Advanced therapies have already demonstrated that they can deliver extraordinary clinical outcomes. The challenge facing the sector now is no longer simply proving that these therapies can work. It is ensuring that promising science can survive the far more complex journey from early development to scalable, sustainable and accessible products.
That was the central question opening the panel discussion on designing advanced therapies that survive scale.
For Núria Gavaldà, one of the biggest underestimated challenges is manufacturing itself — not simply the production process, but the operational logistics surrounding it. An innovative therapy can have compelling science behind it, yet still encounter serious problems when developers attempt to translate that science into a repeatable manufacturing model.
Esteve Trias widened the lens further. His concern was the absence of global planning from the beginning of development. Too often, organisations approach development sequentially: first research, then clinical development, then manufacturing, and eventually reimbursement and adoption.
The problem is that decisions made at one stage can create limitations much later.
A therapy cannot be designed purely around whether it can produce an encouraging clinical result. Developers also need to consider how research will transfer into the clinic, how the product will ultimately be manufactured, whether it can scale and, crucially, whether healthcare systems will be able and willing to adopt and reimburse it.
For smaller biotechnology companies, however, this creates an obvious challenge.
Margot Pont highlighted the tension facing organisations with limited resources. Small companies cannot necessarily develop expertise across every element of manufacturing and commercialisation themselves. Nor should they need to continually reinvent processes that have already been solved elsewhere in the ecosystem.
This creates an important role for partnerships, CDMOs and shared infrastructure. If established manufacturing knowledge and standardised capabilities can be made accessible to smaller innovators, those companies can concentrate resources on the parts of their technology that are genuinely differentiated.
Jacob Smith introduced another factor that can easily disappear from development models: time.
Every part of the advanced therapy lifecycle takes time, yet different stakeholders often have very different expectations about how long individual activities should take. When development is viewed as a series of isolated steps, those timing assumptions can become disconnected.
And time is not simply a scheduling problem. As the panel noted, time is also cost.
A delayed technology transfer, an underestimated analytical programme or a manufacturing process that later requires substantial redevelopment can affect not only timelines but financing requirements and commercial viability.
Smith therefore argued for a much more integrated framework linking product selection and biology with manufacturing, analytical development and supply-chain strategy.
The common thread across all of these perspectives was striking.
The greatest barriers to advanced therapy scale-up may not be spectacular scientific failures. They may instead be the accumulation of decisions made without sufficient consideration of what comes next.
Manufacturing, logistics, cost, time, reimbursement and access cannot be treated as separate problems that will eventually be solved by separate teams.
They are connected parts of the same development pathway.
For the next generation of advanced therapies, success may therefore depend on changing the question developers ask themselves.
Instead of simply asking, “Can we make this therapy work?”, organisations may increasingly need to ask:
“Can we design this therapy so that it can still work when everything around it has to scale?”
That subtle change in perspective could prove decisive. Because scientific promise may open the door to development — but planning for the entire lifecycle is what gives a therapy the chance to make it through.
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