uniQure has built the world’s leading gene therapy platform.
Focused on hemophilia, Huntington's disease and cardiovascular diseases.
Gene therapy offers the prospect of long-term and potentially curative benefits to patients with genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration. > Gene therapy information (PDF)
Genes are the specific areas of DNA that provide the blueprint used by the body’s cellular machinery to make proteins. A defect or mutation in a specific gene can result in the inability or reduced ability to express a protein, or the reduced functionality of a protein. For example, when the gene associated with the production of a protein required for blood clotting is missing or mutated in hemophilia B patients, these patients’ blood cannot clot enough to stop bleeding, even after a minor trauma or surgery. Introducing a copy of the proper gene into the cell would, in principle, restore the presence and natural function of the blood-clotting factor, which would prevent bleeding.
uniQure develops its gene therapies using an innovative and modular technology platform, including our proprietary, cost effective manufacturing process.
Paired with our state-of the art manufacturing process, uniQure brings together all key elements for a successful gene therapy product.
The platform is designed to be modular, in that it may allow us to efficiently develop, manufacture and seek regulatory approval for multiple gene therapies generally using the same principal components. In some cases, we believe that the disease-specific gene cassette will be the only component that needs to be changed to target a new disease in a particular tissue. As a result, we may be able to reduce the overall preclinical and potentially clinical development activities required to obtain regulatory approval, and to significantly reduce the overall development risk, time and cost.
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