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Cleveland's University Hospitals partners with Oxford to find cures for rare diseases

The UH Harrington Discovery Institute and University of Oxford formed the Oxford-Harrington Rare Disease Centre Therapeutics Accelerator.

CLEVELAND — University Hospitals' leadership traveled to London to sign a deal with the University of Oxford to create treatments and hopefully cures for some of the 7,000 rare diseases affecting more than 400 million people around the world. 

An official signing took place at Oxford. 

The OHC connects world-leading research across the U.K. and U.S. with expertise in therapeutics development by combining University of Oxford’s scientific leadership and resources.  Currently there are more than 250 Oxford scientists investigating more than 350 rare diseases. 

Harrington Discovery Institute’s track record is what brought Oxford to the table says HDI President and Founder, Jonathan Stamler, MD.

"We have funded 177 medicines, launched  36 companies, have 19 medicines in clinic, and 13 licenses to pharma companies since we began in 2012," Dr. Stamler said.

The Accelerator is a first-in-kind, transatlantic initiative to identify, fund and advance breakthrough academic discoveries to deliver new treatments for those who suffer from rare diseases. 

The mission is to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over the next ten years and target multiple approvals from regulators in key markets including the U.S., the U.K. and Europe.

At the helm is former U.K. Prime Minister David Cameron, who will lead the international efforts of the Accelerator under his role as Chair of the Oxford-Harrington Rare Disease Centre Advisory Board.

Cameron lost his seven-year-old son to a rare disease and as Prime Minister launched Genomics England and the 100,000 Genomes Project that sequenced five million adults across the U.K.  That data has helped create several invaluable data bases that help researchers understand genetic mutations.

 The OHC prioritizes rare genetic diseases affecting children or adults where there is urgent need and opportunity for greatest impact and that fall into the areas of rare neurological diseases, cancers and developmental diseases. Projects will be sourced from academic labs across the U.K. and U.S.

The Accelerator will operate a unique non-profit/for-profit model with the intention to commit up to $250 million into new projects. Project funding will be paired with research, drug development, commercial strategy and business development expertise from pharma-experienced industry leaders with a track record of bringing new drugs to market. 

The first investment by the Accelerator was made in September 2023 in conjunction with the creation of AlveoGene, a new U.K. company launched to develop innovative inhaled gene therapies and transform outcomes in patients with rare respiratory diseases.

The partners are also collaborating to create a dedicated Rare Disease Impact Fund to enable investments in mission-aligned projects and accelerate the development of rare disease therapeutics.

But it also goes beyond rare diseases. 

"As our understanding becomes deeper we realize that major diseases are comprised of many rarer diseases so this represents a therapeutic entry if you will of many, many diseases," Dr. Stamler said. 

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