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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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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