In-body gene editing
Human trial
- What happened
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.
- 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- How far from your life
Real-world testing
Gene editing delivered by lipid nanoparticles is on step 3 of 5. Could reach you in a few years if the tests go well.
- Source
“The treatment was administered safely, and he is now growing well and thriving.”Children's Hospital of Philadelphia, 15 May 2025
The bigger picture
More on In-body gene editing
On step 3 of 5: Real-world testing. Everything we track on it
- 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 - 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