Building Scalable, Reliable AAV Gene Therapy Manufacturing

The AMT designation reflects increased interest in platform-centric development in advanced therapies. What we are seeing from the FDA is a move in that direction, with increased emphasis on evaluating the platform early, which is a meaningful change because it allows the underlying processes to be understood, trusted, and reused across programs.

The AMT designation reinforces that Cytiva is helping enable this shift, with technologies designed to deliver consistency and regulatory alignment from the outset. Stronger platforms are what will ultimately make these therapies scalable and accessible.

It is difficult because once host cell DNA is encapsidated, it is effectively protected from removal by downstream processing. At that point, there is no reliable way to remove it using conventional purification methods.

The implications are significant. In gene therapy, we are deliberately introducing genetic material into patients. The presence of unintended host cell DNA is a risk that is not yet fully characterized, but one that developers need to minimize as much as possible.

That is why regulators have set a high bar, with expectations for residual DNA below detectable limits.

It is not just a quality consideration; it is fundamental to patient safety and to building confidence in these therapies.

The ELEVECTA transient cell line addresses the problem at the source, during production. It is a genetically engineered human embryonic kidney (HEK) 293 suspension cell line designed to significantly reduce the amount of host cell DNA that gets packaged into the capsid, with up to a 99 percent reduction compared to other commercially available transient cell lines.

That upstream control is essential. Once DNA is inside the capsid, it cannot be removed efficiently, so the only effective strategy is to prevent it from being encapsidated in the first place.

This is also about platform maturity. Robust platforms are designed to control risk at the source rather than trying to correct it later.

It changes the model from product-centric to platform-centric evaluation. Instead of presenting a finished process at the end, developers can engage with the FDA earlier to align on the platform during development. When they file with the FDA, they can refer to the AMT designation, which helps accelerate the filing, which is critical for start-up companies.

That is a meaningful shift because it enables earlier scrutiny of the approach itself, supports a more efficient chemistry, manufacturing, and controls review, and reduces the risk of late-stage surprises.

In practical terms, it moves the industry toward platform-based approval pathways, where a well-understood and validated platform can support multiple therapies.

It improves confidence by strengthening both the platform and how it is evaluated.

In this space, we have to acknowledge that there have been and will continue to be challenges related to patient outcomes. An adverse event does not necessarily mean the platform is flawed. What matters is having the right level of scrutiny to distinguish between a specific outcome and the underlying approach.

That is where platform maturity and early regulatory engagement are critical. By rigorously evaluating the platform, we can support innovation while maintaining a strong focus on patient safety.

They will be foundational. The next generation of genomic medicines will depend on mature, scalable platforms that can be applied consistently across therapies.

That means investing in automation, standardized cell lines, and scalable manufacturing solutions so developers can build with the end in mind from day one.

If we want to move beyond one-off therapies and reach more patients, platform maturity is essential. It is what enables both innovation and long-term sustainability in this field.

For more information about this topic, you can watch the panel “Manufacturing the impossible—cell & gene therapies from bench to billions” from the 2026 Danaher Summit.

The introduction to this interview includes text that has been created with the assistance of generative AI and has undergone editorial review before publishing. Technology Networks' AI policy can be found here.