JP2016522674A - 遺伝子発現を調節するための組成物及び方法 - Google Patents

遺伝子発現を調節するための組成物及び方法 Download PDF

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JP2016522674A
JP2016522674A JP2015512857A JP2015512857A JP2016522674A JP 2016522674 A JP2016522674 A JP 2016522674A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2016522674 A JP2016522674 A JP 2016522674A
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oligonucleotide
nucleotide
stranded oligonucleotide
nucleotides
sequence
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JP2016522674A5 (enExample
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アーサー エム. クリーグ,
アーサー エム. クリーグ,
ロメシュ スブラマニアン,
ロメシュ スブラマニアン,
ジェイムズ マクスウィゲン,
ジェイムズ マクスウィゲン,
ジェニー ティー. リー,
ジェニー ティー. リー,
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ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
ザ ジェネラル ホスピタル コーポレイション
ザ ジェネラル ホスピタル コーポレイション
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    • 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
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    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3517—Marker; Tag

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2015512857A 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法 Pending JP2016522674A (ja)

Applications Claiming Priority (3)

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US201261648077P 2012-05-16 2012-05-16
US61/648,077 2012-05-16
PCT/US2013/041437 WO2013173637A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating gene expression

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JP2016522674A true JP2016522674A (ja) 2016-08-04
JP2016522674A5 JP2016522674A5 (enExample) 2016-09-15

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US (1) US20150141320A1 (enExample)
EP (1) EP2850184A4 (enExample)
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WO (1) WO2013173637A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021514182A (ja) * 2018-02-09 2021-06-10 ジェネンテック, インコーポレイテッド Tmem106b発現を調節するためのオリゴヌクレオチド
JP2023503202A (ja) * 2020-02-12 2023-01-26 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP2023523622A (ja) * 2020-04-29 2023-06-06 サリオジェン セラピューティクス インコーポレイテッド 遺伝性黄斑変性症の治療のための組成物及び方法

Families Citing this family (93)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
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
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
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
CA2913596C (en) * 2013-06-26 2021-05-04 Elanco Us Inc. Markers to predict macrocyclic lactone resistance in dirofilaria immitis, the causative agent of heartworm disease
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
AU2014317961B2 (en) 2013-09-05 2020-07-30 Murdoch University Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3052632A4 (en) 2013-10-04 2017-03-29 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
SI3119888T1 (sl) 2014-03-19 2021-11-30 Ionis Pharmaceuticals, Inc. Sestavki za moduliranje izražanja ataksina 2
HUE050704T2 (hu) 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
WO2016061487A1 (en) * 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10793855B2 (en) * 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
MX391217B (es) 2015-02-26 2025-03-21 Ionis Pharmaceuticals Inc Moduladores de rodopsina p23h con especificidad de alelo.
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10801024B2 (en) * 2015-05-20 2020-10-13 Indiana University Research And Technology Corporation Inhibition of lncRNA HOTAIR and related materials and methods
EP3124619B1 (en) * 2015-07-31 2019-03-06 Menicon Co., Ltd Reagents, method and kit for across and within dog breed glaucoma diagnosis
CA3005247A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
WO2017117496A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
KR20180103166A (ko) 2016-01-29 2018-09-18 교와 핫꼬 기린 가부시키가이샤 핵산 복합체
WO2017134252A1 (en) * 2016-02-05 2017-08-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides effective to reduce the expression of menin in cancer cells of a subject
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
WO2017184883A1 (en) * 2016-04-20 2017-10-26 JBS Science Inc. Kit and method for detecting mutations in ctnnb1 and htert, and use thereof in hcc detection and disease management
AU2017286811A1 (en) 2016-06-17 2018-11-22 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
AU2017287536B9 (en) 2016-06-30 2021-03-25 Kyowa Kirin Co., Ltd. Nucleic acid complex
RU2019101298A (ru) 2016-07-01 2020-08-03 Ф. Хоффманн-Ля Рош Аг Антисмысловые олигонуклеотиды для модулирования экспрессии htra1
US11655470B2 (en) * 2016-07-05 2023-05-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing COL6-related disorders and methods for treating same
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
EP3541939A4 (en) * 2016-11-17 2020-06-24 Ramot at Tel-Aviv University Ltd. HUMAN KAI1 METASTASE SUPPRESSOR MODULATORS METHOD AND USES THEREOF
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2018132755A1 (en) * 2017-01-12 2018-07-19 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
WO2018180005A1 (ja) * 2017-03-31 2018-10-04 国立研究開発法人日本医療研究開発機構 コンドロイチン硫酸生合成を阻害するアンチセンス核酸
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
US20190055564A1 (en) * 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
WO2019048632A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
US20230201357A1 (en) * 2017-11-08 2023-06-29 Aptamer Diagnostic, Inc. D-dimer-specific aptamers and methods of use in diagnostics, therapeutic and theranostic purposes
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
PL3717646T3 (pl) 2017-12-01 2025-10-27 The Texas A&M University System Leczenie antysensowe zespołu angelmana
WO2019165067A1 (en) * 2018-02-21 2019-08-29 Bristol-Myers Squibb Company Camk2d antisense oligonucleotides and uses thereof
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
WO2019200172A1 (en) 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
JP2021522800A (ja) 2018-05-09 2021-09-02 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. Atxn3発現を低減するための化合物及び方法
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
WO2020007700A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007772A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020011653A1 (en) * 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020015959A1 (en) * 2018-07-19 2020-01-23 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4.
AU2019310097A1 (en) 2018-07-25 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
JP7529286B2 (ja) * 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
TW202214854A (zh) 2019-01-31 2022-04-16 美商Ionis製藥公司 Yap1表現之調節劑
CA3133241A1 (en) * 2019-03-15 2020-09-24 University Of Massachusetts Oligonucleotides for tissue specific apoe modulation
TW202536179A (zh) * 2019-03-29 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之化合物及方法
US11739324B2 (en) 2019-06-25 2023-08-29 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of ABCA4
CN114585633A (zh) 2019-08-19 2022-06-03 米纳治疗有限公司 寡核苷酸缀合物组合物和使用方法
KR20220049619A (ko) * 2019-08-30 2022-04-21 베이롤 칼리지 오브 메드신 유전자 발현 조절 시스템
EP3822370A1 (en) * 2019-11-15 2021-05-19 Curiosity Diagnostics Sp. z o.o. Method of determining the presence of a hyper-virulent clostridioides difficile strain of the b1/nap1/027 group in a sample
WO2021126997A1 (en) * 2019-12-18 2021-06-24 Petdx, Inc. Methods and compositions for cancer detection, characterization or management in companion animals
CN114929876A (zh) * 2020-01-31 2022-08-19 株式会社三和化学研究所 Atn1的反义寡核苷酸
WO2021177418A1 (ja) * 2020-03-04 2021-09-10 日産化学株式会社 Calm2のアンチセンスオリゴヌクレオチド
TW202216996A (zh) 2020-06-29 2022-05-01 美商Ionis製藥公司 調節plp1之化合物及方法
CN116368227A (zh) * 2020-07-22 2023-06-30 弗洛里神经科学与心理健康研究所 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法
MX2023001222A (es) 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.
WO2022032017A2 (en) * 2020-08-07 2022-02-10 The General Hospital Corporation Human xist antisense oligonucleotides for x reactivation therapy
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
EP4015040A1 (en) * 2020-12-16 2022-06-22 Fundación para la Formación e Investigación Sanitarias de la Región de Murcia Method for treating blood diseases
AR124408A1 (es) 2020-12-18 2023-03-22 Ionis Pharmaceuticals Inc Compuestos y métodos para modular el factor xii
EP4329799A4 (en) * 2021-04-30 2025-04-30 Greenlight Biosciences, Inc. Messenger rna therapeutics and compositions
US11753644B2 (en) 2021-06-18 2023-09-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing IFNAR1 expression
WO2023004049A1 (en) * 2021-07-21 2023-01-26 AcuraStem, Inc. Unc13a antisense oligonucleotides
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
CN118541484A (zh) * 2021-12-17 2024-08-23 豪夫迈·罗氏有限公司 反义寡核苷酸
WO2023139360A1 (en) * 2022-01-19 2023-07-27 Autolus Limited Nucleic acid construct
WO2024064608A2 (en) * 2022-09-20 2024-03-28 Intergalactic Therapeutics, Inc. Best1 vectors and uses thereof
KR20260021596A (ko) 2023-04-05 2026-02-13 아이오니스 파마수티컬즈, 인코포레이티드 Pln 발현을 감소시키는 화합물 및 방법
GB202312098D0 (en) * 2023-08-08 2023-09-20 Ucl Business Ltd Therapeutic molecules
AU2024339330A1 (en) * 2023-09-13 2026-04-02 Haya Therapeutics Sa Compositions and methods for modulating wisp2 super-enhancer-associated rna
EP4784753A2 (en) * 2023-09-27 2026-08-05 Judo Bio Inc. Slc6a19-targeting modulatory nucleic acid agents
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid
WO2026033502A1 (en) * 2024-08-09 2026-02-12 Associação Biopolis Kit, method, and uses thereof
WO2026074434A1 (en) * 2024-10-01 2026-04-09 Fondazione Istituto Italiano Di Tecnologia Rad51/brca2 complex aptamers and uses thereof
WO2026133251A1 (en) 2024-12-20 2026-06-25 Novartis Ag Combinations of antisense agents, pharmaceutical compositions and methods of use

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04320700A (ja) * 1991-04-17 1992-11-11 Nippon Shoji Kk アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
JP2010516256A (ja) * 2007-01-19 2010-05-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン Adrb2癌マーカー
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008025069A1 (en) * 2006-08-28 2008-03-06 The Walter And Eliza Hall Institute Of Medical Research Methods of modulating cellular activity and compositions therefor
JP2012503651A (ja) * 2008-09-26 2012-02-09 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ 3−デアザネプラノシン誘導体

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04320700A (ja) * 1991-04-17 1992-11-11 Nippon Shoji Kk アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
JP2010516256A (ja) * 2007-01-19 2010-05-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン Adrb2癌マーカー
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
KANHERE A. ET AL., MOLECULAR CELL, vol. 38(2010), JPN6017005739, pages 675 - 688, ISSN: 0003506431 *
PRENSNER J.R. ET AL., NAT. BIOTECHNOL., vol. 29(8)(2011), JPN6017005745, pages 742 - 749, ISSN: 0003506433 *
SARMA K. ET AL., PROC. NATL. ACAD. USA, vol. 107(51)(2010), JPN6017005742, pages 22196 - 22201, ISSN: 0003506430 *
ZHAO J. ET AL., SCIENCE, vol. 322(5902)(2008), JPN6017005741, pages 750 - 756, ISSN: 0003506429 *
神武洋二郎 他: "長鎖ncRNAによるクロマチン修飾因子のリクルートメント機構", 実験医学, vol. Vol.29 No.11(2011), JPN7017000587, pages 1716 - 1721, ISSN: 0003506432 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021514182A (ja) * 2018-02-09 2021-06-10 ジェネンテック, インコーポレイテッド Tmem106b発現を調節するためのオリゴヌクレオチド
JP7281474B2 (ja) 2018-02-09 2023-05-25 ジェネンテック, インコーポレイテッド Tmem106b発現を調節するためのオリゴヌクレオチド
JP2023503202A (ja) * 2020-02-12 2023-01-26 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP7321614B2 (ja) 2020-02-12 2023-08-07 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP2023523622A (ja) * 2020-04-29 2023-06-06 サリオジェン セラピューティクス インコーポレイテッド 遺伝性黄斑変性症の治療のための組成物及び方法

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