Not only was a Nobel Prize awarded for the discovery of gene silencing by short interfering RNA (siRNA), but siRNA agents also have blockbuster potential because they can successfully treat diseases that were previously untreatable. But the developments to get there were a long, rocky road.
However, siRNA drugs can so far only be applied locally, e.g. in the eye or under the skin, or introduced into liver cells via the bloodstream. PRAMOMOLECULAR has developed a technology with which siRNAs can also silence disease-causing proteins in lung, heart and pancreas tissue. This enables us to develop new drugs against lung and pancreatic cancer.
Problematic delivery of RNA therapeutics through nanoparticles.
The Pharmazeutische Zeitung currently describes that a substance used as a stabilizer in the nano-particles in which RNA vaccines have to be packaged, PEG, is the probable trigger for the (fortunately few) anaphylactic shocks in the BioNTech vaccine.
The introduction of an other class of RNA therapeutics, siRNAs (short interfering RNAs), which can specifically silence disease proteins, into the target cells is also a challenge.
With particularly simply constructed delivery systems, such as those developed by PRAMOMOLECULAR, the transport molecules - fatty acids - are coupled directly to the siRNA. This means that they require neither the interference-prone packaging into nano-particles nor additives .
With these simple, covalent delivery systems, it will probably not be possible to introduce mRNAs into the cell, as mRNAs are considerably larger than siRNAs, but short antisense oligonucleotides and siRNAs that enable the silencing of metabolic diseases and probably also of infections and of defined cancers.
@ Felix Noak