transfer technology functions within the body:

A Modular Platform with Potential for Expansion

uniQure develops its gene therapies using an innovative and modular technology platform, that leverages our proprietary manufacturing process.

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.

Our gene therapy technology consists of three key elements:

  1. AAV-Based Vector Delivery System

    Best-in-Class AAV-based Vector Delivery System

    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.

    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.

    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.

    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.

    Titration and Re-Administration

    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.

  2. Therapeutic Gene in a Gene Cassette”

    A therapeutic gene in a gene cassette” that delivers the missing protein or silences a disease-causing gene. We utilize our proprietary enabling technologies that include potent liver and brain-specific promoters, micro-RNA scaffolds and methods to effectively deliver transgenes and biologics to the liver and brain.

    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.

  3. State-of-the-Art Manufacturing Process

    Our state-of-the-art manufacturing process brings together all key elements for a successful gene therapy product.

    We produce AAV-based gene therapies in our own facilities with our proprietary manufacturing process, which uses insect cells and baculoviruses, a common family of harmless viruses found in invertebrates. uniQure is committed to manufacturing excellence in gene therapy. We believe that our leading manufacturing capabilities provide us a significant competitive advantage in rapidly delivering gene therapies to patients. And with our high-speed and larger-scale manufacturing competencies for sizeable indications beyond orphan diseases, we aspire to lead the disruptive technology field in this area.

    Our facility in Lexington, Massachusetts, applies more than two decades of gene therapy manufacturing expertise to provide highly scalable, flexible production capabilities to support uniQure programs.

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