Multiple approved therapeutic options exist for patients with ATTR-CM and hATTR-PN1,2

TTR silencers3,4

Antisense oligonucleotides (ASOs) are short, chemically modified oligonucleotides that reduce the production of TTR protein
RNA interference (RNAi) therapeutics are double-stranded small interfering RNAs (siRNAs) that reduce the production of TTR protein

TTR stabilizers1,3

TTR tetramer stabilizers are drug molecules that bind to the TTR protein, preventing the dissociation into monomers
Indication(s) vary by specific FDA-approved therapeutic.

Mechanisms to target TTR1-3

TTR silencing works upstream of tetramer formation by suppressing TTR production at its source in the liver.
TTR stabilizing works by reducing TTR dissociation and misfolding.
LEARN ABOUT A TREATMENT OPTION FOR ATTR‑CM AND hATTR‑PN
Liver cell image was AI generated.
ATTR=transthyretin-mediated amyloidosis; ATTR-CM=cardiomyopathy of transthyretin-mediated amyloidosis; FDA=Food and Drug Administration; hATTR=hereditary transthyretin-mediated amyloidosis; hATTR-PN=polyneuropathy of transthyretin-mediated amyloidosis; TTR=transthyretin; wtATTR=wild-type transthyretin-mediated amyloidosis.
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References:

  1. Kittleson MM et al. J Am Coll Cardiol. 2023;81(11):1076-1126.
  2. Kittleson MM et al. Circulation. 2020;142(1):e7-e22.
  3. Sekijima Y. J Neurol Neurosurg Psychiatry. 2015;86(9):1036-1043.
  4. Brannagan TH et al. J Peripher Nerv Syst. 2022;27(4):228-237.