Streamlining Time-Intensive Processes in Gene Therapy Discovery and Development: A Focus on AAV Vectors

Adeno-associated viruses (AAVs) are an attractive choice for delivering novel gene therapies: they have low pathogenicity and immunogenicity and can safely and effectively deliver a range of transgenes. However, during production, it is difficult to maintain the AAV viral genomes that code for complex RNA structures without evolution in cell cultures. Because of the relative novelty of viral vectors as a therapeutic delivery method and the potential for evolution and genetic mutation, AAV production, including R&D, pre-GMP process development, and manufacturing, is subject to strict quality checks and regulations.

Advanced, highly sensitive platforms that offer rapid and accurate analysis of genome size and integrity are needed to quickly assess viral vectors throughout development and manufacturing. The SCIEX Biophase 8800 and RNA 9000 Purity and Integrity Kit can help speed up the AAV genome verification process across discovery and development. The Biophase 8800 can run eight samples in parallel, enabling faster routine assessments, such as detection of batch-to-batch variability and contamination in samples.

Another challenge with AAV production is next-generation sequencing (NGS) library preparation, which can be time-consuming even for shorter genomes like AAVs. Even automating these preparation protocols can take a long time with traditional tip-based liquid handling. The Beckman Coulter Life Sciences Echo Acoustic Liquid Handler can speed up NGS library preparation and save reagents through precise, contact-free acoustic transfers of small reaction volumes.

Due to their versatility, AAV vectors have become indispensable tools in targeted gene therapies. By using advanced automation technology, researchers can overcome the limitations of AAV production and bring life-saving gene therapies to market faster. For a more comprehensive list of solutions for accelerating AAV production, contact an expert at the Life Sciences companies of Danaher.