AAV-based gene therapies require rigorous characterization at every production step. SPR provides real-time binding data that fits naturally into the bioproduction workflow.
The innovation in gene therapy is poised to create a huge impact in the area of personalized medicine. Gene therapy holds promise for treating a wide range of diseases, from cancer and diabetes to rare genetic disorders, potentially revolutionizing treatment modality and making it an area of research of utmost importance. Although some drugs have been discovered and approved, gene therapy has yet to reach its full potential due to the complexity and regulatory procedures involved.
Amongst different gene therapy materials, viral vectors are a popular medium used to deliver genetic material for therapeutic application. Adeno-associated virus, or AAV, is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction, featuring high efficiency and excellent safety in human clinical trials. AAV-based products have been licensed to date in the US and EU for treatment of rare genetic diseases.
The AAV product is a function of vector and process design
During the production of AAV vectors, there are several quality control (QC) parameters that should be rigorously monitored to comply with clinical safety and efficacy. Two critical aspects are viral potency and empty capsid impurities.
Viral potency determination is a critical characteristic that must be well established prior to use. Quantitative PCR techniques are the most frequent dose-determining assay. However, digital PCR is time consuming, expensive, and not easily integrable to the production process. This is where Affinité portable SPR can play an important role in terms of reducing time and cost, as well as integration into the quality control procedure.
Another key project objective is to determine empty/full capsid ratios. Empty viral capsids are a challenging and unique type of impurities for AAV vectors with the potential to induce toxicity and undesirable immune reactions, particularly if administered systematically in large doses. Transmission Electron Microscopy (TEM) is a de facto technique today but is labour intensive, time consuming, and requires specialized trained personnel.
With the use of portable SPR, we hope to demonstrate the improvement of the manufacturing processes for pre-clinical and clinical AAV viral vectors with reliable and in-process AAV characterisation.
Portable SPR — a game changer for assay development and quality control
A portable SPR can improve inefficient assay workflows by providing an orthogonal technique to ELISA to offer step-by-step debugging and real-time data. Using SPR requires less reagents and no labeling, making it less expensive and time consuming than ELISA. Experimental results are available in real time without the need for labeling.
Democratizing SPR can help to democratize gene therapy
Though promising, the road to democratize gene therapy can be a long winding road due to the need for large biomanufacturing facilities with expensive and slow quality control equipment. With the collaboration with CERVO Brain Research Center, we hope to enable smaller labs and facilities to also participate in the research of gene therapy using viral vectors.
Contact us to learn how we are applying SPR protocols to set up a new measure of quality control.