In the rapidly evolving field of gene therapy, the use of adeno-associated viral (AAV) vectors holds significant promise. However, to unlock the full potential of these therapies, scientists must navigate challenges associated with safety and manufacturability. These issues are pivotal in ensuring that innovative treatments can be produced efficiently and safely at scale, benefiting patients globally.
An upcoming webinar hosted by GEN will delve into these challenges, featuring expert insights from Mark Stockdale, a key figure in the field. Stockdale's presentation will focus on the intersection of synthetic biology and artificial intelligence (AI) as tools to enhance the safety, potency, and scalability of gene therapies.
A central feature of the webinar will be the introduction of "AAV Edge", a comprehensive platform designed to optimise the end-to-end process of gene therapy development. This platform aims to refine every stage from payload design to the creation of production cell lines and the management of model-guided processes.
A significant portion of the discussion will explore how an internally developed, transformer-based AI model is utilised to design tissue-specific promoters. This innovation has resulted in a notable ability to achieve a dynamic range in gene expression that is over 200 times greater in on-target tissues compared to off-target ones. This specificity is crucial for reducing unintended effects and improving the efficacy of gene therapies.
Also in focus will be advancements in sequence optimisation, which can significantly boost expression levels for clinically relevant transgenes, reportedly by as much as sevenfold. Such improvements are critical for the effectiveness and efficiency of therapeutic applications.
The webinar will also examine how strategic silencing of transgene expression in the production phase can minimise cellular stress and toxicity. These factors are vital to ensuring the viability of manufacturing processes and maintaining the integrity of the therapeutic product.
Further, participants will learn about the innovative combination of Asimov’s clonal HEK293 cell line with an optimised two-plasmid system and model-driven process development. This methodology is said to achieve high unconcentrated titers, reaching up to 1E12 viral genomes per millilitre across various serotypes. Such advancements underscore the progress being made in stabilising and enhancing production yields, which are essential for meeting global therapeutic demands.
The event will conclude with a live question-and-answer session, where attendees will have the opportunity to engage directly with the expert panelist. This interactive segment aims to provide deeper insights into the topics discussed and address specific questions from the audience.
This webinar represents a significant opportunity for professionals in the field to gain a deeper understanding of the current advancements and strategies in overcoming the inherent challenges of AAV gene therapies.
Source: Noah Wire Services