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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
oligonucleotide
nucleotide
stranded oligonucleotide
nucleotides
sequence
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
JP2015512857A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016522674A5 (cg-RX-API-DMAC7.html
Inventor
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，
Original Assignee
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
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 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 filed Critical ラナ セラピューティクス インコーポレイテッド
Publication of JP2016522674A publication Critical patent/JP2016522674A/ja
Publication of JP2016522674A5 publication Critical patent/JP2016522674A5/ja
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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)

Application Number Priority Date Filing Date Title
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

Publications (2)

Publication Number Publication Date
JP2016522674A true JP2016522674A (ja) 2016-08-04
JP2016522674A5 JP2016522674A5 (cg-RX-API-DMAC7.html) 2016-09-15

Family

ID=49584304

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015512857A Pending JP2016522674A (ja) 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

Country Status (4)

Country Link
US (1) US20150141320A1 (cg-RX-API-DMAC7.html)
EP (1) EP2850184A4 (cg-RX-API-DMAC7.html)
JP (1) JP2016522674A (cg-RX-API-DMAC7.html)
WO (1) WO2013173637A1 (cg-RX-API-DMAC7.html)

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 (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101900770B1 (ko) 2010-07-19 2018-09-20 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
DK2638163T3 (en) 2010-11-12 2017-07-24 Massachusetts Gen Hospital POLYCOMB-ASSOCIATED NON-CODING RNAs
CN104583401A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节atp2a2表达的组合物和方法
CA2873779A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating mecp2 expression
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EP2850185A4 (en) 2012-05-16 2015-12-30 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING UTRN GENE EXPRESSION
KR20150030205A (ko) 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
JP2016523103A (ja) * 2013-06-26 2016-08-08 エランコ・ユーエス・インコーポレイテッドＥｌａｎｃｏ ＵＳ Ｉｎｃ． イヌ糸状虫病の病因であるディロフィラリア・イミティス（Ｄｉｒｏｆｉｌａｒｉａ ｉｍｍｉｔｉｓ）において大環状ラクトン耐性を予測するためのマーカー
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
WO2015035231A1 (en) * 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, 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
HRP20211397T1 (hr) 2014-03-19 2021-12-10 Ionis Pharmaceuticals, Inc. Pripravci za modulaciju ekspresije ataksina 2
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
LT3126499T (lt) 2014-04-01 2020-07-27 Biogen Ma Inc. Kompozicijos, skirtos sod-1 ekspresijos moduliavimui
WO2016061487A1 (en) * 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
CA2966044A1 (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
ES2848377T3 (es) 2015-02-26 2021-08-09 Ionis Pharmaceuticals Inc Moduladores específicos de alelo de RODOPSINA P23H
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
CA3006015A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
CA3012967A1 (en) 2016-01-29 2017-08-03 Kyowa Hakko Kirin Co., Ltd. Nucleic acid conjugate
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
US10689709B2 (en) 2016-04-20 2020-06-23 JBS Science Inc. Kit and method for detecting mutations in CTNNB1 and hTERT, and use thereof in HCC detection and disease management
WO2017219017A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
CN109415401B (zh) 2016-06-30 2023-02-03 协和麒麟株式会社 核酸复合物
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
WO2018092137A1 (en) 2016-11-17 2018-05-24 Ramot At Tel-Aviv University Ltd. Modulators of human kai1 metastasis suppressor gene, methods and uses thereof
JP2019535839A (ja) 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
US11352625B2 (en) 2017-01-12 2022-06-07 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
JP7141621B2 (ja) * 2017-03-31 2022-09-26 学校法人 愛知医科大学 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
EP4219715A3 (en) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
US11447773B2 (en) 2017-09-08 2022-09-20 Mina Therapeutics Limited Stabilized HNF4A saRNA compositions and methods of use
WO2019094315A1 (en) * 2017-11-08 2019-05-16 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
RU2020121752A (ru) 2017-12-01 2021-12-30 ТЕХАССКАЯ УНИВЕРСИТЕТСКАЯ СИСТЕМА А энд М Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты
MX2020008581A (es) * 2018-02-21 2020-09-21 Bristol Myers Squibb Co Oligonucleotidos antisentido de la proteina cinasa del tipo ii delta dependiente del calcio/calmodulina (camk2d) y sus usos.
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING 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
AU2019252667A1 (en) 2018-04-11 2020-10-01 Ionis Pharmaceuticals, Inc. Modulators of EZH2 expression
TW202003541A (zh) 2018-05-09 2020-01-16 美商Ionis製藥公司 用於減少ａｔｘｎ３表現之化合物及方法
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.
CA3106986A1 (en) 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
EP3870237A1 (en) * 2018-10-26 2021-09-01 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods and use thereof
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
WO2020160453A1 (en) 2019-01-31 2020-08-06 Ionis Pharmaceuticals, Inc. Modulators of yap1 expression
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸
UA128437C2 (uk) 2019-03-29 2024-07-10 Айоніс Фармасьютикалз, Інк. Сполуки і способи модулювання ube3a-ats
US11739324B2 (en) 2019-06-25 2023-08-29 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of ABCA4
JP2022545101A (ja) 2019-08-19 2022-10-25 ミナ セラピューティクス リミテッド オリゴヌクレオチドコンジュゲート組成物および使用方法
AU2020335909A1 (en) * 2019-08-30 2022-04-14 Baylor College Of Medicine System for regulating gene expression
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
CA3169474A1 (en) * 2020-01-31 2021-08-05 Sanwa Kagaku Kenkyusho Co., Ltd. Antisense oligonucleotide of atn1
JPWO2021177418A1 (cg-RX-API-DMAC7.html) * 2020-03-04 2021-09-10
AU2021299290A1 (en) 2020-06-29 2023-01-05 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating PLP1
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
KR20230043914A (ko) 2020-07-28 2023-03-31 아이오니스 파마수티컬즈, 인코포레이티드 App 발현을 감소시키기 위한 화합물 및 방법
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
MX2023012833A (es) * 2021-04-30 2024-01-23 Greenlight Biosciences Inc Opciones terapéuticas y composiciones de arn mensajero.
JP2024523364A (ja) 2021-06-18 2024-06-28 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 Ｉｆｎａｒ１発現を低減するための化合物及び方法
MX2024000996A (es) * 2021-07-21 2024-06-28 AcuraStem Incorporated Oligonucleótido antisentido unc13a.
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression
EP4448764A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Antisense oligonucleotide
WO2023139360A1 (en) * 2022-01-19 2023-07-27 Autolus Limited Nucleic acid construct
EP4590841A2 (en) * 2022-09-20 2025-07-30 Aldevron, LLC Best1 vectors and uses thereof
GB202312098D0 (en) * 2023-08-08 2023-09-20 Ucl Business Ltd Therapeutic molecules
WO2025056973A2 (en) * 2023-09-13 2025-03-20 Haya Therapeutics Sa Compositions and methods for modulating wisp2 super-enhancer-associated rna
WO2025072672A2 (en) * 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid

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
US20110237606A1 (en) * 2008-09-26 2011-09-29 Agency Of Science, Technology And Research 3-Deazaneplanocin Derivatives

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

Also Published As

Publication number Publication date
EP2850184A1 (en) 2015-03-25
US20150141320A1 (en) 2015-05-21
WO2013173637A1 (en) 2013-11-21
EP2850184A4 (en) 2016-01-27
WO2013173637A8 (en) 2014-12-24

Similar Documents

Publication Publication Date Title
JP2016522674A (ja) 遺伝子発現を調節するための組成物及び方法
JP2016528873A (ja) 遺伝子発現を調節するための組成物及び方法
JP2015518710A (ja) ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法
JP2015518714A (ja) 遺伝子発現を調節するための組成物及び方法
JP2015523853A (ja) Ａｔｐ２ａ２発現を調節するための組成物及び方法
JP2015518712A (ja) Ｍｅｃｐ２発現を調節するための組成物及び方法
JP2015518713A (ja) Ｕｔｒｎ発現を調節するための組成物及び方法
JP2015519057A (ja) Ｐｔｅｎ発現を調節するための組成物及び方法
JP2015523855A (ja) Ａｐｏａ１及びａｂｃａ１発現を調節するための組成物及び方法
ES2586593T3 (es) Liposomas de retinoide para potenciar la modulación de la expresión de hsp47
US10041074B2 (en) Euchromatic region targeting methods for modulating gene expression
JP2015518711A (ja) Ｂｄｎｆ発現を調節するための組成物及び方法
JP6129844B2 (ja) 多量体オリゴヌクレオチド化合物
JP2015523854A (ja) 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
JP2016521556A (ja) Ｆｏｘｐ３発現を調節するための組成物及び方法
US20150225722A1 (en) Methods for selective targeting of heterochromatin forming non-coding rna
JP2016534035A (ja) 筋萎縮性側索硬化症を治療するための組成物及び方法
US11464873B2 (en) RNA-modulating agents
CN105658797A (zh) 用于调节rna的组合物和方法
JP2014527819A5 (cg-RX-API-DMAC7.html)
KR20180057608A (ko) 치료용 올리고뉴클레오타이드
US20180055869A1 (en) Compositions and methods for modulating rna
US20200054746A1 (en) Methods for increasing neuronal survival
WO2022026648A1 (en) Inhibition of incexact1 to treat heart disease
JP7788561B2 (ja) アンチセンスオリゴヌクレオチド及びその使用

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150428

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160513

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20160513

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20170228

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20171012