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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial

NCT ID NCT06672237

Recruiting now Disease control Sponsor: Intellia Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatment or a placebo infusion. The study measures changes in nerve function and quality of life over time to see if the therapy can slow or stop disease progression.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
nexiguran ziclumeran (NTLA-2001), a gene-editing therapy given as a single IV infusion
What this could lead to
If it works, this could be a one-time treatment that stops or slows nerve damage in people with this rare, inherited disease.
What could go wrong
This is still an early-phase 3 trial with only 60 participants. The placebo group means some won't get the real treatment. Long-term safety and effectiveness are not yet known.

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.

Amyloid Neuropathies Amyloid Neuropathies, Familial amyloidosis familial amyloid neuropathy hereditary amyloidosis hereditary disease inborn errors of metabolism inherited neurodegenerative disorder metabolic disease nervous system disorder neurodegenerative disease neuromuscular disease peripheral nervous system disorder polyneuropathy

As listed by the trial registrant

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

AMYLOID NEUROPATHIES AMYLOID NEUROPATHIES, FAMILIAL AMYLOIDOSIS AMYLOIDOSIS, FAMILIAL AMYLOIDOSIS, HEREDITARY GENETIC DISEASE, INBORN METABOLIC DISEASES METABOLISM, INBORN ERRORS NERVE DISORDERS NERVOUS SYSTEM DISEASE NERVOUS SYSTEM DISEASES NEURODEGENERATIVE DISEASE NEURODEGENERATIVE DISEASE, HEREDITARY NEURODEGENERATIVE DISEASES NEUROMUSCULAR DISEASE NEUROMUSCULAR DISEASES NEUROMUSCULAR DISEASES (NMD) PERIPHERAL NERVOUS SYSTEM DISEASE PERIPHERAL NERVOUS SYSTEM DISEASES POLYNEUROPATHIES

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Chang Gung Medical Foundation - Linkou Branch

    ACTIVE_NOT_RECRUITING

    Taoyuan, Taiwan

  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto

    RECRUITING

    Ribeirão Preto, Brazil

  • Hospital Británico de Buenos Aires

    RECRUITING

    Buenos Aires, Argentina

  • Hospital El Cruce

    RECRUITING

    San Juan Bautista, Argentina

  • Hospital Italiano de Buenos Aires (HIBA)

    RECRUITING

    Buenos Aires, Argentina

  • Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)

    RECRUITING

    Campinas, Brazil

  • Instituto de Educacao, Pesquisa e Gestao em Saude

    RECRUITING

    Rio de Janeiro, Brazil

  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)

    RECRUITING

    Tlalpan, Mexico

  • National Taiwan University Hospital

    ACTIVE_NOT_RECRUITING

    Taipei, Taiwan

  • National University Hospital

    RECRUITING

    Singapore, Singapore

  • PSEG Centro de Pesquisa Clinica S.A.

    RECRUITING

    São Paulo, Brazil

  • Singapore General Hospital

    RECRUITING

    Singapore, Singapore

  • Siriraj Hospital

    RECRUITING

    Bangkok, Thailand

  • Taipei Veterans General Hospital

    ACTIVE_NOT_RECRUITING

    Taipei, Taiwan

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