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Gene editing delivered by lipid nanoparticles

The same lipid nanoparticles that carry mRNA vaccines can carry gene-editing tools to the liver. Given by infusion, they edit a gene inside the body. Intellia's nex-z uses CRISPR this way to switch off the gene behind ATTR amyloidosis.

NeurologyLive, 28 Jan 2026
Gene editing carrier · how far it has got3/5Real-world testing

How far from your life: Could reach you in a few years if the tests go well.

Independently sourced

From lab to your life

Why it’s on step 3

  1. 1Lab discovery
  2. 2Working prototype
  3. 3Real-world testing
  4. 4Approved or on sale
  5. 5Everyday life

The rule

Tested in people in a clinical trial, in real conditions in a field or road trial, or delivered as samples to named customers.

  • The FDA lifted the hold on MAGNITUDE-2, the trial in the nerve form of the disease, with closer monitoring of liver tests. The hold on the heart trial, MAGNITUDE, stayed. NeurologyLive, 28 Jan 2026
  • Doctors at Children's Hospital of Philadelphia and Penn Medicine treated a baby, KJ, born with a rare metabolic disease, with a gene-editing therapy made just for him and delivered to his liver in lipid nanoparticles. It was given safely, and he is growing well. The case was published in the New England Journal of Medicine. Children's Hospital of Philadelphia, 15 May 2025

The short version

What you need to know

Why it matters

Intellia hopes nex-z could be a one-time treatment for people with the inherited nerve disease ATTRv-PN.

NeurologyLive, 28 Jan 2026
Is it really nano?

Truly nano

The nanoparticle is the delivery system that gets the editing tools into liver cells.

Children's Hospital of Philadelphia, 15 May 2025
Who’s behind it

Intellia Therapeutics with Regeneron (nex-z); Children's Hospital of Philadelphia and Penn Medicine (a custom treatment for one baby)

On the record

Every step, dated

Newest first. Company-reported means the only source so far is the company itself.

  1. Human trial

    The FDA lifted the hold on MAGNITUDE-2, the trial in the nerve form of the disease, with closer monitoring of liver tests. The hold on the heart trial, MAGNITUDE, stayed.

    NeurologyLive, 28 Jan 2026
  2. Setback

    The FDA put Intellia's two phase 3 trials of nex-z on hold after a patient had severe liver injury. The patient died on November 5, 2025.

    NeurologyLive, 28 Jan 2026
  3. Human trial

    Doctors at Children's Hospital of Philadelphia and Penn Medicine treated a baby, KJ, born with a rare metabolic disease, with a gene-editing therapy made just for him and delivered to his liver in lipid nanoparticles. It was given safely, and he is growing well. The case was published in the New England Journal of Medicine.

    Children's Hospital of Philadelphia, 15 May 2025

Who makes it

The companies

Sources

Where every line comes from

Each source with the passage, word for word, that backs what we wrote.

  1. NeurologyLive, 28 Jan 2026investigational CRISPR/Cas9 gene editing therapy delivered via a lipid nanoparticle (LNP) nonviral system
  2. Children's Hospital of Philadelphia, 15 May 2025Within six months, their team designed and manufactured a base editing therapy delivered via lipid nanoparticles

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