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.

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

 

uniQure develops its gene therapies using an innovative and modular technology platform, including our proprietary, cost-effective manufacturing process. An overview of our key patent portfolio can be found here.   

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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Best-in-Class 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 (adeno-associated virus). 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 the AAV5 variant, or serotype, of vector in our product candidates in hemophilia B and Huntington's disease, and have exclusive, worldwide rights to AAV5 for use in therapeutic products delivered to the brain or liver. Our research suggests that a greater portion of patients can be effectively treated with AAV5 gene therapy, compared to other AAV-based gene therapies. 

AAV5 gene therapies present a favorable immunogenicity profile.  To date, our insect-cell manufactured AAV5 gene therapies have been administered to 22 patients across three clinical studies without any evidence of AAV5 capsid-specific cellular immune responses or long-term safety complications.   

One of the major challenges in AAV-based gene therapy is the presence of circulating anti-AAV neutralizing antibodies, which can pre-exist in patients and may prevent successful gene transfer.  We have presented preclinical data demonstrating successful and effective transduction of AAV5 in non-human primates with pre-existing anti-AAV5 neutralizing antibodies (NABs).  At all observed levels, pre-existing neutralizing antibodies for AAV5 did not have a negative impact on the transduction effectiveness of the AAV5 vector. This suggests a much broader potential population of eligible patients than previously expected for AAV5-based gene therapies, as it appears that patients with pre-existing anti-AAV5 NABs may still be able to be successfully treated with AAV5 gene therapies.  

High levels of circulating anti-AAV neutralizing antibodies can develop after a single administration of gene therapy and can prevent successful gene transfer in patients.  We have made tremendous progress in optimizing and advancing re-administration and cross-administration protocols that we believe will significantly impact the application of our gene therapies and potentially enable repeated administration.  We have presented preclinical data demonstrating successful re-administration of gene therapy with our AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). 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.

AAV5 is a highly differentiated, best-in-class vector with the potential to more effectively and safely deliver gene therapies to a greater portion of patients in need of treatment.

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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.