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Expert view: Call to action: establishing the protein-binding properties of oligonucleotides

Posted: 12 September 2019 | | No comments yet

The number of oligonucleotide-based platforms on the market is constantly increasing and has led to the emergence of innovative nucleic acid-based therapeutic modalities such as CRISPR-Cas, sgRNAs or mRNAs. 

The increasing number of investigational new drugs (INDs) and investigational medicinal product dossiers (IMPDs) reflects the importance and potential of these technologies. Over the last three years, several nucleic acid-based drugs have been approved, including three antisense oligonucleotides (Spinraza®, Tegsedi® and Waylivra®) and one lipid nanoparticle (LNP)-formulated siRNA for the treatment of hATTR amyloidosis (Onpattro®).

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