Alnylam Pharmaceuticals, Inc.

Genzyme Corporation

Donald Foster

Martin A Maier

Klaus Charisse

Stuart Milstein

Kallanthottathil G Rajeev

Kevin Fitzgerald

Brian R Bettencourt

Satyanarayana Kuchimanchi

Muthiah Manoharan

James Butler

Vasant R Jadhav

Gregory Hinkle

Mark K Schlegel

Alfica Sehgal

Anna Borodovsky

Muthusamy Jayaraman

Jayaprakash K Nair

Shigeo Matsuda

Alexander V Kel'in

Maja Janas

Christopher Brown

Shannon Fishman

Akin Akinc

Ivanka Toudjarska

Christopher S Theile

Muthiah Monaharan

COMPOSITIONS AND METHODS FOR SILENCING UGT1A1 GENE EXPRESSION

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 present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the AGT gene. The invention also relates to methods of using such RNAi agents to inhibit expression of an AGT gene and to methods of preventing and treating an AGT-associated disorder, e.g., high blood pressure.

Angiotensinogen (AGT) 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

One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2'-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.

Modified RNA agents with reduced off-target effect

The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the angiotensinogen (AGT) gene, and methods of using such RNAi agents to inhibit expression of AGT and methods of treating subjects having an AGT-associated disorder, e.g., hypertension.

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Attleboro, MA, United States of America

Donald Foster