“Regulatory Readiness is Built Early” and Other Lessons for CGT Developers
Advanced therapy developers are used to making hard decisions under pressure. In early development, the priority is often clear: reach the clinic, generate meaningful data, and keep the program moving.
That urgency is normal and understandable. But for cell and gene therapies (CGTs), some of the decisions made earliest in development can become the hardest to change later. Things like cryopreservation strategy, shipping lane selection, packaging qualification, site readiness, documentation, and cross-functional ownership may seem like small, operational details at first, but in practice they can shape everything that comes later. Regulatory readiness, scalability, comparability, cost, commercial execution… all of these variables can get defined (or influenced) from decisions made in the earliest stages.
This was the central theme of the recent webinar, “Prevent Advanced Therapy Program Delays with Early Supply Chain Strategy.” The expert panel explored why early supply chain decisions deserve strategic attention long before a program reaches pivotal trials or filing preparation.
The discussion made one point especially clear. Regulatory readiness isn’t something that gets assembled at the end, it’s not a late-stage milestone. It’s built over time through the decisions made along the way, the rationale captured, partners engaged, and the risks tested as the program evolved.
Regulatory readiness starts before the filing
For many early-stage organizations, regulatory strategy becomes more formal as the program approaches IND. The webinar panel challenged that timeline, emphasizing that the regulatory story starts much earlier – even when documentation is still informal and processes are still evolving.
Lorraine Hicks captured this well, explaining that documentation is not just an administrative cleanup. “The regulatory readiness,” she pointed out, “it’s not necessarily just about the paperwork. It’s being able to tell your story and present that to the regulators as they come. That’s not something you can build historically.”
That idea is especially relevant for advanced therapy programs, where development can involve multiple sites, specialized handling requirements, cross-border movement, changing processes, and critical starting materials. If teams don’t capture why decisions were made as the program develops, it can become a struggle to reconstruct that logic later.
The issue is not that every early decision must be final. Science evolves. Processes improve. Sites change. Supply chain models grow more complex as programs scale. The point is that teams need to document the path clearly enough to explain how decisions were made and why the strategy remains appropriate.
Regulatory readiness is a habit built into development.
Cryopreservation is a strategic decision, not a late-stage workaround
Cryopreservation often enters the conversation as a logistics decision. If fresh material creates too much timing pressure, freezing may offer a more flexible path.
The discussion made clear that this view is too narrow. Cryopreservation is not just a question of shipment timing, but can affect the biological characteristics of the material, which has implications for compatibility, scalability, and regulatory expectations.
Dominic Clarke explained the issue directly. “Now that material is frozen,” he highlighted, “the characteristics such as viability, the phenotype, the function, those are all aspects now that you need to understand.”
That single point has broad consequences. If a team begins with fresh material and later decides to introduce cryopreservation, it may not be making a simple operational adjustment. It may be introducing a change that requires additional characterization and, depending on the stage of development, comparability work.
Dominic also noted the potential cost and timing impact of waiting too long, stating, “The difference between having [a cryopreservation strategy] already there before you go to the clinic versus having to decide after you’ve been in the clinic to go back and develop a cryopreserved starting material, it’s 10 to 20x in cost and a significant loss in time because you essentially have to start over.”
This does not mean every program must use cryopreservation from the beginning, but it does mean the option should be evaluated early enough that the team understands the tradeoffs. Fresh material may still make sense in certain early, local, or tightly controlled settings. But if the program is expected to expand across more sites or longer distances (including international borders), the assumptions behind a fresh-material model need to be tested before they become embedded in the program. The earlier cryopreservation is evaluated, the more options the program preserves.
Phase-appropriate planning does not mean waiting
One of the more practical themes from the webinar was the need to balance early rigor with phase-appropriate expectations.
Advanced therapy teams do not need to validate everything on day one. Overbuilding too early can waste time and resources. But underplanning can create its own costs, especially when decisions that should have been staged across development are compressed into the months before filing.
Gwendolyn Erskine described a practical, phase-based approach, where in Phase I, teams should begin with shipping risk assessments, while in Phase II they may need updated assessments or shipping lane studies as sites change. And by Phase III, packaging and shipping systems should be moving toward formal qualification and finalization.
“You don’t have to have all your decisions made until phase three,” Gwen pointed out. “Your phase three is when you need to have your, again, to mimic Lorraine, all of your documentation finalized, all your packaging finalized, so that you’re validating and you’re also repeating that multiple times.”
The message is not to rush every decision. It’s to understand what work must happen at each phase so the program is not forced into rushed studies or reactive decision-making downstream. A phase-appropriate strategy gives teams room to adapt while still building toward the evidence package they will eventually need.
Site selection is also a supply chain decision
Site feasibility is often evaluated through the lens of clinical capability, patient access, enrollment potential, and investigator experience. Those factors are essential. But for advanced therapy programs, they’re not enough.
The discussion highlighted that logistics constraints should influence site selection from the beginning. Shipping lanes, airport capabilities, customs requirements, dry ice restrictions, temperature-control requirements, site training, packaging familiarity, and mode of transport can all determine whether a site can realistically support the program.
When asked how early logistics constraints should influence site selection, Lorraine didn’t hesitate. “At the beginning,” she emphasized. “That needs to be a part of the consideration at the very beginning, at early stages of planning, because it matters, right?”
Site-level logistics can influence everything from chain of custody and condition to scheduling, patient experience, product quality, and the ability to scale. A site that looks strong clinically may still introduce risk if its location or handling capabilities are not compatible with the supply chain strategy.
That risk only becomes more visible as programs move beyond early, small-scale trials. A model that works with a few sites in one country, for example, may not hold up as the program expands across regions and regulatory environments.
Cross-functional alignment prevents hidden gaps
Advanced therapy supply chains bring together stakeholders across many different functions. Clinical operations, manufacturing, quality, regulatory, logistics, packaging, commercial teams, CDMOs, clinical sites… all of these roles have a role to play in whether the end-to-end supply chain for the program works under real-world constraints.
The webinar discussion repeatedly returned to the risk of siloed planning. Where a decision made for early clinical convenience could create later issues for commercial launch, or a packaging approach may not align with regulatory expectations. Or where a site may not be prepared to receive or handle a specific shipping system, or a logistics plan fails to account for customs or airport constraints.
Gwen emphasized that the right stakeholders need to be involved early, asking listeners, “Do you have the right decision-makers in the room to make sure you’re following that journey in a timely and effective way?”
Lorraine expanded on that point, reminding listeners, “Within your organization, you need that cross-functional alignment, but you also need it with your partners, your logistics service providers.”
That external alignment is easy to underestimate. Advanced therapy programs depend on a chain of specialized partners, and the evidence package is only as strong as the visibility across that chain. When information remains fragmented, teams may not see gaps until they are already under pressure.
The cost of waiting is rarely just financial
Late-stage rework can be expensive. But the panel made it clear that the bigger problem is often the combination of cost, delay, operational disruption, and regulatory uncertainty.
Gwen shared an example of a company that delayed full shipping qualification work, then faced additional requests for data. The result was more studies, a much bigger budget impact, and a delay of nearly six months. “Time can be your best friend or your enemy, so use it wisely,” she cautioned.
That line could apply to almost every supply chain decision discussed. Teams may feel that deferring a decision preserves speed. And sometimes it does. But if a deferred decision later requires new studies, repeated qualifications, comparability assessments, site retraining, partner realignment (or reselection), or filing support, the time saved early can disappear quickly.
“The corners that you’ve cut now come back to haunt you,” Dominic pointed out, “and you’re just simply not prepared to move forward.”
Build the evidence package before pressure builds
Advanced therapy developers don’t need a commercial-ready supply chain on the first day of development. But they do need a strategy that can mature with the program.
That strategy should account for the realities of the product, the patient population, the clinical footprint, the manufacturing model, the regulatory pathway, and the intended commercial future. It should be documented as it evolves. It should involve the right internal and external stakeholders, and it should test assumptions before those assumptions become embedded in the program.
Supply chain strategy involves how materials move, sure. But it also needs to build the evidence and flexibility that gives teams (plus investors and regulators) confidence in moving an advanced therapy program forward.
For teams working toward clinical and commercial success, the best time to address these questions is before they become urgent.
Watch the on-demand webinar, “Prevent Advanced Therapy Program Delays with Early Supply Chain Strategy,” to hear the full expert discussion.
