Antisense oligonucleotides
Mode of Action

Antisense oligonucleotides (ASOs) can control the expression of therapeutically relevant genes. They suppress gene expression by enzyme-dependent degradation of target messenger RNAs (mRNAs). For that, the technology makes use of RNase H1, a cellular enzyme, to cleave the target RNA and consequently suppress the expression of encoded proteins. By inhibiting the expression of disease-inducing and -exacerbating proteins, ASOs have the potential to play a critical role as new therapeutics to treat diseases that have been non-treatable or have been treated ineffectively with currently available therapies.

Intracellular activity of ASOs

ASOs enter the target cell via endocytosis and then specifically bind to target mRNA. Afterwards, target mRNA is degraded through interaction with RNAse H. Consequently, downstream protein expression is inhibited.

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Intracellular activity of ASOs

ASOs enter the target cell via endocytosis and then specifically bind to target mRNA. Afterwards, target mRNA is degraded through interaction with RNAse H. Consequently, downstream protein expression is inhibited.

Antisense oligonucleotides
Chemistry Generations

Over the years, antisense oligonucleotides have been optimized through chemical modifications. These alterations improve ASO stability, pharmacokinetic properties and productive cellular uptake. Moreover, they reduce unwanted stimulation/activation of the immune system and other possible side effects.

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Since its inception, Secarna has become a global leader in discovering and developing the next generation ASOs.