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The Oxford-Harrington Rare Disease Centre (OHC) Announces Inaugural 2024 Rare Disease Scholar Award Recipients

October 24, 2024

The Oxford-Harrington Rare Disease Centre 2024 Scholar Award

10 scientists have been selected for OHC’s first annual award to advance academic discoveries into clinical practice for rare diseases 

OXFORD, UK and CLEVELAND, Ohio, US, 24th October 2024 – The Oxford-Harrington Rare Disease Centre – a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio – today announces the first class of Oxford-Harrington Rare Disease Scholars. The scholar awards will support breakthrough treatments for rare and ultra-rare diseases across neurologic, oncologic, and metabolic disease areas, using small molecule, nucleic acid, viral, and cellular therapies. The recipients represent institutions across the UK, US, and Canada.  

The OHC brings together world-leading research, expertise in therapeutics development and new financial models to advance solutions to critical challenges in rare diseases. 

Each Scholar will receive £100,000 (UK) / $100,000 (US and Canada), personalised therapeutics development support from industry experts to help bring new therapeutics to market, and the opportunity to receive acceleration and investment funds of up to £1,000,000 based on project milestones. 

Matthew Wood, MD, PhD, Director and Chief Scientific Officer of the OHC, commented:

“This first cohort of OHC Scholars is a testament to the outstanding quality of proposals we received from academics at institutions across the UK and North America. The breadth and standard of research into innovative approaches to treat rare diseases made our selection process very difficult, with successful proposals representing breakthrough discoveries defined by innovation, creativity and potential for clinical impact. Our congratulations go to the 2024 OHC Scholars and our sincere thanks go to all who submitted proposals – their continued efforts to make a meaningful difference for patients in need and their families are an inspiration to us all.”  

Jonathan S. Stamler, MD, President of Harrington Discovery Institute, added:

“By supporting each OHC Scholar with funding and an experienced therapeutics development team, we are maximising the potential of their discoveries to advance into the clinic. We have seen success with this approach at Harrington Discovery Institute and we are excited about replicating the model at the OHC. I look forward to seeing the progress of these innovative initiatives and sharing their advancements as the OHC continues to work towards providing scalable solutions to address the rare disease challenge globally.” 

The 2024 OHC Rare Disease Scholar Award recipients, their organisations and fields of research are:  

Jacquelyn Bower, PhD – University of North Carolina at Chapel Hill, NC, USA  
Novel viral gene therapy to treat lethal rare eye tumours 

Louis Chesler, MD, PhD – Institute of Cancer Research, London, UK 
Regulatable cell therapies to treat brain cancers 

Charles Gersbach, PhD – Duke University, Durham, NC, USA 
Reprogramming immune cells for treatment of Hodgkin’s lymphoma 

Xianxin Hua, MD, PhD – University of Pennsylvania, Philadelphia, PA, USA 
A new cellular strategy for treating acute myeloid leukaemia 

Michele Jacob, PhD – Tufts University School of Medicine, Medford, MA, USA 
A drug to treat brain abnormalities caused by CTNNB1 defects, an ultra-rare disease 

Bowen Li, PhD – University of Toronto, Toronto, Canada 
A nucleic acid therapy to treat cystic fibrosis, an inherited lung disease 

Michael Pacold, MD, PhD – New York University, New York City, NY, USA 
Drugs restoring brain function in rare metabolic diseases 

Carlo Rinaldi, MA, MD, PhD – University of Oxford, Oxford, UK 
Nucleic acid therapy for spinal and bulbar muscular atrophy (SBMA), a rare X-linked neuromuscular condition 

Timothy Yu, MD, PhD – Boston Children’s Hospital, Boston, MA, USA 
Custom DNA-based therapy for a rare genetic disorder (PEX1 Zellweger Syndrome) 

Haiyan Zhou, MD, PhD – University College London, London, UK 
Nucleic acid therapy to treat loss of sensation and paralysis caused by a rare mutation 

For those interested in submitting a proposal for the Oxford-Harrington Rare Disease Scholar Award, the next funding call will open on the 15th of January 2025. For more information, visit: OxfordHarrington.org/funding/overview 

About Rare Diseases 

More than 400 million people worldwide are living with a rare disease, and approximately 50 percent are children. There are about 7,000 known rare diseases, with new diseases being discovered every day. A rare disease affects 10 percent of the US population. Similarly, Europe has approximately 30 million people who suffer from a rare disease. The majority of rare diseases are genetic in origin, which means they are present throughout a person’s life. Only five percent of rare diseases have a treatment approved by the US Food and Drug Administration (FDA) and similar estimates have been made for treatments approved by the European Medicine Agency (EMA). Therefore, someone with a rare disease today faces a lifelong, often life-threatening, condition with little hope for a cure, or even an effective treatment option.  

About Oxford-Harrington Rare Disease Centre 

The Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, in Cleveland, Ohio, US. The OHC combines world-leading strengths in research and therapeutics development from across these organizations to set the science and innovation agenda and drive cutting-edge rare disease breakthroughs to address the unmet need in rare diseases across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | LinkedIn | X 

About the University of Oxford 

The University of Oxford is rated the best in the world for medicine and life sciences, and it is home to the UK’s top-ranked medical school. It has one of the largest clinical trial portfolios in the UK and great expertise in taking discoveries from the lab into the clinic. Partnerships with the local NHS Trusts enable patients to benefit from close links between medical research and healthcare delivery. The University of Oxford’s Medical Sciences Division, which includes the Department of Paediatrics where the OHC is based in Oxford, is one of the largest biomedical research centres in Europe. For more information, visit: www.ox.ac.uk   

About Harrington Discovery Institute 

Harrington Discovery Institute at University Hospitals in Cleveland, Ohio aims to advance academic discoveries into medicines to address unmet need. It was created in 2012 with a $50 million founding gift from the Ronald G. Harrington family and has supported more than 200 scientists throughout the U.S., U.K. and Canada. Harrington Discovery Institute uses an innovation model that surrounds scientists with drug development and business expertise and employs both philanthropic funding and catalytic investment capital to advance projects into the clinic and through commercialization. For more information, visit: HarringtonDiscovery.org| LinkedIn | X 

Media contacts: 

UK/ EU – MEDiSTRAVA  
Sylvie Berrebi / Mark Swallow 

OHC@medistrava.com  

US – For University Hospitals 
Ansley Kelm 

ansley.kelm@UHhospitals.org