公开(公告)号：WO2021092513A1

公开(公告)日：2021-05-14

申请号：PCT/US2020/059568

申请日：2020-11-07

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： HUNT, Charleen ,  LIU, Yang ,  GONG, Guochun ,  ROMANO, Carmelo ,  ZAMBROWICZ, Brian

IPC: A61K48/00

Abstract: 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.

公开(公告)号：WO2022182768A1

公开(公告)日：2022-09-01

申请号：PCT/US2022/017533

申请日：2022-02-23

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： PATEL, Gaurang ,  HU, Ying ,  PRAVEEN, Kavita ,  COPPOLA, Giovanni ,  ABECASIS, Goncalo ,  BARAS, Aris ,  ROMANO, Carmelo

IPC: C12N15/113 ,  A61K31/713 ,  A61K31/573 ,  A61P27/06

Abstract: 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.

公开(公告)号：WO2019108983A1

公开(公告)日：2019-06-06

申请号：PCT/US2018/063390

申请日：2018-11-30

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： MUJICA, Alexander O. ,  TANG, Yajun ,  LEE, Jeffrey D. ,  GAO, Min ,  CROLL, Susan ,  MACDONALD, Lynn ,  HU, Ying ,  ROMANO, Carmelo

IPC: A01K67/027 ,  C12N5/10

Abstract: 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 .

公开(公告)号：WO2023064850A1

公开(公告)日：2023-04-20

申请号：PCT/US2022/078037

申请日：2022-10-13

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： GELFMAN, Sahar ,  LIGOCKI, Ann ,  COPPOLA, Giovanni ,  BARAS, Aris ,  MOSCATI, Arden ,  STAHL, Eli A. ,  ROMANO, Carmelo ,  MENDEZ HUERGO, Santiago ,  WEYNE, Jonathan ,  VAN ZYL, Tave

IPC: C12N15/113 ,  A61K31/7088 ,  A61P27/02

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

公开(公告)号：WO2022216832A1

公开(公告)日：2022-10-13

申请号：PCT/US2022/023685

申请日：2022-04-06

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： GELFMAN, Sahar ,  LIGOCKI, Ann ,  COPPOLA, Giovanni ,  BARAS, Aris ,  MOSCATI, Arden ,  STAHL, Eli A. ,  KOSMICKI, Jack A. ,  FERREIRA, Manuel Allen Revez ,  ROMANO, Carmelo

IPC: C07K16/40 ,  A61K31/7088 ,  C12N9/48 ,  A61P37/02 ,  A61P27/00 ,  A61P17/00 ,  A61P19/00 ,  A61K31/713 ,  A61K39/3955 ,  C07K16/2833 ,  C12N9/485 ,  C12Q1/00 ,  C12Q1/6881

Abstract: 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.

公开(公告)号：WO2016089807A1

公开(公告)日：2016-06-09

申请号：PCT/US2015/063084

申请日：2015-12-01

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： YUAN, Ming ,  HU, Ying ,  CAO, Jingtai ,  ROMANO, Carmelo

IPC: A61K31/14 ,  A61K31/46 ,  A61P27/02

CPC classification number: A01K67/027 ,  A01K2207/20 ,  A01K2227/10 ,  A01K2227/105 ,  A01K2267/03 ,  A61K31/14 ,  A61K31/46 ,  A61K49/0008

Abstract: Provided herein are methods for creating an animal model for the study of dry eye and methods of using such animal models to test candidate treatments for ocular conditions and determine the efficacy of such treatments.

Abstract translation: 本文提供了创建用于干眼症研究的动物模型的方法和使用这种动物模型来测试候选治疗眼睛状况并确定这些治疗的功效的方法。

公开(公告)号：WO2022026336A1

公开(公告)日：2022-02-03

申请号：PCT/US2021/043062

申请日：2021-07-23

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： GELFMAN, Sahar ,  LIGOCKI, Ann ,  COPPOLA, Giovanni ,  BARIS, Aris ,  MOSCATI, Arden ,  STAHL, Eli A. ,  KOSMICKI, Jack A. ,  FERREIRA, Manuel Allen Revez ,  ROMANO, Carmelo

IPC: C07K16/40 ,  A61K31/7088 ,  C12N9/48 ,  A61P37/02 ,  A61P27/00 ,  A61P17/00 ,  A61P19/00 ,  A61K31/713 ,  A61P25/00 ,  C07K14/8103 ,  C12N9/485 ,  C12Y304/11

Abstract: The present disclosure provides methods of treating subjects having an immune disorder by administering a therapeutically effective amount of an Endoplasmic Reticulum Aminopeptidase 2 (ERAP2) inhibitor to the subject, and optionally an Endoplasmic Reticulum Aminopeptidase 1 (ERAP1) agonist or inhibitor and/or an HLA-Aw19 inhibitor, and also provides methods of identifying subjects having an increased risk for developing an MHC-I-opathy.

公开(公告)号：WO2017218515A1

公开(公告)日：2017-12-21

申请号：PCT/US2017/037226

申请日：2017-06-13

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： HU, Ying ,  LATUSZEK, Adrianna ,  ROMANO, Carmelo ,  OLSON, William

IPC: C07K16/18

Abstract: 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.

Abstract translation: 本发明提供了结合补体因子5（C5）蛋白的单克隆抗体及其使用方法。 在本发明的各种实施方案中，抗体是与C5蛋白结合的完全人抗体。 在一些实施方案中，本发明的抗体可用于抑制或中和C5活性，由此提供治疗或预防人类C5相关疾病或病症的手段。 在一些实施方案中，本发明提供了具有改善的药代动力学和药效学性质（例如，半衰期超过10天）的抗C5抗体。

公开(公告)号：WO2020176747A1

公开(公告)日：2020-09-03

申请号：PCT/US2020/020134

申请日：2020-02-27

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： LIGOCKI, Ann J. ,  ROMANO, Carmelo

IPC: A61K9/51 ,  A61P27/02 ,  C12N15/10

Abstract: Provided herein are methods for selectively delivering therapeutics to the eye using AAV vectors. For example, the cornea can be specifically targeted using the methods described. Also provided herein are compositions comprising AAV vectors packaged with CRISPR complexes, which can be delivered directly to the eye, for example the cornea, and in particular the cornea endothelium. Diseases and conditions comprising abnormalities or deterioration of tissues in the eye, such as the cornea endothelium (e.g. FECD), can be treated using the methods and compositions described herein.

公开(公告)号：WO2018157058A1

公开(公告)日：2018-08-30

申请号：PCT/US2018/019721

申请日：2018-02-26

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： BRYDGES, Susannah ,  TANG, Yajun ,  LIU, Yang ,  CAO, Jingtai ,  ROMANO, Carmelo

IPC: A01K67/027 ,  G01N33/50 ,  C07K14/47 ,  C12N15/85

Abstract: 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.