Lonvoguran ziclumeran (lonvo-z)

Name: Lonvoguran ziclumeran (lonvo-z) in Hereditary angioedema: Mechanism of action
Published: 2026-09-14T15:39:06-04:00

Hereditary angioedema

Also known as NTLA-2002

Manufacturer: Intellia Therapeutics
Regulatory submission: Rolling BLA initiated April 2026 and accepted September 2026
Launch: Anticipated in the first half of 2027
Last updated: September 19, 2026

Section

Product information and disease description Product description Product information Pharmacology

Mechanism of action

3 evidence topics · 4 sources

Ownership, therapeutic class, and proposed mechanism

“NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the KLKB1 gene in the liver” (opens the source at this quote in a new tab)

Delivery components

“lipid nanoparticles” (opens the source at this quote in a new tab)
“guide RNA” (opens the source at this quote in a new tab)
“messenger RNA that encodes the Cas9 enzyme” (opens the source at this quote in a new tab)

Hepatic targeting for novel prophylactic therapies: review

“This report reviews currently available data on hepatic-focused interventions for HAE that have advanced into human trials.” (opens the source at this quote in a new tab)