公开(公告)号：US20240279650A1

公开(公告)日：2024-08-22

申请号：US18474327

申请日：2023-09-26

申请人： Shape Therapeutics Inc.

发明人： David HUSS ,  Prashant MALI ,  Anupama LAKSHMANAN ,  Christopher NYE ,  Yiannis SAVVA ,  Liana STEIN ,  Richard SULLIVAN ,  Rafael PONCE ,  Susan BYRNE

IPC分类号： C12N15/11 ,  A61K48/00 ,  A61P25/28 ,  C07K14/47 ,  C12N9/78 ,  C12N15/86

CPC分类号： C12N15/11 ,  A61K48/0066 ,  A61P25/28 ,  C07K14/47 ,  C12N9/78 ,  C12N15/86 ,  C12Y305/04004 ,  C12N2310/122 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/336 ,  C12N2310/531 ,  C12N2310/533 ,  C12N2750/14143 ,  C12N2750/14171

摘要： Disclosed herein are compositions that comprise engineered polynucleotides, pharmaceutical compositions comprising the same, methods of making the same, and methods of treatment comprising the compositions that comprise the engineered polynucleotides.

公开(公告)号：US20240141351A1

公开(公告)日：2024-05-02

申请号：US18530795

申请日：2023-12-06

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 ,  A61K31/713 ,  A61P1/16 ,  C12Q1/6883 ,  G01N33/68

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

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.

公开(公告)号：US20190024081A1

公开(公告)日：2019-01-24

申请号：US15954561

申请日：2018-04-16

申请人： INSTYTUT CHEMII BIOORGANICZNEJ PAN

发明人： Wlodzimierz KRZYZOSIAK ,  Marta OLEJNICZAK ,  Paulina GALKA-MARCINIAK ,  Agnieszka FISZER

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/531 ,  C12N2310/533 ,  C12N2320/34 ,  C12N2330/51

摘要： Subjects of the invention are: nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG-type repeats. Solution relates to the new concept of treating hereditary human neurological diseases caused by expansion of CAG-type trinucleotide repeats using RNA interference technology.

公开(公告)号：US10059942B2

公开(公告)日：2018-08-28

申请号：US14403581

申请日：2013-05-30

申请人： QBI ENTERPRISES LTD. ,  BIO-LAB LTD.

发明人： Sharon Avkin-Nachum ,  Jean-Christophe Truffert ,  Myriam Lefoix ,  Jean Hildesheim ,  Tirtsa Kleinman

IPC分类号： C12N15/113 ,  A61K31/7115

CPC分类号： C12N15/113 ,  A61K31/7115 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/321 ,  C12N2310/333 ,  C12N2310/336 ,  C12N2310/344 ,  C12N2320/51 ,  C12N2320/53 ,  C12N2330/30 ,  C12N2310/3521

摘要： Disclosed herein are double-stranded RNA nucleic acid molecules, which include at least one pyrazolotriazine nucleotide analog and have been modified to exhibit one of the following, increased on-target activity, increased target specificity, enhanced nuclease stability, reduced off target activity and/or reduced immunogenicity when compared to an unmodified or similarly modified dsRNA; pharmaceutical compositions comprising such molecules and methods of use thereof in therapy.

公开(公告)号：US20180155725A1

公开(公告)日：2018-06-07

申请号：US15878161

申请日：2018-01-23

申请人： SomaLogic, Inc.

发明人： Dominic Zichi ,  Sheri K. Wilcox ,  Chris Bock ,  Daniel J. Schneider ,  Bruce Eaton ,  Larry Gold

IPC分类号： C12N15/115 ,  C12N15/10 ,  G01N33/53

CPC分类号： C12N15/115 ,  C12N9/6429 ,  C12N15/1048 ,  C12N2310/16 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2330/31 ,  C12Q1/6811 ,  C12Q1/6816 ,  C12Q1/6832 ,  C12Q1/6834 ,  G01N33/5308 ,  G01N33/58 ,  C12Q2525/205 ,  C12Q2565/514 ,  C12Q2525/161 ,  C12Q2537/101 ,  C12Q2523/313

摘要： The present disclosure describes improved SELEX methods for producing aptamers that are capable of binding to target molecules and improved photoSELEX methods for producing photoreactive aptamers that are capable of both binding and covalently crosslinking to target molecules. Specifically, the present disclosure describes methods for producing aptamers and photoaptamers having slower dissociation rate constants than are obtained using prior SELEX and photoSELEX methods. The disclosure further describes aptamers and photoaptamers having slower dissociation rate constants than those obtained using prior methods. In addition, the disclosure describes aptamer constructs that include a variety of functionalities, including a cleavable element, a detection element, and a capture or immobilization element.

公开(公告)号：US20170298354A1

公开(公告)日：2017-10-19

申请号：US15587538

申请日：2017-05-05

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： Jinyun Chen ,  Kalyani Gampa ,  Dieter Huesken ,  Frank Stegmeier ,  Mark Stump ,  Chandra Vargeese ,  Jan Weiler ,  Wenlai Zhou

IPC分类号： C12N15/113 ,  A61K45/06 ,  A61K31/5377 ,  A61K31/713

CPC分类号： C12N15/113 ,  A61K31/5377 ,  A61K31/713 ,  A61K45/06 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/351 ,  C12N2320/31 ,  C12N2310/3521 ,  C12N2310/3525

摘要： The present disclosure relates to methods of treating heat stock factor 1 (HSF1)-related diseases such as cancer, autoimmune and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.

公开(公告)号：US20170137819A1

公开(公告)日：2017-05-18

申请号：US15410414

申请日：2017-01-19

申请人： SomaLogic, Inc.

发明人： Dominic Zichi ,  Sheri K. Wilcox ,  Chris Bock ,  Daniel J. Schneider ,  Bruce Eaton ,  Larry Gold

IPC分类号： C12N15/115 ,  C12N15/10 ,  G01N33/53

CPC分类号： C12N15/115 ,  C12N9/6429 ,  C12N15/1048 ,  C12N2310/16 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2330/31 ,  C12Q1/6811 ,  C12Q1/6816 ,  C12Q1/6832 ,  C12Q1/6834 ,  G01N33/5308 ,  G01N33/58 ,  C12Q2525/205 ,  C12Q2565/514 ,  C12Q2525/161 ,  C12Q2537/101 ,  C12Q2523/313

摘要： The present disclosure describes improved SELEX methods for producing aptamers that are capable of binding to target molecules and improved photoSELEX methods for producing photoreactive aptamers that are capable of both binding and covalently crosslinking to target molecules. Specifically, the present disclosure describes methods for producing aptamers and photoaptamers having slower dissociation rate constants than are obtained using prior SELEX and photoSELEX methods. The disclosure further describes aptamers and photoaptamers having slower dissociation rate constants than those obtained using prior methods. In addition, the disclosure describes aptamer constructs that include a variety of functionalities, including a cleavable element, a detection element, and a capture or immobilization element.

公开(公告)号：US09540651B2

公开(公告)日：2017-01-10

申请号：US14791749

申请日：2015-07-06

申请人： Idera Pharmaceuticals, Inc.

发明人： Ekambar R. Kandimalla ,  Daqing Wang ,  Ireneusz Nowak ,  Sudhir Agrawal

IPC分类号： A61K31/7115 ,  C12N15/117 ,  A61K45/06

CPC分类号： C12N15/117 ,  A61K31/7115 ,  A61K31/7125 ,  A61K45/06 ,  C12N2310/17 ,  C12N2310/31 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/336 ,  C12N2310/51 ,  C12N2310/52 ,  Y02A50/401 ,  Y02A50/411 ,  Y02A50/414 ,  A61K2300/00

摘要： The invention provides novel immune regulatory oligonucleotides (IRO) as antagonist of TLRs and methods of use thereof. These IROs have unique sequences that inhibit or suppress TLR-mediated signaling in response to a TLR ligand or TLR agonist. The methods may have use in the prevention and treatment of cancer, an autoimmune disorder, airway inflammation, inflammatory disorders, infectious disease, skin disorders, allergy, asthma or a disease caused by a pathogen.

摘要翻译： 本发明提供作为TLR拮抗剂的新型免疫调节寡核苷酸（IRO）及其使用方法。 这些IRO具有独特的序列，其响应于TLR配体或TLR激动剂抑制或抑制TLR介导的信号传导。 该方法可用于预防和治疗癌症，自身免疫性疾病，气道炎症，炎症性疾病，感染性疾病，皮肤病，变态反应，哮喘或由病原体引起的疾病。

公开(公告)号：US20160376586A1

公开(公告)日：2016-12-29

申请号：US14916039

申请日：2014-09-02

申请人： INSTYTUT CHEMII BIOORGANICZNEJ PAN

发明人： Wlodzimierz KRZYZOSIAK ,  Marta OLEJNICZAK ,  Paulina GALKA-MARCINIAK ,  Agnieszka FISZER

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/141 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/531 ,  C12N2310/533 ,  C12N2320/34 ,  C12N2330/51 ,  C12N2310/14

摘要： Subjects of the invention are: nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG-type repeats. Solution relates to the new concept of treating hereditary human neurological diseases caused by expansion of CAG-type trinucleotide repeats using RNA interference technology.

摘要翻译： 本发明的主题是：核酸分子，表达盒，表达载体，真核宿主细胞，真核宿主中RNA干扰的诱导方法和核酸分子在三核苷酸CAG型重复扩增诱导的疾病治疗中的应用。 解决方案涉及使用RNA干扰技术治疗由CAG型三核苷酸重复扩增引起的遗传性人类神经系统疾病的新概念。

公开(公告)号：US20160354491A1

公开(公告)日：2016-12-08

申请号：US15240512

申请日：2016-08-18

申请人： Moderna Therapeutics, Inc.

发明人： Atanu ROY ,  Christopher R. CONLEE ,  Antonin DE FOUGEROLLES ,  Andrew W. FRALEY

IPC分类号： A61K48/00

CPC分类号： A61K48/0066 ,  A61K38/1816 ,  A61K38/193 ,  A61K48/00 ,  C07H19/10 ,  C07H21/00 ,  C12N15/11 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2320/30 ,  C12P19/34 ,  C12P21/00 ,  C12P21/005

摘要： The present disclosure provides modified nucleosides, nucleotides, and nucleic acids, and methods of using them.