Muscular Dystrophy UK welcomes this exciting news which was revealed earlier this week.
Detailed information can be found on PepGen’s website.
PepGen’s work into molecular patches seeks to help people with Duchenne muscular dystrophy (DMD) and other muscle-wasting conditions.
What are molecular patches and how do they work?
Molecular patches are made of small, synthetic pieces of DNA.
The patches effectively ‘mask’ the mutation in the affected gene, for example in the dystrophin gene in people with DMD.
This allows the cell to ‘read’ the gene properly and produce a slightly shorter – but still functional version – of the dystrophin protein.
Molecular patches will not cure the condition, but they could help lessen the symptoms.
Molecular patches are currently being used in clinical trials for people with DMD and other muscle-wasting conditions.
However, there have been problems associated with the delivery of patches to the body, especially to the heart and diaphragm. Researchers have been looking at ways of improving the delivery.
What will the $45million investment help with?
The funding will advance PepGen’s research into molecular patches, with the aim of moving towards clinical trials.
PepGen’s technology will significantly enhance its unique delivery to key tissues affected by DMD – particularly the heart – and other genetic neuromuscular and cardiovascular conditions.
It will do this by adding short, cell-penetrating peptides to the molecular patch. These will help the molecular patch get into the cell.
This technology has been developed over more than a decade of research in the UK.
Who is behind this approach?
Professor Matthew Wood, from the University of Oxford, pioneered the cell-penetrating peptide (CPP) technology.
MDUK has supported Professor Wood’s research group over a number of years.
We have funded research projects into the development of potential molecular patch therapies for myotonic dystrophy type 1, spinal muscular atrophy and Duchenne muscular dystrophy.
What does MDUK think?
We are delighted to see this investment which will help drive research into clinical trials.
Dr Kate Adcock, Director of Research and Innovation at MDUK, said:
“This is an exciting step in the drug development journey that builds on important research supported by Muscular Dystrophy UK. The $45million investment secured by PepGen will enable work to be taken from an academic laboratory to a commercial laboratory. We are looking forward to seeing how PepGen’s molecular patches could help treat the symptoms of DMD as research develops.”
Who led the funding round?
The funding round was led by top tier investors RA Capital Management with participation from Oxford Sciences Innovation (OSI), The University of Oxford and CureDuchenne Ventures.
You can find more information on exon skipping here.