Eplontersen is an anti-sense oligonucleotide conjugated to an N-acetylene galactosamine ligand that is taken up by hepatocytes, the principal source of symmetrically circulating Transthyretin protein.

This conjugation increases the potency of anti-sense oligonucleotide molecules by 20 to 30 fold, allowing for use of substantially lower effective doses. 

When given subcutaneously at 45 mg every four weeks, it results in a mean 86% reduction from baseline in serum transthyretin concentration. 

In patients with ATTRV polyneuropathy the Eplontersen group consistently had lowered serum Transthyretin concentrations, less neuropathy impairment, and better quality of life than with placebo. 

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