In 2019, an initial gift of £5 million by the

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Sigrid Rausing Trust

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established a Neurogenetic Therapies Programme (NgTP) at UCL under the leadership of Professor Nick Fox (UCL Queen Square Institute of Neurology).

The unrestricted nature of this donation has allowed UCL to respond rapidly to new opportunities and pursue emerging ideas for nucleic acid-based therapies. Over the last five years, the NgTP has provided awards ranging from £90,000 to £250,000 in support of numerous therapeutic approaches.

A total of 15 projects have so far been supported under a portfolio adoption process which facilitates the initiation of promising, scientifically robust, projects whilst – relative to traditional funders – reducing timescales and streamlining research set up processes.

In addition, the NgTP has systematically identified pinch points in the delivery of early phase clinical trials to inform a sustainable improvement in capacity. This work has been a major factor in the UK Government’s decision to provide

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£49.9 million in new funding for a UK-wide network for dementia clinical trials

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led by Professor Cath Mummery (UCL

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Queen

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Square

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Institute

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of

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Neurology).

Demonstrating the breadth and width of impact of the NgTP, recent examples of Translational Portfolio and Capacity Building work supported by the Sigrid Rausing Trust include:

Results from the first genetic therapy trial in dementia

The results of the first-ever genetic therapy trial in dementia, led by UCL, were published in the journal Nature Medicine in April 2023.

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This first-in-human trial represents the first time that a ‘gene silencing’ approach has been taken in dementia and Alzheimer’s disease. It shows that a drug called BIIB080 (IONIS-MAPTRx), an antisense oligonucleotide, is the first to have a significant effect on levels of tau, a critical protein in the development of Alzheimer’s disease.

Principal Investigator/Lead Author: Professor Cath Mummery

An AAV gene therapy for a subtype of Frontotemporal Dementia/Amyotrophic Lateral Sclerosis

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This project investigates a novel genetic therapy approach to correct the molecular abnormalities underlying a subtype (C9orf72) of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).

The data generated from this early preclinical project shows that the gene therapy approach can dramatically decrease the toxic C9orf72 repeat RNAs, supporting next steps towards clinical trials in patients.

Principal Investigator: Professor Adrian Isaacs

NIO-SILK Phase 1b UK/US clinical trial investigating the efficacy and safety of a novel genetic therapy drug for Alzheimer’s disease

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NIO-SILK is a clinical research collaboration sitting at the patient end of the translational pipeline. The inclusion of the novel SILK technique to this clinical trial allows investigators to see the real-time effects of the genetic therapy (a new antisense oligonucleotide) on tau protein levels.

Data generated by this project will allow us to understand the impact of this promising therapy with potential to help achieve regulatory approval and reach patients sooner.

Principal Investigator: Dr Ross Paterson

Targeting muscle to restore axonal transport as a therapeutic strategy in Charcot-Marie-Tooth disease (CMT)

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Supported by strong preliminary data, the goal of this project is to restore the process of neuronal axonal transport in CMT by the virus mediated delivery of a genetically encoded growth factor to the nerve cells via the skeletal muscle.

Principal Investigator: Dr James Sleigh

Building upon the progress of the last five years, the Trust has now renewed its commitment with a further donation of £5 million to support the work of the NgTP until 2029.

The funding will enable the continued contribution of the programme to a field that has been revolutionised over the last five years, with increasing translation of gene-based research to patient treatments of a range of diseases, and the regulatory approval of the first disease-modifying therapies for Alzheimer’s disease in both the UK and US.

“The rate at which gene-based therapies have developed over the last 5 years reinforces the immense promise they hold for patients and their families,” said Prof Nick Fox, Principal Investigator for the Neurogenetic Therapies Programme and Director of the

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UCL Dementia Research Centre. “Our deeply valued partnership with the Sigrid Rausing Trust has and will continue to be instrumental to UCL research with real potential to improve treatments for a range of neurodegenerative conditions.”
“UCL was privileged to receive one of the Sigrid Rausing Trust’s largest ever philanthropic gifts for health research in 2019 and we feel that same sense of pride and responsibility today,” said Dr Michael Spence AC, UCL President & Provost. “The renewal of our work together is testament to what we achieve when we unite far-sighted donors and partners with researchers at the frontiers of their field.
“I am immensely grateful to the Trust for their continued belief in the power of our partnership to change the world for the better.”
“The sheer complexity of genetic research for neuro-degenerative conditions – both in terms of identifying causation and in terms of inventing therapeutic remedies and finding a way to convey them to the brain – means that ideas first posited years or even decades ago are only now beginning to give results,” said Sigrid Rausing, Chair of the Sigrid Rausing Trust. “It’s a privilege to be part of a genuinely revolutionary approach to dementia, one which is collaborative, and, importantly, as concerned with humane care and patient experience as it is with pure science and medicine.

“Nick and his team at UCL have built an extraordinary model for medical research, valuing cooperation, compassion, and outstanding science equally.”

Neurological diseases remain amongst the most urgent healthcare challenges, with dementia alone responsible for more deaths in the UK than any other cause between 2012 and 2021.

However, advances in many areas of medicine – from childhood neuromuscular disease to haemophilia – demonstrate the efficacy and potential of gene-based therapies. With increased investment also bringing similar steps forward in Alzheimer’s disease and other degenerative conditions, philanthropy holds the key to saving and changing millions of lives.

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Professor Selina Wray, UCL Queen Square Institute of Neurology.

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