Basic Information

Gene symbol PCSK9 Synonyms FH3, FHCL3, HCHOLA3, LDLCQ1, NARC-1, NARC1, PC9 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description proprotein convertase subtilisin/kexin type 9

GTO ID GTC3054
Trial ID NCT05398029
Disease Heterozygous Familial Hypercholesterolemia | Atherosclerotic Cardiovascular Disease
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapyGene editing
Treatment VERVE-101
PhasePhase1
Recruitment statusActive, Not Recruiting
TitleOpen-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia
Year2022
CountryNew Zealand|United Kingdom
Company sponsorVerve Therapeutics, Inc.
Other ID(s)VT-1001
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort 1
Administration route intravenous infusion
Pts 44
Age Adult, Older_Adult

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