Tom3 schreef op 21 oktober 2020 07:57:
Weer een belangrijke bijdrage van BioBoyScout: het toont aan dat de anti sense benadering van rna therapie een belangrijk nadeel heeft tov rnai. TRIM is in feite een tailor made constructie die leidt tot (waarschijnlijk) minder bijwerkingen dan het nieuwste anti sense platform.
Citaat ontleend aan het Yahoo forum:
Additional observations on the Ionis ENaC news. If you go to the Ionis webpage and look under the tab "Ionis Innovation" and select "Optimizing Antisense", you'll see an explanation of the technology that Ionis uses for its drugs. There are four different technologies: Gen 2+, Gen 2+ LICA, Gen 2.5, and Gen 2.5 LICA. What is LICA? Ionis explains it on that page, it's basically their antisense drugs with targeting ligands (LIgand Conjugated Antisense).
If you recall, Arrowhead in its presentations well over a year ago showed plenty of data on how using targeting ligands with their triggers was much more effective than without using targeting ligands. This is because you're able to target drug into areas where you want it to go. Without a targeting ligand, the drug ends up being much more dispersed throughout the body. The drug may "work" without a targeting ligand, but it will be much less effective because less of the drug gets to where you're wanting it to go (i.e. the liver, or the lung).
With that said, if you go to Ionis's pipeline page, head down to their ENaC drug and click on it, it will expand to show additional information about the drug. This additional info states that the technology used for its ENaC drug is Gen 2.5. It is NOT Gen 2.5 LICA or Gen 2+ LICA, therefore Ionis's ENaC drug is without a targeting ligand. What does this mean? It means that the drug is not specifically targeted like Arrowhead's is to the lung. No doubt Ionis's drug will get absorbed by the lung, but it will also get dispersed elsewhere and will most likely not get the same concentration of to the lung as Arrowhead can.