UCL showcases its leading advanced therapies portfolio

The Success from Lab to Market 2025 document shows that the university, together with its associated hospitals, has a world-leading pipeline of Advanced Therapy Medicinal Products (ATMPs), which includes 34 preclinical ATMP projects and a further 34 in Phase I/II clinical trials.

Its publication coincides with the Advanced Therapies Symposium, held at the Institute of Child’s Health (ICH) at which over 250 scientists, clinicians, funders and industry leaders today (Weds 9th April) at which the latest breakthroughs in advanced medical therapies were discussed.  

The symposium reinforces UCL’s position as a powerhouse of biomedical innovation and underlines the importance of UCL and UCL Ventures as key players in 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 UCL’s position 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.

As one of the world’s leading research-intensive universities with particular expertise in Life Sciences, UCL’s academic research combined with UCL Ventures’s expertise in commercialising that research, is translating into therapies which are bringing real-world benefits to patients:

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 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 Ventures Ltd, who chaired the symposium’s panel on innovation and commercialisation, added: “The scale and success of UCL’s ATMP pipeline demonstrate 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.”

Read our Success from Lab to Market 2025 brochure here.