Unlocking the UK’s advanced therapy pipeline

The UK’s leadership in ATMP clinical trials is built on a world-class academic base and discovery research capability. There are world-leading universities and research institutes in the UK that generate foundational science in ATMPs, while national clinical research networks, specialist manufacturing facilities and coordinated regulatory efforts have pushed breakthrough science into first-in-human studies at pace. There is a strong appetite from the UK government to maintain a position as a global leader, for instance, having declared its ambition to grow the number of domestic commercial clinical trials. This recognises the importance of these trials to both patient outcomes and economic growth.

The UK is building on this foundation with recent reforms like the introduction of accelerated pathways for rare disease therapies. This is helping to create a more agile environment for therapy developers through compressing trial design and making development and approval timelines flexible for diseases with small patient populations. Reforms like these are proving to be effective; in 2025, clinical trial applications increased by 9% compared to 2024. With 21 new ATMP clinical trial applications received in 2025, this highlights the clear impact of regulatory reform.

The UK’s ATMP ecosystem doesn’t run on academic research and commercial investment alone; continued government support, such as early-stage grants and translational accelerators, is essential to ensure promising discoveries can enter the development funnel. The ecosystem is further supported by a healthy infrastructure of support networks. The ATTC network, funded by NIHR, supports ATMP-specific clinical trial acceleration, site readiness and workforce capability through its coordination of national centres operating within the NHS framework and the CGT Catapult offers support across the whole life cycle of advanced therapy development and commercialisation.

CGT Catapult worked with an early-stage European biotechnology company developing cancer immunotherapy products using induced pluripotent stem cell (iPSC) technology. CGT Catapult designed a non-clinical strategy to guide the company in generating the evidence needed to progress its experimental therapy through to first-in-human trials, as well as regulatory advice. This project exemplifies the support it provides to companies looking to move into clinical trials.

The 2025 Clinical Trial Database published by the CGT Catapult confirms the UK’s early-stage trial pathway remains strong, with growth in phase 1 and 2 trials reflecting sustained innovation. Moreover, 80% of trials in 2025 were commercially sponsored and they have been for many recent years, far higher than conventional pharmaceutical research, a clear signal of the sector’s strength. However, the real vulnerability is the gap between demonstrating early efficacy and getting a therapy into routine NHS use. ATMPs require bespoke manufacturing approaches, HTA approval, highly specialised clinical delivery and long-term follow-up with patients. A lack of targeted support at this transition point can cause promising therapies to stall.

Bridging this translation gap will require translational scientific expertise to be coupled with stronger infrastructure for clinical trials. The ATTC network is contributing to this effort. For example, the network conducted a comprehensive review of complex clinical trials that included a detailed national survey and in-depth interviews with a wide range of healthcare and industry stakeholders. The findings provided a clear picture of the main barriers and highlighted the most effective practices. These recommendations are now being implemented across the network, with the aim to reduce trial start-up times and improve the experience for patients who enter future studies.

Coordinated projects like this show how government-led initiatives can build the ecosystem to improve trial efficiency and ensure the NHS is equipped with the skills needed to deliver these therapies.

Clinical trials face several challenges. NHS workforce skills must keep pace with increasingly complex therapies. Identifying patients with rare conditions relies on genomic screening and better data integration, both of which need further development. Geographic inequity persists because specialist centres are concentrated in major urban hospitals and coordination across the devolved nations remains inconsistent. The NHS also needs to embed real-world data collection into routine practice. These issues are difficult to resolve but reflect a system that is continuing to mature rather than one that is fundamentally weak.

A further challenge comes from ever-increasing international competition, particularly from the US and China, where large-scale investment and fast commercialisation pathways are accelerating growth in both markets.

Efforts from regulators aim to ensure the UK remains attractive in light of these challenges, an example being the MHRA’s improved 30-day start-up timelines and high volume of trial approvals, but regulation alone cannot unlock maximum growth. It will be essential for the UK to build a strong commercialisation ecosystem and attract inward investment if it is to remain a global leader.

For ATMP clinical trials, oncology remains dominant, accounting for roughly 35% of trials. But diversification is accelerating. Metabolic conditions represent around 12% of trials, haematological diseases represent 10% and the number of trials for neuromuscular, cardiovascular and neurological disorders is increasing. The expansion of CAR-Ts into autoimmune conditions is a clear example of this growth. As therapies move beyond oncology, NHS infrastructure will face additional pressures to deliver these therapies at scale as the technology demands new delivery models, specialist skills and long-term monitoring systems.

The MHRA has launched a consultation to expand real-world evidence in regulatory decision-making, enabling supplementary data collection alongside traditional clinical trial evaluations. This aligns with broader government ambitions to position the UK as a leading life sciences economy. There is also ongoing discussion around re-evaluating health technology appraisal frameworks of single-administration therapies, recognising their distinct cost and value profiles.

Macroeconomic benefits of these therapies are also starting to be quantified by experts in the field. A recent report by the Office of Health Economics and the CGT Catapult estimated that cell and gene therapies could bring billions to the UK economy by easing the financial burden on patients and caregivers, reducing net NHS and social care costs and increasing productivity and labour market participation. For Alzheimer’s disease alone, the analysis projected benefits of £39.6bn for individuals and their carers, resulting from extra years in work and reduced levels of care, £19.9bn in NHS savings and £21.5bn added to the UK economy.

Digitalisation will play a central role in supporting ATMP production as clinical trials advance and will be essential for maintaining future competitiveness. It will enable more precise tailoring of treatments to individual patient needs and strengthen the use of NHS data to track long-term product performance. To support this shift, CGT Catapult is working with the Data and Healthcare Innovation Centre in Scotland to standardise data collection, which will provide the foundations needed for future data-driven capabilities and the responsible use of artificial intelligence in healthcare innovation.

The UK holds a strong position in ATMP clinical research. A robust and diversified early-stage research pipeline, powered by SMEs and spin-outs from renowned academic institutions, coupled with government support to increase commercial trials and an evolving regulatory environment, provides a solid foundation. However, long-term success depends on sustained support as therapies progress towards commercialisation. Coordinated NHS adoption pathways, streamlined patient identification and funding across all stages of the innovation pathway will be critical.

Continued collaboration across government, regulators, NHS and industry will enable the UK to convert its research excellence into tangible clinical and economic benefits. What needs to be addressed is the translation gap, to ensure that potentially life-changing therapies reach the patients that need them the most.