PL2920304T3 - Koniugaty oligonukleotydowe - Google Patents

Koniugaty oligonukleotydowe

Info

Publication number
PL2920304T3
PL2920304T3 PL13791808T PL13791808T PL2920304T3 PL 2920304 T3 PL2920304 T3 PL 2920304T3 PL 13791808 T PL13791808 T PL 13791808T PL 13791808 T PL13791808 T PL 13791808T PL 2920304 T3 PL2920304 T3 PL 2920304T3
Authority
PL
Poland
Prior art keywords
oligonucleotide conjugates
conjugates
oligonucleotide
Prior art date
Application number
PL13791808T
Other languages
English (en)
Inventor
Nanna ALBÆK
Henrik Frydenlund Hansen
Susanne KAMMLER
Jacob Ravn
Henrik ØRUM
Original Assignee
Roche Innovation Center Copenhagen A/S
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Roche Innovation Center Copenhagen A/S filed Critical Roche Innovation Center Copenhagen A/S
Publication of PL2920304T3 publication Critical patent/PL2920304T3/pl

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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/30Chemical structure
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    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12N2330/30Production chemically synthesised
PL13791808T 2012-11-15 2013-11-14 Koniugaty oligonukleotydowe PL2920304T3 (pl)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
EP12192773 2012-11-15
EP13153296 2013-01-30
EP13157237 2013-02-28
EP13174092 2013-06-27
PCT/EP2013/073858 WO2014076195A1 (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates
EP13791808.2A EP2920304B1 (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates

Publications (1)

Publication Number Publication Date
PL2920304T3 true PL2920304T3 (pl) 2019-07-31

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ID=49584733

Family Applications (1)

Application Number Title Priority Date Filing Date
PL13791808T PL2920304T3 (pl) 2012-11-15 2013-11-14 Koniugaty oligonukleotydowe

Country Status (22)

Country Link
US (4) US20150291958A1 (pl)
EP (3) EP3406718A1 (pl)
JP (3) JP6452614B2 (pl)
KR (2) KR20150083920A (pl)
CN (3) CN117126846A (pl)
AU (2) AU2013346767B2 (pl)
BR (2) BR112015011112A2 (pl)
CA (2) CA2889596C (pl)
DK (2) DK2920304T3 (pl)
ES (1) ES2724853T3 (pl)
HK (2) HK1210213A1 (pl)
HR (1) HRP20190826T1 (pl)
IL (2) IL238448B (pl)
MX (2) MX2015005792A (pl)
MY (1) MY173826A (pl)
NZ (1) NZ708171A (pl)
PL (1) PL2920304T3 (pl)
RU (3) RU2018112970A (pl)
SG (2) SG10201804331TA (pl)
SI (1) SI2920304T1 (pl)
WO (2) WO2014076196A1 (pl)
ZA (1) ZA201503375B (pl)

Families Citing this family (256)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2994089A1 (en) 2002-11-18 2004-06-03 Roche Innovation Center Copenhagen A/S Antisense gapmer oligonucleotides
CN103820450B (zh) 2008-10-15 2018-08-21 Ionis制药公司 因子11表达的调节
JP5645840B2 (ja) 2008-12-02 2014-12-24 株式会社Wave Life Sciences Japan リン原子修飾核酸の合成方法
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
US11083444B2 (en) 2010-03-11 2021-08-10 Globus Medical, Inc. Tissue retractor and methods of use
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP2756080B1 (en) 2011-09-14 2019-02-20 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
WO2013173598A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating atp2a2 expression
WO2013173608A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating mecp2 expression
JP2015518710A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
BR112015000784A8 (pt) 2012-07-13 2018-04-03 Wave Life Sciences Japan Grupo auxiliar assimétrico
PL2872147T3 (pl) 2012-07-13 2023-09-25 Wave Life Sciences Ltd. Sposób wytwarzania chiralnych oligonukleotydów
JP2015529469A (ja) 2012-09-14 2015-10-08 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物
CA2889596C (en) 2012-11-15 2022-08-23 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
SG11201505387PA (en) * 2013-01-30 2015-08-28 Hoffmann La Roche Lna oligonucleotide carbohydrate conjugates
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロRNA化合物およびmiR−122をモジュレートする方法
CN105378085B (zh) 2013-05-01 2019-02-15 Ionis制药公司 用于调节hbv和ttr表达的组合物和方法
TWI680767B (zh) 2013-05-01 2020-01-01 美商雷格勒斯治療公司 用於增強的細胞攝取之化合物及方法
WO2015042447A1 (en) 2013-09-20 2015-03-26 Isis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
SG10201808063PA (en) 2014-03-19 2018-10-30 Ionis Pharmaceuticals Inc Compositions for modulating ataxin 2 expression
MX2016012922A (es) 2014-04-01 2017-01-26 Ionis Pharmaceuticals Inc Composiciones para modular la expresion de sod-1.
ES2849600T3 (es) 2014-05-01 2021-08-19 Ionis Pharmaceuticals Inc Conjugados de oligonucleótidos antisentido modificados y su uso para modular la expresión de PKK
CN106459969B (zh) 2014-05-01 2019-11-15 Ionis制药公司 用于调节生长激素受体表达的组合物和方法
MX2016014102A (es) 2014-05-01 2017-05-03 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de similar a la angiopoyetina tipo 3.
PE20190626A1 (es) 2014-05-01 2019-04-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
GB201408623D0 (en) 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
BR112017001931A2 (pt) * 2014-08-07 2017-11-28 Regulus Therapeutics Inc direcionamento de micrornas para distúrbios metabólicos
US20170327524A1 (en) 2014-10-10 2017-11-16 Hoffmann-La Roche, Inc. Galnac phosphoramidites, nucleic acid conjugates thereof and their use
EP3218486A1 (en) * 2014-11-10 2017-09-20 Glaxosmithkline Intellectual Property (No. 2) Limited Combination long acting compositions and methods for hepatitis c
CN107208092B (zh) 2014-12-16 2021-09-10 罗氏创新中心哥本哈根有限公司 手性毒性筛选方法
AU2015364508A1 (en) * 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
WO2016130943A1 (en) * 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
PT3277815T (pt) 2015-04-03 2021-11-11 Beth Israel Deaconess Medical Ct Inc Compostos de oligonucleótidos para o tratamento de pré-eclâmpsia e outros distúrbios angiogénicos
CN116004624A (zh) 2015-04-03 2023-04-25 马萨诸塞大学 用于靶向亨廷汀mRNA的寡核苷酸化合物
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP6835826B2 (ja) 2015-08-24 2021-02-24 ロシュ イノベーション センター コペンハーゲン エーエス Lna−gプロセス
WO2017053995A1 (en) * 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
CN108136041B (zh) 2015-10-02 2022-12-30 罗氏创新中心哥本哈根有限公司 寡核苷酸缀合方法
IL258230B (en) 2015-10-09 2022-09-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods thereof
WO2017068087A1 (en) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Oligonucleotide detection method
US10557137B2 (en) 2015-11-06 2020-02-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
WO2017079745A1 (en) * 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
WO2017081254A1 (en) 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Methods for determining the efficacy profile of a drug candidate
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
US10907160B2 (en) 2016-01-05 2021-02-02 Ionis Pharmaceuticals, Inc. Methods for reducing LRRK2 expression
EP3409780B1 (en) * 2016-01-29 2021-01-20 Kyowa Kirin Co., Ltd. Nucleic acid complex
JP2019503394A (ja) 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts 分岐オリゴヌクレオチド
CN113797348A (zh) 2016-03-07 2021-12-17 箭头药业股份有限公司 用于治疗性化合物的靶向配体
CN114085836B (zh) 2016-03-14 2024-01-26 豪夫迈·罗氏有限公司 用于减少pd-l1表达的寡核苷酸
CA3013799A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Methods of modulating keap1
US10577607B2 (en) 2016-03-16 2020-03-03 Ionis Pharmaceuticals, Inc. Modulation of DYRK1B expression
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
EP3228326A1 (en) * 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
KR102369898B1 (ko) * 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
KR102468177B1 (ko) 2016-04-14 2022-11-16 에프. 호프만-라 로슈 아게 트리틸-모노-GalNAc 화합물 및 이의 용도
KR20230095130A (ko) * 2016-05-06 2023-06-28 아이오니스 파마수티컬즈, 인코포레이티드 Glp-1 수용체 리간드 모이어티 컨쥬게이트된 올리고뉴클레오티드 및 이의 용도
MA45496A (fr) 2016-06-17 2019-04-24 Hoffmann La Roche Molécules d'acide nucléique pour la réduction de l'arnm de padd5 ou pad7 pour le traitement d'une infection par l'hépatite b
US11236339B2 (en) 2016-06-17 2022-02-01 Ionis Pharmaceuticals, Inc. Modulation of GYS1 expression
US10519450B2 (en) 2016-07-01 2019-12-31 Hoffmann-La Roche Inc. Antisense oligonucleotides for modulating HTRA1 expression
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
KR102403408B1 (ko) 2016-09-02 2022-05-30 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
EP3568478A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rel expression
EP3568479A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb1 expression
EP3568481A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating relb expression
EP3568477A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rela expression
WO2018130584A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb2 expression
US11512312B2 (en) 2017-03-10 2022-11-29 The Board Of Regents Of The University Of Texas System Treatment of Fuchs' endothelial corneal dystrophy
WO2018215049A1 (en) 2017-05-23 2018-11-29 F. Hoffmann-La Roche Ag Process for galnac oligonucleotide conjugates
WO2018216785A1 (ja) 2017-05-26 2018-11-29 国立研究開発法人国立循環器病研究センター Pcsk9を標的としたアンチセンス核酸
US20190055564A1 (en) 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
JP2020522265A (ja) * 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物及びその使用方法
EP3599844A4 (en) * 2017-06-07 2021-01-13 University of Massachusetts ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED PROCESSES
WO2019030574A1 (en) 2017-08-10 2019-02-14 Cerenis Therapeutics Holding CARGOMÈRES
WO2019030313A2 (en) 2017-08-11 2019-02-14 Roche Innovation Center Copenhagen A/S OLIGONUCLEOTIDES FOR MODULATION OF UBE3C EXPRESSION
EP3668984A4 (en) 2017-08-18 2021-09-08 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATH FOR THE TREATMENT OF RESPIRATORY DISORDERS
WO2019038228A1 (en) 2017-08-22 2019-02-28 Roche Innovation Center Copenhagen A/S OLIGONUCLEOTIDES FOR MODULATION OF TOM1 EXPRESSION
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
WO2019075357A1 (en) * 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
WO2019073018A1 (en) 2017-10-13 2019-04-18 Roche Innovation Center Copenhagen A/S METHODS OF IDENTIFYING ANTISENSE OLIGONUCLEOTIDE IMPROVED OLIGONUCLEOTIDE PHOSPHOROTHIOATE STEREODEFINIS VARIANTS USING PARTIALLY STEREODEFINIS OLIGONUCLEOTIDE SUB LIBRARIES
EP3645723A1 (en) 2017-10-16 2020-05-06 H. Hoffnabb-La Roche Ag NUCLEIC ACID MOLECULE FOR REDUCTION OF PAPD5 AND PAPD7 mRNA FOR TREATING HEPATITIS B INFECTION
WO2019090359A1 (en) 2017-11-06 2019-05-09 Nitto Denko Corporation Fusogenic compounds for delivery of biologically active molecules
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
CN110997917B (zh) 2017-12-01 2024-04-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
EP3719128A4 (en) 2017-12-01 2021-10-27 Suzhou Ribo Life Science Co., Ltd. DOUBLE STRANDED OLIGONUCLEOTIDE, COMPOSITION AND CONJUGATE WITH DOUBLE STRANDED OLIGONUCLEOTIDE, METHOD OF MANUFACTURING THEREOF AND USE THEREOF
MA51103A (fr) 2017-12-06 2020-10-14 Avidity Biosciences Inc Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique
EP3724333A2 (en) 2017-12-11 2020-10-21 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating fndc3b expression
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
CA3085211A1 (en) 2017-12-21 2019-06-27 F. Hoffmann-La Roche Ag Companion diagnostic for htra1 rna antagonists
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
WO2019122277A1 (en) 2017-12-22 2019-06-27 Roche Innovation Center Copenhagen A/S Novel thiophosphoramidites
BR112020012427A8 (pt) 2017-12-22 2024-04-02 Roche Innovation Ct Copenhagen As Oligonucleotídeos, sal farmaceuticamente aceitável, conjugado, composição farmacêutica e processo para a fabricação de um oligonucleotídeo
EP4092117A1 (en) 2017-12-22 2022-11-23 Roche Innovation Center Copenhagen A/S Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage
CN110959011B (zh) 2017-12-29 2023-03-28 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
CN111699258A (zh) 2018-01-10 2020-09-22 哥本哈根罗氏创新中心 用于调控pias4表达的寡核苷酸
JP2021510295A (ja) 2018-01-12 2021-04-22 ロシュ イノベーション センター コペンハーゲン エーエス Gsk3b発現を調節するためのオリゴヌクレオチド
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
AU2019208006A1 (en) 2018-01-12 2020-07-23 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
MX2020006973A (es) * 2018-01-12 2020-09-09 Roche Innovation Ct Copenhagen As Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos.
TW201934129A (zh) 2018-01-15 2019-09-01 美商Ionis製藥公司 Dnm2表現之調節劑
JP2021510525A (ja) 2018-01-17 2021-04-30 ロシュ イノベーション センター コペンハーゲン エーエス Erc1発現を調節するためのオリゴヌクレオチド
JP2021511029A (ja) 2018-01-18 2021-05-06 ロシュ イノベーション センター コペンハーゲン エーエス Srebp1を標的とするアンチセンスオリゴヌクレオチド
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
BR112020010436A2 (pt) * 2018-01-26 2020-11-24 F. Hoffmann-La Roche Ag oligonucleotídeo radiomarcado, processos para a preparação de um oligonucleotídeo radiomarcado, uso do oligonucleotídeo radiomarcado, método para determinar a biodistribuição e farmacocinética e oligonucleotídeo
CA3088071A1 (en) 2018-02-09 2019-08-15 Genentech, Inc. Oligonucleotides for modulating tmem106b expression
TW202000199A (zh) 2018-03-02 2020-01-01 美商Ionis製藥公司 Irf4 表現之調節劑
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
CN115976028A (zh) 2018-03-09 2023-04-18 第一三共株式会社 糖原病Ia型治疗药
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
CN112055749A (zh) 2018-04-05 2020-12-08 豪夫迈·罗氏有限公司 Fubp1抑制剂用于治疗乙型肝炎病毒感染的用途
CA3094020A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
WO2019215066A1 (en) 2018-05-07 2019-11-14 Roche Innovation Center Copenhagen A/S Quality control of lna oligonucleotide therapeutics using massively parallel sequencing
US20210371860A1 (en) 2018-05-08 2021-12-02 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
SG11202010012PA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing fxi expression
CA3098144A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ. ホフマン-ラ ロシュ アーゲー Atxn2発現を制御するためのオリゴヌクレオチド
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
MX2020013653A (es) 2018-06-27 2021-03-02 Ionis Pharmaceuticals Inc Compuestos y metodos para la reduccion de la expresion de lrrk2.
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
KR102585973B1 (ko) 2018-07-03 2023-10-10 에프. 호프만-라 로슈 아게 타우 발현을 조절하기 위한 올리고뉴클레오티드
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020011653A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020011743A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mafb
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
SG11202100348SA (en) 2018-07-13 2021-02-25 Hoffmann La Roche Oligonucleotides for modulating rtel1 expression
TW202020154A (zh) 2018-07-25 2020-06-01 美商Ionis製藥公司 用於減少atxn2表現之化合物及方法
CA3098267A1 (en) * 2018-07-31 2020-02-06 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
TW202020155A (zh) 2018-07-31 2020-06-01 丹麥商羅氏創新中心哥本哈根有限公司 含有三硫代磷酸酯核苷間連結之寡核苷酸
SG11202101288TA (en) 2018-08-10 2021-03-30 Univ Massachusetts Modified oligonucleotides targeting snps
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
WO2020038968A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2020038973A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting sptlc1
WO2020038971A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting vcan
WO2020043750A1 (en) 2018-08-28 2020-03-05 Roche Innovation Center Copenhagen A/S Neoantigen engineering using splice modulating compounds
EP3620519A1 (en) 2018-09-04 2020-03-11 F. Hoffmann-La Roche AG Use of isolated milk extracellular vesicles for delivering oligonucleotides orally
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
WO2020089260A1 (en) 2018-11-01 2020-05-07 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting tia1
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
US11208650B2 (en) 2018-11-15 2021-12-28 Ionis Pharmaceuticals, Inc. Modulators of IRF5 expression
US20220056455A1 (en) 2018-12-20 2022-02-24 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
EP3898975A2 (en) 2018-12-21 2021-10-27 Boehringer Ingelheim International GmbH Antisense oligonucleotides targeting card9
WO2020150636A1 (en) * 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
CN113365607A (zh) 2019-01-25 2021-09-07 豪夫迈·罗氏有限公司 用于口服药物递送的脂质囊泡
SG11202107399WA (en) 2019-01-31 2021-08-30 Ionis Pharmaceuticals Inc Modulators of yap1 expression
CA3129646A1 (en) 2019-02-20 2020-08-27 Roche Innovation Center Copenhagen A/S Novel phosphoramidites
AU2020225687A1 (en) 2019-02-20 2021-08-19 Roche Innovation Center Copenhagen A/S Phosphonoacetate gapmer oligonucleotides
CN113474633A (zh) 2019-02-26 2021-10-01 罗氏创新中心哥本哈根有限公司 寡核苷酸配制方法
US11279932B2 (en) 2019-02-27 2022-03-22 Ionis Pharmaceuticals, Inc. Modulators of MALAT1 expression
JP2022522898A (ja) * 2019-03-05 2022-04-20 エフ.ホフマン-ラ ロシュ アーゲー 分子の細胞内標的化
SG11202110679YA (en) 2019-03-29 2021-10-28 Ionis Pharmaceuticals Inc Compounds and methods for modulating ube3a-ats
EP3947678A1 (en) 2019-04-02 2022-02-09 ProQR Therapeutics II B.V. Antisense oligonucleotides for immunotherapy
MX2021012098A (es) 2019-04-03 2021-11-03 Bristol Myers Squibb Co Oligonucleotidos antisentido del transcrito tipo 2 de angiopoyetina (angptl2) y sus usos.
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
CN113785060A (zh) 2019-04-04 2021-12-10 豪夫迈·罗氏有限公司 用于调节atxn2表达的寡核苷酸
CA3141874A1 (en) 2019-05-31 2020-12-03 Aligos Therapeutics, Inc. Modified gapmer oligonucleotides and methods of use
CN113950529A (zh) 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
EP3956450A4 (en) 2019-07-26 2022-11-16 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATION OF GFAP
CA3144467A1 (en) * 2019-07-30 2021-02-04 Jonathan Miles Brown Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity
JPWO2021049504A1 (pl) 2019-09-10 2021-03-18
AU2020356931A1 (en) * 2019-10-02 2022-05-26 Sirnaomics, Inc. Oligonucleotides with nucleoside analogs
JP2023506540A (ja) 2019-12-19 2023-02-16 エフ. ホフマン-ラ ロシュ エージー. B型肝炎ウイルス感染を処置するためのscamp3阻害剤の使用
JP2023506550A (ja) 2019-12-19 2023-02-16 エフ. ホフマン-ラ ロシュ エージー. B型肝炎ウイルス感染症を処置するためのsbds阻害剤の使用
CN114901821A (zh) 2019-12-19 2022-08-12 豪夫迈·罗氏有限公司 Sept9抑制剂用于治疗乙型肝炎病毒感染的用途
JP2023506547A (ja) 2019-12-19 2023-02-16 エフ. ホフマン-ラ ロシュ エージー. B型肝炎ウイルス感染を処置するためのcops3阻害剤の使用
EP4058032A4 (en) * 2019-12-19 2024-01-10 Entrada Therapeutics Inc COMPOSITIONS FOR ADMINISTRATION OF ANTISENSE COMPOUNDS
CN114867856A (zh) 2019-12-19 2022-08-05 豪夫迈·罗氏有限公司 Saraf抑制剂用于治疗乙型肝炎病毒感染的用途
AR120817A1 (es) 2019-12-20 2022-03-23 Hoffmann La Roche Oligonucleótidos mejorados para inhibir la expresión de scn9a
EP4081217A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
TW202137987A (zh) 2019-12-24 2021-10-16 瑞士商赫孚孟拉羅股份公司 用於治療hbv之靶向hbv的治療性寡核苷酸及tlr7促效劑之醫藥組合
EP4097234A1 (en) 2020-01-28 2022-12-07 Université de Strasbourg Antisense oligonucleotide targeting linc00518 for treating melanoma
WO2021158810A1 (en) 2020-02-05 2021-08-12 Bristol-Myers Squibb Company Oligonucleotides for splice modulation of camk2d
WO2021170697A1 (en) 2020-02-28 2021-09-02 F. Hoffmann-La Roche Ag Oligonucleotides for modulating cd73 exon 7 splicing
US20220064638A1 (en) 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
CN115916262A (zh) * 2020-04-21 2023-04-04 旗舰创业股份有限公司 双官能分子及其使用方法
CA3181546A1 (en) 2020-05-01 2021-11-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atxn1
CN115605592A (zh) 2020-05-11 2023-01-13 基因泰克公司(Us) 用于治疗神经系统疾病的补体组分c1r抑制剂以及相关的组合物、系统和使用它们的方法
WO2021231211A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
WO2021231204A1 (en) 2020-05-11 2021-11-18 Genentech, Inc. Complement component 4 inhibitors for treating neurological diseases, and related compositons, systems and methods of using same
CN116096888A (zh) 2020-05-13 2023-05-09 豪夫迈·罗氏有限公司 靶向颗粒蛋白前体的寡核苷酸激动剂
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
JP2023526096A (ja) 2020-05-22 2023-06-20 エフ. ホフマン-ラ ロシュ アーゲー Card9のスプライス調節のためのオリゴヌクレオチド
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
CN115702243A (zh) 2020-06-09 2023-02-14 罗氏创新中心哥本哈根有限公司 用于治疗性多核苷酸的鸟苷类似物
AR122731A1 (es) 2020-06-26 2022-10-05 Hoffmann La Roche Oligonucleótidos mejorados para modular la expresión de fubp1
MX2022016338A (es) 2020-06-29 2023-01-24 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la plp1.
EP4185696A1 (en) 2020-07-23 2023-05-31 F. Hoffmann-La Roche AG Oligonucleotides targeting rna binding protein sites
WO2022018155A1 (en) 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
CN111671913B (zh) * 2020-07-30 2022-02-08 四川大学 一种量子点-小核酸偶联物及其用途
EP4200419A2 (en) 2020-08-21 2023-06-28 F. Hoffmann-La Roche AG Use of a1cf inhibitors for treating hepatitis b virus infection
JP2023545406A (ja) * 2020-10-02 2023-10-30 サーナオミクス インコーポレイテッド ウイルス性疾患を治療するためのヌクレオシド含有siRNA
US11447521B2 (en) 2020-11-18 2022-09-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
WO2022117747A2 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
EP4263831A1 (en) 2020-12-18 2023-10-25 F. Hoffmann-La Roche AG Antisense oligonucleotides for targeting progranulin
EP4267734A1 (en) 2020-12-22 2023-11-01 F. Hoffmann-La Roche AG Oligonucleotides targeting xbp1
CN114716518A (zh) * 2021-01-06 2022-07-08 圣诺制药公司 一种能够抑制pcsk9表达的分子构造及药物组合物
EP4284513A1 (en) 2021-01-26 2023-12-06 Universite Brest Bretagne Occidentale Novel stim1 splicing variants and uses thereof
TW202246500A (zh) 2021-02-02 2022-12-01 瑞士商赫孚孟拉羅股份公司 用於抑制 rtel1 表現之增強型寡核苷酸
AU2022234522A1 (en) 2021-03-08 2023-10-26 Les Laboratoires Servier Antisense oligonucleotides for inhibiting alpha-synuclein expression
WO2022232650A1 (en) * 2021-04-30 2022-11-03 Ionis Pharmaceuticals, Inc. Methods for reducing agt expression
WO2022246023A1 (en) 2021-05-20 2022-11-24 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4352222A1 (en) 2021-06-08 2024-04-17 F. Hoffmann-La Roche AG Oligonucleotide progranulin agonists
EP4355759A1 (en) 2021-06-18 2024-04-24 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
WO2022271786A1 (en) 2021-06-23 2022-12-29 University Of Massachusetts Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023034561A2 (en) * 2021-09-02 2023-03-09 Vanderbilt University Lipophilic oligonucleotide conjugates
AU2022345098A1 (en) 2021-09-16 2024-04-04 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
CA3234835A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023078883A1 (en) 2021-11-03 2023-05-11 F. Hoffmann-La Roche Ag Oligonucleotides for modulating apolipoprotein e4 expression
AU2022384619A1 (en) 2021-11-11 2024-04-11 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
WO2023102188A1 (en) * 2021-12-03 2023-06-08 Quralis Corporation Gapmer antisense oligonucleotides with modified backbone chemistries
WO2023104693A1 (en) 2021-12-07 2023-06-15 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting actl6b
WO2023111336A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Oligonucleotide gba agonists
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
WO2023117738A1 (en) 2021-12-20 2023-06-29 F. Hoffmann-La Roche Ag Threose nucleic acid antisense oligonucleotides and methods thereof
WO2023141507A1 (en) 2022-01-20 2023-07-27 Genentech, Inc. Antisense oligonucleotides for modulating tmem106b expression
TW202346587A (zh) 2022-02-21 2023-12-01 瑞士商赫孚孟拉羅股份公司 反義寡核苷酸
WO2023217890A1 (en) 2022-05-10 2023-11-16 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting cfp-elk1 intergene region
WO2023222858A1 (en) 2022-05-18 2023-11-23 F. Hoffmann-La Roche Ag Improved oligonucleotides targeting rna binding protein sites
WO2023242324A1 (en) 2022-06-17 2023-12-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for targeting progranulin
EP4332221A1 (en) 2022-08-29 2024-03-06 Roche Innovation Center Copenhagen A/S Threose nucleic acid antisense oligonucleotides and methods thereof
WO2024052403A1 (en) 2022-09-06 2024-03-14 F. Hoffmann-La Roche Ag Double-stranded rna molecule for administration to the eye

Family Cites Families (207)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US603095A (en) 1898-04-26 Machine for dishing metal
US2699808A (en) 1944-10-06 1955-01-18 Mark W Lowe Apparatus for peeling tomatoes
US2699508A (en) 1951-12-21 1955-01-11 Selectronics Inc Method of mounting and construction of mounting for low frequency piezoelectric crystals
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US4962029A (en) 1987-10-02 1990-10-09 Cetus Corporation Covalent oligonucleotide-horseradish peroxidase conjugate
US4914210A (en) 1987-10-02 1990-04-03 Cetus Corporation Oligonucleotide functionalizing reagents
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5354844A (en) 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US6395492B1 (en) 1990-01-11 2002-05-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
EP0556301B1 (en) 1990-11-08 2001-01-10 Hybridon, Inc. Incorporation of multiple reporter groups on synthetic oligonucleotides
DE4104186A1 (de) 1991-02-12 1992-08-13 Genentech Inc Neue, ueber endozytose in hoehere eukaryotische zellen aufnehmbare, nukleinsaeure enthaltende komplexe
DE4110409C2 (de) 1991-03-29 1999-05-27 Boehringer Ingelheim Int Neue Protein-Polykation-Konjugate
KR950014915B1 (ko) 1991-06-19 1995-12-18 주식회사녹십자 탈시알로당단백-포함화합물
EP0724447B1 (en) 1991-10-24 2003-05-07 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
DE69433036T2 (de) 1993-09-03 2004-05-27 Isis Pharmaceuticals, Inc., Carlsbad Aminoderivatisierte nukleoside und oligonukleoside
KR100357839B1 (ko) 1994-03-07 2003-08-02 더 다우 케미칼 캄파니 생체활성및/또는표적화된덴드리머콘쥬게이트
US5646262A (en) 1994-07-28 1997-07-08 Georgetown University Antisense oligonucleotides against hepatitis B viral replication
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
JP3307945B2 (ja) 1994-10-06 2002-07-29 アイシス・ファーマシューティカルス・インコーポレーテッド ペプチド核酸コンジュゲート
US5684142A (en) 1995-06-07 1997-11-04 Oncor, Inc. Modified nucleotides for nucleic acid labeling
US5672662A (en) 1995-07-07 1997-09-30 Shearwater Polymers, Inc. Poly(ethylene glycol) and related polymers monosubstituted with propionic or butanoic acids and functional derivatives thereof for biotechnical applications
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
CN1120707C (zh) 1995-11-22 2003-09-10 约翰斯·霍普金斯大学 增强生物分子的细胞摄取的配体
US6344436B1 (en) 1996-01-08 2002-02-05 Baylor College Of Medicine Lipophilic peptides for macromolecule delivery
US5656408A (en) 1996-04-29 1997-08-12 Xerox Corporation Coated carrier particles
US5776907A (en) 1996-05-20 1998-07-07 Texas Biotechnology Corporation Mitomycin oligonucleotide conjugates
US6001991A (en) * 1996-10-04 1999-12-14 Isis Pharmaceuticals Inc. Antisense oligonucleotide modulation of MDR P-glycoprotein gene expression
US20030014318A1 (en) 1996-11-08 2003-01-16 Matthew Byrne International trading system and method
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US5770716A (en) 1997-04-10 1998-06-23 The Perkin-Elmer Corporation Substituted propargylethoxyamido nucleosides, oligonucleotides and methods for using same
US6723706B2 (en) * 1997-05-05 2004-04-20 Aventis Pharma Deutschland Gmbh Modified antisense nucleotides complementary to a section of the human Ha-ras gene
GB2341390B (en) 1997-05-21 2000-11-08 Univ Leland Stanford Junior Composition and method for enhancing transport across biological membranes
NZ503765A (en) 1997-09-12 2002-04-26 Exiqon As Bi-cyclic and tri-cyclic nucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6096875A (en) 1998-05-29 2000-08-01 The Perlein-Elmer Corporation Nucleotide compounds including a rigid linker
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6335432B1 (en) 1998-08-07 2002-01-01 Bio-Red Laboratories, Inc. Structural analogs of amine bases and nucleosides
US6335437B1 (en) 1998-09-07 2002-01-01 Isis Pharmaceuticals, Inc. Methods for the preparation of conjugated oligomers
US6365379B1 (en) 1998-10-06 2002-04-02 Isis Pharmaceuticals, Inc. Zinc finger peptide cleavage of nucleic acids
ATE260337T1 (de) 1998-12-02 2004-03-15 Idm Immuno Designed Molecules Neue oligomere konjugate zum transfer biologischer moleküle in zellen
CA2372085C (en) 1999-05-04 2009-10-27 Exiqon A/S L-ribo-lna analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US20040146516A1 (en) 1999-06-17 2004-07-29 Utah Ventures Ii L.P. Lumen-exposed molecules and methods for targeted delivery
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
EP1224280A2 (en) 1999-10-04 2002-07-24 Exiqon A/S Design of high affinity rnase h recruiting oligonucleotide
US20080039414A1 (en) 2002-02-20 2008-02-14 Sima Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
CA2442092A1 (en) 2001-03-26 2002-10-17 Ribozyme Pharmaceuticals, Inc. Oligonucleotide mediated inhibition of hepatitis b virus and hepatitis c virus replication
EP1572067A4 (en) 2001-05-18 2009-05-13 Sirna Therapeutics Inc CONJUGATES AND COMPOSITIONS FOR CELL DELIVERY
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US7259150B2 (en) 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
US20030191075A1 (en) * 2002-02-22 2003-10-09 Cook Phillip Dan Method of using modified oligonucleotides for hepatic delivery
AU2003237875A1 (en) 2002-05-15 2003-12-02 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein b expression
GB2389824B (en) 2002-06-21 2005-08-24 Paul Sinclair Improvements in or relating to funeral vehicles
US7655790B2 (en) 2002-07-12 2010-02-02 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US7087229B2 (en) 2003-05-30 2006-08-08 Enzon Pharmaceuticals, Inc. Releasable polymeric conjugates based on aliphatic biodegradable linkers
US6878805B2 (en) 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
WO2004044141A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Conjugated oligomeric compounds and their use in gene modulation
AU2003290596B2 (en) 2002-11-05 2011-05-12 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
JP4986109B2 (ja) 2002-11-13 2012-07-25 ジェンザイム・コーポレーション アポリポタンパク質b発現のアンチセンス調節
US7511131B2 (en) 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
CA2994089A1 (en) 2002-11-18 2004-06-03 Roche Innovation Center Copenhagen A/S Antisense gapmer oligonucleotides
US7816337B2 (en) 2003-02-18 2010-10-19 Roche Madison Inc. Reversible attachment of a membrane active polymer to a polynucleotide
WO2004087931A1 (en) 2003-04-03 2004-10-14 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
WO2005002507A2 (en) * 2003-06-03 2005-01-13 Isis Pharmaceuticals, Inc. Modulation of survivin expression
DE60319354T2 (de) 2003-07-11 2009-03-26 Lbr Medbiotech B.V. Mannose-6-Phosphat Rezeptor vermittelter Gentransfer zu Muskelzellen
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
ATE555118T1 (de) 2003-08-28 2012-05-15 Takeshi Imanishi Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung
EP1677822B1 (en) 2003-09-18 2014-04-23 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
US7615539B2 (en) * 2003-09-25 2009-11-10 Coley Pharmaceutical Group, Inc. Nucleic acid-lipophilic conjugates
DE602004027163D1 (de) 2003-12-23 2010-06-24 Santaris Pharma As Oligomere verbindungen zur modulation von bcl-2
US20050261170A1 (en) 2004-01-22 2005-11-24 Immunomedics, Inc. Folate conjugates and complexes
DK1713912T3 (da) 2004-01-30 2013-12-16 Santaris Pharma As Modificerede Korte Interfererende RNA (Modificerede siRNA)
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
EP1737879B1 (en) 2004-04-19 2012-10-10 Archemix LLC Aptamer-mediated intracellular delivery of therapeutic oligonucleotides
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
WO2006112872A2 (en) * 2004-08-04 2006-10-26 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
EP2409713B1 (en) 2004-08-10 2015-07-22 Genzyme Corporation Oligonucleotides for use in modulating lipoprotein and cholesterol levels in humans
WO2006036916A2 (en) * 2004-09-24 2006-04-06 Alnylam Pharmaceuticals, Inc. Rnai modulation of apob and uses thereof
WO2006050734A2 (en) * 2004-11-09 2006-05-18 Santaris Pharma A/S Potent lna oligonucleotides for the inhibition of hif-1a expression
AU2005304110B2 (en) * 2004-11-09 2009-06-11 Enzon Pharmaceuticals, Inc. LNA oligonucleotides and the treatment of cancer
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
WO2006078217A1 (en) 2005-01-24 2006-07-27 Avaris Ab COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
EP2338992A3 (en) 2005-08-29 2011-10-12 Regulus Therapeutics, Inc Antisense compounds having enhanced anti-microRNA activity
EP2338991B1 (en) 2005-08-29 2017-01-18 Regulus Therapeutics Inc. Methods for use in modulating MIR-122a
EP1931778A2 (en) * 2005-09-15 2008-06-18 Santaris Pharma A/S RNA ANTAGONIST COMPOUNDS FOR THE INHIBITION OF APO-Bl00 EXPRESSION
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
WO2007035759A1 (en) 2005-09-19 2007-03-29 Johnson & Johnson Pharmaceutical Research & Development L.L.C. Modulation of glucocorticoid receptor expression
JP2009515523A (ja) 2005-11-10 2009-04-16 サンタリス ファーマ アー/エス Tnfスーパーファミリー受容体に対するスプライス切替オリゴマーならびに疾病治療における該オリゴマーの使用
EP1984381B1 (en) 2006-01-27 2010-09-29 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
WO2007085485A2 (en) 2006-01-27 2007-08-02 Santaris Pharma A/S Lna modified phosphorothiolated oligonucleotides
AU2007229161B2 (en) 2006-03-23 2012-07-12 Roche Innovation Center Copenhagen A/S Small internally segmented interfering RNA
GB0606415D0 (en) 2006-03-31 2006-05-10 Univ Southampton Topical drug delivery
CA2649045C (en) 2006-04-03 2019-06-11 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
ES2569558T3 (es) * 2006-04-03 2016-05-11 Roche Innovation Center Copenhagen A/S Composición farmacéutica que comprende oligonucleótidos antisentido anti-ARNmi
JP5825754B2 (ja) 2006-05-05 2015-12-02 アイシス ファーマシューティカルズ, インコーポレーテッド Apobの発現を調節するための化合物および方法
ES2389737T3 (es) 2006-05-11 2012-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en 5'
AU2007296054B2 (en) 2006-09-15 2013-03-28 Enzon Pharmaceuticals, Inc. Hindered ester-based biodegradable linkers for oligonucleotide delivery
WO2008034123A2 (en) 2006-09-15 2008-03-20 Enzon Pharmaceuticals, Inc. Polymeric conjugates containing positively-charged moieties
MX2009003398A (es) 2006-09-27 2009-08-12 Coley Pharm Gmbh Analogos de oligonucleotidos cpg que contienen analogos t hidrofobos con actividad inmunoestimuladora mejorada.
WO2008040355A2 (en) 2006-10-06 2008-04-10 Exiqon A/S Novel methods for quantification of micrornas and small interfering rnas
US20100216864A1 (en) 2006-10-09 2010-08-26 Ellen Marie Straarup RNA Antagonist Compounds for the Modulation of PCSK9
CA2666981A1 (en) 2006-10-20 2008-05-02 Ercole Biotech, Inc. Soluble tnf receptors and their use in treatment of disease
MX2009005527A (es) 2006-11-27 2009-06-08 Isis Pharmaceuticals Inc Metodos para el tratamiento de hipercolesterolemia.
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
WO2008109369A2 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
DE102007012908A1 (de) 2007-03-19 2008-09-25 Momentive Performance Materials Gmbh Neue Polyamid-Polysiloxan-Verbindungen
CA2681406A1 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
KR20090128494A (ko) 2007-03-24 2009-12-15 겐자임 코포레이션 인간 아포리포프로틴 b에 상보적인 안티센스 올리고뉴클레오타이드 투여
JP2010524476A (ja) 2007-05-01 2010-07-22 サンタリス ファーマ アー/エス Tnfスーパーファミリー受容体についてのスプライススイッチオリゴマーおよび疾患の治療におけるそれらの使用方法
TW200848077A (en) 2007-05-01 2008-12-16 Santaris Pharma As Compounds for the modulation of beta-cantenin expression
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
JP5864100B2 (ja) 2007-06-29 2016-02-17 サレプタ セラピューティクス インコーポレイテッド 組織特異的ペプチドコンジュゲートおよび方法
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
CN101816010A (zh) 2007-07-19 2010-08-25 数据匙电子有限公司 Rf令牌和接纳器系统与方法
EP2192914A4 (en) 2007-08-20 2014-05-07 Belrose Pharma Inc POLYMER BINDERS CONTAINING PYRIDYL DISULFIDE GROUPS
BRPI0817527A2 (pt) 2007-10-01 2017-05-02 Isis Pharmaceuticals Inc modulação antissenso da expressão do receptor de fator de crescimento de fibroblasto humano 4
WO2009043353A2 (en) * 2007-10-04 2009-04-09 Santaris Pharma A/S Micromirs
US20100280098A1 (en) 2007-10-05 2010-11-04 Juliano Rudolph L Receptor targeted oligonucleotides
KR20100110298A (ko) 2007-11-26 2010-10-12 산타리스 팔마 에이/에스 안드로겐 수용체를 표적화하는 lna 길항제
MX2010006039A (es) 2007-12-03 2010-06-23 Enzon Pharmaceuticals Inc Compuestos antagonistas del acido ribonucleico para la modulacion de la expresion de la fosfoinositida-3-cinasa catalitica.
CA2708173C (en) * 2007-12-04 2016-02-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
US8530640B2 (en) 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
AU2009221064B2 (en) 2008-03-07 2014-12-11 Roche Innovation Center Copenhagen A/S Pharmaceutical compositions for treatment of microRNA related diseases
EP2285819B1 (en) 2008-04-04 2013-10-16 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
WO2009126933A2 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2009148605A2 (en) 2008-06-04 2009-12-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
JP2011526482A (ja) 2008-07-03 2011-10-13 サンタリス ファーマ アー/エス ミトコンドリアグリセロール−3−リン酸アシルトランスフェラーゼ1(mtgpat1)の発現を阻害するためのrnaアンタゴニスト化合物
NZ591057A (en) 2008-07-10 2012-11-30 Az Univ Amsterdam Complement antagonists and uses thereof
US9033197B2 (en) 2008-07-17 2015-05-19 Nir Bar Spool holder
US7932256B2 (en) 2008-07-31 2011-04-26 Bristol-Myers Squibb Company (S)-4-(1-cyclopropyl-2-methoxyethyl)-6-(6-(difluoromethoxy)-2,5-dimethylpyridin-3-ylamino)-5-oxo-4,5-dihydropyrazine-2-carbonitrile: a pyrazinone modulator of corticotropin-releasing factor receptor activity
EP2323667A4 (en) 2008-08-07 2012-07-25 Isis Pharmaceuticals Inc MODULATION OF TRANSTHYRETIN EXPRESSION BY TREATMENT OF CNS DISEASES
WO2010045584A1 (en) 2008-10-17 2010-04-22 Endocyte, Inc. Folate targeting of nucleotides
JP5793423B2 (ja) * 2008-12-31 2015-10-14 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブRoche Innovation Center Copenhagen A/S 急性冠症候群の治療のためのLNAApoBアンチセンスオリゴマーの使用
AT507215B1 (de) 2009-01-14 2010-03-15 Boehler Edelstahl Gmbh & Co Kg Verschleissbeständiger werkstoff
WO2010120508A2 (en) 2009-03-31 2010-10-21 The General Hospital Corporation Regulation of mir-33 micrornas in the treatment of cholesterol-related disorders
EP2421970B1 (en) 2009-04-24 2016-09-07 Roche Innovation Center Copenhagen A/S Pharmaceutical compositions for treatment of hcv patients that are non-responders to interferon
ES2555057T3 (es) 2009-06-12 2015-12-28 Roche Innovation Center Copenhagen A/S Nuevos potentes compuestos antisentido anti-ApoB
EP2456870A1 (en) 2009-07-21 2012-05-30 Santaris Pharma A/S Antisense oligomers targeting pcsk9
BR112012008865A2 (pt) 2009-10-16 2019-09-24 Glaxo Group Ltd inibidores de hbv anti-sentido.
KR101663617B1 (ko) 2009-10-29 2016-10-07 엘지전자 주식회사 하향링크 기준신호 송수신 방법 및, 이를 이용한 기지국 및 사용자기기
US20130023578A1 (en) * 2009-12-31 2013-01-24 Samyang Biopharmaceuticals Corporation siRNA for inhibition of c-Met expression and anticancer composition containing the same
DK2521556T3 (en) 2010-01-08 2018-08-13 Ionis Pharmaceuticals Inc MODULATION OF ANGIOPOIETIN-LIKE 3 EXPRESSION
WO2011088309A1 (en) 2010-01-14 2011-07-21 Regulus Therapeutics Inc. Microrna compositions and methods
JP6137834B2 (ja) 2010-02-24 2017-05-31 アローヘッド ファーマシューティカルズ インコーポレイテッド siRNAの標的化送達のための組成物
WO2011123621A2 (en) 2010-04-01 2011-10-06 Alnylam Pharmaceuticals Inc. 2' and 5' modified monomers and oligonucleotides
WO2011126937A1 (en) 2010-04-06 2011-10-13 The University Of North Carolina At Chapel Hill Targeted intracellular delivery of oligonucleotides via conjugation with small molecule ligands
WO2011130458A2 (en) 2010-04-13 2011-10-20 John Rossi Rna aptamers against baff-r as cell-type specific delivery agents and methods for their use
BR112012026471A2 (pt) 2010-04-19 2016-11-29 Nlife Therapeutics S L conjugado, processo para a síntese do mesmo, método para imagem de uma célula que expressa um transportador de neurotransmissor que compreende o contato da referida célula com o conjugado, processo para preparar um comporto de fórmula (ii) e (xiv) e composto
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
KR20180026798A (ko) 2010-04-29 2018-03-13 아이오니스 파마수티컬즈, 인코포레이티드 트랜스티레틴 발현의 조절
US8815826B2 (en) 2010-07-23 2014-08-26 Regulus Therapeutics, Inc. Targeting microRNAs for the treatment of fibrosis
EP2612914B1 (en) 2010-08-31 2017-06-21 Osaka University Oligonucleotide, and therapeutic agent for dyslipidemia containing oligonucleotide as active ingredient
US10378016B2 (en) 2010-12-06 2019-08-13 Children's Medical Center Corporation Composition and method for oligonucleotide delivery
EP3843313A1 (en) 2010-12-13 2021-06-30 Telefonaktiebolaget LM Ericsson (publ) Exchange of parameters relating to measurement periods
BR112013013434A2 (pt) 2010-12-17 2016-08-16 Arrowhead Res Corp metade que tem por alvo modulador fármaco cinético em cacho de galactose para sirna
BR122020024394B1 (pt) 2010-12-29 2021-05-11 F. Hoffmann-La Roche Ag conjugado e composição farmacêutica
CN103491982B (zh) 2010-12-29 2017-09-12 箭头研究公司 具有酶敏感性连接的多核苷酸体内递送偶联物
WO2012099755A1 (en) 2011-01-11 2012-07-26 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
US20120295961A1 (en) 2011-04-21 2012-11-22 Swayze Eric E Modulation of hepatitis b virus (hbv) expression
EP2699583A4 (en) 2011-04-21 2015-04-15 Isis Pharmaceuticals Inc MODULATION OF EXPRESSION OF HEPATITIS B VIRUS (HBV)
HUE031595T2 (en) 2011-04-25 2017-07-28 Regulus Therapeutics Inc Micro RNA Compounds s Methods for Modulating Mir-21 Activity
CN102226185A (zh) * 2011-05-09 2011-10-26 华中科技大学同济医学院附属同济医院 靶向肝细胞的rna适配子及其核苷酸序列
WO2012174154A1 (en) 2011-06-13 2012-12-20 Isis Pharmaceuticals, Inc. Modulation of inflammatory responses by factor vii
AU2012271357A1 (en) 2011-06-16 2013-05-02 Ionis Pharmaceuticals, Inc. Antisense modulation of fibroblast growth factor receptor 4 expression
MX342731B (es) 2011-06-30 2016-10-11 Arrowhead Res Corp Composiciones y metodos para inhibir la expresion genica del virus de hepatitis b.
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2756080B1 (en) * 2011-09-14 2019-02-20 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
AU2012327227A1 (en) 2011-11-07 2013-05-23 Isis Pharmaceuticals, Inc. Administration of Factor XI antisense oligonucleotides
JP2015507924A (ja) 2012-02-08 2015-03-16 アイシス ファーマシューティカルズ, インコーポレーテッド 第vii因子の発現を調節するための方法と組成物
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013159109A1 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Modulation of hepatitis b virus (hbv) expression
US9984408B1 (en) 2012-05-30 2018-05-29 Amazon Technologies, Inc. Method, medium, and system for live video cooperative shopping
CA2889596C (en) 2012-11-15 2022-08-23 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
SG11201505387PA (en) 2013-01-30 2015-08-28 Hoffmann La Roche Lna oligonucleotide carbohydrate conjugates
CN105378085B (zh) * 2013-05-01 2019-02-15 Ionis制药公司 用于调节hbv和ttr表达的组合物和方法
CA2928349A1 (en) 2013-11-14 2015-05-21 Roche Innovation Center Copenhagen A/S Apob antisense conjugate compounds
US9778708B1 (en) 2016-07-18 2017-10-03 Lenovo Enterprise Solutions (Singapore) Pte. Ltd. Dual sided latching retainer for computer modules

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