Hi Hottod,
I remember reading the article you highlighted a few months ago below. I had another read of it on the weekend and I'm glad I did. Thank you for sharing.
Simply amazing how fast that scientist was able to develop that drug. I found the section on dianophore fascinating. Interesting to note that all the approved drugs use old chemistry.
"And since changing the sequence, or pharmacophore, of an oligonucleotide is easy, when scientists land on a really great dianophore, “that’s when you see these really rapid explosions in the field,” Watts says.
That explosion is underway with oligonucleotides targeting diseases in the spine and brain, thanks to Spinraza. It’s also ongoing with antisense drugs designed to treat liver diseases, because of a molecule called N-acetylgalactosamine, or GalNAc, that selectively shuttles oligonucleotides into hepatocytes, a kind of liver cell."https://cen.acs.org/business/antisense-oligonucleotides-hit-stride/96/i45 Interesting with regards to what the company highlighted on page two in the announcement dated 17 January 2019.
Quick recap...
* A diagram highlighting targeted delivery in the liver.
* The GalNac cargo was what bound to the Asialoglycoprotein receptor and as a result increased its potency and delivery into the liver cell by 30-60 fold.
* It improved the uptake of cargo over conventional CPPs.
* It was able to do so more efficiently directly into the cell type of interest i.e., the liver cell.
* PYC's second generation peptides are able to deliver 25-150 times (in vitro) as much cargo into a target cell compared to a cargo alone.
How effective will PYC's technology be? I guess it all comes down to the upcoming in-vivo read-outs.
Tony
(20min delay)