Ann: Operational Update, page-3

Good news re the oligonucleotide delivery collaboration with Murdoch. In the June announcement, improved intracellular delivery using an FPP had been demonstrated. The next challenge was to demonstrate efficacy without toxicity. Preliminary signs of this have now been shown in delivery to a range of muscles in both Duchenne and Spinal Muscular Atrophy models.

There is also evidence of increased levels of dystrophin following treatment with FPP-delivered exon-skipping nucleotides. This is significant when you consider that it was failure to clearly demonstrate that eteplirsen produced sufficient dystrophin to generate a clinically meaningful outcome in Duchenne patients that was the main source of conflict between the FDA and Sarepta.