公开(公告)号：WO2023034870A2

公开(公告)日：2023-03-09

申请号：PCT/US2022/075772

申请日：2022-08-31

申请人： IONIS PHARMACEUTICALS, INC.

发明人： RIGO, Frank ,  DWYER, Chrissa, A.

IPC分类号： C12N15/113 ,  A61K47/06 ,  A61K47/26 ,  A61K47/68 ,  A61K31/7088 ,  A61K47/549 ,  A61K47/6807 ,  A61K47/6849 ,  C12N15/1137 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/351

摘要： Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.

公开(公告)号：WO2022174000A2

公开(公告)日：2022-08-18

申请号：PCT/US2022/016046

申请日：2022-02-11

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： CASTORENO, Adam ,  GILBERT, Jason ,  KAITTANIS, Charalambos ,  MCININCH, James, D. ,  MILSTEIN, Stuart ,  SCHLEGEL, Mark, K.

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P25/28 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515

摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g. Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD) and Down's syndrome (DS), using such dsRNAi agents and compositions.

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

申请人： SANOFI

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

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

摘要： 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：WO2021126734A1

公开(公告)日：2021-06-24

申请号：PCT/US2020/064776

申请日：2020-12-14

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： TREMBLAY, Frederic ,  MCININCH, James, D. ,  CASTORENO, Adam ,  SCHLEGEL, Mark, K. ,  KAITTANIS, Charalambos

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P1/16 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/3183 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351

摘要： The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a PNPLA3 gene and to methods of preventing and treating an PNPLA3 -associated disorder, e.g., Nonalcoholic Fatty Liver Disease (NAFLD).

公开(公告)号：WO2022218941A2

公开(公告)日：2022-10-20

申请号：PCT/EP2022/059663

申请日：2022-04-11

申请人： BOEHRINGER INGELHEIM INTERNATIONAL GMBH

发明人： BROWN, Bob Dale ,  DUDEK, Henryk T. ,  SAXENA, Utsav ,  PARK, Jihye ,  ABRAMS, Marc ,  KOSER, Martin Lee

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P1/16 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/531

摘要： Oligonucleotides are provided herein that inhibit KHK expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with KHK expression.

公开(公告)号：WO2022056266A8

公开(公告)日：2022-03-17

申请号：PCT/US2021/049871

申请日：2021-09-10

申请人： ARROWHEAD PHARMACEUTICALS, INC.

发明人： DING, Zhi-Ming ,  VAN DYKE, Jonathan ,  LI, Xiaokai ,  NICHOLAS, Anthony ,  SCHIENEBECK, Casi, M. ,  PEI, Tao ,  XU, Zhao ,  AI, Teng ,  PHAN, Susan ,  RAMOS-HUNTER, Susan

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P21/00 ,  C12N2310/14 ,  C12N2310/312 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2320/32

摘要： Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a double homeobox 4 ( DUX4 ) gene. The DUX4 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a DUX4 gene. Pharmaceutical compositions that include one or more DUX4 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described DUX4 RNAi agents to skeletal muscle cells in vivo, provides for inhibition of DUX4 gene expression and a reduction in DUX4 levels, which can provide a therapeutic benefit to subjects, including human subjects, suffering from certain skeletal muscle-related diseases or disorders including Facioscapulohumeral Muscular Dystrophy (FSHD).

公开(公告)号：WO2022026387A1

公开(公告)日：2022-02-03

申请号：PCT/US2021/043182

申请日：2021-07-26

申请人： ALIGOS THERAPEUTICS, INC.

发明人： HONG, Jin ,  BEIGELMAN, Leonid ,  BHATTACHARYA, Aneerban ,  DE COSTA, N. Tilani S.

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7115 ,  A61P31/20 ,  A61K31/713 ,  A61K45/06 ,  C12N15/1131 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/351 ,  C12N2320/31

摘要： Oligonucleotides that target hepatitis B virus (HBV) viral sequences, such as rcDNA, cccDNA, and HBV transcripts, are described herein. In addition, compositions and kits comprising such oligonucleotides are further described. Further disclosed herein are uses of such oligonucleotides and compositions to reduce rcDNA to cccDNA conversion, reduce cccDNA levels, and/or treat an HBV infection.

公开(公告)号：WO2021262911A1

公开(公告)日：2021-12-30

申请号：PCT/US2021/038785

申请日：2021-06-24

申请人： THE PROCTER & GAMBLE COMPANY

发明人： VELASQUEZ, Juan, Esteban ,  RUPARD, Spencer, Christopher ,  TREJO, Amy, Violet ,  PITZ, Adam, Michael ,  SCHMEICHEL, Kelly, Lee ,  SWIGART, Erin, Nicole ,  PENNER, Gregory, Allen

IPC分类号： C12N15/115 ,  A61K31/712 ,  A61K9/12 ,  A61K9/20 ,  A61P31/16 ,  A61K31/7088 ,  A61K47/549 ,  A61K47/60 ,  C12N15/1138 ,  C12N2310/16 ,  C12N2310/351 ,  C12N2320/31 ,  C12N2320/32

摘要： An aptamer composition is disclosed which has one or more oligonucleotides that include at least one of deoxyribonucleotides, ribonucleotides, derivatives of deoxyribonucleotides, derivatives of ribonucleotides, or mixtures thereof. The aptamer composition has a binding affinity for one or more cellular membrane glycoproteins selected from the group consisting of: intercellular adhesion molecule 1 (ICAM-1), low-density lipoprotein receptor (LDLR) family members, and cadherin-related family member 3 (CDHR3), preferably intercellular adhesion molecule 1 (ICAM-1), and is configured to reduce the binding of one or more human rhinoviruses to the intercellular adhesion molecule 1 (ICAM-1).

公开(公告)号：WO2021222065A8

公开(公告)日：2021-11-04

申请号：PCT/US2021/029081

申请日：2021-04-26

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  BOSTWICK, Bret, Lee ,  PENG, Haiyan ,  MCININCH, James, D. ,  CASTORENO, Adam ,  SCHLEGEL, Mark, K.

发明人： BOSTWICK, Bret, Lee ,  PENG, Haiyan ,  MCININCH, James, D. ,  CASTORENO, Adam ,  SCHLEGEL, Mark, K.

IPC分类号： C12N15/113 ,  A61K31/713 ,  C07H21/00 ,  A61P25/28 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/31 ,  C12N2320/34

摘要： The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an APOE gene, as well as methods of inhibiting expression of an APOE gene and methods of treating subjects having an APOE-associated neurodegenerative disease or disorder, e.g., Alzheimer's disease and Parkinson's disease, using such dsRNAi agents and compositions.

公开(公告)号：WO2021122735A1

公开(公告)日：2021-06-24

申请号：PCT/EP2020/086405

申请日：2020-12-16

申请人： F. HOFFMANN-LA ROCHE AG ,  HOFFMANN-LA ROCHE INC.

发明人： PEDERSEN, Lykke ,  LUANGSAY, Souphalone ,  WALTHER, Johanna Marie ,  MUELLER-BRECKENRIDGE, Alan James

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  C07H21/02 ,  A61P31/20 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351

摘要： The present invention relates to a SEPT9 inhibitor for use in treatment of an HBV infection, in particular a chronic HBV infection. The invention in particular relates to the use of SEPT9 inhibitors for destabilizing cccDNA, such as HBV cccDNA. The invention also relates to nucleic acid molecules which are complementary to SEPT9 and capable of reducing the level of a SEPT9 mRNA. Also comprised in the present invention is a pharmaceutical composition and its use in the treatment of a HBV infection.