Gene therapy represents an expanding area of medicine with the potential to change the lives of people with life-limiting conditions. As the interest in gene therapies has increased over the years, so has the number of clinical trials, with 372 gene therapy trials active in the first half of 2022. Adeno-associated Viral (AAV) vectors are the most common gene delivery agents for most of these trials, creating a substantial industry need for AAV vector manufacture.1
When bringing a new AAV-based gene therapy through the pipeline and towards clinical trials, it is important for a drug developer to onboard a robust, efficient manufacturing process. Although it is important for a Contract Development and Manufacturing Organization (CDMO) to build custom manufacturing processes when needed, many projects can make use of manufacturing platforms pre-built and pre-tested by the CDMO.
The advantages of platform processes are numerous compared with building a new process every time. The standardisation of processes improves consistency between runs. Using pre-validated, pre-optimised technology helps guarantee good performance while minimising delays. Staff are already trained on the specific processes, with no need for comprehensive onboarding. Keeping materials in stock helps to guarantee supply chain security. These factors combine to create a shorter end-to-end process and an accelerated time to market, as well as reduced manufacturing costs compared with a custom platform.
OXGENE and WuXi Advanced Therapies have combined our technologies and expertise to create a platform approach for transient AAV manufacture. As a Contract Testing Development and Manufacturing Organization (CTDMO), WuXi Advanced Therapies provides end-to-end services to cell and gene therapy companies seeking to discover, develop, manufacture and test Gene therapy represents an expanding area of medicine with the potential to change the lives of people with life-limiting conditions. As the interest in gene therapies has increased over the years, so has the number of clinical trials, with 372 gene therapy trials active in the first half of 2022. Adeno-associated Viral (AAV) vectors are the most common gene delivery agents for most of these trials, creating a substantial industry need for AAV vector manufacture.1 When bringing a new AAV-based gene therapy through the pipeline and towards clinical trials, it is important for a drug developer to onboard a robust, efficient manufacturing process. Although it is important for a Contract Development and Manufacturing Organization (CDMO) to build custom manufacturing processes when needed, many projects can make use of manufacturing platforms pre-built and pre-tested by the CDMO. Summer 2022 Volume 5 Issue 2 innovative drug candidates at scale for global commercialisation. We incorporated OXGENE’s AAVEX™ plasmids into our transient production system, creating a tried-and-tested workflow suitable for a wide range of projects. This AAVEX™ platform combines plasmid and cell line engineering, upstream and downstream process development, GMP manufacture and testing to deliver consistent improvements in AAV yields and quality.