The first siRNA drug, patisiran, was approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat a debilitating genetic disorder called hereditary transthyretin amyloidosis.

which is used in patisiran. But most of the pipeline uses an elegant (Alnylam-patented) solution involving a conjugate which attaches the siRNA to a sugar ligand. This molecule, called GalNAc is ...

Now results from its phase 3 Apollo trial show patisiran, which is co-developed with Sanofi, reduced nerve pain in the rare inherited disease familial amyloid polyneuropathy (FAP). If approved ...

The first siRNA drug, patisiran, was approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat a debilitating genetic disorder called hereditary transthyretin amyloidosis.

It wasn’t until 2018 that Alnylam gained approval for the first siRNA drug, the hATTR amyloidosis therapeutic patisiran. Since then, interest in siRNA therapies has overtaken ASOs.

Reporting in Nature, Kumar and colleagues have now developed a method to target small-interfering RNA (siRNA) to the CNS, providing a safe and non-invasive approach for the delivery of this new ...