Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

Angela Russell and Wyatt Yue
Angela Russell (left) Wyatt Yue (right)

The Harrington Discovery Institute (HDI) in the US has announced five winners of its inaugural UK Rare Disease Scholar Award competition. Through its UK charity, Fund for Cures UK, with Morgan Stanley GIFT Cures, HDI issued a call for proposals for cutting-edge research in rare diseases and rare variants of more common diseases. The goal, aligning with the vision of the Oxford-Harrington Rare Disease Centre (OHC) established in 2019, is to advance novel treatments for rare diseases. The OHC is based in the Department of Paediatrics and is directed by Professor Matthew Wood.

HDI was established in 2012 to accelerate promising discoveries into new medicines to address unmet patient need. 350 million people worldwide have a rare disease, and half of them are children. Of the 7,000 known rare diseases, only five percent have an approved treatment. Rare diseases represent one of the greatest unmet needs in global healthcare today.

Of 50 applications from 17 UK institutions, two Oxford scientists have received an award. Angela Russell is Professor of Medicinal Chemistry in the Department of Chemistry and Department of Pharmacology, and member of the MDUK Oxford Neuromuscular Centre. She aims to develop new drugs for the treatment of Duchenne Muscular Dystrophy, a severe muscle disease with childhood onset.

Wyatt Yue, Associate Professor, leads the metabolic and rare diseases group in the Centre for Medicines Discovery, Nuffield Department of Medicine, and aims to develop inhibitors for Primary Hyperoxaluria, a rare metabolic disorder that causes kidney failure.

In addition to grant funding, the award provides Scholars with dedicated drug discovery and development support from HDI's Therapeutics Development team, while intellectual property remains with the Scholar and their institution.

"In eight years we have built a strong portfolio of breakthrough science throughout the US and Canada, and we are excited to extend our reach to the United Kingdom. Together with our Scholars and their institutions, we are pleased to move one step closer to changing the rare disease landscape in a meaningful way," said Jonathan S. Stamler, President of the HDI.

"This program is an extraordinary opportunity for UK researchers to help them translate scientific discoveries into medicines that improve the lives and health outcomes of those living with a rare disease," said Peter Worrall, former CFO at Rex Bionics plc, and member of the Board of Trustees, Fund for Cures UK.

The Director of the OHC and Deputy Head of Oxford's Medical Sciences Division, Professor Matthew Wood, said "The response to this call is testament to the excellence of rare disease research in the UK. We're delighted that two of the recipients are Oxford scientists who will have the opportunity to advance their rare disease research with the outstanding support offered by these awards and by the Harrington Discovery Institute".

Similar stories

Oxford spinout trials revolutionary bioelectronic implant to treat incontinence

The first participants in a clinical trial of a bioelectrical therapy to treat incontinence have received their “smart” bioelectronic implants.

COVID-19 is a leading cause of death in children and young people in the US

A new study led by researchers at the University of Oxford’s Department of Computer Science has found that, between 2021 and 2022, COVID-19 was a leading cause of death in children and young people in the United States, ranking eighth overall. The results demonstrate that pharmaceutical and public health interventions should continue to be applied to limit the spread of the coronavirus and protect again severe disease in this age group.

Three or more concussions linked with worse brain function in later life

Experiencing three or more concussions is linked with worsened brain function in later life, according to new research.

New blood test could save lives of heart attack victims

Researchers in the Department of Physiology, Anatomy and Genetics (DPAG) have developed a blood test that measures stress hormone levels after heart attacks. The test – costing just £10 – could ensure patients receive timely life-saving treatment.

COVID-19 increased public trust in science, new survey shows

A survey of over 2000 British adults has found that public trust in science, particularly genetics, increased significantly during the pandemic. However, those with extremely negative attitudes towards science tend to have high self-belief in their own understanding despite low textbook knowledge.

Gero Miesenböck awarded 2023 Japan Prize

Congratulations to Professor Gero Miesenböck, Department of Physiology, Anatomy and Genetics (DPAG), who has been awarded the 2023 Japan Prize in the field of Life Sciences, together with Professor Karl Deisseroth, for pioneering work in the field of optogenetics.