Gene-Editing breakthrough: single dose may stop rare fatal disease
NCT ID NCT04601051
First seen Feb 01, 2026 · Last updated May 24, 2026 · Updated 11 times
Summary
This early-stage trial tested a new gene-editing drug, NTLA-2001, in 72 adults with hereditary ATTR amyloidosis, a condition that causes nerve damage and heart problems. The goal was to see if a single dose is safe and can lower the abnormal protein (TTR) that builds up in the body. While not a cure, this approach aims to control the disease by reducing the harmful protein.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Clinical Trial Site
Paris, France
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Clinical Trial Site
Auckland, New Zealand
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Clinical Trial Site
Umeå, Sweden
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Clinical Trial Site
London, United Kingdom
Conditions
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