Benitec Biopharma (ASX:BLT, OTC:BTEBD) is continuing development of a treatment for an inherited muscle disease that causes difficulty in swallowing towards a clinical trial.
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.
Development of a ddRNAi based therapeutic for the treatment of oculopharyngeal muscular dystrophy (OPMD) is carried out in collaboration with the Royal Holloway University of London (RHUL) and the Institut de Myologie (IM) in Paris.
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.
OPMD is an inherited muscle disease characterized by difficulty in swallowing due to the loss of esophageal muscle strength, as well as muscle weakness in multiple parts of the body, in particular the head.
OPMD is a rare disease (1:100,000 in Europe), with a worldwide distribution; it is the most common form of dystrophy in Quebec (1:1,000) due to a founder effect. Benitec is not aware of any effective current medical treatment (apart from surgical intervention) or cure for OPMD.
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