How can we expedite patient access to life-changing advanced therapies? This was the central question driving Advanced Therapies Integrates 2024, recently held at GSK’s state-of-the-art R&D hub in Stevenage. Bringing a vibrant atmosphere of collaboration and innovation, the event brought together a passionate community of experts – from industry leaders and academic researchers to biotech pioneers and patient advocates.
This year’s event served as a platform to address the challenges and opportunities surrounding Advanced Therapy Medicinal Products (ATMPs), including cell and gene therapies, regenerative medicines and tissue engineering. With the overarching goal of translating cutting-edge research into transformative therapies, we explored key issues such as regulatory and commercial barriers, workforce development and patient-centric approaches to R&D. See agenda page here.
We were fortunate to hear from a fantastic array of speakers and panellists, who shared valuable insights and diverse perspectives from across the advanced therapies R&D pipeline and beyond. Adding to the event’s collaborative spirit, the GSK Stevenage venue served as the perfect backdrop. Home to leading players in the cell and gene therapy space, the thriving science R&D hub exemplifies the power of partnerships in accelerating advanced therapies.
Opening up the day’s discussions with an optimistic outlook, Matthew Durdy, CEO, Cell & Gene Therapy Catapult welcomed attendees to our 2024 event with a stimulating address that emphasised now as being pivotal moment for the advanced therapies sector. He likened the industry’s progress to the evolution of mobile technology, suggesting that while cell and gene therapies may still feel like the “clunky phones” of their early days, they hold immense potential for transformative impact.
Matthew outlined four critical conditions to drive growth in the sector:
“Cell and gene therapy is at an inflection point—it’s time to turn potential into universal, accessible solutions that patients can depend on.”
Following his welcome address, Matthew Durdy was joined by Nicole Mather, Health Data, AI, and Life Sciences Lead at IBM, for the first of the day’s fireside chats. These focused, conversational sessions pair industry leaders to explore pressing challenges, share real-world insights, and highlight opportunities in the advanced therapies sector.
Titled “How do we use IT to better serve patients in the advanced therapies sector?”, the first fireside chat of the day explored how digital technologies can transform advanced therapies by improving scalability, efficiency and patient outcomes.
Nicole Mather shared insights into IBM’s contributions, including the development of AI tools to streamline regulatory submissions and predictive analytics designed to optimise manufacturing. She highlighted the value of digital twins in accelerating research and manufacturing processes, citing their ability to simulate and refine therapies before clinical trials. The speakers stressed the importance of integrating technology into existing workflows to ensure practical and efficient adoption, and looked into the exciting potential of quantum computing to revolutionise therapeutic development with advanced modelling and data analysis.
“We now have a real opportunity to scale up manufacturing and reduce costs—these therapies need to reach millions in the next few years, and technology is the key to making that possible.”
The second fireside chat was titled “For the people who need them”. In this deeply moving and inspiring session, James Kelly, Chair of the MEF2C Foundation, shared his journey as a father navigating life with a child diagnosed with MEF2C haploinsufficiency syndrome—a rare and profound neurodevelopmental disorder. For James’ son, daily life involves complete dependence on caregivers, including specialised schooling and a complex regimen of medical and therapeutic interventions.
Joined by Nicola Redfern, an experienced consultant in cell and gene therapy, the discussion highlighted the critical role of patient-led strategies in therapeutic development. James shared how he and his wife, both engineers, transformed their heartbreak into action. By founding the MEF2C Foundation, they connected with researchers globally, secured funding, and launched therapeutic development programmes. Their astonishing achievements include advancing ASO-based therapies that have doubled MEF2C expression in preclinical models and pioneering natural history studies to better understand the disease trajectory.
Thanks to advancements in cell and gene therapies, there is hope for those affected by rare genetic disorders like MEF2C. The speakers emphasised the transformative potential of advanced therapies in rare diseases, and the importance of integrating patient perspectives early in therapeutic development, advocating for greater collaboration between researchers, clinicians and families. The audience were also urged to “lean into rare,” highlighting the innovation potential of rare disease research and its ripple effects on more common conditions.
“There’s nobody more motivated than a parent of someone with a rare disease—engaging them and leaning into their lived experience is essential.”
Following the morning’s energising fireside chats, delegates and speakers broke into broader panel discussions, tackling the key challenges and opportunities shaping the advanced therapies landscape. These larger discussions provided the opportunity to hear from a diverse array of voices and perspectives across the field.
“Make them better” tackled the industry’s pressing need to scale up the production of advanced therapies to serve growing patient populations. Panellists identified critical challenges in scaling cell and gene therapy manufacturing, including the lack of standardisation, inefficiencies in clean room operations, and the limited use of automation. They emphasised that while emerging technologies hold transformative potential, there is still significant “low-hanging fruit” in optimising existing processes and infrastructure.
A central theme was the urgent need to reduce costs and improve efficiency by leveraging data integration and digital solutions. Speakers highlighted the necessity of building a robust digital ecosystem, and called for cross-sector collaboration and learning from industries like automotive and biologics to unlock greater scalability.
“We need to get to a point where our data flows faster than our physical product, so the rate-limiting step is no longer quality control but getting therapies to patients faster.”
“Investing in our health” explored strategies for driving investment in ATMP programmes to ensure their development and accessibility for patients in need. The engaging panellists covered the unique challenges of advanced therapies, including high manufacturing costs, regulatory complexities, and difficulties securing investment for rare disease therapies.
Speakers agreed on the necessity of treating ATMPs differently from traditional innovations, with calls for streamlined regulatory pathways and greater collaboration between regulators, health technology assessors, and payers. The UK’s potential to lead in this space was highlighted, with its strong academic base and flexible regulatory environment. Notwithstanding, it was clear that hurdles remain in scaling workforce capacity and securing reimbursement pathways.
A central theme was the need for cross-sector cooperation to demonstrate the broader economic and societal value of ATMPs, encouraging policy makers and private investors to view them as transformative opportunities rather than financial risks.
“Bringing therapies to patients is the only way to create real value in this space. Every step that delays or inflates costs is a step away from delivering life-changing treatments.”
“Funding battles” explored the challenges and opportunities in securing funding for advanced therapies amid a tightening investment climate. The panellists discussed the evolving expectations of investors, stressing the importance of differentiation, clinical validation, and robust commercialisation strategies. Advanced therapies face the unique hurdle of proving their necessity over more traditional approaches, making a compelling case for why these innovative solutions are indispensable.
A key theme was the growing importance of partnerships with pharmaceutical companies and non-traditional funding models, such as equity-sharing agreements with CDMOs. Panellists also noted the untapped potential of public and university funds, which could play a vital role in bridging early-stage financing gaps. However, despite these avenues, the consensus remained that proving clear patient benefit and cost-effectiveness is critical to attracting investment.
“Differentiation is everything—investors need to see not just innovation, but clear proof that your therapy can deliver what nothing else can.”
After a bustling networking lunch, we were treated to another fireside chat and further panel discussions. The third and final fireside chat of the day, “From lab to market” featured Catriona Crombie, Head of Rare Disease Research at LifeArc, and Robert Panting, General Manager of Rentschler Biopharma’s Advanced Therapy Production unit. Together, they shared insights on the complex journey of translating scientific breakthroughs into market-ready therapies, particularly for rare diseases.
Catriona Crombie brought her deep expertise in rare disease research, highlighting the difficulty of creating a sustainable value proposition when working with small patient populations, where high costs and limited scalability often deter investment. Catriona advocated for innovation in manufacturing processes and infrastructure to reduce costs and accelerate time to market, stressing the need for creative, resilient approaches to overcome these hurdles.
Robert Panting highlighted the practical challenges of manufacturing scale-up. He discussed the potential for modular, platform-based manufacturing systems while acknowledging the limitations and nuances of bespoke therapies. Both speakers voiced the need for collaboration between biotech companies, regulators, and manufacturers to develop innovative, efficient solutions that balance speed, cost and quality.
“If we want to get therapies to rare disease patients, we must push boundaries at every step—reducing costs, streamlining processes, and delivering drugs at a price the market can bear.”
Moving seamlessly into the next panel discussions, we next delved into the world of scientific hubs. The lively panel discussion, aptly named “hub-le bubble” examined the role of pharma-biotech hubs in accelerating advanced therapy innovation. Panellists explored how hubs act as collaborative ecosystems, offering critical mass, shared resources, and connectivity with investors, advisors, and regulators. They emphasised the importance of creating environments that support not just research but also commercialisation and talent retention.
Key themes included addressing the UK’s fragmented approach to biotech development. While the “Golden Triangle” of London, Oxford, and Cambridge remains a focal point, there is growing recognition of the need to adopt a nationwide strategy, leveraging regional strengths through resources like Catapult centres and academia-industry networks. The panel also stressed the role of hubs in nurturing early-stage companies by offering access to top-tier facilities, commercialisation support and soft skills training.
Collaboration was highlighted as essential, both within hubs and across regions, to ensure knowledge-sharing and innovation. The discussion also touched on funding challenges and the value of hubs in connecting SMEs with the expertise and mentorship they need to thrive.
“Hubs are more than facilities—they’re ecosystems that build trust, foster collaboration, and connect people with the expertise and resources needed to turn great science into great products.”
Marco Delise, Stevenage Bioscience Catalyst
The final panel discussion of the day, “It’s an upskill battle” was a spirited and eye-opening discussion that explored the growing skills gap in our industry, and how we can bridge it through innovation, collaboration, and education. Panellists urged the need for both technical and soft skills, such as project management, communication and networking, to navigate the complexities of advanced therapy development.
The conversation highlighted the critical role of academic-industry partnerships and in tailoring education programmes, like those offered by Cell and Gene Therapy Catapult, to meet real-world demands. A key theme was the importance of starting early by introducing STEM careers to younger students and providing hands-on opportunities through apprenticeships, industrial placements, and taster sessions.
Panellists called for greater focus on instilling a healthy feedback culture and continuous upskilling across all levels, from junior staff to senior management, to foster adaptability in a rapidly evolving industry. The session underscored the necessity of collaboration between academia, industry and government to address systemic challenges and build a workforce equipped for the future of advanced therapies.
“We need to start early, engage students at every level, and invest in upskilling to ensure our workforce is equipped to meet the demands of our fast-growing industry.”
As the final discussions drew to a close, Advanced Therapies Integrates 2024 left an undeniable impression and a heartening sense of a shared purpose and forward momentum. This year’s gathering wasn’t simply about identifying challenges; it was about celebrating how far we’ve come and reimagining what’s possible in advanced therapies.
The day’s discussions reflected a sector on the brink of transformation. Whether it was the game-changing potential of digital tools, the power of patient-led innovation, or the urgency of addressing workforce skills and funding gaps, one message rang loud and clear: this is the time for action. Speakers and delegates alike brought bold ideas and pragmatic solutions, leaving attendees with not just hope but a clear sense of direction.
Lively networking sessions punctuated the day, buzzing with the energy of new collaborations and shared ambitions. These moments of connection underscored our primary goal: bringing together visionaries and innovators determined to make life-changing therapies a reality. As we look to the future, we hope that the energy from Advanced Therapies Integrates 2024 will fuel our industry’s mission to ensure these therapies reach the patients who need them most, as quickly as possible.
Thank you for everyone who joined us this year, and a special thanks to our sponsors for helping to make it happen!
