Worldwide patent opens doors to treatment of muscular dystrophy

The University of Western Australia has signed an exclusive worldwide license agreement with US-based pharmaceutical company AVI BioPharma to a patent application related to the treatment of Duchenne muscular dystrophy (DMD).

Among the inventors on the licensed patent application are Professor Steve Wilton and Associate Professor Sue Fletcher, of the Molecular Genetic Therapies Group at UWA, renowned pioneers in the use of exon skipping to treat DMD and other diseases.

AVI BioPharma is a developer of RNA-analogue drugs in Portland, Oregon, USA.

"Exon skipping has the potential to reduce the severity of the disease in about 80% of DMD individuals.  We have been working in this area of personalised gene medicine for over a decade and are very excited and optimistic about the possibility of our treatment making a difference to the lives of people suffering from this devastating disease," said Professor Wilton.

"We look forward to an ongoing collaboration with AVI BioPharma Inc, and plan to extend the technology of splice intervention to other amenable diseases, in addition to DMD."

Mr Simon Handford, Senior Commercialisation Manager with the University’s Office of Industry and Innovation, said the license was an important step in translating university research into clinical outcomes and demonstrated the global nature of commercialising biotechnology innovations.

"Partnering is critical in terms of resourcing the development of new treatments and we are looking forward to collaborating with AVI BioPharma to evaluate new sequences that Professor Wilton and his team identify."

The patent application claims compositions and methods for treating DMD in humans by skipping exons in the dystrophin gene.

(Source: University of Western Australia: November 2008)