SG11201503821YA - Oligonucleotide conjugates - Google Patents

Oligonucleotide conjugates

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Publication number
SG11201503821YA
SG11201503821YA SG11201503821YA SG11201503821YA SG11201503821YA SG 11201503821Y A SG11201503821Y A SG 11201503821YA SG 11201503821Y A SG11201503821Y A SG 11201503821YA SG 11201503821Y A SG11201503821Y A SG 11201503821YA SG 11201503821Y A SG11201503821Y A SG 11201503821YA
Authority
SG
Singapore
Prior art keywords
oligonucleotide conjugates
conjugates
oligonucleotide
Prior art date
Application number
SG11201503821YA
Inventor
Nanna Albæk
Henrik Frydenlund Hansen
Susanne Kammler
Jacob Ravn
HENRIK øRUM
Original Assignee
Roche Innovation Ct Copenhagen As
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 Ct Copenhagen As filed Critical Roche Innovation Ct Copenhagen As
Publication of SG11201503821YA publication Critical patent/SG11201503821YA/en

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
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    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
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    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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SG11201503821YA 2012-11-15 2013-11-14 Oligonucleotide conjugates SG11201503821YA (en)

Applications Claiming Priority (5)

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

Publications (1)

Publication Number Publication Date
SG11201503821YA true SG11201503821YA (en) 2015-06-29

Family

ID=49584733

Family Applications (2)

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SG10201804331TA SG10201804331TA (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates
SG11201503821YA SG11201503821YA (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG10201804331TA SG10201804331TA (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates

Country Status (22)

Country Link
US (4) US20150291958A1 (en)
EP (3) EP2920307B1 (en)
JP (3) JP2016503300A (en)
KR (2) KR102112892B1 (en)
CN (3) CN104884618A (en)
AU (2) AU2013346767B2 (en)
BR (2) BR112015010116A2 (en)
CA (2) CA2889596C (en)
DK (2) DK2920307T3 (en)
ES (1) ES2724853T3 (en)
HK (2) HK1210213A1 (en)
HR (1) HRP20190826T1 (en)
IL (2) IL238448B (en)
MX (2) MX2015005792A (en)
MY (1) MY173826A (en)
NZ (1) NZ708171A (en)
PL (1) PL2920304T3 (en)
RU (3) RU2653438C2 (en)
SG (2) SG10201804331TA (en)
SI (1) SI2920304T1 (en)
WO (2) WO2014076195A1 (en)
ZA (1) ZA201503375B (en)

Families Citing this family (257)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2284269T3 (en) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense design
CA2966011C (en) 2008-10-15 2021-10-19 Ionis Pharmaceuticals, Inc. Modulation of factor 11 expression
KR101881596B1 (en) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 Method for the synthesis of phosphorous atom modified nucleic acids
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
WO2012039448A1 (en) 2010-09-24 2012-03-29 株式会社キラルジェン Asymmetric auxiliary group
WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
CA2848753C (en) 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
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
JP2015518713A (en) 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 Compositions and methods for modulating UTRN expression
EP2850188A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc Compositions and methods for modulating hemoglobin gene family expression
EA201492116A1 (en) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. COMPOSITIONS AND METHODS FOR MODULATING THE EXPRESSION OF MECP2
CA2873794A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating smn gene family expression
KR102213609B1 (en) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 Chiral control
EP2872485B1 (en) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetric auxiliary group
CA2884608A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US20150291958A1 (en) 2012-11-15 2015-10-15 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
RU2018108405A (en) * 2013-01-30 2019-02-26 Ф. Хоффманн-Ля Рош Аг CARBOHYDRATE AND LNA-OLIGONUCLEOTIDE CONJUGATES
CA2909868C (en) 2013-05-01 2021-10-19 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
AU2014259759B2 (en) 2013-05-01 2020-06-18 Ionis Pharmaceuticals, Inc. Compositions and methods
TW201446791A (en) 2013-05-01 2014-12-16 Regulus Therapeutics Inc MicroRNA compounds and methods for modulating miR-122
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
WO2015061536A1 (en) 2013-10-25 2015-04-30 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
JPWO2015108048A1 (en) 2014-01-15 2017-03-23 株式会社新日本科学 Chiral nucleic acid adjuvant and antitumor agent having antitumor activity
WO2015108047A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
AU2015207773B2 (en) 2014-01-16 2021-06-17 Wave Life Sciences Ltd. Chiral design
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
DK3119888T3 (en) 2014-03-19 2021-09-06 Ionis Pharmaceuticals Inc COMPOSITIONS FOR MODULATING ATAXIN-2 EXPRESSION
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
EP3757214B1 (en) 2014-04-01 2022-06-15 Biogen MA Inc. Compositions for modulating sod-1 expression
JP6667453B2 (en) 2014-05-01 2020-03-18 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Compositions and methods for modulating growth hormone receptor expression
EP3845547A1 (en) 2014-05-01 2021-07-07 Ionis Pharmaceuticals, Inc. Galnac3 conjugated modified oligonucleotide for modulating angiopoietin-like 3 expression
PL3608406T3 (en) 2014-05-01 2023-05-22 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating complement factor b expression
MX2016014140A (en) 2014-05-01 2017-09-15 Ionis Pharmaceuticals Inc Compositions and methods for modulating pkk expression.
GB201408623D0 (en) 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
US9487783B2 (en) * 2014-08-07 2016-11-08 Regulus Therapeutics Inc. Targeting microRNAs for metabolic disorders
KR20170068469A (en) 2014-10-10 2017-06-19 에프. 호프만-라 로슈 아게 Galnac phosphoramidites, nucleic acid conjugates thereof and their use
PE20170922A1 (en) * 2014-11-10 2017-07-12 Glaxosmithkline Intellectual Property (No 2) Ltd LONG-ACTING COMBINATION COMPOSITIONS AND PROCEDURES FOR HEPATITIS C
CN107208092B (en) 2014-12-16 2021-09-10 罗氏创新中心哥本哈根有限公司 Chiral toxicity screening method
US20170369872A1 (en) * 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 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
CA2980337A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
EP3277815B1 (en) 2015-04-03 2021-09-22 University of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
CA2995110A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP6835826B2 (en) 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 process
EP3353305A4 (en) * 2015-09-25 2019-09-18 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
JP6893505B2 (en) 2015-10-02 2021-06-23 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 Oligonucleotide Conjugation Method
CR20180233A (en) 2015-10-09 2018-05-25 Wave Life Sciences Ltd OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS
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
US20190046555A1 (en) * 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
ES2852998T3 (en) 2015-11-12 2021-09-14 Hoffmann La Roche Oligonucleotides to induce expression of paternal UBE3A
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
WO2017131236A1 (en) * 2016-01-29 2017-08-03 協和発酵キリン株式会社 Nucleic acid complex
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
KR102515329B1 (en) 2016-03-07 2023-03-29 애로우헤드 파마슈티컬스 인코포레이티드 Targeting ligands for therapeutic compounds
ES2857702T3 (en) 2016-03-14 2021-09-29 Hoffmann La Roche Oligonucleotides for reduction of PD-L1 expression
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
WO2017161172A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Methods of modulating keap1
MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
EP3228326A1 (en) * 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
CA3020343A1 (en) * 2016-04-08 2017-10-12 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
CN109153697A (en) 2016-04-14 2019-01-04 豪夫迈·罗氏有限公司 Mono- GalNAc compound of trityl-and application thereof
EP3452049A4 (en) * 2016-05-06 2020-01-08 Ionis Pharmaceuticals, Inc. Glp-1 receptor ligand moiety conjugated oligonucleotides and uses thereof
MA45496A (en) 2016-06-17 2019-04-24 Hoffmann La Roche NUCLEIC ACID MOLECULES FOR PADD5 OR PAD7 MRNA REDUCTION FOR TREATMENT OF HEPATITIS B INFECTION
WO2017219017A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
SG10202005885VA (en) 2016-07-01 2020-07-29 Hoffmann La Roche Antisense oligonucleotides for modulating htra1 expression
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
US10294474B2 (en) 2016-09-02 2019-05-21 Arrowhead Pharmaceuticals, Inc. Targeting ligands
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (en) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
EP3548620A4 (en) 2016-12-02 2020-07-22 Cold Spring Harbor Laboratory Modulation of lnc05 expression
WO2018130583A1 (en) 2017-01-13 2018-07-19 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb1 expression
US20190345495A1 (en) 2017-01-13 2019-11-14 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating nfkb2 expression
US20190338286A1 (en) 2017-01-13 2019-11-07 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rel expression
US20200216845A1 (en) 2017-01-13 2020-07-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating rela expression
EP3568481A1 (en) 2017-01-13 2019-11-20 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides for modulating relb expression
CN110536964A (en) 2017-03-10 2019-12-03 国立研究开发法人国立成育医疗研究中心 Antisense oligonucleotides and the prevention of glycogen storage disease Ia type or therapeutic composition
EP3592365A4 (en) 2017-03-10 2021-01-13 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
JP6952366B2 (en) 2017-05-26 2021-10-20 国立研究開発法人国立循環器病研究センター Antisense nucleic acid targeting PCSK9
US20190055564A1 (en) 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
US20210198305A1 (en) * 2017-06-02 2021-07-01 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP3599844A4 (en) * 2017-06-07 2021-01-13 University of Massachusetts Anti-adam33 oligonucleotides and related methods
TW201919712A (en) 2017-08-10 2019-06-01 法商塞勒尼斯醫療控股公司 Cargomers
WO2019030313A2 (en) 2017-08-11 2019-02-14 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating ube3c expression
WO2019036613A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
WO2019038228A1 (en) 2017-08-22 2019-02-28 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating tom1 expression
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. Modulators of smad7 expression
SG11202001783YA (en) 2017-10-12 2020-03-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN111226114A (en) 2017-10-13 2020-06-02 罗氏创新中心哥本哈根有限公司 Method for identifying improved variants of antisense oligonucleotides using a subset of sterically defined oligonucleotides
BR122020018622A2 (en) 2017-10-16 2021-02-09 F. Hoffmann-La Roche Ag NUCLEIC ACID MOLECULE FOR REDUCING PAPD5 AND PAPD7 OF MRNA FOR THE TREATMENT OF HEPATITIS B INFECTION
EP3706729A4 (en) 2017-11-06 2021-12-01 Nitto Denko Corporation Fusogenic compounds for delivery of biologically active molecules
TWI809004B (en) 2017-11-09 2023-07-21 美商Ionis製藥公司 Compounds and methods for reducing snca expression
WO2019105419A1 (en) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 Nucleic acid, composition and conjugate containing same, preparation method, and use
EP3719128A4 (en) 2017-12-01 2021-10-27 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
MX2020005860A (en) 2017-12-06 2020-09-09 Avidity Biosciences Inc Compositions and methods of treating muscle atrophy and myotonic dystrophy.
US20200385714A1 (en) 2017-12-11 2020-12-10 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
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
US20200378970A1 (en) 2017-12-21 2020-12-03 Hoffmann-La Roche Inc. Companion diagnostic for htra1 rna antagonists
MX2020005754A (en) 2017-12-22 2020-08-20 Roche Innovation Ct Copenhagen As Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage.
KR20200104302A (en) 2017-12-22 2020-09-03 로슈 이노베이션 센터 코펜하겐 에이/에스 New thiophosphoramidite
WO2019122279A1 (en) 2017-12-22 2019-06-27 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage
CN116375774A (en) 2017-12-29 2023-07-04 苏州瑞博生物技术股份有限公司 Conjugate, preparation method and application thereof
JP2021511022A (en) 2018-01-10 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 Oligonucleotides for regulating PIAS4 expression
SG11202006142PA (en) * 2018-01-12 2020-07-29 Roche Innovation Ct Copenhagen As Alpha-synuclein antisense oligonucleotides and uses thereof
CA3087966A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
JP2021510295A (en) 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 Oligonucleotides for regulating GSK3B expression
US11447775B2 (en) 2018-01-12 2022-09-20 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
US20200392510A1 (en) 2018-01-15 2020-12-17 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US20210095276A1 (en) 2018-01-17 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating erc1 expression
US20210095277A1 (en) 2018-01-18 2021-04-01 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting srebp1
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
CN111867638A (en) * 2018-01-26 2020-10-30 豪夫迈·罗氏有限公司 Radiolabeled oligonucleotides and methods of making same
CR20200346A (en) 2018-02-09 2020-10-19 Genentech Inc Oligonucleotides for modulating tmem106b expression
SG11202007652UA (en) 2018-02-21 2020-09-29 Bristol Myers Squibb Co Camk2d antisense oligonucleotides and uses thereof
JP7239597B2 (en) 2018-03-02 2023-03-14 アイオーニス ファーマシューティカルズ， インコーポレーテッド Regulators of IRF4 expression
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
KR20210130854A (en) 2018-03-09 2021-11-01 다이이찌 산쿄 가부시키가이샤 Therapeutic agent for glycogen storage disease type ia
EP3768694A4 (en) 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
MX2020010383A (en) 2018-04-05 2020-12-10 Hoffmann La Roche Use of fubp1 inhibitors for treating hepatitis b virus infection.
JP7275164B2 (en) 2018-04-11 2023-05-17 アイオーニス ファーマシューティカルズ， インコーポレーテッド Regulators of EZH2 expression
EP3790990A1 (en) 2018-05-07 2021-03-17 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
BR112020020957B1 (en) 2018-05-09 2022-05-10 Ionis Pharmaceuticals, Inc Oligomeric compounds, population and pharmaceutical composition thereof and their uses
KR20210008497A (en) 2018-05-09 2021-01-22 아이오니스 파마수티컬즈, 인코포레이티드 Compounds and methods for reducing ATXN3 expression
CN112912500A (en) 2018-06-05 2021-06-04 豪夫迈·罗氏有限公司 Oligonucleotides for modulating expression of ATXN2
JP2021526823A (en) 2018-06-14 2021-10-11 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Compounds and methods for increasing STMN2 expression
JP7315594B2 (en) 2018-06-27 2023-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド Compounds and methods for reducing LRRK2 expression
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
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
EP3818159A1 (en) 2018-07-03 2021-05-12 F. Hoffmann-La Roche AG Oligonucleotides for modulating tau expression
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
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
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
CR20210015A (en) 2018-07-13 2021-03-22 Hoffmann La Roche Oligonucleotides for modulating rtel1 expression
EP3826645A4 (en) 2018-07-25 2023-05-17 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
CA3098267A1 (en) * 2018-07-31 2020-02-06 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
BR112021001756A2 (en) 2018-07-31 2021-05-11 Roche Innovation Center Copenhagen A/S oligonucleotides, gapmer oligonucleotide, pharmaceutically acceptable salt, conjugate, pharmaceutical composition, uses of an oligonucleotide, method for the treatment or prophylaxis of a disease, process for making an oligonucleotide and invention
JP2021533762A (en) 2018-08-10 2021-12-09 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts Modified oligonucleotides that target SNPs
WO2020038377A1 (en) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
CN112585280A (en) 2018-08-23 2021-03-30 罗氏创新中心哥本哈根有限公司 Micro RNA-134 biomarkers
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
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 (en) 2018-11-14 2020-08-01 美商Ionis製藥公司 Modulators of foxp3 expression
BR112021008967A2 (en) 2018-11-15 2021-08-17 Ionis Pharmaceuticals, Inc. irf5 expression modulators
CN113631709A (en) 2018-12-20 2021-11-09 普拉克西斯精密药物股份有限公司 Compositions and methods for treating KCNT 1-related disorders
WO2020136125A2 (en) 2018-12-21 2020-07-02 Boehringer Ingelheim International Gmbh Antisense oligonucleotides targeting card9
CN113614232A (en) * 2019-01-18 2021-11-05 马萨诸塞大学 Dynamic pharmacokinetic modified anchor
JP2022518500A (en) 2019-01-25 2022-03-15 エフ．ホフマン－ラ ロシュ アーゲー Lipid vesicles for oral drug delivery
MX2021008918A (en) 2019-01-31 2021-08-24 Ionis Pharmaceuticals Inc Modulators of yap1 expression.
CA3130431A1 (en) 2019-02-20 2020-08-27 Roche Innovation Center Copenhagen A/S Phosphonoacetate gapmer oligonucleotides
KR20210132681A (en) 2019-02-20 2021-11-04 로슈 이노베이션 센터 코펜하겐 에이/에스 new phosphoramidite
CN113474633A (en) 2019-02-26 2021-10-01 罗氏创新中心哥本哈根有限公司 Process for preparing oligonucleotide
EP3931328A4 (en) 2019-02-27 2023-09-13 Ionis Pharmaceuticals, Inc. Modulators of malat1 expression
US20220184216A1 (en) * 2019-03-05 2022-06-16 Hoffmann-La Roche Inc. Intracellular targeting of molecules
AU2020253821A1 (en) 2019-03-29 2021-10-28 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating UBE3A-ATS
WO2020201144A1 (en) 2019-04-02 2020-10-08 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
BR112021019182A2 (en) 2019-04-03 2022-05-31 Bristol Myers Squibb Co Antisense angptl2 oligonucleotides and their uses
EP3947677A1 (en) 2019-04-04 2022-02-09 F. Hoffmann-La Roche AG Oligonucleotides for modulating atxn2 expression
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
US11466274B2 (en) 2019-05-31 2022-10-11 Aligos Therapeutics, Inc. Modified gapmer oligonucleotides and methods of use
JP7155302B2 (en) 2019-06-06 2022-10-18 エフ．ホフマン－ラ ロシュ アーゲー Antisense oligonucleotides targeting ATXN3
EP3956450A4 (en) 2019-07-26 2022-11-16 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
CA3144467A1 (en) * 2019-07-30 2021-02-04 Jonathan Miles Brown Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity
US20220378920A1 (en) 2019-09-10 2022-12-01 Daiichi Sankyo Company, Limited CONJUGATE OF GalNAc-OLIGONUCLEOTIDE FOR DELIVERY TO LIVER AND MANUFACTURING METHOD THEREOF
KR20220110729A (en) * 2019-10-02 2022-08-09 서나오믹스, 인크. Oligonucleotides Comprising Nucleoside Analogs
EP4077671A1 (en) 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of saraf inhibitors for treating hepatitis b virus infection
CN114901821A (en) 2019-12-19 2022-08-12 豪夫迈·罗氏有限公司 Use of SEPT9 inhibitors for treating hepatitis B virus infection
WO2021122921A1 (en) 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
WO2021127650A1 (en) * 2019-12-19 2021-06-24 Entrada Therapeutics, Inc. Compositions for delivery of antisense compounds
JP2023506540A (en) 2019-12-19 2023-02-16 エフ． ホフマン－ラ ロシュ エージー． Use of SCAMP3 inhibitors to treat hepatitis B virus infection
JP2023506550A (en) 2019-12-19 2023-02-16 エフ． ホフマン－ラ ロシュ エージー． Use of SBDS inhibitors to treat hepatitis B virus infection
EP4077672A1 (en) 2019-12-20 2022-10-26 F. Hoffmann-La Roche AG Enhanced oligonucleotides for inhibiting scn9a expression
AU2020415322A1 (en) 2019-12-24 2022-06-16 F. Hoffmann-La Roche Ag Pharmaceutical combination of antiviral agents targeting HBV and/or an immune modulator for treatment of HBV
TW202137987A (en) 2019-12-24 2021-10-16 瑞士商赫孚孟拉羅股份公司 Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
WO2021152005A1 (en) 2020-01-28 2021-08-05 Universite 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
US20220064638A1 (en) 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
EP4110916A1 (en) 2020-02-28 2023-01-04 F. Hoffmann-La Roche AG Oligonucleotides for modulating cd73 exon 7 splicing
JP2023537798A (en) 2020-03-19 2023-09-06 アビディティー バイオサイエンシーズ，インク． Compositions and methods for treating facioscapulohumeral muscular dystrophy
AU2021260934A1 (en) * 2020-04-21 2022-11-24 Flagship Pioneering, Inc. Bifunctional molecules and methods of using thereof
IL297435A (en) 2020-05-01 2022-12-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating atxn1
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
JP2023527693A (en) 2020-05-11 2023-06-30 ジェネンテック， インコーポレイテッド Complement Component C1R Inhibitors and Related Compositions, Systems, and Methods of Using The Same for Treating Neurological Disorders
WO2021229036A1 (en) 2020-05-13 2021-11-18 F. Hoffmann-La Roche Ag Oligonucleotide agonists targeting progranulin
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)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
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)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
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)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
CN115884777A (en) 2020-05-22 2023-03-31 豪夫迈·罗氏有限公司 Oligonucleotides for splicing modulation of CARD9
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
JP2023529457A (en) 2020-06-09 2023-07-10 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 Guanosine analogs for use in therapeutic polynucleotides
AR122731A1 (en) 2020-06-26 2022-10-05 Hoffmann La Roche IMPROVED OLIGONUCLEOTIDES TO MODULATE FUBP1 EXPRESSION
US11732263B2 (en) 2020-06-29 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating 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 (en) * 2020-07-30 2022-02-08 四川大学 Quantum dot-small nucleic acid conjugate and application thereof
EP4200419A2 (en) 2020-08-21 2023-06-28 F. Hoffmann-La Roche AG Use of a1cf inhibitors for treating hepatitis b virus infection
WO2022072950A1 (en) * 2020-10-02 2022-04-07 Sirnaomics, Inc. NUCLEOSIDE CONTAINING siRNAS FOR TREAT VIRAL DISEASES
AU2021381363A1 (en) 2020-11-18 2023-06-15 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
TW202237843A (en) 2020-12-03 2022-10-01 瑞士商赫孚孟拉羅股份公司 Antisense oligonucleotides targeting atxn3
AR124229A1 (en) 2020-12-03 2023-03-01 Hoffmann La Roche ANTISENSE OLIGONUCLOTIDES ACTING ON ATXN3
JP2023553710A (en) 2020-12-18 2023-12-25 エフ． ホフマン－ラ ロシュ アーゲー Antisense oligonucleotides for targeting progranulin
EP4267734A1 (en) 2020-12-22 2023-11-01 F. Hoffmann-La Roche AG Oligonucleotides targeting xbp1
CN114716518A (en) * 2021-01-06 2022-07-08 圣诺制药公司 Molecular structure capable of inhibiting expression of PCSK9 and pharmaceutical composition
US20240083990A1 (en) 2021-01-26 2024-03-14 Universite Brest Bretagne Occidentale Novel stim1 splicing variants and uses thereof
TW202246500A (en) 2021-02-02 2022-12-01 瑞士商赫孚孟拉羅股份公司 Enhanced oligonucleotides for inhibiting rtel1 expression
JP2024508956A (en) 2021-03-08 2024-02-28 レ ラボラトワール セルヴィエ Antisense oligonucleotides for inhibiting alpha-synuclein expression.
WO2022232650A1 (en) * 2021-04-30 2022-11-03 Ionis Pharmaceuticals, Inc. Methods for reducing agt expression
EP4341405A1 (en) 2021-05-20 2024-03-27 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
AR126085A1 (en) 2021-06-08 2023-09-13 Hoffmann La Roche OLIGONUCLEOTIDE PROGRANULIN AGONISTS
IL309217A (en) 2021-06-18 2024-02-01 Ionis Pharmaceuticals Inc Compounds and methods for reducing ifnar1 expression
EP4359539A1 (en) 2021-06-23 2024-05-01 University Of Massachusetts Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
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
WO2023111210A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Combination of oligonucleotides for modulating rtel1 and fubp1
WO2023111336A1 (en) 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Oligonucleotide gba agonists
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
WO2023156652A1 (en) 2022-02-21 2023-08-24 F. Hoffmann-La Roche Ag Antisense oligonucleotide
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 (en) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk Oligonucleotide derivative and its preparation
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 (en) 1987-11-12 1989-05-24 Max Planck Gesellschaft MODIFIED OLIGONUCLEOTIDS
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
ATE198598T1 (en) 1990-11-08 2001-01-15 Hybridon Inc CONNECTION OF MULTIPLE REPORTER GROUPS ON SYNTHETIC OLIGONUCLEOTIDES
DE4104186A1 (en) 1991-02-12 1992-08-13 Genentech Inc NEW COMPLEXES INCLUDING ENDOCYTOSIS IN HIGHER EUKARYOTIC CELLS, NUCLEIC ACID
DE4110409C2 (en) 1991-03-29 1999-05-27 Boehringer Ingelheim Int New protein-polycation conjugates
KR950014915B1 (en) 1991-06-19 1995-12-18 주식회사녹십자 Asialoglycoprotein-conjugated compounds
ATE239484T1 (en) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc DERIVATIZED OLIGONUCLEOTIDES WITH IMPROVED ABSORPTION CAPACITY
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
NL9201440A (en) 1992-08-11 1994-03-01 Univ Leiden Triantennary cluster glycosides, their preparation and application.
EP0673559A1 (en) 1992-12-14 1995-09-27 Honeywell Inc. Motor system with individually controlled redundant windings
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
AU679566B2 (en) 1993-09-03 1997-07-03 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
ZA951877B (en) 1994-03-07 1996-09-09 Dow Chemical Co Bioactive and/or targeted dendrimer conjugates
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
PT804456E (en) 1994-10-06 2003-01-31 Peter Eigil Nielsen CONJUGATES OF NUCLEIC ACIDS OF PEPTIDES
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
WO1997020563A1 (en) 1995-11-22 1997-06-12 The Johns-Hopkins University Ligands to enhance cellular uptake of biomolecules
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
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
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 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
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
AU744417B2 (en) * 1997-05-05 2002-02-21 Sanofi-Aventis Deutschland Gmbh Modified antisense nucleotides complementary to a section of the human Ha-Ras gene
ATE251913T1 (en) 1997-05-21 2003-11-15 Univ Leland Stanford Junior COMPOSITION AND METHOD FOR DELAYING TRANSPORT THROUGH BIOLOGICAL MEMBRANES
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
IL135000A0 (en) 1997-09-12 2001-05-20 Exiqon As Bi- and tri-cyclic nucleoside, nucleotide and 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
AU770963B2 (en) 1998-12-02 2004-03-11 I.D.M. Immuno-Designed Molecules New oligomeric conjugates liable to transfer biological molecules into cells
KR100782896B1 (en) 1999-05-04 2007-12-06 엑시콘 에이/에스 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
IL148916A0 (en) 1999-10-04 2002-09-12 Exiqon As 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
JP2004532022A (en) 2001-03-26 2004-10-21 サーナ・セラピューティクス・インコーポレイテッド Oligonucleotide-mediated inhibition of hepatitis B virus and hepatitis C virus replication
EP2415486B1 (en) 2001-05-18 2017-02-22 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7259150B2 (en) 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US20030191075A1 (en) 2002-02-22 2003-10-09 Cook Phillip Dan Method of using modified oligonucleotides for hepatic delivery
WO2003097662A1 (en) 2002-05-15 2003-11-27 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
WO2004044139A2 (en) 2002-11-05 2004-05-27 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
WO2004044141A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Conjugated oligomeric compounds and their 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
CA2505801A1 (en) 2002-11-13 2004-05-27 Rosanne Crooke Antisense modulation of apolipoprotein b expression
US7511131B2 (en) 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
DK2284269T3 (en) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense design
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
CN1984921B (en) * 2003-06-03 2010-06-16 Isis药物公司 Modulation of survivin expression
DE60319354T2 (en) 2003-07-11 2009-03-26 Lbr Medbiotech B.V. Mannose-6-phosphate receptor mediated gene transfer to muscle cells
CA2533701A1 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, 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
ES2382807T3 (en) 2003-08-28 2012-06-13 Takeshi Imanishi New artificial nucleic acids of the N-O link type with cross-linking
AU2004275876B2 (en) * 2003-09-25 2011-03-31 Coley Pharmaceutical Gmbh Nucleic acid-lipophilic conjugates
KR20070006709A (en) 2003-12-23 2007-01-11 산타리스 팔마 에이/에스 Oligomeric compounds for the modulation of bcl-2
JP2007524412A (en) 2004-01-22 2007-08-30 イムノメディクス， インコーポレイテッド Folic acid conjugates and complexes
EP1713912B1 (en) 2004-01-30 2013-09-18 Santaris Pharma A/S Modified short interfering rna (modified 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
AU2005330637B2 (en) 2004-08-04 2012-09-20 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
EP2982754A1 (en) * 2004-09-24 2016-02-10 Alnylam Pharmaceuticals, Inc. Rnai modulation of apob and uses thereof
EP1833840B9 (en) * 2004-11-09 2010-11-10 Santaris Pharma A/S Potent lna oligonucleotides for the inhibition of hif-1a
BRPI0517613A (en) * 2004-11-09 2008-10-14 Santaris Pharma As oligonucleotides lna and cancer treatment
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20080206869A1 (en) 2005-01-24 2008-08-28 Avaris Ab Nucleic Acid Complex
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
JP5523705B2 (en) 2005-08-29 2014-06-18 レグルス・セラピューティクス・インコーポレイテッド Method of using to modulate MIR-122A
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
CA2622583A1 (en) 2005-09-15 2007-03-22 Henrik Frydenlund Hansen Rna antagonist compounds for the inhibition of apo-b100 expression
WO2007035759A1 (en) 2005-09-19 2007-03-29 Johnson & Johnson Pharmaceutical Research & Development L.L.C. Modulation of glucocorticoid receptor expression
KR101789603B1 (en) 2005-11-10 2017-11-21 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
DK1984382T3 (en) 2006-01-27 2012-09-03 Santaris Pharma As LNA modified phosphorothiolated oligonucleotides
KR20130042043A (en) 2006-01-27 2013-04-25 아이시스 파마수티컬즈 인코포레이티드 6-modified bicyclic nucleic acid analogs
EP2089521A1 (en) 2006-02-08 2009-08-19 Ercole Biotech, Inc. Soluble tnf receptors and their use in treatment of disease
US8329888B2 (en) 2006-03-23 2012-12-11 Santaris Pharma A/S Small internally segmented interfering RNA
GB0606415D0 (en) 2006-03-31 2006-05-10 Univ Southampton Topical drug delivery
DK2666859T3 (en) 2006-04-03 2019-04-08 Roche Innovation Ct Copenhagen As PHARMACEUTICAL COMPOSITION CONTAINING ANTI-miRNA ANTISENSE OLIGONUCLEOTIDES
CN102851291B (en) * 2006-04-03 2016-03-09 斯特拉有限责任公司 Comprise the pharmaceutical composition of anti-miRNA antisense oligonucleotides
US20090326042A1 (en) * 2006-05-05 2009-12-31 Isis Pharmaceuticals, Inc Compounds and methods for modulating expression of crp
DK2066684T3 (en) 2006-05-11 2012-10-22 Isis Pharmaceuticals Inc 5'-Modified Bicyclic Nucleic Acid Analogs
AU2007296055A1 (en) 2006-09-15 2008-03-20 Enzon Pharmaceuticals, Inc. Polymeric conjugates containing positively-charged moieties
CN101534643A (en) 2006-09-15 2009-09-16 安佐制药股份有限公司 Hindered ester-based biodegradable linkers for oligonucleotide delivery
MX2009003398A (en) 2006-09-27 2009-08-12 Coley Pharm Gmbh Cpg oligonucleotide analogs containing hydrophobic t analogs with enhanced immunostimulatory activity.
WO2008040355A2 (en) 2006-10-06 2008-04-10 Exiqon A/S Novel methods for quantification of micrornas and small interfering rnas
CA2666191C (en) 2006-10-09 2017-07-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
EA200900741A1 (en) 2006-11-27 2010-04-30 Айзис Фармасьютикалз, Инк. METHODS OF TREATMENT OF HYPERHOLESTERYNEMIA
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis 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 (en) 2007-03-19 2008-09-25 Momentive Performance Materials Gmbh New polyamide-polysiloxane compounds
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
CA2681406A1 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
RU2015127794A (en) 2007-03-24 2018-12-21 Касл Терапьютикс, ЭлЭлСи Introduction of antisense oligonucleotides complementary to human apolipoprotein B
WO2008132234A2 (en) 2007-05-01 2008-11-06 Santaris Pharma A/S Rna antagonist compounds for the modulation of beta-catenin
CN101889086B (en) 2007-05-01 2014-07-02 桑塔里斯制药公司 Splice switching oligomers for TNF superfamily receptors and their use in treatment of disease
WO2008150729A2 (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
ES2694726T3 (en) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2171648A4 (en) 2007-07-19 2011-06-29 Datakey Electronics Inc Rf token and receptacle system and method
BRPI0721948A2 (en) 2007-08-20 2014-04-08 Enzon Pharmaceuticals Inc POLYMERIC LEADERS CONTAINING FRACTIONS PYRIDYL DISULFECTURE
EP2215102B1 (en) 2007-10-01 2016-02-17 Ionis Pharmaceuticals, Inc. Antisense modulation of fibroblast growth factor receptor 4 expression
ES2463665T3 (en) 2007-10-04 2014-05-28 Stella Aps Combination treatment for the treatment of hepatitis C virus infection
WO2009045536A2 (en) 2007-10-05 2009-04-09 The University Of North Carolina At Chapel Hill Receptor targeted oligonucleotides
NZ585327A (en) 2007-11-26 2012-10-26 Santaris Pharma As Lna antagonists targeting the androgen receptor
JP2011505124A (en) 2007-12-03 2011-02-24 エンゾン ファーマシューティカルズ，インコーポレーテッド RNA antagonist compounds for modulating PIK3CA expression
AU2008333811B2 (en) * 2007-12-04 2014-05-01 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
CA2717792A1 (en) 2008-03-07 2009-09-11 Santaris Pharma 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
EP2274425A2 (en) 2008-04-11 2011-01-19 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
MX2010013552A (en) 2008-07-03 2011-03-24 Santaris Pharma As Rna antagonist compounds for the inhibition of expression of mitochondrial glycerol-3-phosphate acyltransferase 1 (mtgpat1).
SI2320925T1 (en) 2008-07-10 2016-05-31 Regenesance B.V. 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
US20110237646A1 (en) 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
US20120022245A1 (en) 2008-10-17 2012-01-26 Purdue Research Foundation Folate targeting of nucleotides
JP5793423B2 (en) * 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 Use of LNAApoB antisense oligomers for the treatment of acute coronary syndromes
AT507215B1 (en) 2009-01-14 2010-03-15 Boehler Edelstahl Gmbh & Co Kg WEAR-RESISTANT MATERIAL
DK2414521T3 (en) 2009-03-31 2017-01-30 Massachusetts Gen Hospital Regulation of miR-33 microRNAs in the treatment of cholesterol-related diseases
JP5773535B2 (en) * 2009-04-24 2015-09-02 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 Pharmaceutical composition for the treatment of HCV patients unresponsive to interferon
NZ596608A (en) 2009-06-12 2014-01-31 Santaris Pharma As New potent anti apob antisense compounds
EP2456870A1 (en) 2009-07-21 2012-05-30 Santaris Pharma A/S Antisense oligomers targeting pcsk9
KR20120095397A (en) 2009-10-16 2012-08-28 글락소 그룹 리미티드 Hbv antisense inhibitors
KR101663617B1 (en) 2009-10-29 2016-10-07 엘지전자 주식회사 A method for transmitting and receiving downlink reference signals, and a base station and a user equipment thereof
US20130023578A1 (en) * 2009-12-31 2013-01-24 Samyang Biopharmaceuticals Corporation siRNA for inhibition of c-Met expression and anticancer composition containing the same
AU2011203986C1 (en) 2010-01-08 2015-03-05 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
WO2011088309A1 (en) 2010-01-14 2011-07-21 Regulus Therapeutics Inc. Microrna compositions and methods
NZ601737A (en) 2010-02-24 2013-06-28 Arrowhead Res Corp Compositions for targeted delivery of sirna
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
JP5819401B2 (en) 2010-04-19 2015-11-24 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． Methods of selectively delivering compositions and oligonucleotide molecules to specific neuron types
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
WO2011139917A1 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression
US8815826B2 (en) 2010-07-23 2014-08-26 Regulus Therapeutics, Inc. Targeting microRNAs for the treatment of fibrosis
US9127280B2 (en) 2010-08-31 2015-09-08 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
ES2858365T3 (en) 2010-12-13 2021-09-30 Ericsson Telefon Ab L M Exchange of parameters related to measurement periods
EP3192800A1 (en) 2010-12-17 2017-07-19 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
RU2619453C2 (en) 2010-12-29 2017-05-16 Эрроухэд Фармасьютикалз, Инк. Conjugates for delivery of polynucleotides in vivo, containing bonds sensitive to enzymatic degradation
CA2976966C (en) 2010-12-29 2021-11-09 F. Hoffmann-La Roche Ag Small molecule conjugates for intracellular delivery of nucleic acids
AU2012207606B2 (en) 2011-01-11 2017-02-23 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
CN111172162A (en) 2011-04-21 2020-05-19 葛兰素史克公司 Modulation of Hepatitis B Virus (HBV) expression
TW201303013A (en) 2011-04-21 2013-01-16 Isis Pharmaceuticals Inc Modulation of hepatitis B virus (HBV) expression
CN113249381A (en) 2011-04-25 2021-08-13 赛诺菲 microRNA compounds and methods for modulating MIR-21 activity
CN102226185A (en) * 2011-05-09 2011-10-26 华中科技大学同济医学院附属同济医院 RNA aptamer of targeting hepatocyte and nucleotide sequence thereof
WO2012174154A1 (en) 2011-06-13 2012-12-20 Isis Pharmaceuticals, Inc. Modulation of inflammatory responses by factor vii
CN103597074A (en) 2011-06-16 2014-02-19 Isis制药公司 Antisense modulation of fibroblast growth factor receptor 4 expression
SG194751A1 (en) 2011-06-30 2013-12-30 Arrowhead Res Corp Compositions and methods for inhibiting gene expression of hepatitis b virus
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CA2848753C (en) * 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
WO2013070771A1 (en) 2011-11-07 2013-05-16 Isis Pharmaceuticals, Inc. Administration of factor xi antisense oligonucleotides
AU2013216852A1 (en) 2012-02-08 2014-08-21 Isis Pharmaceuticals, Inc. Methods and compositions for modulating Factor VII expression
WO2013142514A1 (en) 2012-03-19 2013-09-26 Isis 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
US20150291958A1 (en) 2012-11-15 2015-10-15 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
RU2018108405A (en) 2013-01-30 2019-02-26 Ф. Хоффманн-Ля Рош Аг CARBOHYDRATE AND LNA-OLIGONUCLEOTIDE CONJUGATES
AU2014259759B2 (en) 2013-05-01 2020-06-18 Ionis Pharmaceuticals, Inc. Compositions and methods
JP2017501684A (en) 2013-11-14 2017-01-19 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 APOB antisense conjugate compound
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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