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Quality from the start: designing a scalable AAV production platform

This Feature Partnership discusses AAVs and how manufacturers can overcome the key development challenges and create a scalable AAV production platform.

Adeno-associated viruses

What are the greatest challenges in getting AAV-based gene therapies to clinic, and how can the industry work together to overcome them?

A common challenge cited by developers of AAV therapies is the difficulty of scaling up production. Production processes established during small-scale preclinical production often require significant modification to meet the demands of clinical scales, which necessitate the production of larger volumes of material in order to dose more patients. Such modifications can lead to costly re-validations, delaying the delivery of important treatment to patients.

At the core of this challenge is the design and maintenance of a high-performing process. This means that our team at Ascend considers quality to be the most critical barrier to getting AAV-based gene therapies to the clinic. Scaling up production without establishing a reliable, reproducible process from the beginning can cause a marked decline in product quality as production scales increase. At the same time, AAV developers must seek to lower the cost-per-dose of their therapies, as high costs are a significant obstacle to broadening patient access to advanced therapies.

For the industry to streamline regulatory approvals and meet patient demand, it is essential that safety and quality are prioritised from early in development. This requires the design of productive and reliable processes that generate the desired quality and volumetric yield, while minimising overall development costs.

Register your details to read more.