公开(公告)号：US09957505B2

公开(公告)日：2018-05-01

申请号：US14954653

申请日：2015-11-30

申请人： HALO-BIO RNAI THERAPEUTICS, INC.

发明人： Todd M. Hauser

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/51 ,  C12N2310/52 ,  C12N2310/53 ,  C12N2320/30

摘要： The present invention includes bivalent or multivalent nucleic acid molecules or complexes of nucleic acid molecules having two or more target-specific regions, in which the target-specific regions are complementary to a single target gene at more than one distinct nucleotide site, and/or in which the target regions are complementary to more than one target gene or target sequence. Also included are compositions comprising such nucleic acid molecules and methods of using the same for multivalent RNA interference and the treatment of a variety of diseases and infections.

公开(公告)号：US09783804B2

公开(公告)日：2017-10-10

申请号：US15217007

申请日：2016-07-22

申请人： LIFE TECHNOLOGIES CORPORATION

发明人： Christopher Burnett ,  Nitin Puri ,  Susan Magdaleno ,  Alexander Vlassov

IPC分类号： A61K31/70 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C12N15/11 ,  A61K31/713 ,  A61K47/48 ,  C12N5/00

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/50 ,  C12N15/111 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/332 ,  C12N2310/3515 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/531 ,  C12N2330/31

摘要： The invention relates generally to compositions and methods for inhibiting the function of target nucleic acids by sequence specific binding. The compositions and methods can be used for inhibition of micro RNAs and other relatively short non-coding RNAs.

公开(公告)号：US20170275630A1

公开(公告)日：2017-09-28

申请号：US15508973

申请日：2015-09-09

申请人： The University of Tokyo

发明人： Shinsuke Sando ,  Ryosuke Ueki

IPC分类号： C12N15/115 ,  C12N5/00

CPC分类号： C12N15/115 ,  C12N5/0018 ,  C12N2310/151 ,  C12N2310/16 ,  C12N2310/51 ,  C12N2310/531 ,  C12N2501/12

摘要： Provided is an aptamer including a polynucleotide of any of the following (a) to (c) and capable of binding to an HGF receptor to exhibit an activity of inhibiting the binding of HGF to the HGF receptor. (a) A polynucleotide consisting of a base sequence set forth in SEQ ID NO: 1, (b) A polynucleotide consisting of a base sequence having the deletion, substitution, insertion and/or addition of one to several bases in the base sequence set forth in SEQ ID NO: 1, and (c) A polynucleotide consisting of a base sequence having a sequence identity of 80% or more to the base sequence set forth in SEQ ID NO: 1.

公开(公告)号：US20170240899A1

公开(公告)日：2017-08-24

申请号：US15519359

申请日：2015-10-14

申请人： Texas Tech University System

发明人： Haoquan WU ,  Jang-gi CHOI

IPC分类号： C12N15/113 ,  A61K48/00

CPC分类号： C12N15/1132 ,  A61K48/005 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/31 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2310/531 ,  C12N2330/51 ,  C12N2740/15042 ,  C12N2750/14142

摘要： Nucleic acid molecules such as shRNA clusters and artificial miRNA clusters are disclosed, Also disclosed are methods of use, compositions, cells, viral particles, and kits relating to the nucleic acid molecules disclosed herein. The disclosure provides, at least in part nucleic acid molecules such as shRNA clusters encoding shRNA-like molecules and artificial miRNA clusters encoding modified pri-miRNA-like molecules. The shRNA clusters and artificial miRNA clusters disclosed herein can be used, for example, to produce artificial RNA molecules, e.g., RNAi molecules. Cells, viral particles, compositions (e.g., pharmaceutical compositions), kits, and methods relating to the nucleic acid molecules, e.g., shRNA clusters and artificial miRNA clusters, are also disclosed. The nucleic acid molecules (e.g., shRNA clusters and artificial miRNA clusters), artificial RNA molecules (e.g., RNAi molecules), cells, viral particles, compositions (e.g., pharmaceutical compositions), and kits and methods disclosed herein can be used to treat or prevent a disease, e.g., HIV infection and/or AIDS.

公开(公告)号：US20170211065A1

公开(公告)日：2017-07-27

申请号：US15416100

申请日：2017-01-26

申请人： RaNA Therapeutics, Inc.

发明人： Eugen Uhlmann ,  Markus Weber ,  Romesh Subramanian ,  Thomas Dino Rockel ,  Arthur M. Krieg

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/51 ,  C12N2310/52

摘要： The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided

公开(公告)号：US20170204388A1

公开(公告)日：2017-07-20

申请号：US15460642

申请日：2017-03-16

申请人： Caribou Biosciences, Inc.

发明人： Paul Daniel Donohoue ,  Andrew Paul May

IPC分类号： C12N9/22 ,  C12N15/90 ,  C12N15/113

CPC分类号： C12N9/22 ,  C12N9/96 ,  C12N15/102 ,  C12N15/113 ,  C12N15/902 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2310/51

摘要： The present disclosure provides engineered cross-type-nucleic-acid targeting nucleic acids and compositions thereof. Nucleic acid sequences encoding the engineered cross-type-nucleic-acid targeting nucleic acids, as well as expression cassettes, vectors and cells comprising such nucleic acid sequences, are described. Also, methods are disclosed for making and using the engineered cross-type-nucleic-acid targeting nucleic acids and compositions thereof.

公开(公告)号：US09695425B2

公开(公告)日：2017-07-04

申请号：US15166793

申请日：2016-05-27

申请人： CITY OF HOPE

发明人： John Rossi ,  Katrin Tiemann ,  Jiehua Zhou ,  Britta Vallazza

IPC分类号： C12N15/11 ,  C12N15/115 ,  C12N15/113 ,  C07H21/02 ,  A61K31/713 ,  A61K47/48

CPC分类号： C12N15/115 ,  A61K31/713 ,  A61K47/48092 ,  A61K47/549 ,  C07H21/02 ,  C12N15/111 ,  C12N15/1135 ,  C12N2310/14 ,  C12N2310/16 ,  C12N2310/3519 ,  C12N2310/51 ,  C12N2320/32

摘要： In one embodiment, a B cell specific aptamer-siRNA chimera is provided. The B cell specific aptamer-siRNA chimera may include an RNA aptamer that binds BAFF-R and an siRNA molecule conjugated to the RNA aptamer via a nucleotide linker. In another embodiment, a B cell specific RNA aptamer is provided. The RNA aptamer may be a molecule that binds to BAFF-R that has the sequence SEQ ID NO:37, SEQ ID NO:38 or SEQ ID NO:39. In some embodiments, the RNA aptamer is conjugated, via a nucleotide linker, to an siRNA molecule that suppresses expression of one or more target oncogenes in one or more B cells. In one aspect, the one or more target oncogenes are selected from Bcl6, Bcl2, STAT3, Cyclin D1, Cyclin E2 and c-myc. In another embodiment, methods for treating a B cell malignancy in a cancer patient are provided. Such methods may include administering a therapeutically effective amount of a therapeutic composition, the therapeutic composition comprising a B cell specific RNA aptamer that binds BAFF-R.

公开(公告)号：US09664676B2

公开(公告)日：2017-05-30

申请号：US14480130

申请日：2014-09-08

申请人： Cornell University

发明人： Samie R. Jaffrey ,  Rita L. Strack ,  Grigory Filonov ,  Wenjiao Song

IPC分类号： C12N15/11 ,  G01N33/53 ,  C12N15/115

CPC分类号： G01N33/5308 ,  C12N15/115 ,  C12N2310/16 ,  C12N2310/51 ,  C12N2320/10

摘要： The present invention relates to novel nucleic acid molecules, called aptamers, that bind specifically to a small molecule fluorophore and thereby enhance the fluorescence signal of the fluorophore upon exposure to radiation of suitable wavelength. Molecular complexes formed between the novel fluorophores, novel nucleic acid molecules, and their target molecules are described, and the use of multivalent aptamer constructs as fluorescent sensors for target molecules of interest are also described.

公开(公告)号：US09644209B2

公开(公告)日：2017-05-09

申请号：US14312530

申请日：2014-06-23

申请人： KAIST IP Co. Ltd.

发明人： Tae Gwan Park ,  Hye Jung Mok ,  Soo Hyeon Lee

IPC分类号： C12N15/11 ,  C12N15/113

CPC分类号： C12N15/1136 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/51

摘要： The present invention relates to a multi-conjugate of small interfering RNA (siRNA) and a preparing method of the same, more precisely a multi-conjugate of siRNA prepared by direct binding of double stranded sense/antisense siRNA monomers or indirect covalent bonding mediated by a cross-linking agent or a polymer, and a preparing method of the same. The preparing method of a siRNA multi-conjugate of the present invention is characterized by simple and efficient reaction and thereby the prepared siRNA multi-conjugate of the present invention has high molecular weight multiple times the conventional siRNA, so that it has high negative charge density, suggesting that it has excellent ionic interaction with a cationic gene carrier and high gene delivery efficiency.

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。