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Gene Editing's lasting impact: a Long-Term look at NTLA-2001

NCT ID NCT05697861

Knowledge-focused Sponsor: Intellia Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Study facts

What this study's own registry entry says, in plain language.

Participants

About 72 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2023

Expected to finish

Mar 2038

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Participating sites in an Intellia-sponsored clinical study of NTLA-2001.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received. 2. A subject has provided informed consent for the LTFU study. Exclusion Criteria: None

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Conditions

The condition(s) this trial relates to.

Amyloid Neuropathies, Familial familial amyloid neuropathy wild type ATTR amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Transthyretin-related (ATTR) familial amyloid cardiomyopathy Transthyretin-related (ATTR) familial amyloid polyneuropathy Wild-type transthyretin cardiac amyloidosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    4 sites in 4 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Clinical Trial Site

    Paris, France

  • Clinical Trial Site

    Auckland, New Zealand

  • Clinical Trial Site

    Umeå, Sweden

  • Clinical Trial Site

    London, United Kingdom

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