Join us as we explore the Future of Gene Therapy 

The promise of gene therapy

The rapid development in molecular biology over the last few decades have revolutionized how we think of disease. A wide range of disorders have been found to have a genetic basis and many of the underlying gene mutations have been identified.

Gene therapy holds great promise as an effective treatment of these diseases. Over the coming decades, gene therapy treatment of a wide range of disorders, such as inherited diseases, cancer and viral infections, may be commonplace.

The principle of gene therapy is straightforward: deliver a functional gene to target cells in the patient to restore normal gene function. This is most commonly achieved using a recombinant Adeno-Associated Virus (AAV) vector where some of the viral genetic material has been replaced by a therapeutic cargo. Once the virus has infected the target cell, it uses the cell’s molecular machinery to produce the therapeutic protein.

Moving from promise to reality – Hurdles to overcome

Although the principle of gene therapy may be straightforward, the complexity and scope of the analytical methods associated with this technology are not.

Gene therapy introduces a number of analytical challenges that are not encountered with conventional biopharmaceutical products. Complex and time-consuming bioassays are needed throughout the study to assess product potency, confirm product identity, identify impurities and test for immunogenicity.

Immunogenicity testing

A common first step in gene therapy is to find out if the patient carries antibodies against the virus vector. Such antibodies are present if the patient has been subjected to the virus previously. If such neutralizing antibodies (NAbs) are present, the virus capsid will be destroyed and not able to infect the cells and deliver the gene, thus rendering the gene therapy ineffective.

Svar’s iLite® cell-based platform has a long track record of measuring NAbs against biological drugs in patient material. By combining the reproducibility and ease-of-use of assay-ready cells with the robustness of dual reporter gene assays, iLite® assays offer fast and high-quality results for a wide range of targets. We are now bringing the benefits of iLite® technology to gene therapy by introducing AAV responsive cell lines. These assays are ideal for detecting antibodies against the viral vector in human serum.  

Potency testing

Another very important step in gene therapy is analyzing the finished therapeutic product. Potency testing is performed in a pre-clinical, clinical or CMC setting to show a clinical effect and ensure batch-to-batch consistency. This is another area where iLite® technology is ideally suited. We are developing a robust in vitro assay for the measurement of therapeutic product potency.

Get rapid, reliable and reproducible results using our functional cell-based assays.

What we offer

Svar is in a unique position to offer assistance in any gene therapy project. We have robust platforms suited for all phases of drug development, as well as the experience and knowledge from our diagnostic and bioanalytical services. We are currently developing cell-based assays for immunogenicity and potency testing. Furthermore, we can create custom projects tailored to your specific needs to create truly tailor-made assays. Our Bioanalytical Services offer flexible solutions adapted to your needs and requirements.  

Together, we can move the promise of gene therapy to reality.   

 

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iLite® Cell-Based Solutions

Cell-based reporter gene assays in an assay-ready, rapid and accurate test format 

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Wieslab Bioanalytical Services

Delivering bioanalytical excellence through a collaborative approach