University joins forces with US pharmaceutical company to improve drug treatments

October 2, 2015

Professor Sue Fletcher and Professor Steve Wilton from the Centre for Comparative Genomics

Professor Sue Fletcher and Professor Steve Wilton from the Centre for Comparative Genomics at Murdoch

Murdoch University has signed a four-year agreement with Sarepta Therapeutics, with the purpose of using Sarepta’s phosphorodiamidate morpholino oligomer (PMO) technology and chemistry in researching and developing treatment solutions for diseases outside of Duchenne Muscular Dystrophy (DMD).

“Murdoch’s translational research endeavours to bring positive change through new discoveries,” said Acting Vice Chancellor Professor Andrew Taggart.

“Our partnership with Sarepta is yet another example of our successes in international health research, for it will see our accomplished researchers use innovative technologies to devise treatments for children suffering from debilitating genetic diseases.”

The partnership will allow Murdoch researchers from the Centre for Comparative Genomics, Professors Steve Wilton and Sue Fletcher to build on their research, which won them an Australian Museum Eureka Prize in 2013. The researchers were recognised for developing a new investigational drug designed to ‘skip over’ the faulty part of the gene message in children suffering from DMD.

“We have been personalising medicines that can correct disease-causing mutations in DMD patients,” Professor Wilton said.

“In essence we must design drugs to treat different mutations in the dystrophin gene and now we can translate the technology to other genetic diseases such as cystic fibrosis, metabolic disorders and perhaps even Alzheimer’s.”

Professor Wilton said the agreement with US-based Sarepta allows their Murdoch laboratory to become a pipeline of drug development, and the Sarepta association will facilitate getting the investigational drugs from laboratory to clinical trials.

“We are delighted, excited and proud to now have a more formal and focussed academic:industry partnership, combining Sarepta’s morpholino technology and translational experience with our expertise and creative flair to develop treatments for many other human diseases,” added Professor Wilton.

“A commercial party like Sarepta is essential to bring these investigational drugs to the market. It’s a team effort and Sarepta’s experience is invaluable. Watch this space; much more is yet to come!”

The collaborative research agreement provides the Murdoch University researches with access to Sarepta’s PMO platform technology, as well as funding for researchers and materials. As part of the collaborative agreement, Sarepta will have exclusive rights to license technology and/or products resulting from the research projects.

“We’re very excited about this opportunity to support important early stage research and, through that, generate data on the applicability of our PMO technology for additional disease targets,” said Edward Kaye, Sarepta’s Interim Chief Executive Officer and Chief Medical Officer. “We are hopeful that through this collaboration our technology may lead to important breakthrough treatments for patients.”

The Murdoch researchers will work in conjunction with the Western Australian Neuroscience Research Institute (WANRI) who will provide additional research staff members for the project.

"The agreement with Sarepta Therapeutics represents a breakthrough moment for medical research in Western Australia,” said Steve Arnott, WANRI Chief Executive Officer.

"The fact that a leading US company is willing to provide such a high level of support over so many years is testimony to the importance of the research undertaken by Steve Wilton and Sue Fletcher.

“This world-leading research began in our institute in the 1990s and it is gratifying to see it move to another level at Murdoch University.”

Initially the collaborative research agreement will support the Murdoch researchers to focus on developing investigational drugs to treat several diseases, including cystic fibrosis, multiple sclerosis, Facioscapulohumeral Muscular Dystrophy and Huntington’s disease.

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