Gene editing breakthrough? CRISPR therapy targets rare amyloidosis
NCT ID NCT04601051
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests NTLA-2001, a CRISPR-based gene editing therapy, in 72 adults with hereditary ATTR amyloidosis—a condition where abnormal protein builds up in nerves or the heart. The treatment aims to turn off the faulty gene to reduce protein levels. The study focuses on safety and how the body processes the drug, not yet on curing the disease.
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 (a CRISPR gene editing therapy delivered via lipid nanoparticles)
What this could lead to
If successful, this could lead to a one-time treatment that reduces the harmful protein buildup in ATTR amyloidosis, potentially controlling the disease without lifelong medication.
What could go wrong
This is an early Phase 1 trial with only 72 participants, so safety and effectiveness are not yet proven. Gene editing is new and may have unforeseen risks or side effects.
Disclaimer
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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.
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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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
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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