Thursday, July 02, 2015 9:15:35 PM
• Benitec’s OPMD program in collaboration with RHUL and IM to progress towards the clinic
• Gene therapy treatment combining silencing and replacement of mutant gene
• Positive pre-clinical proof of concept established in vivo
Sydney, Australia: Benitec Biopharma is pleased to announce the progression of the company’s collaboration with the Royal Holloway University of London (RHUL) and the Institut de Myologie (IM) in Paris to continue the development of a ddRNAi based therapeutic for the treatment of oculopharyngeal muscular dystrophy (OPMD). This follows successful pre-clinical proof of concept data that show using ddRNAi to silence the mutant gene responsible for the disease and replacement with the healthy gene can restore muscle strength to near normal levels in vivo.
The work to be undertaken in the collaboration with RHUL and IM will evaluate the efficacy and safety of a clinical construct in animal studies with the ultimate goal of generating sufficient data to advance the program to a clinical trial.
Professor George Dickson and Dr Capucine Trollet will lead the team at RHUL and IM, which is a national reference centre for muscle disorders in France. These teams are experts in the area of treatment development for this disease.
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com.
Good luck and GOD bless,
George
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