UCL cemented as global leader in developing and trialling advanced medicinal therapies

The Success from Lab to Market 2025 report shows that the university, together with its associated hospitals (including UCLH, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital) has a world-leading pipeline of Advanced Therapy Medicinal Products (ATMPs) – medicines using human genes, cells or tissues to treat diseases and disorders – which includes 34 preclinical ATMP projects and a further 34 in Phase I/II clinical trials. 

It coincides with the Advanced Therapies Symposium, hosted by UCL, at which over 250 scientists, clinicians, funders and industry leaders gathered on the 9^th April to discuss the latest breakthroughs in advanced medical therapies.

The symposium reinforced UCL’s position as a powerhouse of biomedical innovation and a key player in the capital’s growth strategy, driving collaborations that accelerate healthcare impact and shaping the future of life sciences.

Researchers at the event presented groundbreaking work on cell and gene therapies targeting conditions such as dementia, Motor Neurone Disease, retinal diseases, diabetic retinopathy, Alzheimer’s disease, choroideremia, thalassaemia, and autoimmune disorders. 

Professor Geraint Rees, Vice-Provost (Research, Innovation & Global Engagement), said: “UCL is committed to driving innovation that transforms lives, not just in the UK but globally. Our partnerships with industry, funders, hospitals and clinical colleagues ensure that breakthrough treatments move swiftly from the lab to patients. We are proud to be at the heart of the UK’s thriving life sciences ecosystem.” 

Success from Lab to Market 2025 also highlights that UCL is at the forefront of ATMP clinical trials, with close to half (49%) of the UK’s academically sponsored ATMP clinical trials and over 40% of the UK’s commercially sponsored ATMP trials conducted by UCL and its partners. 

It underlines the importance of UCL’s multidisciplinary expertise and innovation, with a focus on rare diseases, ophthalmology, haematology/oncology, neurology and immunology.

Key achievements include: 

• 120+ projects in development, spanning all stages from discovery to market launch 

• Over £66 million in public funding invested in Phase I/II trials and preclinical projects 

• 14 spinouts and licensees commercialising UCL’s ATMP innovations, contributing to UCL’s track record of raising over £3bn in external investment through the commercialisation office of UCL, UCL Business Ltd (UCLB) 

• Two treatments already approved for use in patients in various countries (including the U.S) - Roctavian™ for haemophilia A and AUCATZYL® for an aggressive blood cancer.

UCL is also a key player in the newly established London Advanced Therapy Treatment Centre (ATTC), supported by the National institute for Health and Care Research (NIHR) and Innovate UK, which aims to accelerate cell and gene therapy clinical trials and expand patient access to cutting-edge treatments. 

As one of the world’s leading research-intensive universities with particular expertise in Life Sciences, UCL’s academic research is already translating into therapies which are bringing real-world benefits to patients. These include next-generation technologies such as gene therapies and immunotherapies providing hope in areas such as cancers, neurodegenerative diseases and inherited conditions, such as: 

• The first ever ‘one shot and done’ gene therapy for adults with severe haemophilia A, which was developed at UCL and licensed to BioMarin Pharmaceuticals. This revolutionary treatment is now approved for use in patients in the U.S and EU under the drug name Roctavian 

•  A gene therapy for inherited childhood sight loss which has helped young children gain life-changing improvements in sight through a novel gene therapy treatment at UCL’s Institute of Ophthalmology and Moorfields Eye Hospital. This therapy is being developed and trialled by UCL spinout MeiraGTx 

• A breakthrough therapy for treatment of the most common form of Motor Neurone Disease (MND). This treatment has been developed by UCL spinout Trace Neuroscience, co-founded by UCL researchers, which is backed by $101 million (£85 million) in Series A funding. The therapy has recently entered phase 1 clinical trials 

• Autolus’s CAR-T cell therapy, AUCATZYL®, approved for treatment of adults with aggressive recurrent type A Leukaemia, which was developed in collaboration with UCL, and recently secured FDA approval. 

Dr Anne Lane, CEO of UCL Business Ltd, who chaired the symposium’s panel on innovation and commercialisation, added: “The scale and success of UCL’s ATMP pipeline demonstrates our ability to turn pioneering research into commercial opportunities that attract global investment. By fostering spinouts and licensing innovative therapies, we are helping to build a dynamic life sciences sector that benefits both patients and the economy.” 

Links

• UCL ATMP Report 2025 
• UCL Translational Research Office 
• UCL Business  
• The UCL Therapeutic Innovation Networks (TINs) - Cell and Gene Therapy 
• UCL Innovation & Enterprise  

Image

• Credit: James Tye

Media contact

Kate Corry

k.corry@ucl.ac.uk