Alnylam Pharmaceuticals, Inc.

Curis, Inc.

Wave Life Sciences Ltd.

Arbutus Biopharma Corporation

Tekmira Pharmaceuticals Corporation

Maria Frank-Kamenetsky

Kevin Fitzgerald

Stuart Milstein

William Querbes

Brian R Bettencourt

Svetlana Shulga Morskaya

Akin Akinc

Pamela Tan

Victor E Kotelianski

Birgit Bramlage

Muthiah Manoharan

Kallanthottathil G Rajeev

Philipp Hadwiger

Martin A Maier

Antonin De Fougerolles

Stephen Colin Price

Chandra Vargeese

Klaus Charisse

Edward Andrew Boyd

Lee L Rubin

Oivin M Guicherit

Anthony David Baxter

John Harry Alexander Stibbard

Jeffery Porter

Hans-Peter Vornlocher

Satyanarayana Kuchimanchi

Genliang Lu

Anke Geick

Anna Borodovsky

Naoki Iwamoto

Subramanian Marappan

Ingo Roehl

David Charles Donnell Butler

Jason Jingxin Zhang

Christopher J Francis

Hailin Yang

Gregory Lawrence Verdine

Dinah Wen-Yee Sah

Meena

Dinah Sah

Sethumadhavan Divakaramenon

Kallanthottahil G Rajeev

Aaron Jay Morris

Vinod Vathipadiekal

Luciano Henrique Apponi

Young Jin Cho

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

Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

Oligonucleotide compositions and methods thereof

Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.

Oligonucleotide compositions and methods of use thereof

Growing community of inventors

Brookline, MA, United States of America

Maria Frank-Kamenetsky