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

The adeno-associated virus (AAV) therapeutic market is experiencing rapid evolution, with recent FDA approvals and upcoming regulatory decisions in 2025 signaling a new era for gene therapies. As these treatments expand beyond rare genetic diseases to target more common indications, including cardiovascular and neurological conditions, the industry faces both opportunities and challenges in scaling up production to meet increasing demand.

Expanding Horizons and Manufacturing Complexities

AAV-based therapies are now being developed for larger patient populations, offering transformative potential for conditions such as Alzheimer's and Parkinson's disease. However, this expansion introduces new complexities in the manufacturing process. Scaling up from bench to production scale requires significant process development and optimization to maintain consistent quality and yield across batches.

Eva Fong, Associate Director of Process Development, Viral Vector CDMO at MilliporeSigma, 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."

CDMO Partnerships: A Strategic Approach to AAV Manufacturing

To address these challenges, many developers are turning to Contract Development and Manufacturing Organizations (CDMOs) with established platform solutions for AAV production. These partnerships offer several key benefits:

1. Increased 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.

2. Robust Analytics: CDMOs with strong analytical capabilities can help define quality parameters for AAV production, ensuring purity and consistency throughout the process.

3. Flexibility and Scalability: Effective platforms offer customization options to meet unique program needs while accommodating various serotypes and batch sizes, from 3 liters to 1000 liters or more.

4. Regulatory Support: With evolving regulatory guidelines for AAV-based therapeutics, partnering with experienced CDMOs can help navigate expectations and expedite the approval process across different target markets.

Streamlining Development with Established Platforms

MilliporeSigma, a leader in viral vector manufacturing for over 30 years, has developed the AAV Express platform to address these industry needs. This platform provides a full spectrum of support for streamlined and de-risked AAV production, offering flexibility and scalability while reducing costs and accelerating time to clinic.

By leveraging such established platforms, developers can significantly streamline gene therapy development, benefiting from built-in expertise, reliable scalability, and operational efficiency. This approach helps reduce costs, mitigate risks, and accelerate the path to clinical trials, ultimately bringing gene therapies to patients more quickly.

As the AAV therapeutic market continues to evolve, strategic partnerships between developers and experienced CDMOs will play a crucial role in overcoming manufacturing challenges and advancing the next generation of gene therapies.