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This website is intended for UK Healthcare Professionals only.
The website contains both promotional and non-promotional content.
If you are a patient or a carer of a patient prescribed an AstraZeneca product, please visit myazmed.co.uk
For any other UK residents please visit astrazeneca.co.uk
This website is intended for UK Healthcare Professionals only. Other UK residents please visit astrazeneca.co.uk
WAINZUA is a TTR gene silencer designed to directly bind to hepatocytes and reduce the production of TTR protein.1
The TTR protein, which can misfold to cause ATTR amyloidosis, is primarily synthesised by the liver and normally circulates as a tetramer involved in transporting vitamin A and thyroxine.4,5
WAINZUA uses GalNAc ligand-conjugated antisense (LICA) technology, where the antisense oligonucleotide (ASO) is conjugated to the ligand
N-acetylgalactosamine. Due to this structure, WAINZUA is designed for targeted and enhanced uptake by hepatocytes, the primary source of systemically circulating TTR protein.2,6
Once within hepatocytes, WAINZUA selectively binds to the TTR mRNA causing the degradation of both the mutant and wild-type TTR mRNA. This prevents the synthesis of TTR protein in the liver, resulting in significant reductions in the levels of mutated and wild-type TTR protein secreted by the liver
into circulation.1,6
WAINZUA is the only TTR silencer with LICA technology enabling direct binding to hepatocytes, targeting ASGP receptors on the cell surface.1-3,6,7
The clinical benefit of WAINZUA was demonstrated in the NEURO-TTRansform Phase III clinical study.1,6
By lowering serum TTR levels, WAINZUA helps to slow the accumulation of amyloid fibrils, which contribute to the progression of neuropathy and cardiac complications in patients with mixed phenotypic presentation of ATTRv amyloidosis.1,6,8–10
ASGP=asialglycoprotein; ASO=antisense oligonucleotide; ATTRv=hereditary transthyretin amyloidosis; ATTRv-PN=hereditary transthyretin amyloidosis with polyneuropathy; GaINAc=N acetyl galactosamine; LICA=ligand conjugated antisense; mRNA=messenger ribonucleic acid; TTR=transthyretin.
GB-68497 | November 2025
Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to AstraZeneca by visiting https://contactazmedical.astrazeneca.com/ or by calling 0800 783 0033.
This website was created for UK HCPs and has been designed to provide information to educate, empower and enable them in the great work they are doing for patients across a range of therapy areas. This website is intended for doctors, nurses, and pharmacists in the UK. Other UK residents please visit astrazeneca.co.uk.
