How the Focus of Cell and Gene Clinical Trials in the UK is Shifting

The UK remains a global hub for cell and gene therapy (CGT) innovation, but where is the field heading? What indications are seeing the most investment? How are sponsors adapting their pipelines?

At Lonrú Consulting, we apply data-driven insights to illuminate these questions, helping technology providers and innovators navigate the evolving CGT landscape. Our latest analysis of UK CGT clinical trials (2010–2024) highlights shifting therapeutic priorities, changing sponsor dynamics, and emerging areas of opportunity.

A Recent Decline in CGT Clinical Trials

While the UK has maintained strong CGT activity over the past decade, 2024 saw a decline in new trial initiations. A total of 26 new trials were initiated in 2024, compared to 31 in 2023, 32 in 2022, and 28 in 2021. This decline reflects several industry-wide challenges, including regulatory complexities that extend trial approval timelines, funding constraints that particularly affect early-stage biotech companies, and a strategic shift among sponsors toward refining pipelines post-pandemic.

Although this slowdown is worth monitoring, it may also indicate a shift in the field, with a move toward high-value, well-validated programs rather than a focus on sheer trial volume. Understanding how companies are adapting to this changing landscape is essential for stakeholders in the CGT ecosystem.

Explore Lonrú’s interactive dashboard examples to view UK CGT trial trends for further insights.

A Shifting Sponsor Landscape

The mix of sponsors in UK CGT trials is evolving. Large pharmaceutical companies, such as AbbVie and Novartis, are increasing their presence, signaling long-term investment in CGT. UK-based biotech firms remain active in driving first-in-human trials, though some, like Achilles Therapeutics, have recently pivoted away from clinical-stage programs. Academic institutions such as University College London (UCL) continue to play a critical role in early-stage innovation, particularly in rare diseases and regenerative medicine.

This diverse sponsor base reflects both the increasing commercial interest in CGT and the persistent role of academic centers in developing new approaches. The dynamic between large pharma, biotech startups, and research institutions will be key to shaping the next phase of clinical development.

The Evolution of Indications in CGT Trials

By categorizing CGT clinical trials into broader therapeutic areas, we can see shifts in industry focus.

Oncology remains the dominant area for CGT trials, but the landscape is evolving. Hematologic cancers, including multiple myeloma, leukemia, and lymphoma, continue to drive significant trial activity, largely fueled by CAR-T and other engineered cell therapies. Solid tumors, such as lung cancer, liver cancer, and glioblastoma, remain a major focus, though the pace of new trial initiation has been more gradual. Adoptive cell therapies, including tumor-infiltrating lymphocytes (TILs) and engineered T-cell receptors (TCRs), are emerging as alternatives to traditional CAR-T approaches, offering new avenues for immunotherapy.

Rare and genetic disorders have gained significant momentum in CGT research. Duchenne muscular dystrophy, cystic fibrosis, and spinal muscular atrophy have all seen increased trial activity in recent years. Many of these trials leverage gene therapy and gene editing approaches, reflecting industry confidence in in vivo genetic correction as a viable treatment strategy.

Neurological disorders are an emerging frontier for CGT. While historically underrepresented in CGT trials, conditions such as ALS, Parkinson’s disease, and Huntington’s disease are now receiving increased investment. Challenges remain in delivering therapies effectively across the blood-brain barrier, but clinical interest in gene therapies for neurodegenerative and neuromuscular conditions continues to rise.

Ophthalmology has consistently been a growth area in CGT. Retinal diseases, including retinitis pigmentosa and Leber’s congenital amaurosis, are among the top indications for AAV-based gene therapies. These conditions are well-suited for local gene delivery, reducing systemic risks while improving treatment precision.

Hematological conditions outside of cancer, such as sickle cell disease and hemophilia, continue to attract CGT investment. However, with recent regulatory approvals such as Casgevy for ex vivo gene editing in sickle cell disease, the number of new clinical trials in these areas may moderate over time as companies transition toward commercialization.

Strategic Takeaways for CGT Stakeholders

Understanding these trends is essential for stakeholders across the CGT ecosystem. Enabling technology providers, Biotech and pharmaceutical companies should align their pipeline strategies with the shift beyond oncology into neurology, ophthalmology, and rare diseases, where CGT applications are growing. Technology providers and contract development and manufacturing organizations (CDMOs) must adapt to an evolving landscape where scalable allogeneic cell therapies, in vivo gene editing, and AAV-based delivery are gaining prominence. Investors should look beyond oncology, as emerging CGT applications in neurological and metabolic disorders present new opportunities for value creation.

As the industry moves forward, the ability to navigate these macro trends will be critical for companies developing platforms and enabling technologies. Lonrú Consulting provides real-time data analytics, market intelligence, and commercialization strategies to help companies position themselves effectively in a competitive and evolving field.