Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

Molecular Therapy

Volumn 25, Issue 1, 2017, Pages 71-78

  Zimmermann, Tracy S ^a   Karsten, Verena ^a   Chan, Amy ^a   Chiesa, Joseph ^b   Boyce, Malcolm ^c   Bettencourt, Brian R ^a   Hutabarat, Renta ^a   Nochur, Saraswathy ^a   Vaishnaw, Akshay ^a   Gollob, Jared ^a  

^a ALNYLAM PHARMACEUTICALS   (United States)

^b 206 Carnegie Center   (United Kingdom)

^c Hammersmith Medicines Research Limited and Trio Medicines Limited   (United Kingdom)

Author keywords

asialoglycoprotein receptor; GalNAc siRNA; revusiran; RNAi

Indexed keywords

N ACETYLGALACTOSAMINE; PLACEBO; PREALBUMIN; REVUSIRAN; SMALL INTERFERING RNA; ASIALOGLYCOPROTEIN RECEPTOR;

ADULT; ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG MECHANISM; DRUG SAFETY; DRUG TOLERABILITY; ELIMINATION HALF-LIFE; ERYTHEMA; FEMALE; GENE DELIVERY SYSTEM; GENE TARGETING; HEADACHE; HUMAN; HUMAN EXPERIMENT; INJECTION SITE REACTION; LIVER CELL; MALE; NORMAL HUMAN; PHASE 1 CLINICAL TRIAL; PROTEIN BLOOD LEVEL; RANDOMIZED CONTROLLED TRIAL; SWELLING; CHEMISTRY; CLINICAL TRIAL; DRUG MONITORING; GENE SILENCING; GENETICS; METABOLISM; MIDDLE AGED; YOUNG ADULT;

ACETYLGALACTOSAMINE; ADULT; ASIALOGLYCOPROTEIN RECEPTOR; DRUG MONITORING; FEMALE; GENE SILENCING; HEALTHY VOLUNTEERS; HEPATOCYTES; HUMANS; MALE; MIDDLE AGED; PREALBUMIN; RNA, SMALL INTERFERING; YOUNG ADULT;

EID: 85016271522     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2016.10.019     Document Type: Article

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