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TREATING ATTR1,2



Multiple approved therapeutic options exist for 
patients with ATTR‑CM and hATTR‑PN

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 stabilizers2,3

  • 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.
  Silencers Stabilizers
FDA-approved to treat RNAis ASOs Tetramer stabilizer
wtATTR-CM
 
  
 
hATTR-CM
 
  
 
hATTR-PN
 
 
  

Mechanisms to target TTR1-3

TTR silencing suppresses TTR production at its source in the liver.1-3
TTR stabilizing works by reducing TTR dissociation and misfolding.1-3

LEARN ABOUT A TREATMENT OPTION FOR ATTR‑CM AND hATTR‑PN

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Liver cell image was AI generated.
ATTR=transthyretin-mediated amyloidosis; ATTR‑CM=cardiomyopathy of transthyretin-mediated amyloidosis; hATTR=hereditary transthyretin-mediated amyloidosis; hATTR-CM=cardiomyopathy of transthyretin-mediated amyloidosis; hATTR-PN=polyneuropathy of transthyretin-mediated amyloidosis; TTR=transthyretin; wtATTR=wild-type transthyretin-mediated amyloidosis.

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.