12 June 2018


The UCL Technology Fund (UCLTF) has approved an investment of up to £1m to support Professor Bobby Gaspar’s development of a gene therapy for (perforin deficient) familial haemophagocytic lymphohistiocytosis (FHL type 2 or ‘FHL-2’) through to a clinical trial in which it could potentially cure patients of a rare but deadly inherited disease.

FHL-2 is a genetic disorder in which key immune effector cells (T-lymphocytes) are unable to kill their targets (usually pathogens such as bacteria or fungi, or cancer cells). As a result, the disease is severely life-limiting if untreated, and the only existing curative therapy – bone marrow transplant – is associated with significant complications especially if no suitable donor is available.

A number of genetic diseases resulting from mutations in single genes have been successfully treated in recent years using ex vivo gene therapy based on a lentiviral vector. Using a similar approach, Professor Gaspar and colleagues at the UCL Great Ormond Street Institute for Child Health have developed a potentially curative gene therapy for FHL-2 and have demonstrated in a number of preclinical experiments that their approach has the potential to correct this devastating disease. The preclinical work of Professor Gaspar and colleagues on FHL-2 has been supported by the NIHR Great Ormond Street Hospital Biomedical Research Centre.

The funding provided by the UCLTF is intended to support completion of the final preclinical steps (biodistribution and toxicology studies) leading to a first-in-man, proof-of-concept clinical trial at Great Ormond Street Hospital, with the potential to directly save the lives of children with FHL-2.

Support for this project is enabled by UCLTF’s innovative investment model. In addition to providing funding and commercial support for traditional university spinout companies, UCLTF also provides direct investment for translational projects which have significant commercial potential, by which funding is provided directly to academic labs such as Prof Gaspar’s team. This approach enables university research groups to de-risk technological development of their projects, carrying them through to a much later stage.

Editors Notes:

UCL Technology Fund

The UCL Technology Fund is dedicated to investing in intellectual property commercialisation opportunities arising from UCL’s world-class research base, focusing in particular on the physical and life sciences. The Fund supports UCL in achieving the full potential of innovations that have prospects for outstanding societal and market impact, right through the development journey from initial proof of concept to practical commercial application.

The Fund is managed by Albion Capital, one of the largest independent venture capital investors in the UK, in collaboration with UCL Business PLC. For further information please visit: www.ucltf.co.uk

About the UCL Great Ormond Street Institute of Child Health

The UCL Great Ormond Street Institute of Child Health (ICH), together with its clinical partner Great Ormond Street Hospital for Children (GOSH), forms the largest concentration of children’s health research in Europe. The inspirational mission of the UCL Great Ormond Street Institute of Child Health is to: “improve the health and well-being of children, and the adults they will become, through world-class research, education and public engagement”. For further information, please visit www.ucl.ac.uk/ich

About UCL Business PLC

UCL Business PLC (UCLB), part of UCL Innovation and Enterprise, is a leading technology commercialisation company that supports and commercialises research and innovations arising from UCL, one of the UK’s top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL’s associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market. For further information, please visit: www.uclb.com | Twitter @UCL_Business

About Albion Capital Group LLP

The wider Albion group has just under £1 billion funds under investment management or administration and is a long term investor in UK businesses, from start-ups through to FTSE 100 companies.  In addition to six Albion VCTs, it manages the UCL Technology Fund and provides management services to Albion Community Power PLC and Albion Care Communities.

Funds managed by Albion invest between £250,000 and £12 million into high growth businesses.

For further information please visit www.albion.capital

About the National Institute for Health Research

The National Institute for Health Research (NIHR): improving the health and wealth of the nation through research.

Established by the Department of Health, the NIHR:

  • funds high quality research to improve health
  • trains and supports health researchers
  • provides world-class research facilities
  • works with the life sciences industry and charities to benefit all
  • involves patients and the public at every step

For further information, visit the NIHR website (www.nihr.ac.uk).