Adding Consistency to Gene Therapy Manufacturing

Viralgen developed a dedicated platform and a designed-for-use cell line, which combine to improve efficiency and reduce costs

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Expanding the availability of gene therapy depends on increasing the production of various components, particularly viral vectors. As explained in Viralgen’s “A proprietary mammalian suspension technology for scalable rAAV production: The Pro10™ cell line” (see white paper linked below): “Recombinant adeno-associated viruses (rAAVs) are among the most popular vectors for gene therapies due to recent clinical successes. To meet clinical and market needs of increasing AAV vector amounts, the manufacturing stage poses the biggest bottleneck.”

César Trigueros Viralgen logo
César Trigueros Fernande

César Trigueros Fernandez, PhD, chief scientific officer at Viralgen, says, “In a funny way, I’ve been trying to kill viruses all my life, but now I’m trying to produce more viral vectors.” For him, that production started with lentiviral vectors while for the past five years he’s focused on rAAVs.

From that experience, Trigueros points out several challenges in manufacturing AAVs. “In some indications, we are working with rare diseases,  combined with the fact that it is a treatment based on single doses, and that is a massive challenge for
manufacturing,” he says. “The opposite challenge arises in
working on a common indication, which may require quite a lot of viruses, and we’re still in the phase where we are learning how to produce in a massive, massive amount.”

On top of those challenges, contract development and manufacturing organizations like Viralgen lack guidance. “Basically, there are few guidelines from regulators showing how to produce these viruses and what are the right specifications,” Trigueros explains.

To address some of that uncertainty, Viralgen developed what Trigueros describes as a “corporate platform, which means that we have a production system that we are applying for every single client. It’s always the same production system, with the only difference of working with a different capsid and gene of interest.”

Creating consistency

Fundamentally, the Viralgen platform depends on the Pro10™ platform, a technology licensed from AskBio, which includes a HEK293-derived, animal component-free suspension cell line. The company uses the Pro10™ cell line in a continuous process composed of upstream and downstream steps, as well as analytics and testing. The consistent methodology and technology decrease the numbers of variables and optimization steps in rAAV manufacturing. The Viralgen platform also shortens development and manufacturing timelines and reduces costs.

As Trigueros explains, scaling up is easier when working within the same production system, because it generates abundant data that we can use to build specifications and apply to different serotoypes. From development on one sequence, for example, Viralgen can apply the associated data to the manufacturing of future sequences. “From every single sequence, we learn as much as we can,” Trigueros says.

When asked about the most unique feature of this platform, Trigueros emphasizes the Pro10™ cell line. “These cells have been specifically isolated and developed to produce AAVs,” he says, “and that is the most important feature of our platform.”

A custom and complete service

Despite using a consistent platform, Viralgen customizes development and manufacturing for each customer. “The first thing that we do is analyze the sequence of the client’s gene of interest,” Trigueros says. “From the size of the sequence, we can recommend what is going to be appropriate during manufacturing.”

Beyond customization, Viralgen provides a turn-key service. “We offer our clients a full package, including all the supply chain, all the production, but also the entire regulatory package,” Trigueros explains. “We are working often with regulators from Europe, US, UK, and other countries. Then, Viralgen applies any regulatory feedback to future projects.”

As Viralgen improves the capabilities of its current platform for manufacturing AAVs, it’s also working on a next-generation version for production. “It’s a natural iteration process,” Trigueros explains. Only with such ongoing improvements can we reduce costs of gene therapies and help even more patients.


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