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The Harrington Discovery Institute (HDI) in the US has announced five winners of its inaugural UK Rare Disease Scholar Award competition.

Professor Angela Russell

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 Departments of Chemistry and 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”.

Harrington Discovery Unit press release