Ann: Successful Competitive Drug Evaluation in Animals, page-8

Delivery, delivery and delivery.

We have heard about the delivery challenge in the field of RNA therapeutics and more particularly antisense oligonucleotides. I doubt whether its PYC which is the only entity excited by the latest developments.

Authors from CMMIT and the Perron Institute have recently released an article for publication titled In Vitro Validation of Phosphorodiamidate Morpholino Oligomers. The following is an excerpt;
"It is often stated that there are three great challenges to genetic therapies: delivery, delivery and delivery. While the design of the therapeutic vector, plasmid or antisense oligomer is a fundamentally crucial aspect, without efficient and effective delivery to the target tissue or cells, the most therapeutically active compound may appear inert."

Its difficult to escape the significance of the latest announcement by PYC when judged against the observation by the aforementioned authors that the delivery challenge must be resolved. Yes, you can keep increasing the dosage rate for a naked ASO to get some kind of efficacy but only at ever increasing levels of toxicity. The recent judgement by the FDA in relation to golodirsen provides a level of guidance in relation to the issues of efficacy and toxicity in the antisense space.

If the developments in RNA therapeutics could be described as a major weather event, we would currently be in the eye of the cyclone. Sarepta may have to come on board or otherwise batten down the hatches.