公开(公告)号：US12116573B2

公开(公告)日：2024-10-15

申请号：US17184575

申请日：2021-02-24

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： David B. Rozema ,  Darren H. Wakefield ,  Andrei V. Blokhin ,  Jonathan D. Benson ,  Zhen Li ,  Tao Pei ,  Fred Fleitz

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/54 ,  C07H15/04

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/549 ,  C07H15/04 ,  C12N2310/14 ,  C12N2310/317 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/322 ,  C12N2310/3525

摘要： Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds, that are useful in directing the compounds to the target in vivo. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to an expression-inhibiting oligomeric compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.

公开(公告)号：US20240318183A1

公开(公告)日：2024-09-26

申请号：US18489316

申请日：2023-10-18

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04

CPC分类号： C12N15/1137 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04 ,  C12N15/113 ,  C12Y304/21034 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3525 ,  C12N2320/31 ,  C12N2320/35

摘要： Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

公开(公告)号：US12091662B2

公开(公告)日：2024-09-17

申请号：US18155945

申请日：2023-01-18

申请人： Biogen MA Inc.

发明人： Holly Kordasiewicz ,  Eric E. Swayze ,  Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  A61K31/711 ,  C07H21/02 ,  C12Q1/68

CPC分类号： C12N15/113 ,  A61K31/711 ,  C07H21/02 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525

摘要： Disclosed herein are antisense compounds and methods for decreasing Tau mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Tau-associated diseases, disorders, and conditions.

公开(公告)号：US12030910B2

公开(公告)日：2024-07-09

申请号：US17021510

申请日：2020-09-15

申请人： Ionis Pharmaceuticals, Inc.

发明人： Stanley T. Crooke ,  Xue-hai Liang ,  Wen Shen

IPC分类号： C07H21/04 ,  A61K31/7088 ,  C07H21/00 ,  C12N15/113 ,  C12N15/67 ,  C12P19/34

CPC分类号： C07H21/04 ,  A61K31/7088 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N15/1138 ,  C12N15/67 ,  C12P19/34 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/346 ,  C12Y301/26004 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/321 ,  C12N2310/3525

摘要： In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.

公开(公告)号：US20240117356A1

公开(公告)日：2024-04-11

申请号：US18270324

申请日：2021-12-30

申请人： Dyne Therapeutics, Inc.

发明人： Romesh R. Subramanian ,  Timothy Weeden ,  Cody A. Waltham ,  Stefano Zanotti ,  Kim Tang ,  Mohammed T. Qatanani ,  Brendan Quinn ,  John Najim

IPC分类号： C12N15/113 ,  A61P21/00 ,  C07K16/28

CPC分类号： C12N15/1137 ,  A61P21/00 ,  C07K16/28 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/3513 ,  C12N2310/3525 ,  C12N2320/32

摘要： The present application relates to oligonucleotides (e.g., antisense oligonucleotides such as gapmers) designed to target DMPK RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DMPK.

公开(公告)号：US20240026364A1

公开(公告)日：2024-01-25

申请号：US18358591

申请日：2023-07-25

申请人： Ionis Pharmaceuticals, Inc.

发明人： Shuling Guo ,  Mariam Aghajan ,  Eric E. Swayze

IPC分类号： C12N15/113 ,  A61K47/54 ,  C07H21/00 ,  A61P7/06 ,  A61P7/00

CPC分类号： C12N15/1137 ,  A61K47/549 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1138 ,  A61P7/06 ,  A61P7/00 ,  C12Y304/21109 ,  C12N2310/11 ,  C12N2310/351 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/334 ,  C12N2310/341 ,  C12N2310/3525 ,  C12N2310/3527

摘要： Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating TMPRSS6 and modulating an iron accumulation disease, disorder and/or condition in an individual in need thereof. Iron accumulation diseases in an individual such as polycythemia, hemochromatosis or β-thalassemia can be treated, ameliorated, delayed or prevented with the administration of antisense compounds targeted to TMPRSS6.

公开(公告)号：US11840686B2

公开(公告)日：2023-12-12

申请号：US17367096

申请日：2021-07-02

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  C07H21/04 ,  A61K31/7088 ,  A61K38/36

CPC分类号： C12N15/1137 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04 ,  C12N15/113 ,  C12Y304/21034 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3525 ,  C12N2320/31 ,  C12N2320/35

摘要： Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

公开(公告)号：US11834660B2

公开(公告)日：2023-12-05

申请号：US17393738

申请日：2021-08-04

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Gene Hung ,  C. Frank Bennett

IPC分类号： C12N15/113 ,  A61K48/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525

摘要： Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.

公开(公告)号：US20230374519A1

公开(公告)日：2023-11-23

申请号：US18001848

申请日：2021-06-18

申请人： Ionis Pharmaceuticals, Inc.

发明人： Eric E. Swayze ,  Holly Kordasiewicz ,  Punit P. Seth ,  Hien Thuy Zhao ,  Michael T. Migawa ,  Ruben E. Valas ,  Thazha P. Prakash

IPC分类号： C12N15/113 ,  A61K31/7125 ,  A61P25/28

CPC分类号： C12N15/1138 ,  A61K31/7125 ,  A61P25/28 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/323 ,  C12N2310/322 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2310/3181 ,  C12N2310/315 ,  C12N2310/31 ,  C12N2310/3525 ,  C12N2310/3341 ,  C12N2310/3515 ,  C12N2310/351 ,  C12N2310/3125 ,  C12N2310/346 ,  C12N2320/35

摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PMP22 RNA in a cell or animal, and in certain instances reducing the amount of PMP22 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include demyelination, progressive axonal damage and/or loss, weakness and wasting of foot and lower leg muscles, foot deformities, and weakness and atrophy in the hands. Such neurodegenerative diseases include Charcot-Marie-Tooth disease.

公开(公告)号：US20230279396A1

公开(公告)日：2023-09-07

申请号：US18067574

申请日：2022-12-16

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Eric E. Swayze

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12Y301/03016 ,  C12Y301/03048 ,  C12Y301/03067 ,  C12N15/111 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/343 ,  C12N2310/3525 ,  C12N2320/51 ,  C12N2310/321

摘要： The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.