There are two major types of antisense oligonucleotides;
- PS-ASOs - negatively charged with phosphorothioate backbone
- Morpholinos aka PMOs - neutrally charged with phosphorodiamidate backbone
The Ionis platform is built upon PS-ASOs whereas the Sarepta platform is built upon PMOs. Sarepta have added a peptide to improve delivery of the naked PMO so we get PPMOs. There is also a new kid on the block with a CPP-PMO.
The problem with PMOs is that they have poor cellular uptake. What happens if you solve the delivery problem and create an effective, long lasting drug with an excellent safety profile? Once you have a design you like and establish the backbone chemistry, all you need to do is change the sequence of nucleotides for a new target address. Do you now have a morpholino platform which in time will rival the Ionis platform of PS-ASOs?
The age of RNA therapeutics is opening before us and PYC have found entry with the potential to develop a platform of morpholino drugs in one of the fastest growing therapeutic classes on the planet.
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