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Lonvoguran ziclumeran (lonvo-z)

Hereditary angioedema

Also known as NTLA-2002
Last updated
82 sources

Product information and disease descriptionProduct descriptionProduct informationPharmacology

Pharmacodynamics

4 evidence topics · 5 sources

Phase 2: plasma kallikrein change
OutcomeNTLA-2002 25 mgNTLA-2002 50 mgPlacebo
Plasma kallikrein change from baseline, week 16“-55%”“-86%”“2%”
Phase 3: plasma kallikrein time course

“reached a steady state by week 5 and remained stable through the data cutoff.” (opens the source at this quote in a new tab)

Preclinical studies: editing durability after partial hepatectomy in huKLKB1 mice

“KLKB1 editing is retained following partial hepatectomy-induced liver regeneration, further demonstrating the durability of CRISPR/Cas9-induced gene editing with NTLA-2002.” (opens the source at this quote in a new tab)

Preclinical studies: KLKB1 gene knockout in nonhuman primates

“Kallikrein activity reduction sustained for at least 77 weeks in NHPs” (opens the source at this quote in a new tab)