Gene Editing's lasting impact: a Long-Term look at NTLA-2001
NCT ID NCT05697861
First seen Aug 03, 2026 · Last updated Aug 04, 2026 · Updated 1 time
Summary
This study follows people who previously received NTLA-2001, an experimental gene-editing therapy, for a type of amyloidosis that can affect nerves or the heart. The goal is to monitor long-term safety and see if the therapy's effects on disease markers persist over time. Participants are observed without receiving new treatment, providing valuable data on the durability and potential risks of this one-time intervention.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- NTLA-2001, an investigational gene-editing therapy
- What this could lead to
- If successful, this follow-up could confirm that a single dose of gene-editing therapy provides lasting benefit and safety for people with ATTR amyloidosis.
- What could go wrong
- This is an observational follow-up, not a new treatment test. It cannot prove the therapy works, and long-term risks or loss of effect may still emerge.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
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