Professor Steve Wilton, WA Neuroscience Research Institute (the Perron Institute) Director, and Professor Sue Fletcher, both based at the Centre for Comparative Genomics, lead the research team at Murdoch University which has signed a four-year agreement with Sarepta Pharmaceuticals that will see the type of drugs developed to treat Duchenne Muscular Dystrophy (DMD) patients being redesigned into solutions for people with other genetic diseases.

The researchers were recognised for developing a new drug that works by ‘skipping over’ the faulty part of the gene message in children suffering from DMD. Professors Wilton and Fletcher started their research into DMD when they joined the Perron Institute in 1991. They joined Murdoch University and the Centre for Comparative Genomics in early 2013, and the partnership with Sarepta will allow them to build on the research that won them an Australian Museum Eureka Prize in 2013.

“We have been personalizing 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 drugs from laboratory to clinical trials.

“We are delighted, excited and proud to now have a more formal and focused 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 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 Murdoch researchers will work in conjunction with the Perron Institute 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, the Perron Institute 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.”

The collaboration agreement provides the Murdoch University researchers with access to the Sarepta’s phosphorodiamidate morpholino oligomer (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.

“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,” said Murdoch University Acting Vice Chancellor Professor Andrew Taggart.

“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.”

Initially the new agreement will see the award winning research team focus on developing drugs to treat several diseases, including cystic fibrosis, multiple sclerosis, Facioscapulohumeral muscular dystrophy, and Huntington’s disease.