JP2015529469A5 - - Google Patents

Download PDF

Info

Publication number
JP2015529469A5
JP2015529469A5 JP2015532097A JP2015532097A JP2015529469A5 JP 2015529469 A5 JP2015529469 A5 JP 2015529469A5 JP 2015532097 A JP2015532097 A JP 2015532097A JP 2015532097 A JP2015532097 A JP 2015532097A JP 2015529469 A5 JP2015529469 A5 JP 2015529469A5
Authority
JP
Japan
Prior art keywords
compound
item
compound according
nucleotide
targeting oligonucleotide
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2015532097A
Other languages
English (en)
Japanese (ja)
Other versions
JP2015529469A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2013/059772 external-priority patent/WO2014043544A1/en
Publication of JP2015529469A publication Critical patent/JP2015529469A/ja
Publication of JP2015529469A5 publication Critical patent/JP2015529469A5/ja
Pending legal-status Critical Current

Links

JP2015532097A 2012-09-14 2013-09-13 多量体オリゴヌクレオチド化合物 Pending JP2015529469A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261701351P 2012-09-14 2012-09-14
US61/701,351 2012-09-14
US201361783272P 2013-03-14 2013-03-14
US61/783,272 2013-03-14
PCT/US2013/059772 WO2014043544A1 (en) 2012-09-14 2013-09-13 Multimeric oligonucleotide compounds

Publications (2)

Publication Number Publication Date
JP2015529469A JP2015529469A (ja) 2015-10-08
JP2015529469A5 true JP2015529469A5 (enExample) 2016-11-04

Family

ID=50278726

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015532097A Pending JP2015529469A (ja) 2012-09-14 2013-09-13 多量体オリゴヌクレオチド化合物

Country Status (8)

Country Link
US (4) US20150247141A1 (enExample)
EP (1) EP2895200B1 (enExample)
JP (1) JP2015529469A (enExample)
AU (3) AU2013315225B2 (enExample)
CA (1) CA2884608C (enExample)
DK (1) DK2895200T3 (enExample)
IL (1) IL237634A0 (enExample)
WO (1) WO2014043544A1 (enExample)

Families Citing this family (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
CN104540946A (zh) 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节utrn表达的组合物和方法
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
CN104583402A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节mecp2表达的组合物和方法
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Smn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
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
CA2921556A1 (en) * 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
US10358643B2 (en) 2014-01-30 2019-07-23 Hoffmann-La Roche, Inc. Poly oligomer compound with biocleavable conjugates
MA40463A (fr) 2014-08-07 2017-06-14 Regulus Therapeutics Inc Ciblage de micro-arn pour traiter des troubles métaboliques
EP3180033B1 (de) * 2014-08-14 2023-06-07 Friedrich-Schiller-Universität Jena Peptid zur verwendung in der reduktion von nebenwirkungen in form von immunstimulatorischen reaktionen/effekten
GB201421379D0 (en) * 2014-12-02 2015-01-14 Isis Innovation Ltd And Medical Res Council Molecule
WO2016130943A1 (en) * 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
RS60762B1 (sr) 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
IL316159A (en) * 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
WO2017007886A2 (en) * 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
EP3409779A4 (en) * 2016-01-26 2019-07-03 Nissan Chemical Corporation Single-stranded oligonucleotide
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
JP7188087B2 (ja) * 2017-02-06 2022-12-13 日産化学株式会社 一本鎖オリゴヌクレオチド
CN110248665A (zh) 2017-02-06 2019-09-17 Mpeg La有限责任公司 具有降低的肾清除的多聚体寡核苷酸
WO2018150196A1 (en) 2017-02-17 2018-08-23 Oxford University Innovation Limited Cell penetrating peptides modified by glycosylation
US20200038427A1 (en) * 2017-03-31 2020-02-06 Bonac Corporation Cyclic nucleic acid molecule having gene expression control function
WO2018212271A1 (ja) * 2017-05-18 2018-11-22 国立大学法人京都大学 脊髄小脳変性症36型の予防又は治療用組成物
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
US11555188B2 (en) 2017-07-26 2023-01-17 Nissan Chemical Corporation Single-stranded oligonucleotide
JP6960641B2 (ja) * 2017-11-09 2021-11-05 国立大学法人 東京大学 mRNAの安定化方法
WO2019199945A1 (en) * 2018-04-10 2019-10-17 Board Of Regents, The University Of Texas System Dna-barcoded antigen multimers and methods of use thereof
JP7162918B2 (ja) * 2018-05-31 2022-10-31 コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション マイクロrnaを抑制する変形核酸及びその用途
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US11168141B2 (en) * 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
US12331293B2 (en) 2018-11-02 2025-06-17 Biomarin Technologies B.V. Bispecific antisense oligonucleotides for dystrophin exon skipping
AU2019394177A1 (en) 2018-12-07 2021-06-10 Oxford University Innovation Limited Linkers
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
WO2020191361A2 (en) 2019-03-21 2020-09-24 Codiak Biosciences, Inc. Extracellular vesicles for vaccine delivery
KR20210141554A (ko) 2019-03-21 2021-11-23 코디악 바이오사이언시즈, 인크. 세포외 소포 접합체 및 이의 용도
WO2020215010A1 (en) 2019-04-17 2020-10-22 Codiak Biosciences, Inc. Compositions of exosomes and aav
US20220251200A1 (en) 2019-07-03 2022-08-11 Codiak Biosciences, Inc. Extracellular vesicles targeting t cells and uses thereof
US20230114023A1 (en) * 2019-07-30 2023-04-13 Mpeg La, L.L.C. Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
CN120771302A (zh) 2019-08-14 2025-10-14 隆萨销售股份公司 与分子连接的细胞外囊泡及其用途
EP4013878A1 (en) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extracellular vesicle-aso constructs targeting cebp/beta
CN114729356A (zh) 2019-08-14 2022-07-08 科迪亚克生物科学公司 靶向stat6的细胞外囊泡-aso构建体
CA3147365A1 (en) 2019-08-14 2021-02-18 Joanne LIM Extracellular vesicle-nlrp3 antagonist
CN114729327A (zh) 2019-08-14 2022-07-08 科迪亚克生物科学公司 具有stat3反义寡核苷酸的细胞外囊泡
EP4013872A1 (en) 2019-08-14 2022-06-22 Codiak BioSciences, Inc. Extracellular vesicles with antisense oligonucleotides targeting kras
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
US20240148891A1 (en) 2019-10-18 2024-05-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR102270700B1 (ko) * 2019-11-01 2021-06-30 한국과학기술연구원 폐 섬유증의 예방 또는 치료를 위한 올리고뉴클레오티드 중합체와 생체 적합성 양이온성 펩타이드로 이루어지는 폐 특이적 약물 전달체 및 이의 이용
JP7858532B2 (ja) 2020-01-10 2026-05-14 ダイン セラピューティクス,インコーポレーテッド 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用
WO2021222635A2 (en) * 2020-04-30 2021-11-04 Mpeg La, L.L.C. Multimeric oligonucleotides with divided strands
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2022066883A1 (en) 2020-09-23 2022-03-31 Codiak Biosciences, Inc. Extracellular vesicles comprising kras antigens and uses thereof
WO2022066898A2 (en) 2020-09-23 2022-03-31 Codiak Biosciences, Inc. Methods of producing extracellular vesicles
WO2022076596A1 (en) 2020-10-06 2022-04-14 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting stat6
MX2023004371A (es) 2020-10-14 2023-07-26 George Mason Res Foundation Inc Lípidos ionizables, y métodos de fabricación y uso de los mismos.
EP4237436A4 (en) * 2020-10-29 2025-09-10 Garcia Blanco Mariano A SOLUBLE INTERLEUKIN-7 RECEPTOR (SIL7R) MODULATION THERAPY FOR CANCERS
KR20230117182A (ko) 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
CA3211038A1 (en) 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
EP4294456A1 (en) 2021-02-17 2023-12-27 Lonza Sales AG Extracellular vesicle linked to a biologically active molecule via an optimized linker and an anchoring moiety
IL309334A (en) 2021-06-23 2024-02-01 Univ Massachusetts Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
EP4441224A4 (en) * 2021-12-03 2026-03-11 Quralis Corp Antisense oligonucleotides with modified skeletal chemicals
JP2025509552A (ja) * 2022-03-17 2025-04-11 アリゴス セラピューティクス,インコーポレーテッド 修飾ギャップマーオリゴマー及びその使用方法
WO2024005156A1 (ja) * 2022-06-29 2024-01-04 国立大学法人東京医科歯科大学 核酸医薬の毒性軽減剤
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体(tfr)介导的脑和肌肉递送的组合物和方法
EP4594520A2 (en) 2022-09-30 2025-08-06 Sixfold Bioscience Ltd. Compositions containing oligonucleotides with theranostic applications
AU2023371614A1 (en) 2022-11-04 2025-05-15 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
EP4654982A1 (en) 2023-01-27 2025-12-03 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof
WO2026102034A1 (en) * 2024-11-06 2026-05-15 Junipero Therapeutics, Inc. Compositions and methods for genetic and epigenetic modulation

Family Cites Families (117)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0092574B1 (en) 1981-10-23 1992-04-29 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and methods of making same
DE3329892A1 (de) 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg Verfahren zur herstellung von oligonucleotiden
JP3019994B2 (ja) 1987-11-30 2000-03-15 ユニバーシティ オブ アイオワ リサーチ ファウンデーション 新規なオリゴデオキシヌクレオチド、それを使用した標的遺伝子の発現をブロックする方法、及び新規なオリゴデオキシヌクレオチド並びにそれを使用した標的遺伝子の発現を阻止する方法
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5623065A (en) 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US6395492B1 (en) 1990-01-11 2002-05-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
WO1994008003A1 (en) 1991-06-14 1994-04-14 Isis Pharmaceuticals, Inc. ANTISENSE OLIGONUCLEOTIDE INHIBITION OF THE ras GENE
US5965722A (en) 1991-05-21 1999-10-12 Isis Pharmaceuticals, Inc. Antisense inhibition of ras gene with chimeric and alternating oligonucleotides
US5661134A (en) 1991-10-15 1997-08-26 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating Ha-ras or Ki-ras having phosphorothioate linkages of high chiral purity
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
US20070032446A1 (en) 1991-12-24 2007-02-08 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
TW244371B (enExample) 1992-07-23 1995-04-01 Tri Clover Inc
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
JPH08502723A (ja) 1992-07-27 1996-03-26 ハイブライドン インコーポレイテッド オリゴヌクレオチド・アルキルホスホノチオエート
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
DK0677056T3 (da) 1993-01-25 1996-08-05 Hybridon Inc Oligonukleotid-alkylphosphonater og -alkylphosphonothioater
US5734039A (en) * 1994-09-15 1998-03-31 Thomas Jefferson University Antisense oligonucleotides targeting cooperating oncogenes
US6608035B1 (en) 1994-10-25 2003-08-19 Hybridon, Inc. Method of down-regulating gene expression
DK0882061T3 (da) 1996-02-14 2004-09-27 Isis Pharmaceuticals Inc Sukkermodificerede gapped oligonukleotider
US6015710A (en) 1996-04-09 2000-01-18 The University Of Texas System Modulation of mammalian telomerase by peptide nucleic acids
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US5912332A (en) * 1996-07-26 1999-06-15 Hybridon, Inc. Affinity-based purification of oligonucleotides using soluble multimeric oligonucleotides
US6383808B1 (en) 2000-09-11 2002-05-07 Isis Pharmaceuticals, Inc. Antisense inhibition of clusterin expression
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
CA2326823A1 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
EP0979869A1 (en) 1998-08-07 2000-02-16 Hoechst Marion Roussel Deutschland GmbH Short oligonucleotides for the inhibition of VEGF expression
US6228642B1 (en) 1998-10-05 2001-05-08 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of tumor necrosis factor-(α) (TNF-α) expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6465628B1 (en) 1999-02-04 2002-10-15 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
KR20000065690A (ko) 1999-04-08 2000-11-15 박종구 반응 특이성 및 안정성을 개선시킨 안티센스 올리고 뉴클레오타이드, 안티센스 dna 및 그 제조방법
US6677445B1 (en) 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
AU7406700A (en) 1999-10-04 2001-05-10 Exiqon A/S Design of high affinity rnase h recruiting oligonucleotide
WO2001048190A2 (en) 1999-12-23 2001-07-05 Exiqon A/S Therapeutic uses of lna-modified oligonucleotides
US6602857B1 (en) 2000-01-18 2003-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
CA2419563C (en) 2000-09-06 2018-03-20 Mcgill University Chimeric antisense of arabinofuranose analogue and deoxyribose nucleotides
US20050054836A1 (en) 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
JP2005508634A (ja) * 2001-10-29 2005-04-07 マクギル・ユニヴァーシティ 非環式リンカー含有オリゴヌクレオチド及びその用途
CA2475388A1 (en) * 2002-02-14 2003-08-21 William J. Rutter Chimeric molecules for cleavage in a treated host
US20080207542A1 (en) 2002-03-26 2008-08-28 Sirna Therapeutics, Inc. RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
ES2649817T3 (es) 2002-04-05 2018-01-15 Roche Innovation Center Copenhagen A/S Compuestos oligom¿¿ricos para la modulaci¿®n de la expresi¿®n de HIF-1¿Á
US20030228690A1 (en) 2002-06-10 2003-12-11 Isis Pharmaceuticals Inc. Antisense modulation of IL-1 receptor-associated kinase-1 expression
US7507808B2 (en) 2002-12-12 2009-03-24 Isis Pharmaceuticals, Inc. Modulation of endothelial lipase expression
US20050130924A1 (en) 2002-06-26 2005-06-16 Monia Brett P. Antisense inhibition via RNAse H-independent reduction in mRNA
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2284269B1 (en) 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
WO2004069992A2 (en) 2003-02-10 2004-08-19 Santaris Pharma A/S Oligomeric compounds for the modulation of ras expression
FR2853663B1 (fr) 2003-04-14 2007-08-31 Aventis Pharma Sa Procede d'obtention de lignees de mastocytes a partir de tissus de porcs et procede de production de molecules de type heparine
US20080124317A1 (en) 2003-06-25 2008-05-29 Orna Mor Diagnos and Treatment of Fibrosis Related Pathology
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
NZ546275A (en) 2003-10-30 2009-05-31 Coley Pharm Gmbh C-class oligonucleotides analogs with enhanced immunostimulatory potency
US7431915B2 (en) * 2003-10-31 2008-10-07 The Regents Of The University Of California Peptides whose uptake by cells is controllable
ES2344566T3 (es) * 2003-12-23 2010-08-31 Santaris Pharma A/S Compuestos oligomericos para la modulacion de bcl-2.
WO2005072527A2 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
WO2005072057A2 (en) 2004-01-30 2005-08-11 Quark Biotech, Inc. Oligoribonucleotides and methods of use thereof for treatment of fibrotic conditions and other diseases
US8314226B2 (en) 2004-03-29 2012-11-20 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
WO2006086667A2 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
DE102005036654A1 (de) 2005-08-04 2007-02-15 Bayer Materialscience Ag Selbstvernetzende PUR-Dispersionen mit Uretdionstruktur
EP1951263A4 (en) 2005-11-21 2009-11-18 Johnson & Johnson Res Pty Ltd MULTITARGETING DISTURBING RNAS WITH TWO ACTIVE STRANDS AND DESIGN AND APPLICATION METHODS
US20090011004A1 (en) 2005-12-30 2009-01-08 Philadelphia Health & Education Corp., D/B/A/ Drexel University Of College Of Medicine Improved carriers for delivery of nucleic acid agents to cells and tissues
EP2388328A1 (en) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
US8362229B2 (en) 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
CA3042781C (en) 2006-04-03 2021-10-19 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EP2505646A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
WO2008029619A1 (fr) 2006-09-07 2008-03-13 Daiichi Sankyo Company, Limited Oligonucléotide antisens ena ayant une action spécifique de la séquence
EP2410053B2 (en) 2006-10-18 2020-07-15 Ionis Pharmaceuticals, Inc. Antisense compounds
WO2008109105A2 (en) 2007-03-06 2008-09-12 Flagship Ventures Methods and compositions for improved therapeutic effects with sirna
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
JP2010536787A (ja) 2007-08-15 2010-12-02 イデラ ファーマシューティカルズ インコーポレイテッド Toll様受容体モジュレータ
DK2205737T3 (da) 2007-10-04 2013-05-21 Santaris Pharma As Mikromirer
AU2008308499A1 (en) 2007-10-04 2009-04-09 Board Of Regents, The University Of Texas System Modulating gene expression with agRNA and gapmers targeting antisense transcripts
WO2009090182A1 (en) 2008-01-14 2009-07-23 Santaris Pharma A/S C4'-substituted - dna nucleotide gapmer oligonucleotides
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
AU2009235941A1 (en) 2008-04-07 2009-10-15 Riken RNA molecules and uses thereof
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
DK2310505T3 (en) 2008-06-30 2017-10-16 Roche Innovation Ct Copenhagen As ANTIDOT oligomers
MY188457A (en) 2008-10-03 2021-12-10 Opko Curna Llc Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
CN104382853A (zh) 2008-10-16 2015-03-04 玛瑞纳生物技术有限公司 基因沉默治疗剂的脂质体有效递送方法和组合物
EP2405921A4 (en) 2009-01-26 2013-05-22 Protiva Biotherapeutics Inc COMPOSITIONS AND METHODS FOR VAPORING APOLIPOPROTIEN C-III EXPRESSION
WO2010090452A2 (ko) 2009-02-04 2010-08-12 성균관대학교산학협력단 세포 내 전달능이 증가된 작은 간섭 rna 복합체
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
WO2010120969A1 (en) * 2009-04-15 2010-10-21 Board Of Regents, The University Of Texas System Targeting of the mir-30 family and let-7 family as a treatment for heart disease
EP2456470A1 (en) 2009-07-22 2012-05-30 Cenix Bioscience GmbH Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes
HUE026020T2 (en) * 2009-08-27 2016-05-30 Idera Pharmaceuticals Inc Preparation for inhibiting gene expression and its uses
US9364495B2 (en) 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
AU2010335039B2 (en) 2009-12-24 2015-03-26 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Molecule for treating an inflammatory disorder
WO2011117353A1 (en) * 2010-03-24 2011-09-29 Mirrx Therapeutics A/S Bivalent antisense oligonucleotides
WO2011130371A1 (en) 2010-04-13 2011-10-20 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
WO2011133886A2 (en) * 2010-04-23 2011-10-27 Genentech, Inc. Production of heteromultimeric proteins
ES2625689T3 (es) 2010-04-29 2017-07-20 Ionis Pharmaceuticals, Inc. Modulación de la expresión de transtiretina
WO2011159836A2 (en) 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
GB201010557D0 (en) 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
WO2012068340A2 (en) 2010-11-18 2012-05-24 Opko Curna Llc Antagonat compositions and methods of use
US8557787B2 (en) 2011-05-13 2013-10-15 The Board Of Trustees Of The Leland Stanford Junior University Diagnostic, prognostic and therapeutic uses of long non-coding RNAs for cancer and regenerative medicine
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
EP3254700B1 (en) 2012-03-04 2019-10-23 Bonac Corporation Micro-rna inhibitor
EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
CN104583398A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节基因表达的组合物和方法
EA201492118A1 (ru) 2012-05-16 2015-04-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии pten
CN104582737A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节apoa1和abca1表达的组合物和方法
CN104583402A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节mecp2表达的组合物和方法
US9567581B2 (en) * 2012-08-07 2017-02-14 The General Hospital Corporation Selective reactivation of genes on the inactive X chromosome
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
AU2015346042A1 (en) 2014-11-14 2017-04-27 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins

Similar Documents

Publication Publication Date Title
US11697811B2 (en) Oligomers
CN105838714B (zh) 反义分子和治疗疾病的方法
JP2021533804A5 (enExample)
JP2014527401A5 (enExample)
JP2015519057A5 (enExample)
JP2015523854A5 (enExample)
JP2016522674A5 (enExample)
JP2008509670A5 (enExample)
KR101026502B1 (ko) 마이크로rna 안티센스 pna, 그를 포함하는 조성물, 및 그의 사용 및 평가 방법
JP2015518712A5 (enExample)
JP2015523855A5 (enExample)
JP2015523853A5 (enExample)
JP2015518711A5 (enExample)
KR102804353B1 (ko) Gys2 발현을 억제하기 위한 조성물 및 방법
JP2023506546A (ja) B型肝炎ウイルス感染症を処置するためのsept9阻害剤の使用
EP3532617A1 (en) Antisense oligonucleotides
JPWO2017068791A1 (ja) 少なくとも1つのバルジ構造を有する核酸複合体
CN112534055A (zh) 用于调节rtel1表达的寡核苷酸
CN114829599A (zh) Scamp3抑制剂用于治疗乙型肝炎病毒感染的用途
CN114867856A (zh) Saraf抑制剂用于治疗乙型肝炎病毒感染的用途
EP3898975A2 (en) Antisense oligonucleotides targeting card9
WO2021231211A1 (en) Complement component c1s inhibitors for treating a neurological disease, and related compositions, systems and methods of using same
CN114829601A (zh) Sbds抑制剂用于治疗乙型肝炎病毒感染的用途
JP2023506547A (ja) 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
US11306310B2 (en) MicroRNA inhibitor