Alnylam Pharmaceuticals, Inc.

Novartis Ag

Arrowhead Pharmaceuticals, Inc.

Monsanto Technology LLC

Regeneron Pharmaceuticals, Inc.

Arrowhead Research Corporation

Genzyme Corporation

Gregory Hinkle

Stuart Milstein

Kevin Fitzgerald

Martin A Maier

Antonin De Fougerolles

Satyanarayana Kuchimanchi

Brian R Bettencourt

Muthiah Manoharan

John Louis Diener

James Butler

Emma Hickman

Anne-Marie Pulichino

Andrew Griffin Sprague

Kallanthottathil G Rajeev

Klaus Charisse

Tracy Stage Zimmermann

Ping Zhu

Markus Warmuth

Wenlai Zhou

Tatiana Novobrantseva

Alfica Sehgal

Dinah Wen-Yee Sah

William Querbes

Stephanie Williams

Huilei Xu

David A Bumcrot

Ivanka Toudjarska

Donald Foster

Jared Gollob

Qingmin Chen

Rene Alvarez

Akin Akinc

Barry S Goldman

Steven C Slater

Abigail Liebow

Vasant R Jadhav

Laura Sepp-Lorenzino

Jesper Gromada

Aris I Baras

Mark K Schlegel

Yongwei Cao

Xiping Cheng

Frederick E Dewey

David V Erbe

Yurong Xin

Noura S Abul-Husn

Omri Gottesman

Frederic Tremblay

Alexander Li

Jingxuan Liu

Xianfeng Chen

Jennifer Willoughby

Timothy Ryan Mooney

David Richard Corbin

Thomas M Malvar

Victor E Kotelianski

Anna Borodovsky

Svetlana Shulga Morskaya

Alan R Shuldiner

Karina Krasomil-Osterfeld

Joseph E Huesing

Roger Wiegand

Jinyun Chen

Sergei Spiridonov

Alan Shuldiner

Laura Sepp-lorenzino

Svetlana Shulga-Morskaya

Wen-Yee Sah Dinah

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the HSD17B 13 gene, as well as methods of inhibiting expression of HSD17B13, and methods of treating subjects that would benefit from reduction in expression of HSD17B13, such as subjects having a HSD17B13-associated disease, disorder, or condition, using such dsRNA compositions.

17ß-hydroxysteroid dehydrogenase type 13 (HSD17B13) iRNA compositions and methods of use thereof

The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Kallikrein B, Plasma (Fletcher Factor) 1 (KLKB1) gene, the Factor XII (Hageman Factor (F12) gene, or the Kininogen 1 (KNG1) gene, and methods of using such RNAi agents to inhibit expression of a KLKB1 gene, an F12 gene, and/or a KNG1 gene, and methods of treating subjects having an hereditary angioedema (HAE) and/or a contact activation pathway-associated disorder.

Factor XII (hageman factor) (F12), kallikrein B, plasma (fletcher factor) 1 (KLKB1), and kininogen 1 (KNG1) iRNA compositions and methods of use thereof

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.

Compositions and methods for inhibiting expression of transthyretin

The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.

Organic compositions to treat HSF1-related diseases

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

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 a double-stranded ribonucleic acid (dsRNA) targeting a G-alpha q subunit (GNAQ) of a heterotrimeric G gene, and methods of using the dsRNA to inhibit expression of GNAQ.

GNAQ targeted dsRNA compositions and methods for inhibiting expression

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.

Compositions and methods for inhibiting expression of the LECT2 gene

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a Serum Amyloid A (SAA) gene, and methods of using the dsRNA to inhibit expression of SAA.

Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene

The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), and C3 glomerulonephritis.

Complement component C3 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 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 present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such RNAi agents to inhibit expression of a PD-L1 gene and methods of treating subjects having a PD-L1-associated disorder.

Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof

The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.

Sterol regulatory element binding protein (SREBP) chaperone (SCAP) iRNA compositions and methods of use thereof

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.

Growing community of inventors

Plymouth, MA, United States of America

Gregory Hinkle