Gene therapies plays an increasingly significant role in the future of medicine. Process development and quality testing are yet in the focus of the international authorities. Today, due to their specific characteristics as non self-replicative viral vectors, adeno-associated viruses (AAVs) are a safe approach for delivering the genes of interest.
Innovative companies face analytical challenges to find the most effective ways of process development and standardization. There is a broad range of important quality attributes for delivery vectors which need to be closely controlled for efficacy and biosafety of gene therapy. PPS has established specialized testing packages for the characterization and quality control testing of AAV particles and constituent capsid protein assemblies.
Adeno-associated viruses – benefits and challenges
AAV based gene therapy is driving today’s therapeutic discoveries and has evolved to the leading strategy for the treatment of diseases. Simultaneously, the regulatory demands are increasing and authorities request extensive analytical characterization for the drug vectors (e.g., capsid loading, process impurities). Hence GMP-compliant analytical methods and characterization protocols are required. To assess vector productivity, vector purity, biological activity and safety various analytical assays need to be performed.
Based on more than 20 years of analytical, biopharmaceutical experience, we can address our clients’ specific needs by swift and effective analytical support throughout the whole lifecycle of their gene therapy products and development processes. As a full-service partner PPS offers a broad range of analytical methods to quantify the strength/dose of the AAV vector, to quantify full and empty particles as well as particles carrying wrongly encapsulated DNA. Therefore, our analytical platform enables us to ensure the identity, purity, potency, and safety of the final product. To prove whether the gene of interest is present in the vector is essential for surveilling the production process as well as for the evaluation of the right dosage for the patient.
As a very common example - PPS can provide information on vector loading using several techniques:
- Analytical ultracentrifugation (AUC)
- Size-exclusion chromatography combined with multiangle laser light scattering (SEC-MALLS)
- Capillary gel electrophoresis assay (cGE)
- Ion exchange chromatography (IEX)
AUC analysis in particular can be integrated in existing processes to detect vector loading at particular steps in manufacturing and by that constributes to the quality of the final product.