24/7 BIOPHARMA - issue 1 / October 2024

RECIBIOPHARM

Manufacturing GMP-grade plasmids under the same roof as viral vector development and manufacturing streamlines the sourcing process, saving both time and energy. This in-house approach allows for faster response times and adaptability to evolving project needs. Support for multiple serotypes As viral vector serotypes differ in their tropism, careful selection is crucial for therapeutic efficacy. While AAV-2 is most prevalent, 11 AAV serotypes have been identified. Researchers have also expanded AAV potential through pseudotyping, enhancing transduction efficiency, tissue and cell specificity, and reduced immune responses. Given the diversity of serotypes and their unique benefits, manufacturing must be capable of supporting the production of various serotypes while addressing their specific challenges. Adaptable single-use systems Adopting single-use technologies (SUTs) can provide flexibility throughout the entire manufacturing process, from cell culture to final product fill. By eliminating the need for intricate cleaning and validation steps between batches, SUTs enable rapid changeover and minimise cross-contamination risk. As SUTs are becoming increasingly scalable, fully single use manufacturing platforms offer maximum adaptability and a low-risk approach to scaling production. - Robust development and manufacturing support To help with flexibility and adaptable AAV manufacturing, platforms should demonstrate scalability, allowing for seamless progression from bench-top to pilot-scale production. A holistic approach to development, incorporating a deep understanding of each process step and regulatory requirements, helps identify and mitigate risks early. Additionally, future-proofing platforms through the integration of automation and digital technologies allow for responsiveness to evolving industry trends and patient needs, including the use of alternative viral vectors beyond AAV. - -

therapeutic applications expand. This approach can make these life-changing treatments available at a lower price point.

However, investing in the development of a manufacturing platform can be prohibitively

expensive and time-consuming for AAV developers. One potential solution to achieve the benefits of a well-designed manufacturing platform is to partner with specialist AAV contract development and manufacturing organisations (CDMOs). Partnerships with CDMOs can provide access to flexible AAV manufacturing, including multiple cell lines, plasmid vectors and serotype libraries and analytical, upstream and downstream optimisation. These capabilities can help developers seeking to bring safe, effective and affordable gene therapies to market.

References

1. American Society of Cell & Gene Therapy. Gene, Cell, & RNA Therapy Landscape Report; Q2 2024 Quarterly Data Report. Available at: https://www. asgct.org/publications/landscape-report 2. https://www.pharmaceutical-technology.com/ analyst-comment/lenmeldy-becomes-worlds most-expensive-drug/?cf-view

Leveraging AAV manufacturing platforms for cost effective gene therapies

By leveraging well-designed manufacturing platforms, AAV developers can accelerate timelines, reduce manufacturing costs and improve the overall efficiency of gene therapy production. Platforms that rapidly scale with production needs can help meet the growing demand for gene therapies, particularly as their

JING ZHU Head of Process Development ReciBioPharm

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TWENTYFOURSEVENBIOPHARMA Issue 1 / October 2024