UCL Queen Square Institute of Neurology


New Gene therapy translational research partnership between Institutes of Neurology and Child Health

26 September 2022

The Directors of UCL Queen Square Institute of Neurology and Great Ormond Street Institute of Child Health, together with the Deans of the Faculties of Brain Sciences and Population Health Sciences are pleased to announce the formation of a Genetic Therapy Accelerator Centre.

gene therapy

The Centre will be based at Queen Square and directed by Professor Francesco Muntoni, a Paediatric Neurologist who will have a dual appointment in both institutions. This Centre will spearhead activities in this rapidly evolving area and play a major role in translational neuroscience in UCL.

In the last five years a number of extremely effective RNA and adeno-associated viral vectors therapies have dramatically improved the outcomes for individuals affected by a range of neurological disorders, mostly affecting children.

The focus of this new Centre will be to harness the learning derived from these recent successes, from mechanistic insight discovery, to early and late translational research and eventually regulatory approval, and apply it to a wider group of neurodegenerative disorders.

This new Centre will be established at QSION initially, and subsequently at the new £281m Translational Neuroscience building on Grays Inn Road in 2024. It will combine laboratory and clinical expertise focused on neurological, neuromuscular and neurodegenerative disorders, their mechanisms of disease and cutting-edge RNA and AAV gene therapy expertise and foster cross-institutional collaborations between QSION and GOSICH.

The activities of the Centre will be highly complementary with established activities and expertise in both institutes, including in the Zayed Centre, the Dementia Research Institute, the Sigrid Rausing Neurogenetics Therapies Programme and the two NIHR Biomedical Research Centres translational research programmes (UCLH and GOSH).

Professor Michael Hanna, Director of the UCL Queen Square Institute of Neurology said “I am delighted that we will work in partnership and share expertise with colleagues at ICH in order to develop new gene therapies for adults with neurological and neurodegenerative diseases”

professor Muntoni
Professor Francesco Muntoni said “The area of genetic therapies for neurological diseases is rapidly progressing. The possibility of combining expertise across Institutes to develop therapies for new targets is very exciting and I am very pleased to lead this new joint initiative”
Professor Helen Cross, Director of the UCL Great Ormond Street Institute of Child Health said “There is now huge potential for genetic therapies to be transformative for the lives of children and adults with a range of neurological diseases. Joint working between ICH and ION will accelerate progress”
Professor Alan Thompson, Dean of the UCL Faculty of Brain Sciences, said “This is a wonderful example of cross-faculty collaboration within UCL which will drive forward an exciting evolving area with direct therapeutic benefits for patients with disabling neurological conditions”
Professor Ibrahim Abubakar, Dean of the UCL Faculty of Population Health Sciences said “Delighted to see UCL GOSICH and QSION coming together to solve key challenges using state-of-the-art genetic therapy approaches to benefit patients across the life course.”