uniQure's Technology:

Excellence in gene therapy through innovative modular technology, proprietary manufacturing and the experience to achieve success.

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.

Our gene therapy technology consists of three key elements:

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Therapeutic gene cassettes that carry a transgene that encodes, or provides the blueprint for the expression of, a therapeutic protein;

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Therapeutic Gene Cassettes

We design our own gene therapies to deliver a transgene into the body’s cells to encode, or provide the blueprint for the expression of a therapeutic protein. The transgene is carried in a gene cassette together with DNA promoters that direct expression in specific tissues.

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An AAV-based vector delivery system for delivering the gene cassette

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AAV-based Vector Delivery System

We deliver the gene cassette to the target tissue using an engineered, non-replicating viral vector delivery system based on AAV. We have based all of our current product candidates on our AAV-based vector technologies. Researchers have used AAV-based vectors in pre-clinical research and over 80 clinical trials in which AAV-based vectors have demonstrated a good safety profile. AAVs have also demonstrated lasting therapeutic gene expression following a single treatment in preclinical and clinical studies. We use different variants of AAV vectors, or serotypes, each of which selectively targets particular tissues. For example, the AAV5 serotype has shown a strong affinity for the cells typical to liver tissue and to the neuron cells found in the central nervous system.

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Administration technologies to effectively deliver the relevant transgene into the tissues and organs

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Administration technology

We and our collaborators are developing expertise in utilizing a variety of technologies to administer the vector to the body in order to optimize delivery of our gene therapies into tissues or organs relevant to the indication we are targeting. Administration technologies include intramuscular injection in the leg of the patient using a syringe or intravenous infusion in our hemophilia B program.

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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.