New Era for Splice-Modifying Therapies, page-4

Thanks Hottod, indeed footnote 44 tell us a great deal of what we need to know [from Antisense-mediated splice intervention to treat human disease: the odyssey continues - published May 2019].

The presentation by Phylogica also tells us a great deal about what we need to know but of course many appear to have stopped listening. Odysseus was lashed to the mast and his men's ears were filled with wax.

From the presentation, the quote by Doug Ingram is important re: the delivery challenge of PMO technology. The authors of the May 2019 paper pick up the same issue, "The charge-neutral PMOs have an excellent safety profile but are rapidly cleared from the circulation, showing poor uptake into cells". Segue back to footnote 44 - who will deliver us?

There is another quote in the presentation that's worth reprising; "Haven't heard of RNA therapy yet? You will"...."After a decade of painstaking progress, the underdog is on the brink of treating a broad range of diseases" - Joshua Peters. Lets pick up again comments from the May 2019 paper; "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". The journey ahead into the eye of the storm and inherited retinal diseases will be informed by the past decade of painstaking progress. We might even be surprised by the speed of the progress and the powerful therapeutic impact of two mature and synergistic technologies that have simultaneously come of age.