Gene editing · Gene editing: transthyretin (TTR) · developed by Intellia Therapeutics
NTLA-2001 is a lipid nanoparticle formulation delivering mRNA encoding CRISPR-Cas9 and a guide RNA targeting transthyretin (TTR). It edits the TTR gene in vivo to reduce misfolded TTR protein accumulation. It is studied in transthyretin amyloidosis with cardiomyopathy and neuromuscular manifestations.
Summary written with AI from the sources listed at the end of this page.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT06672237 | Phase 3 | Neuromuscular Disease | Recruiting | NTLA |
| NCT06128629 | Phase 3 | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Recruiting | NTLA |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
AmyloidosisAmyloidosis, FamilialAmyloidosis, HereditaryGenetic Disease, InbornNerve DisordersNervous System DiseaseNeurodegenerative DiseaseNeurodegenerative Disease, HereditaryNeuromuscular DiseaseNeuromuscular Diseases (NMD)PolyneuropathiesTransthyretin Amyloidosis (ATTR) With Cardiomyopathy
Updated Oct 3, 2026. Something wrong or missing? Report it on the NTLA page.
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