For the past year or two, over on the NEU board, I’ve been following the fortunes of various DMD therapeutics as they attempt to gain FDA approval. The latest candidate, Sarepta’s eteplirsen, is still awaiting a final decision from the FDA. I had no idea of eteplirsen's connection with researchers at Murdoch University, yet alone that these same scientists had been assessing the ability of PYC CPPs to improve intracellular delivery! (1, 2)
Today’s announcement focuses on the potential role that PYC CPPs may play in improving efficacy of Duchenne therapies. However, as Richard Hopkins highlights in the announcement, possible applications are much broader. Other inherited and acquired conditions in which Professor Wilton is exploring the use of antisense oligomers include spinal muscular atrophy, cystic fibrosis, multiple sclerosis, Alzheimer’s, Pompe’s disease, congenital muscular dystrophy, Huntington’s and asthma.(3)
A 2013 review of antisense oligonucleotide drug candidates in the pipeline noted 109 drugs in development at the time, with cancer being the most common indication (31 candidates), followed by cardiovascular and related diseases (14), ocular disorders (10), and muscle dystrophies (10). (4, 5)
Effective delivery of oligonucleotides to their intracellular sites of action remains a major issue with this class of drugs, with the endosome escape barrier said to be now generally regarded as perhaps the most important impediment to effective use of oligonucleotides in therapeutics. (6)
That's why it's likely that the pilot study results announced today will attract attention.
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