Accelerating analytics for gene therapy using enhanced mass photometry

基因疗法对许多使用传统药物难以预防或治疗的疾病具有巨大的前景，包括遗传性、罕见和与年龄相关的疾病以及癌症。随着行业的发展，迫切需要更高效、更有效的质量控制测试技术。这造成了处理瓶颈，并意味着通常只在生产结束时进行测量，即使这样的信息在整个开发和生产过程中都是非常必要的。需要单独的化验来测量污染物，这造成了进一步的效率低下。我们相信，这项技术可能会改变这一领域的游戏规则，因为初步实验表明，它的表现可以超过目前最先进的水平。它可以测量负载颗粒的百分比，同时测量较小污染物的量，而且比目前的方法快15倍，便宜100倍。它将推动基因治疗行业的发展，为英国和其他国家带来经济和其他好处。该项目以卓越的创新技术瞄准快速增长的市场，是一项合理的投资。这也是及时的；目前正在制定这一领域的法规，为将这项技术嵌入生产和质量控制过程创造了机会。

Gene therapy holds huge promise for many conditions that have been difficult to prevent or treat using conventional drugs, including inherited, rare and age-related diseases, and cancers. As the industry grows, there is an urgent need for more efficient, effective technologies for quality control tests. Technologies developed for traditional drugs have major limitations when applied to complex gene therapies, such as adeno-associated virus (AAV) gene therapies, which use virus particles to deliver genes that can help treat or cure disease.For AAV therapies, one important factor is how many vector particles are carrying the target gene, but current approaches for measuring this are expensive and time-consuming, and usually need to be outsourced. This creates a processing bottleneck and means the measurement is usually done only at the end of production, even though such information is highly desirable throughout development and production. Separate assays are needed to measure contaminants, creating further inefficiencies. As another important limitation, no available technology can distinguish partially loaded particles from full ones.In this project, we propose to address these challenges using mass photometry, a technology we developed that measures the mass of single particles using minimal sample. We believe this technology could be a game-changer in this area because preliminary experiments show it can outperform the current state-of-the art. It can measure the percentage of loaded particles while simultaneously measuring amounts of smaller contaminants, and it is up to 15-fold faster and 100-fold cheaper than current approaches. Here, we plan to benchmark mass photometry against existing approaches and also make improvements, to better tailor our approach to the requirements of AAV vector characterisation.The project is a collaboration between Refeyn Ltd (the world's sole producer of mass photometry technology), who will develop the instrument, and the Cell and Gene Therapy Catapult, who will provide AAV material and conduct benchmarking.If successful, this project will deliver a transformative technology, helping accelerate the development, production, approval and safe use of promising gene therapies. It will advance the gene therapy sector, bringing economic and other benefits for the UK and beyond. Targeting a rapidly growing market with a superior, innovative technology, this project represents a sound investment. It is also timely; regulations are now being formed in this area, creating an opportunity to embed this technology in production and quality control processes.