公开(公告)号：WO2022020785A2

公开(公告)日：2022-01-27

申请号：PCT/US2021/043081

申请日：2021-07-23

Applicant: ORTHOBIO THERAPEUTICS, INC.

Inventor： MILLETT, Peter, J.

IPC: A61K48/00 ,  C12N15/10 ,  C12N15/85 ,  A61P19/02 ,  C12N15/1136 ,  C12N2310/20

Abstract: The present disclosure provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.

公开(公告)号：WO2022079222A1

公开(公告)日：2022-04-21

申请号：PCT/EP2021/078570

申请日：2021-10-15

Applicant: SANOFI

Inventor： BRUNNER, Bodo ,  HELMS, Mike ,  HOFMEISTER, Armin ,  JAHN-HOFMANN, Kerstin ,  METZ-WEIDMANN, Christiane ,  SCHEIDLER, Sabine ,  RIVAL, Pierrick

IPC: C12N15/113 ,  A61K31/713 ,  C07H21/02 ,  A61P3/06 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/323 ,  C12N2310/341 ,  C12N2310/351

Abstract: The present disclosure relates to dsRNAs targeting ANGPTL3, methods of inhibiting ANGPTL3 gene expression, and methods of treating one or more conditions associated with ANGPTL3 gene expression.

公开(公告)号：WO2021130537A2

公开(公告)日：2021-07-01

申请号：PCT/IB2020/001091

申请日：2020-12-21

Applicant: VERSAMEB AG

Inventor： ANTONY, Selvaraj, Justin ,  METZGER, Friedrich ,  SCHAFFHAUSER, Herve ,  HILMANN-WULLNER, Petra

IPC: C12N15/113 ,  C07K14/54 ,  A61K31/7088 ,  C07K14/65 ,  C07K14/005 ,  A61K31/713 ,  C07K14/52 ,  C07K14/565 ,  A61K38/00 ,  A61K48/005 ,  A61P17/06 ,  A61P19/02 ,  A61P25/28 ,  C07K14/5406 ,  C07K2319/01 ,  C07K2319/02 ,  C12N15/1131 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2320/31 ,  C12N2770/20022

Abstract: The present invention relates to compositions of recombinant polynucleic acid constructs comprising at least one nucleic acid sequence encoding an siRNA capable of binding to a target mRNA and at least one nucleic acid sequence encoding a gene of interest. Also disclosed herein is use of the compositions in treating a disease or a condition and in simultaneously modulating expression of two or more genes.

公开(公告)号：WO2022269356A2

公开(公告)日：2022-12-29

申请号：PCT/IB2022/000358

申请日：2022-06-22

Applicant: VERSAMEB AG

Inventor： SELVARAJ, Justin, Antony ,  ZUIDEVELD, Klaas, Pieter ,  HILLMANN -WULLNER, Petra ,  METZGER, Friedrich

IPC: C07K14/5406 ,  C07K14/65 ,  C12N15/113 ,  C12N15/1136 ,  C12N2310/14

Abstract: The present invention relates to compositions and methods for modulating expression of genes, comprising recombinant polynucleic acid or RNA constructs comprising a first RNA sequence encoding a gene of interest, and a second RNA sequence comprising at least two genetic elements that modulate expression of one or more target RNAs. The recombinant RNA constructs described herein induce an immune response in a human cell that is lower than the immune response induced by a corresponding recombinant RNA construct comprising the first RNA sequence encoding a gene of interest and a corresponding second RNA sequence comprising at most one of the at least two genetic elements. Also disclosed herein is the use of the compositions in treating diseases and in modulating expression of two or more genes.

公开(公告)号：WO2021117009A2

公开(公告)日：2021-06-17

申请号：PCT/IB2020/061852

申请日：2020-12-11

Applicant: MOORE, Tara

Inventor： KOWALCZYK, Amanda

IPC: C07K14/495 ,  C12N15/113 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/344 ,  C12N2320/34 ,  C12N2320/53

Abstract: The present disclosure relates to a ribonucleic acid (RNA) complex and methods of use thereof for preventing, ameliorating or treating symptoms associated with granular corneal dystrophy type 2 in a subject.

公开(公告)号：WO2022016121A2

公开(公告)日：2022-01-20

申请号：PCT/US2021/042100

申请日：2021-07-16

Applicant: ORTHOBIO THERAPEUTICS, INC.

Inventor： MILLETT, Peter, J. ,  RUSSELL, Iain, Alasdair ,  ALLEN, Matthew, J.

IPC: C12N15/11 ,  C12N15/1136 ,  C12N2310/20

Abstract: The present disclosure provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.

公开(公告)号：WO2022016100A2

公开(公告)日：2022-01-20

申请号：PCT/US2021/042048

申请日：2021-07-16

Applicant: ORTHOBIO THERAPEUTICS, INC.

Inventor： MILLETT, Peter, J. ,  RUSSELL, Iain, Alasdair ,  ALLEN, Matthew J.

IPC: A61K35/76 ,  C12N9/22 ,  C12N15/86 ,  C12N15/113 ,  C12N15/90 ,  A61P19/00 ,  C12N15/1136 ,  C12N2310/20

Abstract: The present disclosure provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.

公开(公告)号：WO2020154268A2

公开(公告)日：2020-07-30

申请号：PCT/US2020/014373

申请日：2020-01-21

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： PRAVEEN, Kavita ,  SCHURMANN, Claudia ,  GURSKI, Lauren ,  DOSTAL, Tanya Teslovich ,  ABECASIS, Goncalo ,  BARAS, Aris ,  COPPOLA, Giovanni

IPC: C12N15/113 ,  C07K14/515 ,  C12Q1/6827 ,  A61K31/7088 ,  A61P27/06 ,  C12N15/11 ,  C12N15/1136 ,  C12N2310/20 ,  C12N2800/80 ,  C12N9/22 ,  C12Q1/6883 ,  C12Q2600/106 ,  C12Q2600/156 ,  C12Q2600/158

Abstract: 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.

公开(公告)号：WO2023278787A2

公开(公告)日：2023-01-05

申请号：PCT/US2022/035846

申请日：2022-06-30

Applicant: REGENERON PHARMACEUTICALS, INC.

Inventor： BOVIJN, Jonas ,  SOSINA, Olukayode ,  LOTTA, Luca Andrea ,  BARAS, Aris

IPC: C12N15/113 ,  A61K31/713 ,  A61P19/10 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/20 ,  C12Q1/6883

Abstract: The present disclosure provides methods of treating subjects having decreased bone mineral density or at risk of developing decreased bone mineral density, methods of identifying subjects having an increased risk of developing decreased bone mineral density, methods of detecting Wnt Family Member 5B (WNT5B) variant nucleic acid molecules and variant polypeptides, and WNT5B variant nucleic acid molecules and variant polypeptides.

公开(公告)号：WO2023278576A1

公开(公告)日：2023-01-05

申请号：PCT/US2022/035523

申请日：2022-06-29

Applicant: ALNYLAM PHARMACEUTICALS, INC.

Inventor： HUANG, Stephen, Albert ,  FUJITA, Kenji, P. ,  LIN, Kang ,  MEHROTRA, Nitin ,  ROBBIE, Gabriel ,  LEE, Jongtae ,  AGARWAL, Sagar

IPC: C12N15/113 ,  C12N15/1136 ,  C12N15/1137 ,  C12N2310/14

Abstract: The present invention relates to methods of inhibiting the expression of an AGT gene in a subject, as well as methods for treating subjects having an AGT-associated disorder, e.g., hypertension, using RNAi agents, e.g., double stranded RNAi agents, targeting the AGT gene. The invention also relates to methods of decreasing blood pressure levels in a subject using such RNAi agents to inhibit expression of an AGT gene.