Major progress made in cell and gene therapies at UCL

The Success: From Lab to Market 2026 report, presented at the UCL Advanced Therapies Symposium on 22 April, outlines a portfolio of more than 50 discovery projects, 29 preclinical and IND-enabling programmes preparing therapies for first-in-human clinical trials, and 30 Phase I/II clinical trials. This work is supported by over £70 million in public funding alongside private investment. In this last year alone, one therapy - a CAR-T immonutherapy for Acute Lymphoblastic Leukaemia - got regulatory approval and is now available to NHS patients while four therapies have progressed from the preclinical stage into Phase I/II clinical trials in the past year. 

UCL’s clinical reach is unmatched among UK academic institutions. Partner hospitals host 44% of the UK’s academically sponsored and non-profit ATMP trials and more than 50% of commercially sponsored Advanced Therapy Medicinal Products (ATMP) trials, based on data from the Cell and Gene Therapy Catapult ATMP Clinical Trials Database 2025. 

This strength is underpinned by UCL’s close integration with leading hospitals, research institutes and innovation partners in London and internationally, alongside sustained investment in in-house manufacturing capability. The report also highlights how UCL researchers are advancing next-generation enabling technologies to address complex ATMP scientific and manufacturing challenges, including gene editing, exon skipping, cell therapies, lipid nanoparticles for non-viral delivery, and viral vector optimisation. 

Professor Emma Morris, Dean of UCL’s Faculty of Medical Sciences and Professor of Clinical Cell and Gene Therapy, said: “What sets UCL apart in ATMP development is the ecosystem we have built over many years. Alongside world-class researchers and clinical expertise at our partner hospitals, we have invested in Good Manufacturing Practice (GMP) manufacturing facilities and work closely with commercial partners across the pipeline to accelerate patient access.” 

Several of UCL’s advanced therapies have reached notable milestones in the last 12 months:  

• The first NHS patient treated for Advanced Lymphoblastic Leukaemia with a revolutionary advance in CAR T-cell immunotherapy known as obe-cel. Developed by researchers at the UCL Cancer Institute and by UCL spinout Autolus, this personalised ‘living medicine’ has much improved efficacy and lower side effects than previous CAR-T therapies and was approved by NICE for use on NHS in late 2025. 

• A gene therapy for Huntington’s disease, AMT-130, led by researchers at the UCL Queen Square Institute of Neurology, has demonstrated a 75 per cent reduction in disease progression. 

• Life-changing improvements in vision for several young children following treatment with a gene therapy for rare inherited childhood sight loss.  The therapy was developed by UCL Institute of Ophthalmology and Moorfields Eye Hospital and progressed to the clinic by UCL spinout company  MeiraGTx. 

• A world-first antisense oligonucleotide therapy targeting the most common genetic cause of motor neurone disease entered Phase I clinical trials. It has been developed by researchers at UCL Queen Square Institute of Neurology and UCL spinout Trace Neuroscience, backed by $101 million investment, one of the largest biopharma Series A rounds in recent years. 

•  A gene therapy for p47 chronic granulomatous disease, developed at the UCL Great Ormond Street Institute of Child Health, was the first therapy to be researched, manufactured and delivered to patients entirely within a single centre at the Zayed Centre for Research into Rare Diseases in Children. The first patient described the treatment as having ‘completely changed my life’. 

• The first child received a world-first gene therapy for Hunter syndrome (MPS II), developed at the Zayed Centre, in February 2025. The patient’s family reported marked developmental improvements nine months later. 

More than 250 scientists, clinicians, funders and industry leaders attended the symposium to discuss advances across the pipeline, including CAR T therapies for autoimmune conditions, rare disease gene therapies, cell therapies, and approaches to accelerating commercialisation from lab to market. 

Professor Geraint Rees, UCL Vice-Provost for Research, Innovation and Global Engagement, said: “UCL's ATMP pipeline is one expression of a broader strategic ambition to ensure that UCL's research translates swiftly from lab to market, delivering innovations that benefit citizens in the UK and worldwide. The scale of this pipeline, combined with deep partnerships across London’s life sciences ecosystem, demonstrates UCL’s role in anchoring the UK’s innovation landscape.” 

Dr Anne Lane, Chief Executive Officer of UCL Business, said: “Working side by side with UCL’s academics, clinicians and innovators we turn exceptional research into successful products, spinouts and technologies that change the world. 2025 was truly a turning point in our work to bring advanced cell and gene therapies from the lab to the clinic and the success stories we have seen this year show the effectiveness of the close collaboration between UCL Business, the Translational Research Office, UCL researchers, partner hospitals and funding and investment partners.” 

About the report 

Success: From Lab to Market 2026 is jointly published by the UCL Translational Research Office and UCL Business Ltd. 

For information on translational research and industry-academic collaboration, contact Dr Jane Kinghorn, Director, UCL Translational Research Office: j.kinghorn@ucl.ac.uk 

For commercialisation enquiries, contact UCL Business Ltd: r.fagan@uclb.com 

To receive news and event invitations on UCL’s therapeutic innovations, join the UCL Therapeutic Innovation Networks (TINs): tins@ucl.ac.uk 

Links

• UCL Translational Research Office
• UCL Innovation and Entreprise
• UCL Business
• Professor Emma Morris's academic profile
• Professor Geraint Rees's academic profile
• Dr Anne Lane's profile

Image

• Credit: James Tye