One-Shot gene therapy tested to halt debilitating protein disease
NCT ID NCT04601051
First seen Feb 01, 2026 · Last updated Apr 15, 2026 · Updated 8 times
Summary
This early-stage study tested a new, single-dose gene therapy called NTLA-2001 in 72 adults with hereditary forms of amyloidosis, a disease where a faulty protein damages nerves and the heart. The main goals were to check the treatment's safety, how the body processes it, and to see if it could reduce levels of the harmful protein. Researchers also looked for early signs that the therapy might improve patients' nerve function, heart health, and quality of life.
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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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