Regeneron Pharmaceuticals, Inc.

Regeneran Pharmaceuticals, Inc.

Carmelo Romano

Ying Hu

Aris I Baras

Giovanni Coppola

Yajun Tang

William C Olson

Goncalo Abecasis

Kavita Praveen

Adrianna Latuszek

Yang Liu

Lynn Macdonald

Alexander O Mujica

Jingtai Cao

Susan D Croll

Min Gao

Jeffrey D Lee

Lauren Gurski

Claudia Schurmann

Tanya Teslovich Dostal

Susannah Brydges

Gaurang Patel

Sahar Gelfman

Eli A Stahl

Ann Ligocki

Arden Moscati

Brian Zambrowicz

Manuel Allen Revez Ferreira

Giusy Della Gatta

Trikaldarshi Persaud

Santiago Mendez Huergo

Tave Van Zyl

Jonathan Weyne

Jack A Kosmicki

Ming Yuan

Nucleic acid constructs and compositions that allow insertion and/or expression of a retinoschisin coding sequence are provided. Nuclease agents targeting RS1 loci are provided. Compositions and methods of using such constructs for integration into a target genomic locus and/or expression in a cell are also provided. Methods of treating X-linked juvenile retinoschisis using the nucleic acid constructs and compositions are also provided.

CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TRKB locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TRKB locus express a human TRKB protein or a chimeric transthyretin protein, fragments of which are from human TRKB. Methods are provided for using such non-human animals comprising a humanized TRKB locus to assess in vivo efficacy of human-TRKB-targeting reagents such as nuclease agents designed to target human TRKB.

Mouse comprising a humanized TRKB locus

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 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

Non-human animals suitable for use as animal models for Retinoschisis are provided. In some embodiments, provided non-human animals are characterized by a disruption in a Retinoschisin-1 locus. In some embodiments, provided non-human animals are characterized by a mutant Retinoschisin-1 gene. The non-human animals may be described, in some embodiments, as having a phenotype that includes the development of one or more symptoms or phenotypes associated with Retinoschisis. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat Retinoschisis or eye-related diseases, disorders or conditions are also provided.

Non-human animals models of retinoschisis

The present disclosure provides methods of treating subjects having inflammation with an Angiopoietin-Like 7 (ANGPTL7) inhibitor and a glucocorticoid, methods of decreasing glucocorticoid-induced ophthalmic conditions in subjects, and methods of identifying subjects having an increased risk of developing glucocorticoid-induced ophthalmic conditions.

Treatment of inflammation with glucocorticoids and angiopoietin-like 7 (ANGPTL7) inhibitors

The present disclosure provides methods of treating patients having an ophthalmic condition, methods of identifying subjects having an increased risk of developing an ophthalmic condition, methods of detecting human angiopoietin like 7 (ANGPTL7) variant nucleic acid molecules and variant polypeptides, and ANGPTL7 variant nucleic acid molecules and variant polypeptides.

Treatment of ophthalmic conditions with angiopoietin-like 7 (ANGPTL7) inhibitors

The present invention provides monoclonal antibodies that bind to the complement factor 5 (C5) protein, and methods of use thereof. In various embodiments of the invention, the antibodies are fully human antibodies that bind to C5 protein. In some embodiments, the antibodies of the invention are useful for inhibiting or neutralizing C5 activity, thus providing a means of treating or preventing a C5-related disease or disorder in humans. In some embodiments, the invention provides for an anti-C5 antibody that has improved pharmacokinetic and pharmacodynamic properties, e.g., a half-life of more than 10 days.

Polynucleotides encoding anti-C5 antibodies

Methods of treating C5-associated diseases comprising administering anti-C5 antibodies

[object Object]

Non-human animal models of retinoschisis

Anti-C5 antibodies

Growing community of inventors

Tarrytown, NY, United States of America

Carmelo Romano