In this presentation, Mark Stockdale, Senior Director of Strategic Alliance at Asimov, provides an insightful overview of his company’s cutting-edge approaches in gene therapy development. Here, he highlights Asimov's integrated platform, methodology, and innovative technologies.
Stockdale bean by explaining Asimov's capabilities as a comprehensive solution provider for gene therapy, encompassing everything from payload design and promoter integration to optimizing production systems. He emphasises the use of synthetic biology, computational models, and CAD software, which collectively streamline the design and optimisation of genetic systems for therapeutic applications.
One of the key topics discussed was the AAV Edge production system. This system features a robust HEK293 cell line, a transient plasmid system, and model-guided process development, all of which contribute to achieving high titers and exceptional quality in gene production.
Stockdale then delved into sequence optimisation, where machine learning algorithms play a crucial role in enhancing therapeutic transgenes. This optimisation leads to significantly improved gene expression in HEK293 cells.
Another significant area covered is the development of tissue-specific promoters. Asimov has created a library of these promoters, particularly focusing on cardiac expression, which enhances on-target expression while reducing off-target effects.
Addressing the challenges posed by toxic genes, Stockdale explained that Asimov has developed genetic modules to silence these genes during AAV production. This innovation ensures cell viability and efficient virus production.
He also introduced the two-plasmid system, which includes rep, cap, and helper genes, stating that it improves AAV production and quality compared to traditional methods.
To conclude, Stockdale shared benchmarking results, demonstrating that the AAV Edge system significantly outperforms existing systems in terms of titer, purity, and capsid quality. He also outlines future prospects, with a focus on refining production scalability and therapeutic design. This presentation underscores Asimov's commitment to pushing the boundaries of gene therapy development through innovation and technology.
