Action Duchenne and Duchenne Ireland funds further research for exon skipping therapies

• Thursday, 26 November 2009

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Press Release
26 November 2009

Action Duchenne and Duchenne Ireland funds further research for
exon skipping therapies

Recent successful trials of RNA-based drug provide foundations for new therapies for more patients with Duchenne Muscular Dystrophy


Action Duchenne and Duchenne Ireland have awarded new funding to support further drug development towards clinical trials in exon skipping. The funding awarded to Dr. Matthew Wood at Oxford University and Dr Mike Gait at the MRC Laboratory of Molecular Biology in Cambridge (both members of the MDEX Consortium*), will be used for two development projects. The projects will investigate methods for the improved delivery and reduced dosage of Antisense Oligonucleotids (AO) to all muscle groups. Specifically, one project will investigate exon skipping for the heart muscle and the second will support further work into developing ‘multi-exon’ skipping treatments .The teams will be working with AVI BioPharma, Inc. (Nasdaq: AVII), a developer of RNA-based drugs. Recent clinical trial results published by AVI BioPharma on drug AVI-4658 for exon skipping as a therapy for boys affected by DMD, suggest that this approach could provide possible treatment for affected individuals.

Research to date has demonstrated that Antisense Oligonucleotides – can restore the production of dystrophin protein to affected tissues. Called ‘exon skipping’, the parts of the genetic code affected by a mutation are “patched” by AO’s so that that the code can be correctly read. Drug AVI–4658 is designed to skip exon 51 of the dystrophin gene. Skipping this exon enables restoration of dystrophin production that may potentially improve or significantly slow the disease process, thus prolonging and improving the quality of life for the affected patient.

The new research will investigate the possibility of ‘multi’ exon skipping, thus enabling more exons to be targeted with the treatment of AOs (not just exon 51) and benefiting larger groups of patients.

According to Dr. Matthew Wood; “Although more than 80% of Duchenne boys could potentially benefit from the “exon skipping” approach, each exon targeted requires a unique AO, and thus treating all the different mutations known to cause DMD requires the development of large numbers of AOs, each treating only a small subset of the patient population.

“With this new round of funding we can build on the great work that has been achieved so far and develop the treatments further to benefit more groups of DMD patients. It is scientifically very challenging, but if this new work is successful, we may be able to develop treatments with one Antisense Oligonucleotide that can potentially skip multiple exons. This will enable us to develop treatments for all of the DMD cases that could benefit from AO therapy. This will also reduce development time by five to ten years and speed up the process of making the treatment commercially viable, enabling more patients to receive treatment therapies much sooner.”

The funding will also support development of special peptides that will increase the effectiveness of oligonucleotides in the heart and reduce weakening of the heart muscle. It is hoped that the tests will provide further data on the heart’s ability to be treated by exon skipping. Currently oligonucleotides pass into other muscles more easily, it is believed, due to their ’leaky’ nature, whereas the heart has a stronger and more complex barrier.
Nick Catlin, CEO of Action Duchenne said; “We are delighted that we can provide further funding to build on the progress that AVI and the Consortium have made in the trial of AVI-4658 and the potential for exon skipping drugs. The success to date provides a fantastic foundation on which to carry out further research and widen the opportunities for more DMD patients to benefit from an effective therapy for this disease.”

Duchenne muscular dystrophy is the most severe form of the muscular dystrophy condition, affecting around 3,500 people in the UK and 40,000 people worldwide. It has no cure and is caused by mutations in the dystrophin gene that prevent correct reading of the genetic code, the result of which is that the essential muscle protein dystrophin is not produced. This leads to progressive muscle wasting, severely limiting mobility in teenage patients (boys and men) and resulting in paralysis and premature death in their twenties from respiratory or cardiac problems (the heart muscle is directly affected by the genetic defect).

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NOTES TO EDITORS

About the MDEX Consortium

The MDEX consortium led by Professor Francesco Muntoni, is a multidisciplinary enterprise to promote translational research into muscular dystrophies, and is formed by the clinical groups of Professor Francesco Muntoni (UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle University), and scientists from Imperial College London (Professor Dominic Wells), UCL Institute of Child Health (Dr. Jennifer Morgan), Royal Holloway University of London (Professor George Dickson), Oxford University (Dr. Matthew Wood) and University of Western Australia (Professor Steve Wilton).

In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participate in the Consortium. For more information, visit www.mdex.org.uk.

About Duchenne Ireland

Duchenne Ireland is a patient organisation and registered Irish Charity (CHY 18197) established to faciliate funding translational research into Duchenne Muscular Dystrophy. Duchenne Ireland is affiliated with numerous patient groups and clinical, research and support networks for Duchenne Muscular Dystrophy. Duchenne Ireland works internationally to rapidly advance research into Duchenne and welcomes organisations, parents and supporter who believe they can help to forward progress in this research.

The aims of Duchenne Ireland is to raise awareness of Duchenne Muscular Dystrophy at local, national and government level. Our objective is to raise funds which shall go directly to the researchers and clinicians who we believe have the best chance of developing improved therapies which will benefit this generation. We are also working towards achieving an infrastructure which is on a par with best international practice.

For more information please visit: www. Duchenne.ie


About Action Duchenne

Action Duchenne (formally Parent Project UK) was set up by Duchenne families in 2001 to promote new research for a cure for Duchenne. The charity has a strong record in funding research and has to date funded 8 major projects costing £800,000 and has lead the £1.6m DoH funding of the MDEX project. These projects have enabled much needed early work to be completed on exon skipping and other therapeutic approaches.

Action Duchenne holds an international conference every year to bring together researchers and families to exchange new research developments and provide a vital meeting venue for scientists.

In 2005 Action Duchenne launched the Duchenne Registry, the first National Duchenne database that holds gene information of people living with Duchenne and can be used to speed up the recruitment of patients for clinical trials.

In 2006 Action Duchenne launched a comprehensive learning and behaviour toolkit for use by parents and education professionals.

For more information please visit: www.actionduchenne.org

Editors Contacts
Nick Catlin
CEO,
Action Duchenne
Tel: 0208 556 9955
Email: nick@actionduchenne.org

Andreina West
PR Artistry Limited
Tel: 01491 639500
email: Andreina@pra-ltd.co.uk

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