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

OXFORD, United Kingdom and CLEVELAND, Oct. 24, 2024 /PRNewswire/ — 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 15^th of January 2025. For more information, visit: OxfordHarrington.org/funding/overview

SOURCE Harrington Discovery Institute

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