Ligand Conjugated Antisense Oligonucleotide for the Treatment of Transthyretin Amyloidosis: Preclinical and Phase 1 Data

AIMS: (ION-682884) is a ligand-conjugated antisense drug designed for receptor-mediated uptake by hepatocytes, the primary source of circulating transthyretin (TTR). Enhanced delivery of the antisense pharmacophore is expected to increase drug potency and support lower, less frequent dosing in treatment. METHODS AND RESULTS: . CONCLUSIONS: for the treatment of ATTR polyneuropathy and cardiomyopathy.