Regeneron Pharmaceuticals, Inc.

The Royal Institution for the Advancement of Learning/mcgill University

Duke University

Regeneran Pharmaceuticals, Inc.

Regeneron Phramaceuticals, Inc.

Aris I Baras

Manuel Allen Revez Ferreira

Luca Andrea Lotta

Goncalo Abecasis

Frederick E Dewey

Jesper Gromada

Xiping Cheng

Noura S Abul-Husn

Giovanni Coppola

Yurong Xin

Olukayode Sosina

Kavita Praveen

Omri Gottesman

Alexander Li

Lauren Gurski

Tanya Teslovich Dostal

Claudia Schurmann

Alan R Shuldiner

Carmelo Romano

Parsa Akbari

Niek Verweij

Adam Locke

Joshua Backman

Julie Horowitz

Andrew J Murphy

George D Yancopoulos

Lynn Macdonald

Cagan Gurer

Gregory Hinkle

Stuart Milstein

John R McWhirter

Karolina Meagher

Ying Hu

Israel Lowy

Suzanne Hartford

Meghan Drummond Samuelson

Frederic Tremblay

Garnett Kelsoe

Charles Paulding

Barton F Haynes

John Brent Richards

Mary Haas

Jonas Nielsen

Gaurang Patel

Eric Jorgenson

Amy Damask

Sahar Gelfman

Eli A Stahl

Ann Ligocki

Arden Moscati

Evangelos Pefanis

Jonas Bovijn

Sirui Zhou

Gavin Thurston

Garen Manvelian

Giusy Della Gatta

Stefan Semrau

Alan Shuldiner

Colm O'Dushlaine

Michael Cantor

Shannon Bruse

Shane McCarthy

Xinyuan Zhang

Trikaldarshi Persaud

Michael Kessler

Santiago Mendez Huergo

Tave Van Zyl

Jonathan Weyne

Jonathan Marchini

Jack A Kosmicki

The present disclosure provides methods of treating subjects having sepsis, SIRS, septic shock, and/or MODS, methods of identifying subjects having an increased risk of developing sepsis, SIRS, septic shock, and/or MODS, and methods of detecting Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) and/or Low Density Lipoprotein Receptor (LDLR) variant nucleic acid molecules and variant polypeptides.

Treatment of sepsis with PCSK9 and LDLR modulators

The present disclosure provides methods of treating subjects having an immune disorder by administering to the subject a therapeutically effective amount of an Endoplasmic Reticulum Aminopeptidase 2 (ERAP2) inhibitor in combination with an HLA-A29 or HLA-B27 inhibitory nucleic acid molecule.

Materials and methods of treating MHC-I-opathy risk haplotypes

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 disclosure provides methods of identifying a human subject as a candidate for treating or inhibiting a liver disease by inhibiting HSD17B13. The disclosure also provides methods of treating a subject who is PNPLA3 Ile148Met+ by administering an inhibitor of HSD17B13. The disclosure also provides method of detecting a PNPLA3 Ile148Met variant and functional HSD17B13 in a subject. The disclosure also provides method of identifying a subject having a protective effect against liver disease. The disclosure also provides inhibitors of HSD17B13 for use in the treatment of a liver disease.

Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation

The present disclosure provides methods of reducing cardiovascular risk by administration of a PCSK9 inhibitor to patients having a genetic profile associated with response to PCSK9 inhibitor therapy.

Genome-based methods for reducing cardiovascular risk

The present disclosure provides methods of treating subjects having hearing loss, methods of identifying subjects having an increased risk of developing hearing loss, and methods of detecting Kelch Domain Containing 7B (KLHDC7B) variant nucleic acid molecules and variant polypeptides.

Kelch domain containing 7B (KLHDC7B) variants and uses thereof

The present disclosure provides methods of treating a subject having decreased bone mineral density or at risk of developing decreased bone mineral density, and methods of identifying subjects having an increased risk of developing decreased bone mineral density.

Methods of treating decreased bone mineral density with cluster of differentiation 109 (CD109) inhibitors

The present disclosure provides methods of treating subjects having liver disease, and methods of identifying subjects having an increased risk of developing liver disease.

Treatment of liver disease with mitochondrial glycerol-3-phosphate acyltransferase (GPAM) inhibitors

The present disclosure provides methods of treating subjects having skin cancer or at risk of developing skin cancer, and methods of identifying subjects having an increased risk of developing skin cancer.

Methods of treating skin cancer with histidine ammonia-lyase (HAL) agonists

Methods of treating patients having varicose veins, methods of identifying subjects having an increased risk of developing varicose veins, and methods of diagnosing varicose veins in a human subject, comprising detecting the presence of Piezo Type Mechanosensitive Ion Channel Component 1 (PIEZO1) predicted loss-of-function variant nucleic acid molecules and polypeptides in a biological sample from the patient or subject, are provided herein.

Piezo type mechanosensitive ion channel component 1 (PIEZO1) variants and uses thereof

The present disclosure provides methods of treating subjects having uveitis, and methods of identifying subjects having an increased risk of developing uveitis.

Treatment of uveitis with endoplasmic reticulum aminopeptidase 1 (ERAP1) inhibitors

The present disclosure provides methods of treating a subject having a liver disease or type 2 diabetes, and methods of identifying subjects having an increased risk of developing a liver disease or type 2 diabetes.

Methods of treating liver diseases with phosphodiesterase 3B (PDE3B) inhibitors

The present disclosure provides methods of treating a subject having skin cancer or preventing a subject from developing skin cancer, and methods of identifying subjects having an increased risk of developing skin cancer.

Methods of treating skin cancer with Carboxypeptidase Vitellogenic Like (CPVL) inhibitors

The present disclosure provides methods of treating subjects having a liver disease, and methods of identifying subjects having an increased risk of developing liver disease.

Treatment of liver diseases with cell death inducing DFFA like effector B (CIDEB) inhibitors

The present disclosure provides methods of treating a subject having metabolic disorders and/or cardiovascular diseases, methods of identifying subjects having an increased risk of developing a metabolic disorder and/or a cardiovascular disease, and methods of detecting human Inhibin Subunit Beta E variant nucleic acid molecules and variant polypeptides.

Methods of treating metabolic disorders and cardiovascular disease with inhibin subunit beta E (INHBE) inhibitors

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Mount Kisco, NY, United States of America

Aris I Baras