公开(公告)号：US20240352460A1

公开(公告)日：2024-10-24

申请号：US18764207

申请日：2024-07-04

申请人： GUANGZHOU REFORGENE MEDICINE CO., LTD.

发明人： Junbin Liang ,  Jiayu Ou ,  Hui Xu ,  Simiao Lin

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/333 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/531 ,  C12N2320/33

摘要： Provided is snRNA which targets USH2A pre-mRNA. The recognition domain of the snRNA is the reverse complement of a USH2A pre-mRNA sequence. The snRNA binds to the USH2A pre-mRNA to splice and jump exon 13. The snRNA promotes a higher exon 13 skipping efficiency than AON.

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：US20230250426A1

公开(公告)日：2023-08-10

申请号：US18001284

申请日：2021-06-09

申请人： INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  ECOLE CENTRALE DE NANTES ,  INSTITUT DE CANCÉROLOGIE DE L'OUEST ,  UNIVERSITÉ DE NANTES

发明人： Pierre-François CARTRON ,  Gwenola BOUGRAS-CARTRON ,  Aurélien SERANDOUR ,  Joséphine BRIAND

IPC分类号： C12N15/113 ,  A61P35/00 ,  A61P25/00 ,  C12Q1/6886

CPC分类号： C12N15/113 ,  A61P35/00 ,  A61P25/00 ,  C12Q1/6886 ,  C12N2310/141 ,  C12N2310/333 ,  C12N2310/3521 ,  C12Q2600/178 ,  C12Q2600/158 ,  C12Q2600/118 ,  C12Q2600/154

摘要： The present invention relates the treatment and prognostic of cancer like glioblastoma. Here, the inventors focused their study on the impact of presence of N6-adenosine methylation in miRNA-200b-3p in samples of patients suffering from glioblastoma multiforme (GBM). Their study was particularly focused on the impact of miRNA-200b-3p and its adenosine methylation on the expression of XIAP. XIAP acts as an anti-apoptotic protein via the inhibition of caspase-3 and -7 activation and high XIAP expression is associated with a poor survival in several solid tumors. Thus, the miR-200b-3p-mediated repression of XIAP mRNA expression appears as a mechanism governing the caspase-3 and -7 activity and the apoptosis. In theory, in the presence of miR-200b-3p, XIAP mRNA expression is repressed and caspase-3 and -7 can be activated to promote apoptosis. Thus, the present invention relates to an in vitro method for determining the prognosis of the survival time of a patient suffering from a cancer comprising the steps consisting of i) determining the expression level of the miR-200b-3p and/or the N6-adenosine methylated miRNA-200b-3p (miR-200b-3p m6A) in a sample from said patient and to the N6-adenosine methylated miRNA-200b-3p (miR-200b-3p m6A) for use in the treatment of a cancer in a subject in need thereof.

公开(公告)号：US20190241911A1

公开(公告)日：2019-08-08

申请号：US16269021

申请日：2019-02-06

申请人： Ohio State Innovation Foundation

发明人： Yizhou Dong ,  Bin Li

IPC分类号： C12N15/90 ,  C12N15/85 ,  C12N9/22 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/7105

CPC分类号： C12N15/907 ,  A61K31/7105 ,  A61K31/712 ,  A61K31/7125 ,  C12N9/22 ,  C12N15/85 ,  C12N2310/122 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/3235 ,  C12N2310/333 ,  C12N2310/3341 ,  C12N2310/335

摘要： The present disclosure generally relates to systems, methods and compositions for use in Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cpf1 genome editing systems.

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：US20160032284A1

公开(公告)日：2016-02-04

申请号：US14342193

申请日：2012-08-30

申请人： Jinyun CHEN ,  Kalyani GAMPA ,  Dieter HUESKEN ,  Frank STEGMEIER ,  Mark STUMP ,  Chandra VARGEESE ,  Jan WEILER ,  Wenlai ZHOU

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 shock factor 1 (HSF1)-related diseases such as cancer, autoimmune and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.

摘要翻译： 本公开涉及使用治疗有效量的RNAi剂对HSF治疗热休克因子1（HSF1）相关疾病如癌症，自身免疫性和病毒性疾病的方法。