Ann: Phylogica's Technology is Effective in Human Cells, page-30

We appear to have the answer to the question, does the CPP achieve a 30-60 times increase in cargo delivery relative to the naked drug cargo? At 8% exon skipping for the naked ASO, its not mathematically possible to get 30-60 times increase but we will settle on 100% exon skipping.

The more interesting question is whether it will lead to absolute disease correction. Previously, we noted that "the lessons learnt over the course of translating eteplirsen and nusinersen and from the failure of drispersen will inform the development of emerging and future molecular medicines" - Antisense-mediated splice intervention to treat human disease: the odyssey continues - published May 2019. There is a lot of science, and clinical expertise guiding these experiments as well as a clear pathway to the clinic.

It would be interesting to find out whether 100% exon skipping exceeded expectations. There may even be a little excitement in Perth.