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commonly achieved using a recombinant Adeno-Associated Virus (AAV) vector where some of the viral genetic material has been replaced by a therapeutic cargo.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.
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.
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.