Put together, there’s certainly some interesting connections and overlaps…
October 2, 2015
Professor Steve Wilton, WA Neuroscience Research Institute (the Perron Institute) Director, and Professor Sue Fletcher, both based at the Centre for Comparative Genomics, lead the research team at Murdoch University which has signed a four-year agreement with Sarepta Pharmaceuticals that will see the type of drugs developed to treat Duchenne Muscular Dystrophy (DMD) patients being redesigned into solutions for people with other genetic diseases….
Professor Wilton said the agreement with US-based Sarepta allows their Murdoch laboratory to become a pipeline of drug development, and the Sarepta association will facilitate getting the drugs from laboratory to clinical trials…..
The Murdoch researchers will work in conjunction with the Perron Institute who will provide additional research staff members for the project….
The collaboration agreement provides the Murdoch University researchers with access to the Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform technology, as well as funding for researchers and materials. As part of the collaborative agreement, Sarepta will have exclusive rights to license technology and/or products resulting from the research projects.
Di Huang 1,2,3, Sue Fletcher 1,3, Steve D. Wilton 1,3, Norman Palmer 3, Samuel McLenachan 2, David A. Mackey 2 and Fred K. Chen 2,4
1 Molecular Therapy Laboratory, Murdoch University, Murdoch 6150, Australia
2 Centre for Ophthalmology and Visual Science (Incorporating Lions Eye Institute), The University of Western Australia, Nedlands 6009, Australia
3 Perron Institute, 4th Floor A Block, Queen Elizabeth II Medical Centre, Verdun Street, Nedlands 6009, Australia
4 Department of Ophthalmology, Royal Perth Hospital, Perth 6000, Australia
Efforts to safely and effectively deliver AONs to the retina, increase cellular uptake in vivo, limit off-target effects [72], define the optimal treatment window and examine potential toxicity and immunostimulatory effects [104] reflect the many issues still to be explored.
Phylogica is pleased to advise that it has appointed Dr. Rakesh Veedu to its Scientific Advisory Board (SAB).....
Dr Rakesh Naduvile Veedu is a Perron Institute McCusker fellow and the head of Nucleic Acid Therapeutics Research based at the Centre for Comparative Genomics at Murdoch University.
In January 2015 Dr Veedu began his five year McCusker Research Fellowship with the Perron Institute, then the WA Neuroscience Research Institute, based at the Centre for Comparative Genomics at Murdoch University. In this role, Dr Veedu works closely with internationally-acclaimed molecular genetics researchers, Professors Steve Wilton and Sue Fletcher, and their team.
Antisense-mediated splice intervention to treat human disease: the odyssey continues
Ianthe Pitout 1, Loren L. Flynn 1-3, Steve D. Wilton 1-3, Sue Fletcher 1-3
1 Murdoch University, Murdoch, WA, 6150, Australia
2 The University of Western Australia, Nedlands, WA, 6009, Australia
3 Perron Institute, Nedlands, WA, 6009, Australia
Competing interests: SF and SDW serve as consultants to Sarepta Therapeutics and are named on intellectual property licensed to Sarepta Therapeutics by the University of Western Australia
Systemic delivery of these molecules can be greatly improved by conjugation to cell-penetrating peptides 43,44,..... Successful translation of peptide-phosphorodiamidate morpholino drugs that are currently in development and clinical evaluation (for example, for DMD) may well begin a new era for splice-modifying therapies, particularly if tissue or cell targeting can be achieved, perhaps leading to reduced dosages.
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