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 GTC1672
Trial ID NCT02963311
Disease Familial Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-PCSSC|Inclisiran|KJX839|Leqvio
Co-treatment Standard of Care
Location approved EU, UK, Australia, Canada, Israel, US, Saudi Arabia, Japan, China
PhasePhase2
Recruitment statusCompleted
TitleAn Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
Year2016
CountryNetherlands|South Africa|United States
Company sponsorThe Medicines Company
Other ID(s)MDCO-PCS-16-02
Vector information
VectorGalNAc

Clinical Result

Cohort 1
Administration route subcutaneous injection
Dosage ALN-PCSSC, 300mg
Pts 9
Age Child, Adult, Older_Adult

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