Alnylam Pharmaceuticals, Inc.

Icahn School of Medicine at Mount Sinai

Arbutus Biopharma Corporation

Tekmira Pharmaceuticals Corporation

Kevin Fitzgerald

Brian R Bettencourt

Stuart Milstein

William Querbes

Martin A Maier

Maria Frank-Kamenetsky

Gregory Hinkle

Muthiah Manoharan

James Butler

Svetlana Shulga Morskaya

Kallanthottathil G Rajeev

Klaus Charisse

Satyanarayana Kuchimanchi

Akin Akinc

Donald Foster

Vasant R Jadhav

Pamela Tan

Birgit Bramlage

Anna Borodovsky

Stephanie Williams

Robert J Desnick

Victor E Kotelianski

Makiko Yasuda

Abigail Liebow

Jayaprakash K Nair

Muthusamy Jayaraman

David V Erbe

Alexander V Kel'in

Huilei Xu

Alfica Sehgal

Adam Castoreno

Kirk Brown

Shannon Fishman

Shigeo Matsuda

Mark K Schlegel

Rubina G Parmar

Maja Janas

Christopher S Theile

Timothy Ryan Mooney

Tuyen Nguyen

Jennifer Willoughby

Hans-Peter Vornlocher

Tracy Stage Zimmermann

Satya Kuchimanchi

Christopher Brown

Mark Keating

Mangala Meenakshi Soundarapandian

Patrick Haslett

John Benson

Rajeev G Kallanthottathil

Svetlana Shulga-Morskaya

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.

Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof

The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit expression of PCSK9 and methods of treating subjects having a lipid disorder, such as a hyperlipidemia.

PCSK9 iRNA compositions and methods of use thereof

The invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs at one or more internal positions on at least one strand, optionally via a linker or carrier.

Extrahepatic delivery

The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting a xanthine dehydrogenase (XDH) gene, and methods of using such double stranded RNAi agents to inhibit expression of an XDH gene and methods of treating subjects having an XDH-associated disease.

Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof

One aspect of the present invention relates to a double stranded iRNA agent comprising an antisense strand which is complementary to a target gene; a sense strand which is complementary to said antisense strand; and one or more lipophilic moieties conjugated to one or more internal positions on at least one strand, optionally via a linker or carrier. Another aspect of the invention relates to a method of gene silencing, comprising administering to a cell or a subject in need thereof a therapeutically effective amount of the lipophilic moieties-conjugated double-stranded iRNAs.

The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.

Complement component C5 iRNA compositions and methods of use thereof

The invention relates to double stranded ribonucleic acid (dsRNA) compositions targeting a glucokinase (GCK) gene, as well as methods of inhibiting expression of a glucokinase (GCK) gene, and methods of treating subjects having a glycogen storage disease (GSD), e.g., type Ia GSD.

Glucokinase (GCK) iRNA compositions and methods of use thereof

The invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.

Serpina1 iRNA compositions and methods of use thereof

The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as &#x3b2;-thalassemia or hemochromatosis.

TMPRSS6 iRNA compositions and methods of use thereof

The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the HAO1 gene, and methods of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.

Compositions and methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression

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Brookline, MA, United States of America

Kevin Fitzgerald