CRISPR therapy shows promise against debilitating amyloid disease
NCT ID NCT04601051
First seen Feb 01, 2026 · Last updated May 12, 2026 · Updated 10 times
Summary
This early-stage study tested a single dose of NTLA-2001, a gene-editing treatment, in 72 adults with hereditary transthyretin amyloidosis, a condition that causes nerve damage and heart problems. The goal was to see if the treatment is safe and can lower a harmful protein called TTR. Participants received one infusion and were monitored for side effects and changes in their condition.
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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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