OXB Flash Talks: The Strategic Side of Regulatory Development

Introducing OXB Flash Talks

Michaela Duffy:
Welcome to the first episode of OXB Flash Talks, our series of short discussions focused on key topics in cell and gene therapy development and manufacturing. Each episode brings together OXB experts to share insights and perspectives on the challenges and opportunities facing the industry today.

I’m your host, Michaela Duffy. I’m a Senior Research Associate II at OXB in the Process Development Group, and I’ll be guiding these conversations with colleagues from across the organisation to explore the science, strategy and expertise driving progress in the field.

Joining me for this first episode are Pete Embley and Francesco Lanucara from OXB’s Global Regulatory Team.

Today, we’ll explore how regulatory strategy has evolved and why it plays an increasingly important role in guiding successful cell and gene therapy development programmes, from early-stage development through to commercialisation.

Regulatory expertise within a CDMO

Michaela Duffy:
Let’s start with the big-picture question. What makes OXB’s regulatory team stand out within the CDMO environment?

OXB Global Regulatory Team:
Great question, Michaela.

We’re not simply a team of technical writers producing chemistry, manufacturing and controls, or CMC, documentation. We provide comprehensive, end-to-end regulatory support, from early development through to commercialisation and lifecycle management.

We guide clients through interactions with regulatory agencies, apply learnings from previous programmes and proactively identify regulatory risks and gaps. We have visibility across a considerable portion of the regulatory landscape and have already supported clients through commercialisation.

Because of the number of programmes we support across multiple jurisdictions, we have a wealth of prior knowledge that can inform future decision-making.

I often describe our expertise as the secret ingredient. When companies first engage with a CDMO, they’re usually focused on manufacturing capabilities. They want to know whether the CDMO can produce their product and provide conventional CMC regulatory support for the vector. That is the standard service offering.

However, when a CDMO such as OXB combines manufacturing expertise with strategic regulatory knowledge, particularly within the CMC and viral vector space, it means we’re not simply executing. We’re actively helping to shape the development pathway for our clients.

Integrating regulatory strategy into development programmes

Michaela Duffy:
That makes a lot of sense. Can you explain how the team works with clients to integrate this knowledge into their development programmes?

For someone like me who doesn’t have extensive insight into the regulatory space, can you break down what the team does and why it is so important?

OXB Global Regulatory Team:
Certainly. When clients ask us to be part of their journey from the beginning, we have an opportunity to contribute to collaborative decision-making and help future-proof the strategy throughout development.

Regulatory support for drug development is a lot like using GPS for a long road journey.

First, you need to plan the route. That is your regulatory strategy. Developing a credible regulatory strategy brings clarity to the development programme and can add value from an investor’s perspective.

Next, you have early engagement with regulatory agencies. Interactions such as an FDA INTERACT meeting can help address initial development challenges and support important regulatory designations, including orphan designation.

You then have live traffic updates. This means interpreting evolving guidance from regulatory agencies.

Rerouting represents the ability to adjust plans based on agency feedback, interim clinical data and assessment questions that may arise along the way.

You also have speed limits. Maintaining compliance helps prevent issues such as a clinical hold, which could significantly delay or halt a development programme.

Finally, you have your initial and global destinations. Your first destination may be a marketing authorisation or Biologics License Application, followed by a smooth commercial launch.

In cell and gene therapy, developers are working with highly valuable and complex assets. However, the pathway to approval is high-risk, highly regulated and constantly evolving.

Without strong regulatory guidance, it can be easy to miss critical checks, misinterpret requirements or encounter costly delays.

Regulatory teams act as strategic partners by planning the route, helping to ensure compliance, interpreting real-time feedback and making course corrections when needed. They also maintain close alignment with agencies such as the FDA, EMA and MHRA.

Ultimately, the role of regulatory strategy is not simply to support compliance. It is to de-risk development, accelerate timelines where possible and maximise the probability of approval.

In that sense, regulatory support is not a function on the side. It is central to the success of the entire programme.

The importance of early regulatory planning

Michaela Duffy:
I love that analogy. The point about de-risking development really stood out to me.

It sounds as though your team becomes involved much earlier than many people might expect.

OXB Global Regulatory Team:
Absolutely. Our involvement can begin as early as the translational stage, when a product is moving from the bench into development.

At that point, we can help define the blueprint for the therapy, which may take the form of a Target Product Profile, or TPP, and a Quality Target Product Profile, or QTPP.

We focus on key parameters such as the indication, delivery mechanism, safety profile associated with the modality and product class, and whether the product may qualify for an accelerated pathway or regulatory designation.

From there, we build an early regulatory strategy that maps the journey from preclinical development through to first-in-human clinical trials.

That early strategy is incredibly valuable, not only scientifically but commercially. It gives companies a clear roadmap, which can be important when attracting investment.

Investors want to see a credible plan with defined milestones. That is exactly what a strong regulatory strategy can help deliver.

Regulatory strategy and investor confidence

Michaela Duffy:
That’s interesting. I hadn’t considered it from an investment perspective.

It sounds as though the regulatory strategy can play an important role in securing funding and building investor confidence.

OXB Global Regulatory Team:
Yes. A well-defined regulatory pathway signals that a programme is viable and has been thoroughly considered.

It reduces uncertainty, which is critical to investment decisions concerning a therapy or product.

As the programme progresses, we continue to provide support. This may include preparing CMC sections, facilitating cross-functional input and coordinating interactions with regulatory agencies.

Regulatory challenges in rare and ultra-rare diseases

Michaela Duffy:
I’d like to switch topics and talk about complexity.

Gene therapies are often developed for rare or ultra-rare diseases. Does that create unique challenges from a regulatory or strategic perspective?

OXB Global Regulatory Team:
Absolutely. The ultra-rare disease space is particularly challenging because developers are often working with very small patient populations. This affects clinical development as well as regulatory and CMC expectations.

In an ultra-rare indication, a single manufacturing batch may sometimes be sufficient to supply the entire clinical demand. The CMC strategy must therefore be adjusted accordingly.

Flexibility is essential in these situations. Regulatory agencies can be open to discussions, particularly when sponsors take a clear risk-benefit approach.

For example, alternative strategies may be considered for process performance qualification, or PPQ. This could involve reducing the number of PPQ batches from the conventional three, provided the proposed strategy has been discussed and agreed with the relevant agency in advance.

On the clinical side, finding eligible patients can be like searching for a needle in a haystack, particularly for orphan indications.

Clinical trials may need to operate globally, with sites across multiple regions, to recruit sufficient patients. The regulatory strategy must account for that complexity by coordinating clinical trial submissions across jurisdictions and aligning the global CMC strategy.

Developers must also ensure that the investigational product is available when needed and that it meets the applicable requirements of the different regulatory agencies.

Using platform knowledge to accelerate development

Michaela Duffy:
Efficiency sounds especially important in these scenarios. How can the regulatory team help clients accelerate development?

OXB Global Regulatory Team:
One of the key ways is through platform knowledge.

We can leverage data and experience from previous programmes to streamline activities such as process development and process characterisation for new programmes.

In that sense, the platform can become a more readily deployable tool for new projects.

Michaela Duffy:
That feels like a significant advantage.

Does your support extend beyond CMC regulatory activities into other areas?

Connecting CMC, clinical and non-clinical strategy

OXB Global Regulatory Team:
Yes, and that is another differentiator.

While we are not clinicians or toxicologists, OXB’s regulatory expertise allows us to facilitate cross-functional discussions and build a coherent regulatory narrative.

We interpret data from different functions and identify potential gaps. If needed, we facilitate scientific advice meetings, frame the right questions for the regulatory agencies and present a clear company position.

The goal is to avoid surprises and help keep our clients’ development programmes on track.

Coordinating global regulatory submissions

Michaela Duffy:
Another distinctive aspect of OXB is its global coordination, which you mentioned earlier. How important is that in your work?

OXB Global Regulatory Team:
Global coordination is essential because we operate across multiple regions, including Europe, the US and the UK.

This coordination enables us to support global development strategies and manage submissions efficiently across different jurisdictions.

For clients pursuing parallel pathways in Europe and the US, this becomes even more critical. It gives them greater flexibility to adapt or pivot a programme from one region to another.

Accelerated pathways, such as the FDA’s Breakthrough Therapy designation or the EMA’s PRIority MEdicines, or PRIME, scheme, can be highly beneficial. However, they can also be demanding in terms of resources.

These pathways require careful planning and sufficient organisational capacity. We help clients assess whether they have the bandwidth to pursue them and consider how they can be managed effectively.

The future of regulatory strategy at OXB

Michaela Duffy:
Looking ahead, how is the regulatory function evolving at OXB?

OXB Global Regulatory Team:
We are working closely with OXB’s innovation team to enhance our platform technologies and optimise our manufacturing processes.

The aim is to make development faster and more cost-effective while maintaining strong regulatory alignment.

We are also expanding into new modalities, including in vivo gene therapy approaches. These bring new challenges, particularly around areas such as safety and gene targeting, but they also create exciting opportunities.

The role of the regulatory team is to help ensure that these innovations are supported by a robust regulatory strategy from the outset.

De-risking cell and gene therapy development

Michaela Duffy:
If you had to summarise the value of OXB’s regulatory team in one sentence, what would it be?

OXB Global Regulatory Team:
Regulatory expertise that accelerates every step of development.

We help de-risk programmes before they begin so that clients can move forward with confidence.

Closing remarks

Michaela Duffy:
Those are powerful messages.

Pete and Francesco, thank you both for sharing your insights.

It is clear that regulatory affairs is no longer simply a supporting function. It is a strategic driver of success in modern drug development.

We hope you’ve enjoyed the first episode of OXB Flash Talks. Join us for future episodes as we continue to discuss the science, strategy and innovation driving progress in cell and gene therapy.