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Industry-Academic Center to Boost Rare Disease Therapies

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(Gerd Altmann, Pixabay)

3 Oct. 2023. An alliance of academic labs and venture investors is starting a new research enterprise to speed development of treatments for rare diseases in the U.S. and U.K. The Oxford-Harrington Rare Disease Centre, located in Oxford, U.K. and Cleveland, Ohio, says its Therapeutics Accelerator plans to commit £200 million ($US 241.3 million) to deliver 40 new therapies over the next 10 years.

The Oxford-Harrington Rare Disease Centre is a four year-old collaboration between University of Oxford and Harrington Discovery Institute, a translational research accelerator affiliated with University Hospitals in Cleveland. The Rare Disease Centre seeks to combine research expertise at Oxford’s labs and Harrington’s new-drug development efforts to support new technologies that address unmet needs from rare diseases. The center says about 400 million people worldwide are living with some 700 rare diseases rare diseases, about half of whom are children. Most rare diseases, says the center, are genetic in origin, but only about five percent of rare diseases have treatments approved by regulatory agencies in the U.S. and Europe.

The Therapeutics Accelerator is a program to identify and advance new innovative treatments for rare cancers as well as neurological diseases and developmental disorders from academic labs in the U.S. and U.K. The program expects to operate under a hybrid non-profit and for-profit model that combines research grants with private investment to develop and bring new therapies to market, including the launch of new spin-off companies.

First investment made last month

University Hospitals and Oxford Science Enterprises, the university’s venture capital arm, are providing financial support for the accelerator under their current co-funding and co-investment agreement. The partners are also establishing a separate Rare Disease Impact Fund to make possible further investments in rare disease therapies.

The Therapeutics Accelerator made its first investment in September to AlveoGene, a start-up company in Oxford advancing gene therapies delivered by inhalation through the lungs to treat rare inherited respiratory diseases. AlveoGene is spun-off from the U.K. Respiratory Gene Therapy Consortium, a group of researchers studying inherited respiratory diseases at universities of Oxford and Edinburgh, and Imperial College London. Science & Enterprise reported last month on the AlveoGene launch and seed funding.

Matthew Wood, a neuroscience professor at Oxford and director of the Oxford-Harrington Rare Disease Center or OHC says in a statement, “OHC was set up with a bold vision and ambitious goals to make an impact on the treatment of rare diseases. This new Accelerator initiative takes this a step further with its unique collaboration and for-profit and not-for-profit model. It is only by combining resources and adopting innovative approaches that we can truly accelerate the development of new drugs for patients in need.”

David Cameron, the former U.K. Prime Minister, chairs the the Oxford-Harrington Rare Disease Center advisory board. Cameron notes, “I know from my own experience of having a son born with an incredibly rare and incurable disease, how little we really know about rare diseases.” He adds, “This Accelerator will deliver new, real and tangible therapies for patients and, by doing so, offer huge benefits for patients, their families and wider society.”

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