专利检索 cpc:"C12N2310/3533" 第 1 页

1.

发明授权
Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated diseases 有权

公开(公告)号：US12049628B2

公开(公告)日：2024-07-30

申请号：US17584463

申请日：2022-01-26

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Tracy Zimmermann , Amy Chan , Vasant R. Jadhav , Martin A Maier , Kallanthottathil G. Rajeev

IPC分类号： C12N15/113

CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/322 , C12N2310/344 , C12N2310/351 , C12N2310/3515 , C12N2310/3533 , C12N2320/30 , C12N2310/322 , C12N2310/3533

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

2.

发明公开
METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS 审中-公开

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.

3.

发明授权
Antisense-induced exon exclusion in type VII collagen 有权

公开(公告)号：US11911403B2

公开(公告)日：2024-02-27

申请号：US17080363

申请日：2020-10-26

申请人： Sarepta Therapeutics, Inc.

发明人： Dan V. Mourich

IPC分类号： C07H21/02 , A61K31/70 , C12N15/113 , C07H21/04 , A61P17/00

CPC分类号： A61K31/70 , A61P17/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N2310/11 , C12N2310/3145 , C12N2310/3181 , C12N2310/3231 , C12N2310/3233 , C12N2320/33 , C12N2310/321 , C12N2310/3521 , C12N2310/321 , C12N2310/3525 , C12N2310/322 , C12N2310/3533 , C12N2310/321 , C12N2310/3527

摘要： The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.

4.

发明授权
Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use 有权

公开(公告)号：US11884920B2

公开(公告)日：2024-01-30

申请号：US17523173

申请日：2021-11-10

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Zhen Li , Rui Zhu , Christine I. Wooddell , Tao Pei

IPC分类号： C12N15/113 , A61K9/00 , A61P1/16 , G01N33/68 , C12Q1/6883 , A61K31/713

CPC分类号： C12N15/113 , A61K9/0019 , A61K31/713 , A61P1/16 , C12Q1/6883 , G01N33/6893 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/333 , C12N2310/334 , C12N2310/335 , C12N2310/336 , C12N2310/343 , C12N2310/346 , C12N2310/351 , C12N2320/31 , C12Q2600/156 , C12Q2600/158 , G01N2333/8125 , G01N2800/085 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533

摘要： RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.

5.

发明申请
MOLECULAR RE-ENGINEERING OF EXCITATION-INHIBITION BALANCE IN MEMORY CIRCUITS 审中-公开

公开(公告)号：US20190241894A1

公开(公告)日：2019-08-08

申请号：US16389467

申请日：2019-04-19

申请人： The General Hospital Corporation

发明人： Amar Sahay , Nannan Guo

IPC分类号： C12N15/113 , A61K49/00 , C12Q1/6897

CPC分类号： C12N15/113 , A61K49/0008 , C12N2310/11 , C12N2310/12 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3233 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533 , C12N2310/531 , C12Q1/6897

摘要： Memory-regulating agents and methods that target actin binding LIM protein family, member 3 (ABLIM3).

6.

发明申请
INHIBITION OF PCSK9 THROUGH RNAI 审中-公开

公开(公告)号：US20190218557A1

公开(公告)日：2019-07-18

申请号：US16191396

申请日：2018-11-14

申请人： Phio Pharmaceuticals Corporation

发明人： Joanne Kamens , Anastasia Khvorova

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/344 , C12N2310/3515 , C12N2310/531 , C12N2320/11 , C12Y304/21061 , C12Y304/21112 , C12N2310/3521 , C12N2310/3533

摘要： The invention relates to various PCSK9 RNAi constructs with gene silencing activities, and uses thereof. The construct has a double-stranded region of 19-49 nucleotides, preferably 25, 26, or 27 nucleotides, and preferably blunt-ended. The construct has selective minimal modifications to confer an optimal balance of biological activity, toxicity, stability, and target gene specificity. The sense strand may be modified such that the construct is not cleaved by Dicer or other RNAse III, and the entire length of the antisense strand is loaded into RISC In addition, the antisense strand may also be modified by 2′-O-methyl groups at the 2nd 5′-end nucleotide to greatly reduce off-target silencing. The constructs of the invention largely avoid the interferon response and sequence-independent apoptosis in mammalian cells, exhibits better serum stability, and enhanced target specificity.

7.

发明申请
VON WILLEBRAND FACTOR (VWF)-TARGETING AGENTS AND METHODS OF USING THE SAME 审中-公开

公开(公告)号：US20190218553A1

公开(公告)日：2019-07-18

申请号：US16334307

申请日：2017-09-18

申请人： DUKE UNIVERSITY , OHIO STATE INNOVATION FOUNDATION

发明人： Shahid M. NIMJEE , George A. PITOC , Juliana LAYZER , Bruce SULLENGER

IPC分类号： C12N15/113 , A61P7/02 , A61K31/7088

CPC分类号： C12N15/113 , A61K31/7088 , A61K31/7105 , A61K31/7115 , A61K45/06 , A61K47/554 , A61K47/60 , A61K47/64 , A61P7/02 , C12N15/115 , C12N2310/113 , C12N2310/16 , C12N2310/317 , C12N2310/3183 , C12N2310/3515 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533 , A61K2300/00

摘要： Provided herein are aptamers capable of inhibiting the activity of Von Willebrand Factor (VWF). Pharmaceutical compositions comprising these aptamers are also provided. Methods of preventing blood clot formation in a subject by administering the aptamers are provided and methods of treating a blood clot by administering a VWF-targeting agent are also provided.

8.

发明申请
ANTISENSE-INDUCED EXON EXCLUSION IN TYPE VII COLLAGEN 审中-公开

公开(公告)号：US20190201425A1

公开(公告)日：2019-07-04

申请号：US15578612

申请日：2016-06-01

申请人： Sarepta Therapeutics, Inc.

发明人： Dan V. Mourich

IPC分类号： A61K31/70 , A61P17/00 , C07H21/02 , C12N15/113

CPC分类号： A61K31/70 , A61P17/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N2310/11 , C12N2310/3145 , C12N2310/3181 , C12N2310/3231 , C12N2310/3233 , C12N2320/33 , C12N2310/321 , C12N2310/3521 , C12N2310/3525 , C12N2310/322 , C12N2310/3533 , C12N2310/3527

摘要： The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.