Duchenne UK funds Peptide Generation (PepGen) exon skipping project
Duchenne UK is pleased to announce its support of the development of the next generation of exon skipping drugs, with a £50,000 grant to the University of Oxford.
The grant is for Professor Matthew Wood’s Peptide Generation (PepGen) project. PepGen is focused on improving the delivery and effectiveness of exon skipping drugs as a way of restoring dystrophin.
Matthew Wood is Professor of Neuroscience at the University of Oxford. He has been working within the field of Muscular Dystrophy for over 15 years.
Researchers will look at the peptide-based delivery platform technology for oligonucleotide therapies. If the project develops as planned, clinical trials will be planned for 2019/20.
Pre-clinical data shows that PepGen’s technology enhances oligonucleotide delivery to heart and skeletal muscle versus first generation exon-skipping. This means that the next generation of drugs using the peptide-oligonucleotide conjugate could provide a more effective therapy for Duchenne muscular dystrophy (and related conditions including spinal muscular atrophy).
Professor Matthew Wood said:
“Development of next generation exon skipping oligonucleotide drugs is critical in order to provide improved clinical outcomes for Duchenne patients and we are very excited to have Duchenne UK’s support to advance our PepGen peptide technology.”
Emily Crossley and Alex Johnson from Duchenne UK said:
“We are delighted to be working with the University of Oxford on this new project that has the potential to enhance the efficacy of exon skipping drugs for the treatment of DMD.”
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues, it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected by the disease, and around 300,000 worldwide. It is classified as a rare disease.
Who are Duchenne UK?
Duchenne UK is a lean, ambitious, and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne Muscular Dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children’s Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Kris Radlinski and Andy Farrell.