Gene therapies are changing how we approach a range of diseases, with the potential to reduce or eliminate the need for lifelong medication. Despite regulatory approval of several therapies, access to these treatments is limited. Scaling production to meet wider demand remains a significant hurdle.

The following insights explore what it takes to move from research to clinical-grade production while avoiding costly missteps.

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Space and equipment demands

Scaling a gene therapy product requires substantial investment into specialized laboratory spaces and equipment, with a strong emphasis on contamination control. For example, Grade B cleanrooms or isolators are needed for aseptic preparation, while biosafety level 2 rooms protect staff from biohazards.

When it comes to designing spaces for clinical-grade production, it is important to understand your manufacturing process first, advises Drew Hope, senior GMP compliance consultant and qualified person at eXmoor Pharma. “Understand what the throughput of that needs to be, what the scale of it is, how many batches there are going to be, and design your facility around that process.”

Scaling up to use large bioreactors can look very different than scaling out, which is commonly done for autologous products and involves making more small batches of product. The space and equipment requirements for these options are therefore very different.

When sourcing equipment for transitioning from research to scaled-up GMP-compliant manufacturing, it is important to consider product contact surfaces. According to Hope, “A big change between research and manufacturing is product contact surfaces. In general, single-use systems are adopted so that they're disposable. You don't ever reuse any product contact surfaces.”

Workforce challenges

The gene therapy sector is facing difficulties recruiting and retaining staff with expertise in gene therapy techniques and regulatory compliance. According to a workforce survey analysis by the International Society for Cell and Gene Therapy (ISCT), manufacturing and process development were the most desired skills and the biggest workforce gap in the cell and gene therapy sector.

Adding to staffing challenges, Hope believes that “a real shortfall in the workforce is people that understand quality issues. It's not just the quality managers that do the quality management. Everybody has to understand it.” The ISCT also highlights training gaps from academic degrees, suggesting that academia and industry should work together to bridge these gaps and establish suitable training programs. For example, with basic research and early drug discovery taught by academic institutions, and clinical development and regulatory review stages taught by industry.

Regulatory requirements for transitioning to GMP production

Before transitioning from research to clinical-grade production, Hope encourages companies to reach out to the relevant regulatory body early to discuss their approach to the quality and safety of their products and the study design. Ideally, this should be done before making any pre-clinical batches for safety studies or non-human safety studies. “Because if you're going to be submitting that data to them to support your clinical trial application, they need to understand the approach and consider whether it's acceptable or not.” he explains.

One challenge that Hope often observes when working with other companies is with materials categorization. Many suppliers in the life sciences sector are not GMP manufacturers themselves, so they may not be willing to apply the standards needed for gene therapy products. “So that puts a lot of emphasis on cell and gene manufacturers to choose very wisely their suppliers and the materials that they use and have good systems to assess those materials and those vendors. And I find that frequently, those manufacturers get it wrong,” he notes.

This is important when transitioning from research to GMP production. “For example, the media that you use in growing up your virus, that media might be completely unsuitable for using in a GMP manufacturing process and almost impossible to replace.” Hope continues. Process development staff should therefore consider the compatibility of their materials and processes with GMP guidelines early.

Validating the process and product

During the technology transfer process from the research laboratory to the manufacturing unit, critical information about the production process needs to be considered and documented. This enables the manufacturing unit to assess whether they can make the product in a GMP-compliant way.  

Research teams must provide information about the controls used, testing specifications, and how they can demonstrate safety and potency. Hope finds that “the research teams will sometimes be a bit surprised by that and haven’t really got a standard response to it. So, they'll need to do a lot more research to then go into clinical.”

Using automation solutions to scale

For scale-out, Hope cautions against relying on the same manual manufacturing process used during early-phase production. As volume increases, this approach becomes inefficient and cost-prohibitive.

Instead, he recommends exploring scalable solutions such as automation for both production and testing. While fully automated, end-to-end systems offer significant benefits—like higher throughput, reduced human error, and more consistent product quality—they may not be feasible for every organization right away.

To bridge the gap, labs can take a phased approach: identify high-risk or repetitive tasks that are good candidates for partial automation first, then build toward more integrated systems over time. When evaluating automation options, consider ease of integration with your existing workflows, validation requirements, and vendor support for GMP compliance. Scalability isn’t just about volume; it’s about designing a process that can grow with your product and your team’s capabilities.

Considerations for partnering with a CDMO

For companies considering partnering with a CDMO to support clinical-grade production and overcome scaling challenges, Hope suggests a build-or-buy analysis can be helpful to determine whether a CDMO is the right choice for your product or whether building your own facility is best. Here are examples of considerations to include in your analysis:

• Cost comparison: Assess capital expenditure for building a facility vs. service fees for outsourcing.
• Time to market: Evaluate which option enables faster clinical or commercial production.
• Internal capabilities: Determine whether in-house expertise and infrastructure are sufficient for GMP manufacturing.
• Product complexity and volume: Consider whether your therapy’s process is better suited to internal control or external standardization.
• Long-term flexibility: Weigh the scalability, adaptability, and control each option offers as your program evolves.
• Regulatory readiness: Assess whether your team can meet compliance expectations independently or would benefit from an experienced CDMO’s support.

If choosing a CDMO, Hope advises finding one with deep expertise in cell and gene therapy manufacturing, not just general GMP capabilities. He stresses: “Choose a true partner that you can work with that will implement a process that works well for your product.” To do this, look for a partner that demonstrates transparency in communication, a track record of regulatory compliance, and flexibility in adapting processes to your product’s unique needs. Ask prospective partners how they handle tech transfer, how much control you’ll retain over process decisions, and whether they’ve scaled similar therapies before. A strong CDMO relationship is built on aligned expectations, shared goals, and the ability to navigate complexity together.

Manage costs with long-term goals in mind

Scaling gene therapy manufacturing comes with high upfront costs, but strategic planning can help manage these investments. Phased buildouts, modular facility designs, and early alignment with commercial goals can prevent costly rework and reduce the risk of sunk costs. Choosing scalable, flexible equipment and building partnerships with experienced vendors or CDMOs can also help stretch budgets while maintaining compliance.

Final thoughts

Bridging the gap between research and clinical-grade production in gene therapy requires more than technical know-how. It demands foresight, regulatory awareness, workforce readiness, and a clear path to commercial viability. With the right strategy, labs can avoid common pitfalls and bring promising therapies to more patients, faster.