公开(公告)号：US12013403B2

公开(公告)日：2024-06-18

申请号：US17365365

申请日：2021-07-01

申请人： Biogen MA Inc.

发明人： Frank Rigo ,  Katherine M. Bishop

IPC分类号： C07H21/04 ,  C12N15/113 ,  G01N33/566 ,  G01N33/68

CPC分类号： G01N33/6896 ,  C12N15/113 ,  G01N33/566 ,  G01N33/6854 ,  G01N33/6887 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2320/32 ,  C12N2320/35 ,  G01N2333/47 ,  G01N2800/28 ,  G01N2800/2878 ,  G01N2800/52

摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.

公开(公告)号：US11987792B2

公开(公告)日：2024-05-21

申请号：US17268206

申请日：2019-08-15

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Gregory Hinkle ,  Huilei Xu

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K9/08 ,  A61K9/51 ,  A61K31/7125 ,  A61K47/02 ,  C07H21/02

CPC分类号： C12N15/113 ,  A61K9/0019 ,  A61K9/08 ,  A61K9/5123 ,  A61K47/02 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/32 ,  C12N2320/35

摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.

公开(公告)号：US20240158792A1

公开(公告)日：2024-05-16

申请号：US18504781

申请日：2023-11-08

申请人： NIPPON SHINYAKU CO., LTD.

发明人： Tomonori UNO ,  Takashi NATSUKAWA ,  Youichi EGAWA ,  Youhei SATOU

IPC分类号： C12N15/113 ,  A61P21/00

CPC分类号： C12N15/113 ,  A61P21/00 ,  C12N2310/11 ,  C12N2320/35

摘要： The present invention relates to a composition containing an antisense oligonucleotide, and the use thereof to treat Duchenne muscular dystrophy. The present invention particularly relates to the above-described composition that is effective for the treatment of Duchenne muscular dystrophy when it is administered at a dose for the treatment, and the use thereof.

公开(公告)号：US20240102020A1

公开(公告)日：2024-03-28

申请号：US18202328

申请日：2023-05-26

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Gregory Hinkle

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K9/08 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  A61K47/02 ,  A61K47/28 ,  A61K47/54 ,  G01N33/569 ,  G01N33/574

CPC分类号： C12N15/1138 ,  A61K9/0019 ,  A61K9/08 ,  A61K9/1271 ,  A61K9/5123 ,  A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  A61K47/02 ,  A61K47/28 ,  A61K47/549 ,  G01N33/56983 ,  G01N33/574 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/35

摘要： The invention relates to polynucleotide agents targeting programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such polynucleotide agents to inhibit expression of PD-L1 and to treat subjects having a PD-L1-associated disorder.

公开(公告)号：US20240100080A1

公开(公告)日：2024-03-28

申请号：US18299220

申请日：2023-04-12

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Amy Chan ,  John Vest ,  Gabriel Robbie ,  Husain Z. Attarwala ,  Varun Goel

IPC分类号： A61K31/713 ,  A61K31/7105 ,  C12N15/113

CPC分类号： A61K31/713 ,  A61K31/7105 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3533 ,  C12N2320/31 ,  C12N2320/35

摘要： The present invention provides methods for treating or preventing TTR-associated diseases using RNAi agents, e.g., double stranded RNAi agents, that target the transthyretin (TTR) gene.

公开(公告)号：US11891606B2

公开(公告)日：2024-02-06

申请号：US16622820

申请日：2018-06-15

申请人： Antisense Therapeutics Ltd

发明人： George Tachas

IPC分类号： C12N15/113 ,  A61P25/28 ,  A61K31/437 ,  A61K31/7125 ,  A61K38/07 ,  A61K38/21 ,  C12Q1/68

CPC分类号： C12N15/1138 ,  A61K31/437 ,  A61K31/7125 ,  A61K38/07 ,  A61K38/215 ,  A61P25/28 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/35

摘要： A method for treating a patient suffering from multiple sclerosis, including progressive forms of multiple sclerosis, comprising periodically administering a pharmaceutical composition comprising a therapeutically effective amount of OLIGONUCLEOTIDE I to the patient, thereby treating the patient.

公开(公告)号：US20230407312A1

公开(公告)日：2023-12-21

申请号：US18324191

申请日：2023-05-26

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Tracy L. McGregor ,  John Michael Gansner ,  Ishir Bhan

IPC分类号： C12N15/113 ,  A61P13/02

CPC分类号： C12N15/1137 ,  C12Y101/03015 ,  A61P13/02 ,  C12N2310/14 ,  C12N2320/35 ,  C12N2310/351 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/315

摘要： The invention relates methods of using RNAi agents to inhibit expression of HAO1 and methods of treating subjects having primary hyperoxaluria, e.g., PH1.

公开(公告)号：US20230392155A1

公开(公告)日：2023-12-07

申请号：US18302855

申请日：2023-04-19

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Gabriel Robbie ,  Varun Goel

IPC分类号： C12N15/113 ,  A61P3/00

CPC分类号： C12N15/1137 ,  C12Y101/03015 ,  A61P3/00 ,  C12N2310/14 ,  C12N2320/35 ,  C12N2310/351

摘要： The present invention provides methods and compositions for treating a pediatric subject having primary hyperoxaluria and methods for preventing at least one symptom in a pediatric subject having primary hyperoxaluria. The methods include administering to the subject a therapeutically effective amount or a prophylactically effective amount of an RNAi agent, e.g., double-stranded RNAi agent, targeting HAO1.

公开(公告)号：US11795459B2

公开(公告)日：2023-10-24

申请号：US17692630

申请日：2022-03-11

申请人： miRecule, Inc.

发明人： Robert Place ,  Anthony Saleh ,  Tishan Williams

IPC分类号： C07H21/04 ,  C12N15/113 ,  A61K31/7088 ,  A61K47/54

CPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K47/549 ,  C12N2310/141 ,  C12N2310/316 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/33 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/531 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/35

摘要： Disclosed herein are engineered oligonucleotides for selective inhibition of polypeptide expression and activity. Also disclosed herein are methods of selectively inhibiting polypeptide expression and activity contacting an engineered oligonucleotide with a polynucleotide encoding the polypeptide.

公开(公告)号：US20230265428A1

公开(公告)日：2023-08-24

申请号：US18053447

申请日：2022-11-08

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. FREIER

IPC分类号： C12N15/113 ,  A61P13/02

CPC分类号： C12N15/113 ,  A61P13/02 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/321 ,  C12N2320/35 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/11 ,  C12N2320/32 ,  C12N2310/3341

摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting APOL1 expression, which may be useful for treating, preventing, or ameliorating a disease associated with APOL1.