The Alliance for Regenerative Medicine cites a total of 362 active gene therapy clinical trials and a similar number for gene-modified cell therapies. With recent approvals in both modalities, expansive and growing pipelines and substantial global financings of companies in the field, the pace at which these important new therapeutics will reach the market will continue to accelerate. It has been estimated that by 2025, the FDA will be approving 10 to 20 cell and gene therapy products per year (1).
As gene therapies and gene-modified cell therapies show increasing promise, the need for innovative and proficient viral vector manufacturing continues to grow. Increased regulatory guidance governing the manufacturing and testing of viral vectors adds complexity and increases the timelines to proficient viral vector manufacturing and analytical testing. Using adeno-associated virus (AAV) as one example, this white paper will describe how the implementation of platform characterization and safety assays for gene therapy vectors can increase the likelihood of success in process validation and accelerate the timeline to commercialization for gene therapy products and delivery to patients in need. These platform assays allow for specific parameters to be customized (e.g. gene of interest) and require minimal additional qualification work to maintain the validated state. Because these assays are pre-qualified, they reduce the variability inherent in assay validation and subsequently the time needed to establish readiness for regulatory compliance. While this approach increases the standardization across the testing workflow, it remains flexible and able to address the specific needs of developers and manufacturers. Additionally, the approach is as future-proof as possible, allowing for adaptability as the regulatory landscape of gene therapies evolves.