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Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Hereditary angioedema (HAE) due to C1 inhibitor deficiency — prophylaxis Clinical stage: Phase 3 Modality: Gene editing therapy (in vivo CRISPR-Cas9 via lipid nanoparticle) Mechanism / Target: Single-dose in vivo knock-out of KLKB1 gene (encoding plasma kallikrein) in hepatocytes; permanently reduces plasma kallikrein, preventing bradykinin generation and HAE attacks Route / form: Single intravenous infusion (LNP delivery to liver) Differentiation: Potentially curative single-dose; permanent reduction vs chronic prophylaxis (lanadelumab, berotralstat); Phase 1/2 showed >90% KLKB1 reduction sustained >2 years
Manufacturer #39179

Pioneering in vivo CRISPR gene editing company targeting genetic diseases at the DNA level. Advances single-administration therapies aimed at permanent correction via lipid nanoparticle delivery. Wholly owned HAE programme independent from its TTR collaboration.
 

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