AAV Manufacturing Advances Pave the Way for Next-Generation Gene Therapies

The field of gene therapy continues to evolve rapidly, with adeno-associated virus (AAV) vectors at the forefront of this medical revolution. Recent developments in AAV manufacturing are set to streamline the production process, potentially accelerating the development of new treatments for a wide range of conditions.

Expanding Horizons for AAV Therapies

AAV-based therapeutics are no longer confined to rare genetic disorders. The scope has broadened to include more common indications such as cardiovascular disease, Alzheimer's, and Parkinson's. This expansion brings both opportunities and challenges, as developers must now scale up production to meet larger patient populations while maintaining consistent quality and yield.

Eva Fong, Associate Director of Process Development at MilliporeSigma's Viral Vector CDMO, emphasizes the importance of early planning: "AAV-based therapy development starts with a gene and a dream — and while that spark of innovation is important, it's critical to have plans to de-risk the program by maximizing performance at the scale of production you need."

Leveraging CDMO Expertise to Overcome Manufacturing Hurdles

As the AAV therapeutic market grows, developers face increasing pressure to optimize their manufacturing processes. Partnering with Contract Development and Manufacturing Organizations (CDMOs) that have established platform solutions for AAV production is emerging as a key strategy to overcome these challenges.

Quality and Yield Optimization

CDMOs with specialized platforms can offer significant advantages in increasing quality and yield. Optimized upstream and downstream workflows maximize high-quality AAV production, achieving high titers and recovery while minimizing impurities like empty and partially filled capsids. This results in more fully packaged and intact capsids, essential for reducing manufacturing costs per dose and enhancing the effectiveness of gene therapies.

Robust Analytics and Regulatory Support

Consistency in production is crucial for meeting quality targets. CDMOs with strong analytical capabilities can help define quality parameters for AAV production, ensuring purity and consistency throughout the manufacturing process. Additionally, their expertise in navigating the complex regulatory landscape can be invaluable, especially given the current gray areas in how existing regulations apply to viral vector therapies.

Flexibility and Scalability: Key to Meeting Diverse Needs

An effective AAV manufacturing platform should offer customization options to meet the unique needs of each program while accommodating various serotypes and batch sizes. This flexibility supports cost-efficiency and reduces variability across production scales, whether producing 3 liters or 1000 liters.

MilliporeSigma, with over 30 years of experience in viral vector manufacturing, has developed the AAV Express platform to address these needs. The platform aims to provide a full spectrum of support, enabling streamlined and de-risked AAV production for gene therapies.

As the field of gene therapy continues to advance, the role of specialized CDMOs in AAV manufacturing is likely to become increasingly crucial. By leveraging their expertise and established platforms, developers can potentially reduce costs, mitigate risks, and accelerate the path to clinical trials — ultimately bringing innovative gene therapies to patients more quickly.