Harrington Discovery Institute Announces Award Recipients in New UK Rare Disease Program

Five scientists selected for their breakthrough discoveries with the potential to advance to clinical trials

CLEVELAND, Oct. 14, 2020 /PRNewswire/ -- The Harrington Discovery Institute® at University Hospitals in Cleveland, Ohio, and its registered charity Fund for Cures UK, Ltd., together with Morgan Stanley GIFT (Global Impact Funding Trust) Cures, announced five winners in the inaugural Harrington UK Rare Disease Scholar Award competition to advance novel treatments for rare diseases.

The Harrington Discovery Institute — part of The Harrington Project for Discovery & Development — was established in 2012 to accelerate promising discoveries into new medicines to address unmet patient needs. Approximately 350 million people worldwide are living with a rare disease, and half of them are children. With more than 7,000 known rare diseases, and only five percent of those having an approved treatment, rare diseases represent one of the greatest unmet needs in healthcare today.

Through its UK charity, Fund for Cures UK, Harrington Discovery Institute issued a national call for proposals in January 2020 for cutting-edge research related to rare diseases, ultra-rare diseases, and rare variants of more common diseases. In addition to grant funding, the award provides dedicated drug discovery and development support from Harrington Discovery Institute's Therapeutics Development team (experienced pharma and business development professionals with a track record of bringing new drugs to market), while taking no rights to intellectual property, which is retained by the award recipient ('Harrington Scholar') and their institution.

Nearly 50 applications from scientists and physicians in 17 institutions across England, Scotland and Wales were received in response to the call. The applications progressed through multiple rounds of confidential review from both academic and industry science reviewers who evaluated the projects on the likelihood to improve standard of care, be transformative, and/or result in a licensable package, among other criteria. 

"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, MD, President, Harrington Discovery Institute, Robert S. and Sylvia K. Reitman Family Foundation Distinguished Professor of Cardiovascular Innovation and Professor of Medicine at University Hospitals and Case Western Reserve University.

The national grant award program is part of a broader commitment to advance rare disease treatments by working collaboratively with UK institutions. In 2019, Harrington Discovery Institute and the University of Oxford announced their transatlantic partnership to establish the Oxford-Harrington Rare Disease Centre (OHC). The OHC is focused primarily on rare diseases that have a clear genetic basis, present early in life, and fit one of three research themes: neurological disorders, developmental disorders, and cancer. Harrington UK Rare Disease Scholars may have opportunity to utilize Oxford's core labs as well as the OHC Therapeutic Accelerator (planned opening in 2021) for their projects.

"The Harrington model is a bold approach that de-risks clinical drug candidates and validates their commercial potential, making them attractive to for-profit funders who are able to bring them to market. 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 Chief Financial Officer at Rex Bionics plc, and member, Board of Trustees, Fund for Cures UK.   

Harrington Discovery Institute will raise capital to support the growth of the UK rare disease initiative through Morgan Stanley GIFT Cures, its philanthropic partnership with global financial services firm Morgan Stanley. 

The 2021 Harrington UK Rare Disease Scholar Award recipients, their organizations and fields of research are:

Pietro Fratta, MD, PhD – University College London
Gene Therapy for Kennedy's Disease, a rare neuromuscular disease

Angela Russell, DPhil – University of Oxford 
New Drugs for the Treatment of Duchenne Muscular Dystrophy

Helen Waller-Evans, DPhil – Cardiff University 
Novel Inhibitors to Treat Multiple Lysomal Storage Disorders, causes of widespread organ damage

Wyatt Yue, PhD – University of Oxford 
Inhibitors of Primary Hyperoxaluria, a cause of kidney failure

Haiyan Zhou, MD, PhD – University College London
Novel Therapy for SPTLC1-Related Hereditary Sensory Neuropathy, a cause of shooting pain