公开(公告)号：US12071619B2

公开(公告)日：2024-08-27

申请号：US17141275

申请日：2021-01-05

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Alfica Sehgal ,  Klaus Charisse ,  Brian Bettencourt ,  Martin A. Maier ,  Kallanthottathil G. Rajeev ,  Gregory Hinkle ,  Muthiah Manoharan

IPC分类号： C07H21/02 ,  A61K31/712 ,  A61K31/713 ,  C07H21/04 ,  C12N15/113

CPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/331 ,  C12N2310/332 ,  C12N2310/343 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/32 ,  C12N2320/34 ,  C12N2320/51 ,  C12N2330/30 ,  C12N2310/343 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/343 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/343 ,  C12N2310/351 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/332 ,  C12N2310/322 ,  C12N2320/32 ,  C12N2310/351

摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.

公开(公告)号：US11987794B2

公开(公告)日：2024-05-21

申请号：US17688789

申请日：2022-03-07

申请人： Silence Therapeutics GmbH

发明人： Judith Hauptmann ,  Dmitry Samarsky ,  Adrien Weingärtner ,  Lucas Bethge ,  Christian Frauendorf ,  Alison Gallafent

IPC分类号： C12N15/11 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/344 ,  C12N2320/51

摘要： The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with target gene expression or inhibit target gene expression and therapeutic uses of such products.

公开(公告)号：US11981893B2

公开(公告)日：2024-05-14

申请号：US17493637

申请日：2021-10-04

申请人： Nantomics

发明人： Patrick Soon-Shiong ,  Shahrooz Rabizadeh ,  Kathleen Danenberg

IPC分类号： C12N15/11 ,  C12N15/10 ,  C12Q1/6876

CPC分类号： C12N15/11 ,  C12N15/1003 ,  C12Q1/6876 ,  C12N2320/51 ,  C12Q2565/00

摘要： RNA from a biological fluid is stabilized during isolation and/or storage using DNA. In especially preferred aspects, the RNA is cfRNA and/or ctRNA, and the biological fluid is blood.

公开(公告)号：US20240067964A1

公开(公告)日：2024-02-29

申请号：US18176174

申请日：2023-02-28

申请人： Ionis Pharmaceuticals, Inc.

发明人： Rosanne M. Crooke ,  Mark J. Graham ,  Susan M. Freier ,  Marc Lim ,  Andrew Dibble

IPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C12N15/11

CPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/3525 ,  C12N2320/50 ,  C12N2320/51 ,  C12N2320/52

摘要： Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a). Certain diseases, disorders or conditions related to apo(a) or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The antisense compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

公开(公告)号：US20230348904A1

公开(公告)日：2023-11-02

申请号：US17984486

申请日：2022-11-10

申请人： Sirna Therapeutics, Inc.

发明人： Brian Allen Carr ,  Vasant R. Jadhav ,  Denise M. Kenski ,  David M. Tellers ,  Aarron T. Willingham

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/32 ,  C12N2320/51 ,  C12N2310/321

摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.

公开(公告)号：US20230323344A1

公开(公告)日：2023-10-12

申请号：US17929956

申请日：2022-09-06

申请人： ModernaTx, Inc.

发明人： Gabor BUTORA ,  Andrew W. Fraley ,  Edward John Miracco ,  Jennifer Nelson ,  Amy Rhoden Smith ,  Matthew Stanton

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/351 ,  C12N2320/51

摘要： The disclosure provides polynucleotides encoding a polypeptide including a morpholino linker. In some embodiments, the polynucleotides of the invention have increased stability compared to wild-type polynucleotides.

公开(公告)号：US20230220393A1

公开(公告)日：2023-07-13

申请号：US18155730

申请日：2023-01-17

申请人： Larry J. Smith

发明人： Larry J. Smith

IPC分类号： C12N15/113 ,  C07H21/04 ,  C12N15/11 ,  C07H21/02

CPC分类号： C12N15/113 ,  C07H21/04 ,  C12N15/111 ,  C07H21/02 ,  C12N2320/50 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/321 ,  C12N2310/14 ,  C12N2310/346 ,  C12N2320/51 ,  C12N2310/344 ,  C12N2310/141 ,  C12N2310/32

摘要： Compositions and methods for modulating expression of target nucleic acids using a single strand oligoribonucleotide ss-siRNA compound are disclosed.

公开(公告)号：US20230158150A1

公开(公告)日：2023-05-25

申请号：US17778372

申请日：2020-11-25

申请人： DTX PHARMA, INC.

发明人： Arthur T. Suckow ,  Charles Allerson ,  Fabio C. Tucci

IPC分类号： A61K47/54 ,  C12N15/113

CPC分类号： A61K47/543 ,  C12N15/1137 ,  A61K47/542 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2320/51 ,  C12N2310/346 ,  C12N2320/30

摘要： Disclosed herein are compounds including a nucleic acid (A), their preparation, and their use.

公开(公告)号：US20190233821A1

公开(公告)日：2019-08-01

申请号：US16381288

申请日：2019-04-11

申请人： SIRNA THERAPEUTICS, INC.

发明人： Leonid Beigelman ,  James McSwiggen ,  Chandra Vargeese

IPC分类号： C12N15/113 ,  C12N15/82 ,  C12N15/11 ,  C07H21/02

CPC分类号： C12N15/113 ,  C07H21/02 ,  C12N15/111 ,  C12N15/1131 ,  C12N15/1137 ,  C12N15/1138 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/53 ,  C12N2320/32 ,  C12N2320/51 ,  C12Y304/21021

摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

公开(公告)号：US20190203208A1

公开(公告)日：2019-07-04

申请号：US16325827

申请日：2017-08-11

申请人： Steffen Panzner

发明人： Steffen Panzner

IPC分类号： C12N15/113 ,  C07H19/06 ,  C07H19/10

CPC分类号： C12N15/113 ,  C07H19/06 ,  C07H19/10 ,  C07H19/16 ,  C07H21/00 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/314 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2320/51 ,  C12N2320/52 ,  C12N2310/322 ,  C12N2310/3535

摘要： The invention proves an oligonucleotide comprising one or more carboxylated 2′-amino-LNA nucleotide units. The invention also provides a method of transfecting cells with the oligonucleotide, a method of treating a human or animal by therapy using the oligonucleotide, and a pharmaceutical composition comprising the oligonucleotide.