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Editorial
. 2025 Sep 25;66(3):2501101.
doi: 10.1183/13993003.01101-2025. Print 2025 Sep.

A cure for alpha-1? Novel therapeutics in alpha-1 antitrypsin deficiency

Reem Alluhibi  1   2 , Aileen Marshall  3 , David A Lomas  4 , John R Hurst  4
Affiliations
Editorial

A cure for alpha-1? Novel therapeutics in alpha-1 antitrypsin deficiency

Reem Alluhibi et al. Eur Respir J. .

Abstract

Novel therapies targeting protein misfolding, RNA and DNA offer exciting advances towards a cure for AATD, potentially addressing both liver and lung disease https://bit.ly/4on5Gn5

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Conflict of interest statement

Conflict of interest: R. Alluhibi has no potential conflicts of interest to disclose. A. Marshall is local PI for the TAK 999 3001 clinical trial. D.A. Lomas reports support for the present study from Medical Research Council (UK), Alpha-1 Foundation (US), National Institute for Health Research and Biomarin, royalties or licences from Biomarin, consultancy fees from Biomarin, support for attending meetings from Alpha-1 Foundation, patents planned, issued or pending (PCT/GB2019/051761 describing small molecules to block polymerisation of Z a1-antitrypsin including BMN349) with Biomarin, and leadership roles with Asthma and Lung UK, Alpha-1 Foundation (US), Anti-Doping Laboratory Qatar (ADQL), Association of Physicians of Great Britain and Ireland and Rosetrees. J.R. Hurst reports grants from AstraZeneca, consultancy fees from AstraZeneca, Boehringer Ingelheim, Chiesi, GSK, Sanofi-Regeneron and Takeda, payment or honoraria for lectures, presentations, manuscript writing or educational events from AstraZeneca, Boehringer Ingelheim, Chiesi, GSK, Sanofi-Regeneron, Sanofi and Takeda, and support for attending meetings from AstraZeneca.

Figures

FIGURE 1
FIGURE 1
An overview of novel liver-targeted alpha-1 antitrypsin deficiency treatment strategies. siRNA: small interfering RNA; AAT: alpha-1 antitrypsin; GalNAc: N-acetylgalactosamine; RNAi: RNA interference; ADAR: adenosine deaminase acting on RNA; RISC: RNA-induced silencing complex.
See this image and copyright information in PMC

References

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