MECHANISM OF ACTION (MOA)

WAINUA is the only ASO with LICA technology approved for hATTR-PN, enabling direct binding to hepatocytes1-5

ASO, antisense oligonucleotide.

subpage-topBanner-Gray-Columns

Play this video to understand how WAINUA works

WAINUA mechanism of action

WAINUA Mechanism of Action Step 1

Liver-specific targeting1,2,6

A silencer that directly binds to hepatocytes, reducing off-target exposure through GalNAc conjugation

 
WAINUA Mechanism of Action Step 2

TTR mRNA degradation2,6

Degradation of both mutant and wild-type TTR mRNA

 
WAINUA Mechanism of Action Step 3

Reduction of TTR protein production2,6

Resulting in reduced serum TTR protein and subsequent amyloid formation

Learn how WAINUA can affect serum TTR levels

View TTR suppression

arrow-buttonarrow-button

Explore the NEURO-TTRansform study design

Clinical trial design

arrow-button arrow-button

FOOTNOTES AND ABBREVIATIONS

ASGPR, asialoglycoprotein receptor; GalNAc, N-acetyl galactosamine; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis; LICA, ligand-conjugated antisense; mRNA, messenger ribonucleic acid; TTR, transthyretin.

REFERENCES

  1. 1. Viney NJ, Guo S, Tai L-J, et al. Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data. ESC Heart Fail. 2021;8(1):652-661.
  2. 2. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2026.
  3. 3. AMVUTTRA® (vutrisiran) [prescribing information]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; March 2025.
  4. 4. ONPATTRO® (patisiran) [prescribing information]. San Diego, CA: Alnylam Pharmaceuticals, Inc; September 2025.
  5. 5. TEGSEDI® (inotersen) [prescribing information]. Waltham, MA: Sobi Inc; January 2024.
  6. 6. Crooke ST, Baker BF, Xia S, et al. Integrated assessment of the clinical performance of GalNAc3-conjugated 2'-O-methoxyethyl chimeric antisense oligonucleotides: I. Human volunteer experience. Nucleic Acid Ther. 2019;29(1):16-32.