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  • OUTLOOK

Why rare genetic diseases are a logical focus for RNA therapies

When two pharmaceutical companies turned their attention to a rare condition, they came up with different strategies for targeting RNA. Now, RNA therapies for other hereditary conditions are within the sights of the industry.
  1. Liam Drew

    1. Liam Drew is a freelance writer based in London.

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Ellie Darby-Prangnell has a genetic condition called spinal muscular atrophy. Credit: Marcus Hessenberg/Barcroft Images/Barcroft Media via Getty

In 2018, US and European regulators approved two RNA-targeted drugs for the rare genetic disease hereditary transthyretin (hATTR) amyloidosis, offering hope to around 50,000 people with the condition worldwide.

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Nature 574, S16-S18 (2019)

doi: https://doi.org/10.1038/d41586-019-03075-5

This article is part of Nature Outlook: RNA therapy, an editorially independent supplement produced with the financial support of third parties. About this content.

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