JP2016528897A5 - - Google Patents

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Publication number
JP2016528897A5
JP2016528897A5 JP2016534875A JP2016534875A JP2016528897A5 JP 2016528897 A5 JP2016528897 A5 JP 2016528897A5 JP 2016534875 A JP2016534875 A JP 2016534875A JP 2016534875 A JP2016534875 A JP 2016534875A JP 2016528897 A5 JP2016528897 A5 JP 2016528897A5
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JP
Japan
Prior art keywords
oligonucleotide
item
nucleotides
complementary
composition
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Pending
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JP2016534875A
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English (en)
Japanese (ja)
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JP2016528897A (ja
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Priority claimed from PCT/US2014/051331 external-priority patent/WO2015023975A1/en
Publication of JP2016528897A publication Critical patent/JP2016528897A/ja
Publication of JP2016528897A5 publication Critical patent/JP2016528897A5/ja
Pending legal-status Critical Current

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JP2016534875A 2013-08-16 2014-08-15 Ｒｎａを調節するための組成物および方法 Pending JP2016528897A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361866989P 2013-08-16 2013-08-16
US61/866,989 2013-08-16
US201361898461P 2013-10-31 2013-10-31
US61/898,461 2013-10-31
US201462010417P 2014-06-10 2014-06-10
US62/010,417 2014-06-10
PCT/US2014/051331 WO2015023975A1 (en) 2013-08-16 2014-08-15 Compositions and methods for modulating rna

Publications (2)

Publication Number Publication Date
JP2016528897A JP2016528897A (ja) 2016-09-23
JP2016528897A5 true JP2016528897A5 (enExample) 2017-09-28

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JP2016534875A Pending JP2016528897A (ja) 2013-08-16 2014-08-15 Ｒｎａを調節するための組成物および方法

Country Status (13)

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US (8) US20150050738A1 (enExample)
EP (1) EP3033424A4 (enExample)
JP (1) JP2016528897A (enExample)
KR (1) KR20160036065A (enExample)
CN (1) CN105658797A (enExample)
AU (2) AU2014306416B2 (enExample)
BR (1) BR112016003127A2 (enExample)
CA (1) CA2921556A1 (enExample)
EA (1) EA201690403A1 (enExample)
IL (1) IL244081A0 (enExample)
MX (1) MX2016002044A (enExample)
SG (1) SG11201600987TA (enExample)
WO (1) WO2015023975A1 (enExample)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
EP4074693A1 (en) 2011-06-08 2022-10-19 Translate Bio, Inc. Cleavable lipids
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
EP4279610A3 (en) 2013-03-15 2024-01-03 ModernaTX, Inc. Ribonucleic acid purification
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
WO2015023939A1 (en) * 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
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
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
AU2015289656A1 (en) 2014-07-16 2017-02-16 Modernatx, Inc. Circular polynucleotides
WO2016011226A1 (en) * 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
AU2015346042A1 (en) 2014-11-14 2017-04-27 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins
WO2016130963A1 (en) * 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Compositions and methods for modulating rna
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
EP4101930A1 (en) 2015-09-17 2022-12-14 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
JP2018530587A (ja) 2015-10-16 2018-10-18 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． ｍＲＮＡキャップ類似体およびｍＲＮＡキャッピングの方法
DK3362461T3 (da) 2015-10-16 2022-05-09 Modernatx Inc Mrna-cap-analoger med modificeret phosphatbinding
WO2017066782A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Hydrophobic mrna cap analogs
WO2017066789A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Mrna cap analogs with modified sugar
WO2017066791A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Sugar substituted mrna cap analogs
EP3368089B1 (en) * 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
AU2016359629B2 (en) 2015-11-23 2023-03-09 Ranjan BATRA Tracking and manipulating cellular RNA via nuclear delivery of CRISPR/Cas9
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
US10689689B2 (en) * 2015-12-28 2020-06-23 Roche Molecular Systems, Inc. Generic method for the stabilization of specific RNA
AU2017248189B2 (en) 2016-04-08 2021-04-29 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
WO2017188898A1 (en) 2016-04-29 2017-11-02 Nanyang Technological University G-quadruplex-containing antisense oligonucleotides
CN107841510B (zh) * 2016-09-20 2021-02-09 中国科学院青岛生物能源与过程研究所 一种原核细胞转录后水平控制不同基因表达比例的方法
KR102519171B1 (ko) * 2016-10-07 2023-04-07 세카나 파머씨티컬스 지엠비에이치 엔 씨오. 케이지 암 치료를 위한 새로운 접근법
WO2018089688A1 (en) 2016-11-09 2018-05-17 Jinjun Shi Restoration of tumor suppression using mrna-based delivery system
AU2017359342B2 (en) * 2016-11-09 2022-02-17 Intrexon Corporation Frataxin expression constructs
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
CN106813960A (zh) * 2016-12-26 2017-06-09 广州和实生物技术有限公司 一种血液游离rna保护剂及其制备方法与应用
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
KR20260009385A (ko) 2017-08-25 2026-01-19 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
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
MX2020006150A (es) 2017-12-15 2020-11-11 Flagship Pioneering Innovations Vi Llc Composiciones que comprenden polirribonucleotidos circulares y usos de las mismas.
WO2019183440A1 (en) * 2018-03-22 2019-09-26 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
CN108531496B (zh) * 2018-04-04 2020-11-06 江南大学 一种提高外源基因mRNA数量的DNA及其应用
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
US20220298516A1 (en) * 2018-09-20 2022-09-22 Modernatx, Inc. Compositions and methods for delivery of nucleic acids
CN111041001B (zh) * 2018-10-15 2023-02-28 上海行深生物科技有限公司 治疗kras突变型肿瘤的安全型柯萨奇病毒及其药物组合物
CN113227375A (zh) 2018-12-20 2021-08-06 原住民股份有限公司 合成的微小rna模拟物
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
JP7618577B2 (ja) 2019-03-25 2025-01-21 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 修飾環状ポリリボヌクレオチドを含む組成物及びその使用
US12637682B2 (en) * 2019-04-16 2026-05-26 The Regents Of The University Of California Protein translational control
CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
CN112111524B (zh) * 2020-01-10 2024-02-27 深圳瑞吉生物科技有限公司 mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
CN111744019B (zh) 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
AU2023310896A1 (en) * 2022-07-19 2025-01-30 Shenzhen Shenxin Biotechnology Co., Ltd. Mrna for sars-cov-2 s protein and use thereof

Family Cites Families (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US13402A (en) * 1855-08-07 Forming screw-threads
US21020A (en) * 1858-07-27 Improved combination of the needle and sun-dial to ascertain time
US546A (en) * 1838-01-06 Loom for weaving knotted counterpanes and other fabrics in which the
US1099771A (en) * 1913-05-26 1914-06-09 Guy P Slater Derrick.
US6111094A (en) * 1990-08-14 2000-08-29 Isis Pharmaceuticals Inc. Enhanced antisense modulation of ICAM-1
US20040033977A1 (en) * 1990-08-14 2004-02-19 Bennett C. Frank Oligonucleotide modulation of cell adhesion
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
WO1994001550A1 (en) * 1992-07-02 1994-01-20 Hybridon, Inc. Self-stabilized oligonucleotides as therapeutic agents
CA2167804A1 (en) * 1993-07-20 1995-02-02 Robert H. Singer In vivo nucleic acid hybridization method
US5962332A (en) * 1994-03-17 1999-10-05 University Of Massachusetts Detection of trinucleotide repeats by in situ hybridization
US5866331A (en) * 1995-10-20 1999-02-02 University Of Massachusetts Single molecule detection by in situ hybridization
US5962675A (en) * 1996-02-13 1999-10-05 Ribozyme Pharmaceuticals, Inc. Chemical syntheses of 2'-O-methoxy purine nucleosides
US20080119427A1 (en) * 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US6261836B1 (en) * 1996-10-01 2001-07-17 Geron Corporation Telomerase
CA2287571A1 (en) * 1997-04-23 1998-10-29 Abbott Laboratories Reagents and methods useful for detecting diseases of the prostate
WO1999001139A1 (en) * 1997-07-03 1999-01-14 Thomas Jefferson University An improved method for design and selection of efficacious antisense oligonucleotides
WO1999010509A1 (en) * 1997-08-22 1999-03-04 The Government Of The United States Of America, Represented By The Secretary Of Health And Human Services, National Institutes Of Health Polynucleotide inhibition of rna destabilization and sequestration
US6322978B1 (en) * 1998-04-20 2001-11-27 Joslin Diabetes Center, Inc. Repeat polymorphism in the frataxin gene and uses therefore
EP1190047A1 (en) * 1999-06-23 2002-03-27 Angiogene, Inc. Antisense oligonucleotide modulating cyclin e gene expression and therapeutic uses thereof
WO2001042792A2 (en) * 1999-12-08 2001-06-14 Millennium Pharmaceuticals, Inc. Diagnosis of cervical cancer using marker proteins
EP1130121A3 (en) * 2000-01-26 2003-04-16 Nisshinbo Industries Inc. Immobilized nucleic acid and method for detecting nucleic acid
US6503756B1 (en) * 2000-09-22 2003-01-07 Isis Pharmaceuticals, Inc. Antisense modulation of syntaxin 4 interacting protein expression
US20030125273A1 (en) * 2001-12-05 2003-07-03 Isis Pharmaceuticals Inc, Antisense modulation of MHC class II transactivator expression
GB0101397D0 (en) * 2001-01-19 2001-03-07 Amersham Pharm Biotech Uk Ltd Suppression of non-specific nucleic acid amplication
US20030125241A1 (en) * 2001-05-18 2003-07-03 Margit Wissenbach Therapeutic uses of LNA-modified oligonucleotides in infectious diseases
US20050048529A1 (en) * 2002-02-20 2005-03-03 Sirna Therapeutics, Inc. RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA)
US7399590B2 (en) * 2002-02-21 2008-07-15 Asm Scientific, Inc. Recombinase polymerase amplification
US20040023906A1 (en) * 2002-08-01 2004-02-05 Isis Pharmaceuticals Inc. Antisense modulation of phosphotyrosyl phosphatase activator expression
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
US20040097441A1 (en) * 2002-11-16 2004-05-20 Isis Pharmaceuticals Inc. Modulation of NIMA-related kinase 6 expression
US20040005565A1 (en) * 2002-07-02 2004-01-08 Isis Pharmaceuticals Inc. Antisense modulation of livin expression
US8604183B2 (en) * 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
EP2284269B1 (en) * 2002-11-18 2017-08-09 Roche Innovation Center Copenhagen A/S Antisense design
US20040110153A1 (en) * 2002-12-10 2004-06-10 Affymetrix, Inc. Compleixity management of genomic DNA by semi-specific amplification
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
US20050014168A1 (en) * 2003-06-03 2005-01-20 Arcturus Bioscience, Inc. 3' biased microarrays
EP2182063A3 (en) * 2003-09-18 2012-07-18 Isis Pharmaceuticals, Inc. Modulation of EIF4E expression
US20050108783A1 (en) * 2003-09-23 2005-05-19 Chihiro Koike Porcine invariant chain protein, full length cDNA, genomic organization, and regulatory region
US20050261217A1 (en) * 2004-05-18 2005-11-24 Isis Pharmaceuticals Inc. Modulation of pumilio 1 expression
US20050261216A1 (en) * 2004-05-18 2005-11-24 Isis Pharmaceuticals Inc. Modulation of Nanos 1 expression
US20050287539A1 (en) * 2004-06-29 2005-12-29 Emmanuel Labourier Methods and compositions for preparing capped RNA
DE102004034987A1 (de) * 2004-07-16 2006-02-02 Carl Zeiss Jena Gmbh Lichtrastermikroskop und Verwendung
US7718625B2 (en) * 2005-01-27 2010-05-18 University Of South Florida Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof
EP1854881B1 (en) * 2005-02-02 2010-06-02 Eisai R&D Management Co., Ltd. METHODS FOR IDENTIFYING PURKINJE CELLS USING THE Corl2 GENE AS A TARGET
US8999943B2 (en) * 2005-03-14 2015-04-07 Board Of Regents, The University Of Texas System Antigene oligomers inhibit transcription
CA2629664A1 (en) * 2005-11-17 2007-08-02 Board Of Regents, The University Of Texas System Modulation of gene expression by oligomers targeted to chromosomal dna
CA2630602A1 (en) * 2005-11-21 2007-05-31 Isis Pharmaceuticals, Inc. Modulation of eif4e-bp2 expression
US8685899B2 (en) * 2007-02-14 2014-04-01 Genisphere Inc. Methods, reagents and kits for detection of nucleic acid molecules
NZ580712A (en) * 2007-05-22 2011-12-22 Marina Biotech Inc Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers
US20090082297A1 (en) * 2007-06-25 2009-03-26 Lioy Daniel T Compositions and Methods for Regulating Gene Expression
EP2173913A4 (en) * 2007-08-03 2010-09-01 Biocept Inc IN-SITU HYBRIDIZATION FOR THE DETECTION OF RNA AND DNA MARKERS
US20110009466A1 (en) * 2007-08-29 2011-01-13 President And Fellows Of Harvard College Methods of increasing gene expression through rna protection
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
NZ585327A (en) * 2007-11-26 2012-10-26 Santaris Pharma As Lna antagonists targeting the androgen receptor
EP2235033A4 (en) * 2007-12-28 2011-11-02 Univ California METHOD AND COMPOSITIONS FOR INCREASING GENE EXPRESSION
WO2009093384A1 (ja) * 2008-01-24 2009-07-30 National Institute Of Advanced Industrial Science And Technology ポリヌクレオチド及びポリヌクレオチド類似体並びにこれらを用いた遺伝子発現制御方法
AU2009235941A1 (en) * 2008-04-07 2009-10-15 Riken RNA molecules and uses thereof
WO2010019270A1 (en) * 2008-08-14 2010-02-18 Isis Pharmaceuticals, Inc. Modulation of prion expression
WO2010124231A2 (en) * 2009-04-24 2010-10-28 The Board Of Regents Of The University Of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions
HUE040445T2 (hu) * 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
WO2011130371A1 (en) * 2010-04-13 2011-10-20 Life Technologies Corporation Compositions and methods for inhibition of nucleic acids function
US20110306653A1 (en) * 2010-05-14 2011-12-15 Tagcyx Biotechnologies Stabilization method of functional nucleic acid
GB201010557D0 (en) * 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
WO2012122645A1 (en) * 2011-03-11 2012-09-20 Sarissa Inc. Methods of treating cancer by inhibition of dna repair proteins
US9476043B2 (en) * 2011-04-08 2016-10-25 Rula Zain-Luqman Diagnosis and treatment of friedreich's ataxia
WO2012170771A1 (en) * 2011-06-09 2012-12-13 Curna, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
HK1200484A1 (zh) * 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
US20150247141A1 (en) * 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US20160032273A1 (en) * 2013-03-15 2016-02-04 Moderna Therapeutics, Inc. Characterization of mrna molecules
CN105370259A (zh) * 2014-08-29 2016-03-02 中国石油化工股份有限公司 水平井分段压裂方法
CN107987053B (zh) * 2017-12-08 2023-04-14 植恩生物技术股份有限公司 一种高纯度z型盐酸氟哌噻吨的制备方法
CN108590349A (zh) * 2018-06-11 2018-09-28 太仓市金毅电子有限公司 具有防撬功能的智能锁

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