Modulation of RNA holds the promise for opening up entirely new concepts for pharmaceutical intervention. Providing the precision tools to modulate this wealth of new RNA targets is our mission. Our development programs focus on severe neuromuscular diseases.
Our proprietary alpha anomeric-bicyclo DNA platform reaches unprecedented precision in RNA modulation by eliminating common side effects of current oligonucleotide chemistries and by increasing the specificity and effectiveness of treatment.
Eliminating common side effects
All state of the art oligonucleotide chemistries require a modification of the natural phosphodiester linkage found in DNA and RNA in order to prevent rapid degradation by nucleases that are present in all biological fluids.
Increasing specificity and efficacy
The Watson Crick base pairing mechanism allows for the design of highly specific compounds for interference with RNA, which are called oligonucleotides.