Wednesday, 26 November 2008

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.

About Duchenne Muscular Dystrophy (DMD):

DMD, a relentlessly progressive muscle wasting disorder, is one of the most common fatal genetic disorders to affect children around the world.  Approximately one in every 3,500 boys worldwide is afflicted with Duchenne muscular dystrophy, with one third of cases presenting with no prior family history of disease.

DMD is a devastating and incurable muscle-wasting disease associated with specific errors in the gene that encodes dystrophin, a protein that plays a key structural role in muscle fibre function and stability.

Symptoms usually appear in boys before the age of 6 years.  At this age, affected boys have difficulty in keeping up with their peers, may appear clumsy and fall easily.  By age 10, boys have difficulty walking, and patients are confined to a wheelchair by age 12.  Eventually, all muscles are affected and patients experience increased difficulty in breathing.

The condition is terminal and death usually occurs before the age of 30.  The outpatient cost of care for a non-ambulatory DMD boy is among the highest of any disease.  There is currently no cure for DMD, but for the first time in decades, there are a range of promising therapies under development.

About exon skipping

Most of our genes consist of blocks of protein coding information (exons) which are separated by non-coding sequence (introns).  During the steps of gene expression to make a protein, the non-coding introns are snipped out and the exons are precisely spliced together to make a gene message (mRNA).  "Exon skipping" is the process where a mutated exon, that may cause a severe genetic disease (ie DMD), can be targeted for removal during the splicing process.  In this way, the disease-causing mutation is skipped from the mature gene message and a functional protein can be made.

The dystrophin gene, which is mutated in the DMD, consists of 79 exons and it has been well established that not all are necessary for function.

Media references

Dr Andy Sierakowski (UWA Office of Industry and Innovation)  (+61 8)  6488 7048
Professor Steve Wilton (+61 8)  9346 3967
(UWA Centre for Neuromuscular and Neurological Disorders)  (+61 4) 17 982 365
Janine MacDonald (UWA Public Affairs)  (+61 8)  6488 5563  /  (+61 4) 32 637 716

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