This is underscored by an FDA approval of an antisense oligo for the treatment of familial hypercholesterolemia and a current phase 3 clinical trial by Alnylam, Inc. for the treatment of TTR (Transthyretin)—Mediated Amyloidosis (see [54] for phase 1 Proof-of concept data) [55]. The gene discussed is TTR; the disease is amyloidosis.