MA43072A - Compositions d'oligonucléotides et procédés associés - Google Patents

Compositions d'oligonucléotides et procédés associés

Info

Publication number
MA43072A
MA43072A MA43072A MA43072A MA43072A MA 43072 A MA43072 A MA 43072A MA 43072 A MA43072 A MA 43072A MA 43072 A MA43072 A MA 43072A MA 43072 A MA43072 A MA 43072A
Authority
MA
Morocco
Prior art keywords
oligonucleotide composition
chirally
controlled oligonucleotide
oligonucleotides
compositions
Prior art date
Application number
MA43072A
Other languages
English (en)
Inventor
Christopher J Francis
Susovan Mohapatra
Meena
Anna Sokolovska
Nenad Svrzikapa
Chandra Vargeese
Gregory L Verdine
Original Assignee
Wave Life Sciences Ltd
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 Wave Life Sciences Ltd filed Critical Wave Life Sciences Ltd
Publication of MA43072A publication Critical patent/MA43072A/fr

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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • C12N2310/322—2'-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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Entre autres choses, la présente invention concerne des oligonucléotides à contrôle chirale de conceptions sélectionnées, des compositions oligonucléotidiques à contrôle chiral, et des procédés pour les fabriquer et les utiliser. Dans certains modes de réalisation, une composition d'oligonucléotides à commande chirale fournie fournit différents motifs de clivage d'un polymère d'acide nucléique qu'une composition d'oligonucléotides de référence. Dans certains modes de réalisation, une composition d'oligonucléotides à commande chirale fournie fournit un clivage à site unique dans une séquence complémentaire d'un polymère d'acide nucléique. Dans certains modes de réalisation, une composition oligonucléotidique contrôlée chiralement a une séquence quelconque de bases et / ou des modifications de motif ou de base, des modifications de sucre, des modifications du squelette et / ou une stéréochimie ou une combinaison de ces éléments.
MA43072A 2015-07-22 2016-07-22 Compositions d'oligonucléotides et procédés associés MA43072A (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562195779P 2015-07-22 2015-07-22
US201562236847P 2015-10-02 2015-10-02
US201662331960P 2016-05-04 2016-05-04

Publications (1)

Publication Number Publication Date
MA43072A true MA43072A (fr) 2018-05-30

Family

ID=57835219

Family Applications (1)

Application Number Title Priority Date Filing Date
MA43072A MA43072A (fr) 2015-07-22 2016-07-22 Compositions d'oligonucléotides et procédés associés

Country Status (23)

Country Link
US (6) US10479995B2 (fr)
EP (3) EP4344744A3 (fr)
JP (6) JP2018525357A (fr)
KR (1) KR20180028516A (fr)
CN (3) CN108025089A (fr)
AU (3) AU2016297155B2 (fr)
BR (1) BR112017028636A2 (fr)
CA (1) CA2989682A1 (fr)
CL (1) CL2018000145A1 (fr)
CO (1) CO2018001484A2 (fr)
CR (1) CR20180107A (fr)
EC (1) ECSP18010463A (fr)
HK (2) HK1255370A1 (fr)
IL (2) IL320434A (fr)
MA (1) MA43072A (fr)
MX (2) MX2018000796A (fr)
NI (1) NI201800009A (fr)
PE (1) PE20181174A1 (fr)
PH (1) PH12018500145A1 (fr)
SG (1) SG10201912900XA (fr)
SV (1) SV2018005619A (fr)
TW (1) TW201707711A (fr)
WO (2) WO2017015575A1 (fr)

Families Citing this family (158)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8158768B2 (en) * 2002-12-23 2012-04-17 Dynavax Technologies Corporation Immunostimulatory sequence oligonucleotides and methods of using the same
RU2572826C2 (ru) 2008-12-02 2016-01-20 Чиралджен, Лтд. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
SG10201403841QA (en) 2009-07-06 2014-09-26 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
EP2872147B1 (fr) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Procede de fabrication d'oligonucleotides chiraux
RU2693381C2 (ru) 2012-07-13 2019-07-02 Уэйв Лайф Сайенсес Лтд. Асимметричная вспомогательная группа
TWI657819B (zh) 2013-07-19 2019-05-01 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
EP3095459A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
CA2936712A1 (fr) 2014-01-16 2015-07-23 Meena Conception chirale
CN107208092B (zh) * 2014-12-16 2021-09-10 罗氏创新中心哥本哈根有限公司 手性毒性筛选方法
WO2016097212A1 (fr) 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Édition ciblée d'arn
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3929293A3 (fr) 2015-04-03 2022-03-16 University Of Massachusetts Petit arn interférent asymétrique entièrement stabilisé
CA2980337A1 (fr) 2015-04-03 2016-10-06 University Of Massachusetts Composes oligonucleotidiques pour le ciblage de l'arnm de l'hungtingtine
CR20180107A (es) 2015-07-22 2018-04-03 Wave Life Sciences Ltd Composiciones de oligonucleótidos y métodos de los mismos
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7511326B2 (ja) 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
MX391850B (es) 2015-12-10 2025-03-19 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
CA3015823A1 (fr) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions et procedes de synthese de phosphoramidite et d'oligonucleotides
MA45270A (fr) * 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
EP3463386A4 (fr) 2016-06-03 2020-03-04 Wave Life Sciences Ltd. Oligonucléotides, compositions et méthodes associées
WO2017220751A1 (fr) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Oligonucléotides d'édition d'arn monocaténaire
CA3033368A1 (fr) 2016-08-12 2018-02-15 University Of Massachusetts Oligonucleotides conjugues
KR102501980B1 (ko) 2016-09-01 2023-02-20 프로큐알 테라퓨틱스 Ⅱ 비.브이. 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드
WO2018064593A1 (fr) 2016-09-29 2018-04-05 Ionis Pharmaceuticals, Inc. Composés et procédés pour réduire l'expression de tau
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
PL3554554T3 (pl) 2016-12-19 2022-12-19 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
LT3554553T (lt) 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos
RS63621B1 (sr) 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Oligomerni konjugati za preskakanje egzona za mišićnu distrofiju
EP3571300A1 (fr) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Complexes oligonucléotidiques destinés à être utilisés dans l'édition d'arn
WO2018145009A1 (fr) * 2017-02-06 2018-08-09 University Of Tennessee Research Foundation Méthodes et compositions à base d'adnzyme pour le traitement de la maladie de huntington
US11932856B2 (en) 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
CA3065523A1 (fr) * 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs procedes d'utilisation
EP3630199A4 (fr) 2017-06-02 2021-11-10 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs procédés d'utilisation
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2018237194A1 (fr) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Composés, compositions et procédés de synthèse
US10844377B2 (en) 2017-06-23 2020-11-24 University Of Massachusetts Two-tailed self-delivering siRNA
EP3645121B8 (fr) 2017-06-28 2025-06-18 PTC Therapeutics, Inc. Méthodes de traitement de la maladie de huntington
US11382918B2 (en) 2017-06-28 2022-07-12 Ptc Therapeutics, Inc. Methods for treating Huntington's Disease
CA3072076A1 (fr) * 2017-08-08 2019-02-14 Wave Life Sciences Ltd. Compositions oligonucleotidiques et methodes associees
KR20200052369A (ko) 2017-09-18 2020-05-14 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP2020536060A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
WO2019075357A1 (fr) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
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
MX2020007439A (es) 2018-01-12 2020-09-14 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina y usos de estos.
PE20201501A1 (es) 2018-01-12 2020-12-29 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos
PE20211242A1 (es) 2018-01-15 2021-07-09 Ionis Pharmaceuticals Inc Moduladores de la expresion dnm2
WO2019157531A1 (fr) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Composés modifiés et leurs utilisations
EP3768694A4 (fr) * 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Méthodes de modulation de l'expression de fmr1
MX2020009957A (es) 2018-03-27 2021-01-15 Ptc Therapeutics Inc Compuestos para el tratamiento de enfermedad de hungtinton.
AU2019252680B2 (en) * 2018-04-12 2025-11-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
SG11202010910QA (en) 2018-05-07 2020-12-30 Alnylam Pharmaceuticals Inc Extrahepatic delivery
CN112189053B (zh) 2018-05-09 2024-05-14 Ionis制药公司 用于减少atxn3表达的化合物和方法
CR20200605A (es) 2018-05-09 2021-01-29 Ionis Pharmaceuticals Inc Compuestos y métodos para la reducción de la expresión de fxi
JP7427608B2 (ja) * 2018-05-11 2024-02-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
GB201808146D0 (en) * 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
JP2021525507A (ja) * 2018-05-25 2021-09-27 ニューヨーク インスティチュート オブ テクノロジーＮｅｗ Ｙｏｒｋ Ｉｎｓｔｉｔｕｔｅ Ｏｆ Ｔｅｃｈｎｏｌｏｇｙ 直接核酸配列決定方法
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (fr) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. Oligomères induisant un saut d'exon pour la dystrophie musculaire
CA3103429A1 (fr) * 2018-06-14 2019-12-19 Don W. Cleveland Composes et procedes permettant d'augmenter l'expression de stmn2
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
PL3814357T3 (pl) 2018-06-27 2024-09-16 Ptc Therapeutics, Inc. Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona
CN109030440B (zh) * 2018-07-18 2020-12-04 西北农林科技大学 一种基于三氧化钼量子点检测单宁酸含量的方法
NZ771733A (en) 2018-07-25 2025-10-31 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn2 expression
TW202449155A (zh) 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
KR102916447B1 (ko) 2018-08-10 2026-01-23 유니버시티 오브 매사추세츠 Snp를 표적화하는 변형된 올리고뉴클레오티드
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
TWI856973B (zh) 2018-09-19 2024-10-01 美商Ionis製藥公司 Pnpla3表現之調節劑
WO2020072883A1 (fr) * 2018-10-05 2020-04-09 Ionis Pharmaceuticals, Inc. Composés oligomères enrichis de manière chirale
EP3880821A4 (fr) 2018-11-15 2023-01-25 Ionis Pharmaceuticals, Inc. Modulateurs de l'expression d'irf5
BR112021007476A2 (pt) 2018-11-21 2021-11-03 Ionis Pharmaceuticals Inc Compostos e métodos para reduzir a expressão de príon
US12485136B2 (en) 2018-12-03 2025-12-02 Takeda Pharmaceuticals U.S.A., Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with MLH3 activity
CN113412330A (zh) 2018-12-13 2021-09-17 萨勒普塔医疗公司 用于肌营养不良的外显子跳跃寡聚物缀合物
JP7394137B2 (ja) 2018-12-21 2023-12-07 アイオニス・ファーマシューティカルズ・インコーポレイテッド Ｈｓｄ１７ｂ１３発現のモジュレータ
WO2020150636A1 (fr) 2019-01-18 2020-07-23 University Of Massachusetts Ancres de modification pharmacocinétique dynamique
KR20210121199A (ko) * 2019-02-01 2021-10-07 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
WO2020176771A1 (fr) 2019-02-27 2020-09-03 Ionis Pharmaceuticals, Inc. Modulateurs de l'expression de malat1
JP2022525541A (ja) * 2019-03-20 2022-05-17 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド調製に有用な技術
PL3947684T3 (pl) 2019-03-29 2025-07-21 Ionis Pharmaceuticals, Inc. Związki i sposoby modulacji ube3a-ats
EP3955966A1 (fr) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions pour le traitement de la dystrophie musculaire
BR112021021203A2 (pt) * 2019-04-25 2022-01-04 Wave Life Sciences Ltd Oligonucleotídeo; composição de oligonucleotídeo quiralmente controlada; composição farmacêutica; método para prevenir, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a e/ou prevenir, atrasar o início, desenvolvimento e/ou progresso, e/ou tratar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para aumentar o nível de salto de um éxon prejudicial em uma transcrição de gene ush2a ou seu produto de gene em uma célula; método para prevenir, atrasar o início ou progressão de, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para saltar um éxon prejudicial 13 em um alelo de ush2a em um indivíduo; método para produzir ou aumentar o nível de um éxon de proteína ush2a com salto 13 em um sistema; e composto, oligonucleotídeo, composição ou método
CA3137741A1 (fr) * 2019-04-25 2020-10-29 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs methodes d'utilisation
KR20220022114A (ko) 2019-05-13 2022-02-24 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
LT3976791T (lt) 2019-05-28 2025-07-25 Ionis Pharmaceuticals, Inc. Junginiai ir būdai fus raiškai mažinti
EP3956450B1 (fr) 2019-07-26 2025-08-13 Ionis Pharmaceuticals, Inc. Composés et procédés pour la modulation de gfap
CN114502730A (zh) 2019-08-09 2022-05-13 马萨诸塞大学 经化学修饰的靶向snp的寡核苷酸
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
CN114728017B (zh) 2019-10-14 2025-10-28 阿斯利康(瑞典)有限公司 Pnpla3表达的调节剂
JP2023500251A (ja) 2019-11-01 2023-01-05 ノバルティス アーゲー ハンチントン病の進行を遅らせる処置のためのスプライシングモジュレーターの使用
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
WO2021168183A1 (fr) * 2020-02-21 2021-08-26 Ionis Pharmaceuticals, Inc. Procédés de réduction de l'expression de htt
AR121446A1 (es) 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
JP2023521604A (ja) * 2020-03-30 2023-05-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法
JP2023522961A (ja) 2020-04-21 2023-06-01 フラッグシップ パイオニアリング， インコーポレイテッド 二機能性分子およびその使用方法
CN115885042A (zh) 2020-05-22 2023-03-31 波涛生命科学有限公司 双链寡核苷酸组合物及其相关方法
EP4157289A4 (fr) 2020-05-26 2024-06-26 University Of Massachusetts Oligonucléotides synthétiques ayant des régions de séquences et de modifications de groupe
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
TW202227102A (zh) 2020-09-22 2022-07-16 瑞典商阿斯特捷利康公司 治療脂肪肝病之方法
KR20230108728A (ko) 2020-11-18 2023-07-18 아이오니스 파마수티컬즈, 인코포레이티드 앤지오텐시노겐 발현을 조절하기 위한 화합물 및 방법
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
WO2022189363A1 (fr) 2021-03-08 2022-09-15 Les Laboratoires Servier Oligonucléotides antisens pour inhiber l'expression de l'alpha-synucléine
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
CN113122615B (zh) * 2021-05-24 2024-07-09 湖南赛哲智造科技有限公司 一种应用于绝对定量高通量测序的多重pcr扩增的单分子标签引物及其应用
MX2023015102A (es) 2021-06-22 2024-04-01 AcuraStem Incorporated Oligonucleotidos antisentido pikfyve.
KR20240040729A (ko) 2021-06-23 2024-03-28 유니버시티 오브 매사추세츠 자간전증 및 기타 혈관신생 질환의 치료를 위한 최적화된 항-flt1 올리고뉴클레오타이드 화합물
WO2023004049A1 (fr) 2021-07-21 2023-01-26 AcuraStem, Inc. Oligonucléotides antisens unc13a
CA3233242A1 (fr) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Oligonucleotides antisens ayant une ou plusieurs unites abasiques
MX2024004011A (es) 2021-10-01 2024-07-01 Adarx Pharmaceuticals Inc Composiciones moduladoras de precalicreína y métodos de uso de estas.
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
CN119213127A (zh) 2022-04-28 2024-12-27 阿库拉斯特姆公司 Syf2反义寡核苷酸
WO2024013360A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar
WO2024013361A1 (fr) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucléotides pour édition d'arn médiée par adar et leur utilisation
KR20250051686A (ko) * 2022-08-11 2025-04-17 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
WO2024064237A2 (fr) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Efficacité du saut d'exon médié par un oligonucléotide antisens dans le traitement de la dmd
CN120239607A (zh) 2022-09-23 2025-07-01 Ionis制药公司 用于降低mecp2表达的化合物和方法
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
TW202435897A (zh) 2022-11-24 2024-09-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療遺傳性ｈｆｅ血色沉著病之反義寡核苷酸
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (fr) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024155986A2 (fr) 2023-01-20 2024-07-25 AcuraStem Incorporated Oligonucléotides antisens unc13a
IL322500A (en) 2023-01-31 2025-09-01 AcuraStem Incorporated syf2 molecular-readable oligonucleotides
CN120659879A (zh) 2023-02-06 2025-09-16 爱可施丹股份有限公司 Pikfyve反义寡核苷酸
EP4669753A1 (fr) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire athérosclérotique
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024206175A1 (fr) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles neurologiques
EP4688143A1 (fr) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Oligonucléotides antisens pour traitement des maladies du foie
KR20260007584A (ko) 2023-04-20 2026-01-14 아다르엑스 파마슈티컬스, 인크. Mapt-조절 조성물 및 이의 사용 방법
TW202442246A (zh) 2023-04-27 2024-11-01 美商薩羅塔治療公司 用於治療慢性腎病之反義寡聚物
EP4709422A1 (fr) 2023-05-12 2026-03-18 Adarx Pharmaceuticals, Inc. Composés conjugués à un ligand de nmda et leurs utilisations
WO2024249328A2 (fr) 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Compositions de modulation de sod1 et leurs procédés d'utilisation
WO2024256620A1 (fr) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement des maladies neurodégénératives
KR20260026066A (ko) 2023-06-20 2026-02-25 아다르엑스 파마슈티컬스, 인크. Lrrk2 조절 조성물 및 이의 사용 방법
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
AU2024323589A1 (en) 2023-08-16 2026-03-05 Les Laboratoires Servier Oligonucleotides for modulating kcnt1 expression
WO2025051946A1 (fr) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles métaboliques
TW202526017A (zh) 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少ａｐｏｃｉｉｉ表現的化合物及方法
TW202530406A (zh) 2023-09-21 2025-08-01 美商Ionis製藥公司 用於抑制ｌｐａ的化合物及方法
GB202314724D0 (en) 2023-09-26 2023-11-08 Astrazeneca Ab compounds and methods for reducing psd3 expression
WO2025085810A2 (fr) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Oligomères antisens pour le traitement de myopathies centronucléaires
AU2024369609A1 (en) 2023-10-31 2025-12-11 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025104239A1 (fr) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de galactosémie classique
WO2025128799A1 (fr) 2023-12-12 2025-06-19 Korro Bio, Inc. Oligonucléotides d'édition d'arn double brin et leurs utilisations
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224230A1 (fr) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour traitement de la stéatose hépatique
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (fr) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles métaboliques
WO2026050243A1 (fr) 2024-08-26 2026-03-05 Korro Bio, Inc. Oligonucléotides conjugués à des fractions galnac pour édition d'arn
WO2026060374A2 (fr) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles neurologiques

Family Cites Families (211)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US513030A (en) 1894-01-16 Machine for waxing or coating paper
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
DE3329892A1 (de) 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg Verfahren zur herstellung von oligonucleotiden
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US4659774A (en) 1985-11-01 1987-04-21 American Hoechst Corporation Support for solid-phase oligonucleotide synthesis
US5130300A (en) 1986-03-07 1992-07-14 Monsanto Company Method for enhancing growth of mammary parenchyma
ATE113059T1 (de) 1987-06-24 1994-11-15 Florey Howard Inst Nukleosid-derivate.
US4923901A (en) 1987-09-04 1990-05-08 Millipore Corporation Membranes with bound oligonucleotides and peptides
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5141813A (en) 1989-08-28 1992-08-25 Clontech Laboratories, Inc. Multifunctional controlled pore glass reagent for solid phase oligonucleotide synthesis
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
CA2029273A1 (fr) 1989-12-04 1991-06-05 Christine L. Brakel Compose a base de nucleotide modifie
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5212295A (en) 1990-01-11 1993-05-18 Isis Pharmaceuticals Monomers for preparation of oligonucleotides having chiral phosphorus linkages
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5512668A (en) * 1991-03-06 1996-04-30 Polish Academy Of Sciences Solid phase oligonucleotide synthesis using phospholane intermediates
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US20020183502A1 (en) 1991-05-21 2002-12-05 Mesmaeker Alain De Backbone-modified oligonucleotide analogs and methods for using same
ATE221127T1 (de) 1991-10-15 2002-08-15 Isis Pharmaceuticals Inc Über chirale phosphoratome gebundene oligonukleotide
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US6235887B1 (en) 1991-11-26 2001-05-22 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation directed by oligonucleotides containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
DK1695979T3 (da) 1991-12-24 2011-10-10 Isis Pharmaceuticals Inc Gappede modificerede oligonukleotider
US6015886A (en) 1993-05-24 2000-01-18 Chemgenes Corporation Oligonucleotide phosphate esters
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
AU678085B2 (en) 1993-11-16 1997-05-15 Genta Incorporated Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US6222025B1 (en) 1995-03-06 2001-04-24 Isis Pharmaceuticals, Inc. Process for the synthesis of 2′-O-substituted pyrimidines and oligomeric compounds therefrom
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
WO1999005160A2 (fr) 1997-07-25 1999-02-04 Hybridon, Inc. Oligonucleotides possedant des thiophosphates stereospecifiques aux extremites 3'
US6528640B1 (en) 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
CN100366293C (zh) * 1998-02-20 2008-02-06 泰诺士公司 补体活化的抑制剂
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6242589B1 (en) * 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US6066500A (en) 1999-06-25 2000-05-23 Isis Pharmaceuticals Inc. Antisense modulation of Beta catenin expression
US6147200A (en) 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6949520B1 (en) 1999-09-27 2005-09-27 Coley Pharmaceutical Group, Inc. Methods related to immunostimulatory nucleic acid-induced interferon
US20020082227A1 (en) * 1999-09-30 2002-06-27 Scott Henry Use of oligonucleotides for inhibition of complement activation
US20010055761A1 (en) 1999-10-29 2001-12-27 Agilent Technologies Small scale dna synthesis using polymeric solid support with functionalized regions
DE10019756A1 (de) 2000-04-20 2001-10-25 Bayer Ag Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen
US6492171B2 (en) 2000-05-16 2002-12-10 Isis Pharmaceuticals, Inc. Antisense modulation of TERT expression
GB0024752D0 (en) 2000-10-10 2000-11-22 Univ Belfast Oxidative halogenation of aromatic compounds
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US6440739B1 (en) 2001-07-17 2002-08-27 Isis Pharmaceuticals, Inc. Antisense modulation of glioma-associated oncogene-2 expression
JP4348044B2 (ja) 2002-02-12 2009-10-21 株式会社キラルジェン 立体規則性の高いジヌクレオシドホスホロチオエートの製造法
US8232383B2 (en) 2002-02-20 2012-07-31 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
SI2264172T1 (sl) 2002-04-05 2017-12-29 Roche Innovation Center Copenhagen A/S Oligomerne spojine za modulacijo izražanja HIF-1alfa
CA2489174C (fr) 2002-07-10 2013-02-05 Thomas Tuschl Interference de l'arn (arni) par des molecules d'arn simple brin
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US7045306B2 (en) 2003-04-28 2006-05-16 The General Hospital Corporation Method for identifying compounds in vitro that modulate the dysregulation of transcription of transcription mediated by mutant huntingtin protein
JP4179562B2 (ja) 2003-05-14 2008-11-12 独立行政法人科学技術振興機構 ハンチンチン遺伝子の発現抑制
JP2005089441A (ja) 2003-08-08 2005-04-07 Toudai Tlo Ltd 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法
US7427672B2 (en) 2003-08-28 2008-09-23 Takeshi Imanishi Artificial nucleic acids of n-o bond crosslinkage type
WO2005023828A1 (fr) 2003-09-02 2005-03-17 Takeshi Wada Procede de production de ribonucleotide ou de derive de ribonucleotide
WO2005028494A1 (fr) 2003-09-02 2005-03-31 Takeshi Wada Procede de production d'un monomere 5'-phosphityle et d'un derive d'oligonucleotide h-phosphonate
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2008005562A2 (fr) 2006-07-07 2008-01-10 University Of Massachusetts Compositions de silençage de l'arn, et méthodes de traitement de la chorée de huntington
WO2005027980A1 (fr) 2003-09-12 2005-03-31 University Of Massachusetts Arn interference pour le traitement de troubles a gain de fonction
WO2005070859A1 (fr) 2004-01-27 2005-08-04 Takeshi Wada Supports fluores et procedes de production de derives d'oligonucleotide utilisant ces supports
WO2005085272A1 (fr) 2004-03-05 2005-09-15 Takeshi Wada Monomere de boranophosphate et procede de production de derive oligonucleotide
JP4865544B2 (ja) 2004-03-25 2012-02-01 株式会社キラルジェン 立体規則性の高いリボヌクレオチド類縁体及びデオキシリボヌクレオチド類縁体の製造法
US20060019916A1 (en) 2004-04-02 2006-01-26 Coley Pharmaceutical Group, Inc. Immunostimulatory nucleic acids for inducing IL-10 responses
JP2008513507A (ja) 2004-09-17 2008-05-01 アイシス ファーマシューティカルズ インコーポレイティッド 増強されたアンチセンスオリゴヌクレオチド
WO2006066260A2 (fr) 2004-12-17 2006-06-22 Thiosense, Inc. Compositions de monomeres et oligomeres de phosphore chiraux et procedes de production
US20060257912A1 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
WO2007002904A2 (fr) 2005-06-28 2007-01-04 Medtronic, Inc. Procedes et sequences permettant de supprimer de maniere preferentielle l'expression de la huntingtine mutee
US8501703B2 (en) 2005-08-30 2013-08-06 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007051045A2 (fr) 2005-10-28 2007-05-03 Alnylam Pharmaceuticals, Inc. Compositions et methodes destinees a inhiber l'expression du gene huntingtine
PL2161038T3 (pl) 2006-01-26 2014-06-30 Ionis Pharmaceuticals Inc Kompozycje i ich zastosowanie ukierunkowane na huntingtynę
KR101304071B1 (ko) 2006-01-27 2013-09-06 아이시스 파마수티컬즈 인코포레이티드 6-변형된 바이시클릭 핵산 유사체
WO2007143317A2 (fr) 2006-05-05 2007-12-13 Isis Pharmaceuticals, Inc Composés et procédés destinés à moduler l'expression de crp
WO2007134181A2 (fr) 2006-05-11 2007-11-22 Isis Pharmaceuticals, Inc. Analogues d'acides nucléiques bicycliques modifiés en 5'
KR101065760B1 (ko) 2006-05-31 2011-09-19 오사카 유니버시티 면역자극 올리고뉴클레오티드 및 그 의약 용도
EP3916095A1 (fr) * 2006-10-18 2021-12-01 Ionis Pharmaceuticals, Inc. Composés antisens
WO2008151833A2 (fr) 2007-06-13 2008-12-18 Hochschule Mannheim Composés pour la modulation de l'agrégation de l'huntingtine, procédés et moyens d'identification de tels composés
ATE462787T1 (de) 2007-06-18 2010-04-15 Commissariat Energie Atomique Reversibles sirna-silencing eines mutierten und endogenen huntington-wildtypgens und dessen anwendung zur behandlung von morbus huntington
EP2348112B1 (fr) 2007-07-09 2018-11-28 Idera Pharmaceuticals, Inc. Composés d'ARN modulatoire immunitaire stabilisé (SIMRA)
EP2224912B1 (fr) * 2008-01-02 2016-05-11 TEKMIRA Pharmaceuticals Corporation Compositions et procédés améliorés pour la délivrance d'acides nucléiques
CA2711587A1 (fr) 2008-02-04 2009-08-13 Galapagos Nv Cibles et composes moleculaires, et procedes pour les identifier, destines au traitement de maladies neurodegeneratives
US8679750B2 (en) 2008-05-09 2014-03-25 The University Of British Columbia Methods and compositions for the treatment of Huntington'S disease
WO2009135322A1 (fr) 2008-05-09 2009-11-12 The Universtity Of British Columbia Procédés et compositions pour le traitement de la maladie de huntington
WO2009143391A2 (fr) 2008-05-22 2009-11-26 Isis Pharmaceuticals, Inc Procédés de modulation de l’expression de creb
WO2009148605A2 (fr) 2008-06-04 2009-12-10 Isis Pharmaceuticals, Inc. Procédés pour traiter une hypercholestérolémie
HRP20160261T1 (hr) 2008-07-10 2016-06-03 Regenesance B.V. Komplementarni antagonisti i njihova uporaba
RU2572826C2 (ru) 2008-12-02 2016-01-20 Чиралджен, Лтд. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
US20120264806A1 (en) 2009-02-06 2012-10-18 Bennett C Frank Oligomeric compounds and excipients
US8987222B2 (en) * 2009-04-08 2015-03-24 University Of Massachusetts Single nucleotide polymorphism (SNP) targeting therapies for the treatment of huntington'S disease
SG10201403841QA (en) 2009-07-06 2014-09-26 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
WO2011015572A1 (fr) 2009-08-03 2011-02-10 Galapagos Nv Cibles et composés moléculaires, et procédés pour leur identification utiles dans le traitement de maladies neurodégénératives
WO2011015573A1 (fr) 2009-08-03 2011-02-10 Galapagos Nv Cibles et composés moléculaires, et procédés pour les identifier, utiles dans le traitement de maladies neurodégénératives
US8927553B2 (en) 2009-08-10 2015-01-06 Daljit Singh Dhanoa Deuterium-enriched alkyl sulfonamides and uses thereof
US8906873B2 (en) 2009-09-11 2014-12-09 Isis Pharmaceuticals, Inc. Modulation of huntingtin expression
EP2479182B8 (fr) 2009-09-16 2016-07-13 Wave Life Sciences Japan, Inc. Nouveau groupe protecteur pour synthétiser de l'arn et dérivé de celui-ci
DK2519632T3 (en) 2009-12-31 2018-07-23 Curna Inc TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2- (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPTION TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3)
JP6006120B2 (ja) 2010-02-08 2016-10-12 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． 対立遺伝子多様体の選択的低減
JP6018506B2 (ja) 2010-02-08 2016-11-02 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． 対立遺伝子多様体の選択的低減
JP2011184318A (ja) 2010-03-05 2011-09-22 Univ Of Tokyo リボヌクレシドｈ−ボラノホスホネート
WO2011108682A1 (fr) 2010-03-05 2011-09-09 国立大学法人 東京大学 Procédé de fabrication de ribonucléoside phosphorothioate
MX342764B (es) 2010-04-19 2016-10-12 Nlife Therapeutics S L Composiciones y metodos para la distribucion selectiva de moleculas de oligonucleotidos a tipos especificos de neuronas.
AU2011291582A1 (en) * 2010-08-20 2013-03-07 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
ES2908978T3 (es) 2010-08-31 2022-05-04 Sirna Therapeutics Inc Nuevas entidades químicas simples y métodos para la administración de oligonucleótidos
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2647644B1 (fr) 2010-11-30 2020-09-09 Wave Life Sciences Japan, Inc. Arn modifié par 2'-o
EP2673361B1 (fr) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Composés oligomères comprenant des nucléotides bicycliques et leurs utilisations
WO2012109667A1 (fr) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Composés thérapeutiques
RU2013151301A (ru) 2011-04-20 2015-05-27 Ларри Дж. СМИТ Способы и композиции для модулирования экспрессии генов с использованием компонентов, которые обладают способностью к самосборке в клетках и обусловливают рнкi-активность
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
EP2742136B1 (fr) 2011-08-11 2017-09-27 Ionis Pharmaceuticals, Inc. Composés oligomères à brèche comprenant des désoxyribonucléosides modifiés en 5' dans la brèche, et leurs utilisations
ES2832531T3 (es) 2011-11-30 2021-06-10 Sarepta Therapeutics Inc Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones
EP4043039A1 (fr) * 2012-01-27 2022-08-17 BioMarin Technologies B.V. Oligonucléotides de modulation arn ayant des caractéristiques améliorées pour le traitement de la dystrophie musculaire de duchenne et becker
WO2014010718A1 (fr) 2012-07-13 2014-01-16 株式会社新日本科学 Adjuvant d'acide nucléique chiral
EP2872147B1 (fr) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Procede de fabrication d'oligonucleotides chiraux
RU2693381C2 (ru) 2012-07-13 2019-07-02 Уэйв Лайф Сайенсес Лтд. Асимметричная вспомогательная группа
US20150275208A1 (en) 2012-10-12 2015-10-01 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
AU2013336582A1 (en) 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types
ES2967530T3 (es) 2012-10-26 2024-04-30 Palomo Ltd Composiciones y métodos para el tratamiento de la enfermedad de Parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas
RU2018108405A (ru) 2013-01-30 2019-02-26 Ф. Хоффманн-Ля Рош Аг Конъюгаты углевода и lna-олигонуклеотида
ES2817050T3 (es) 2013-02-04 2021-04-06 Ionis Pharmaceuticals Inc Compuestos antisentido selectivos y usos de los mismos
DK2991656T3 (da) * 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
BR112016007255A2 (pt) * 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
WO2015070212A1 (fr) 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Méthodes et compositions pour traiter la maladie de huntington
EP3095459A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
CA2936712A1 (fr) 2014-01-16 2015-07-23 Meena Conception chirale
EP3137476B1 (fr) 2014-04-28 2019-10-09 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés par liaison
EP3139966B1 (fr) 2014-05-08 2020-11-18 Sangamo Therapeutics, Inc. Compositions pour utilisation dans le traitement de la maladie de huntington
WO2015179525A1 (fr) 2014-05-20 2015-11-26 University Of Iowa Research Foundation Composés thérapeutiques pour la maladie de huntington
US20160017327A1 (en) 2014-07-11 2016-01-21 The Johns Hopkins University Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity
EP3169693B1 (fr) 2014-07-16 2022-03-09 ModernaTX, Inc. Polynucléotides chimériques
EP3221329A1 (fr) 2014-11-19 2017-09-27 Roche Innovation Center Copenhagen A/S Oligonucléotides de type gapmères de lna comprenant des liaisons phosphorothioate chirales
CN107208092B (zh) 2014-12-16 2021-09-10 罗氏创新中心哥本哈根有限公司 手性毒性筛选方法
EP3237618B1 (fr) 2014-12-24 2019-05-22 uniQure IP B.V. Suppression du gène huntingtine induit par l'arni
EP3254104B1 (fr) 2015-02-04 2021-08-25 Bristol-Myers Squibb Company Procédés de sélection de molécules thérapeutiques
WO2016127002A1 (fr) 2015-02-04 2016-08-11 Bristol-Myers Squibb Company Oligonucléotides lna à flancs alternés
MX2017010369A (es) 2015-02-10 2017-12-14 Genzyme Corp Arni variante.
CA2980337A1 (fr) 2015-04-03 2016-10-06 University Of Massachusetts Composes oligonucleotidiques pour le ciblage de l'arnm de l'hungtingtine
CR20180107A (es) 2015-07-22 2018-04-03 Wave Life Sciences Ltd Composiciones de oligonucleótidos y métodos de los mismos
JP7511326B2 (ja) 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
WO2017067970A1 (fr) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Essai de dépistage de la toxicité in vitro
CA3015823A1 (fr) 2016-03-13 2017-09-21 Wave Life Sciences Ltd. Compositions et procedes de synthese de phosphoramidite et d'oligonucleotides
JP2019516679A (ja) 2016-05-04 2019-06-20 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． 生物活性剤に関する方法および組成物
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
EP3463386A4 (fr) 2016-06-03 2020-03-04 Wave Life Sciences Ltd. Oligonucléotides, compositions et méthodes associées
WO2018022473A1 (fr) 2016-07-25 2018-02-01 Wave Life Sciences Ltd. Phasage
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
EP3630199A4 (fr) 2017-06-02 2021-11-10 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs procédés d'utilisation
CA3065523A1 (fr) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs procedes d'utilisation
JP2020522265A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
WO2018237194A1 (fr) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Composés, compositions et procédés de synthèse
CA3072076A1 (fr) 2017-08-08 2019-02-14 Wave Life Sciences Ltd. Compositions oligonucleotidiques et methodes associees
KR20200052369A (ko) 2017-09-18 2020-05-14 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 제조 기술
WO2019075357A1 (fr) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
AU2019252680B2 (en) 2018-04-12 2025-11-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP7427608B2 (ja) 2018-05-11 2024-02-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
WO2020118246A1 (fr) 2018-12-06 2020-06-11 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
KR20210121199A (ko) 2019-02-01 2021-10-07 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
JP2022525541A (ja) 2019-03-20 2022-05-17 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド調製に有用な技術
JPWO2020196662A1 (fr) 2019-03-25 2020-10-01
BR112021021203A2 (pt) 2019-04-25 2022-01-04 Wave Life Sciences Ltd Oligonucleotídeo; composição de oligonucleotídeo quiralmente controlada; composição farmacêutica; método para prevenir, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a e/ou prevenir, atrasar o início, desenvolvimento e/ou progresso, e/ou tratar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para aumentar o nível de salto de um éxon prejudicial em uma transcrição de gene ush2a ou seu produto de gene em uma célula; método para prevenir, atrasar o início ou progressão de, tratar ou melhorar uma afecção, distúrbio ou doença relacionada a ush2a em um indivíduo suscetível à mesma ou que sofre da mesma; método para saltar um éxon prejudicial 13 em um alelo de ush2a em um indivíduo; método para produzir ou aumentar o nível de um éxon de proteína ush2a com salto 13 em um sistema; e composto, oligonucleotídeo, composição ou método
CA3137741A1 (fr) 2019-04-25 2020-10-29 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs methodes d'utilisation
US20220145300A1 (en) 2019-05-09 2022-05-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US20230220384A1 (en) 2019-10-06 2023-07-13 Prashant Monian Oligonucleotide compositions and methods of use thereof
CN114585370A (zh) 2019-10-06 2022-06-03 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
US20230295619A1 (en) 2020-03-01 2023-09-21 Abbie Madeline Maguire Oligonucleotide compositions and methods thereof
CN115885042A (zh) 2020-05-22 2023-03-31 波涛生命科学有限公司 双链寡核苷酸组合物及其相关方法
WO2021237223A1 (fr) 2020-05-22 2021-11-25 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
EP4200616A1 (fr) 2020-08-24 2023-06-28 Wave Life Sciences Ltd. Dosages immunologiques pour détecter une protéine de huntingtine de type sauvage et procédés de traitement utilisant de tels dosages immunologiques
WO2022046667A1 (fr) 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cellules et animaux non humains modifiés pour exprimer adar1 et leurs utilisations
EP4217363A4 (fr) 2020-09-24 2024-09-25 Mayo Foundation for Medical Education and Research Plates-formes de criblage
CN116507724A (zh) 2020-11-08 2023-07-28 波涛生命科学有限公司 寡核苷酸组合物及其方法
MX2023005326A (es) 2020-11-08 2023-07-31 Wave Life Sciences Ltd Composiciones de oligonucleótidos y métodos de las mismas.
US20250135036A1 (en) 2021-09-26 2025-05-01 Wave Life Sciences Ltd. Compositions for editing mecp2 transcripts and methods thereof
IL311625A (en) 2021-09-26 2024-05-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
WO2023075766A1 (fr) 2021-10-27 2023-05-04 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs méthodes d'utilisation
EP4422645A4 (fr) 2021-10-27 2025-12-03 Wave Life Sciences Ltd Compositions oligonucléotidiques et leurs procédés d'utilisation
CN118660876A (zh) 2022-02-11 2024-09-17 波涛生命科学有限公司 手性化合物的立体选择性技术
CA3244794A1 (fr) 2022-03-02 2023-09-07 Wave Life Sciences Ltd. Compositions oligonucléotidiques et procédés associé permettant le saut d'exon
AU2023253657A1 (en) 2022-04-15 2024-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods relating thereto
CN119452084A (zh) 2022-05-12 2025-02-14 波涛生命科学有限公司 寡核苷酸组合物及其方法
KR20250051686A (ko) 2022-08-11 2025-04-17 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
WO2025030155A1 (fr) 2023-08-03 2025-02-06 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés

Also Published As

Publication number Publication date
US12486505B2 (en) 2025-12-02
SV2018005619A (es) 2018-07-20
MX2025000894A (es) 2025-03-07
AU2025220872A1 (en) 2025-10-16
US20220195429A1 (en) 2022-06-23
CO2018001484A2 (es) 2018-07-19
WO2017015555A1 (fr) 2017-01-26
CN108135921B (zh) 2023-10-17
WO2017015555A8 (fr) 2017-06-22
TW201707711A (zh) 2017-03-01
EP3325017A1 (fr) 2018-05-30
US20200080083A1 (en) 2020-03-12
PE20181174A1 (es) 2018-07-20
NZ778379A (en) 2025-05-02
RU2017146349A (ru) 2019-08-22
JP7441816B2 (ja) 2024-03-01
US11634710B2 (en) 2023-04-25
US20200299692A1 (en) 2020-09-24
RU2017146349A3 (fr) 2020-01-14
KR20180028516A (ko) 2018-03-16
US20240150756A1 (en) 2024-05-09
US20180216108A1 (en) 2018-08-02
EP3324978A4 (fr) 2019-01-30
CN108025089A (zh) 2018-05-11
CN117965536A (zh) 2024-05-03
JP2018525357A (ja) 2018-09-06
WO2017015575A1 (fr) 2017-01-26
US10479995B2 (en) 2019-11-19
AU2016297155A1 (en) 2018-01-18
JP7606954B2 (ja) 2024-12-26
IL256603A (en) 2018-02-28
CA2989682A1 (fr) 2017-01-26
JP2024028927A (ja) 2024-03-05
NZ738490A (en) 2025-05-02
CR20180107A (es) 2018-04-03
HK1255369A1 (zh) 2019-08-16
HK1255370A1 (zh) 2019-08-16
AU2016297155B2 (en) 2022-11-17
SG10201912900XA (en) 2020-02-27
JP2018524013A (ja) 2018-08-30
ECSP18010463A (es) 2018-04-30
JP7296724B2 (ja) 2023-06-23
MX2018000796A (es) 2018-03-23
NI201800009A (es) 2018-06-12
IL320434A (en) 2025-06-01
BR112017028636A2 (pt) 2019-09-17
JP7818563B2 (ja) 2026-02-20
EP4344744A3 (fr) 2024-07-17
JP2022009217A (ja) 2022-01-14
PH12018500145A1 (en) 2018-07-23
US20180216107A1 (en) 2018-08-02
CN108135921A (zh) 2018-06-08
EP3324978A1 (fr) 2018-05-30
JP2022000023A (ja) 2022-01-04
AU2023200807A1 (en) 2023-05-11
EP3325017A4 (fr) 2019-06-19
JP2025038091A (ja) 2025-03-18
CL2018000145A1 (es) 2018-08-10
EP4344744A2 (fr) 2024-04-03

Similar Documents

Publication Publication Date Title
MA43072A (fr) Compositions d'oligonucléotides et procédés associés
WO2015107425A8 (fr) Conception chirale
EA201492122A1 (ru) Композиции и способы для модулирования экспрессии utrn
CY1120782T1 (el) Συνθεσεις για την αντιμετωπιση ή την προληψη νοσου που σχετιζεται με οξαλικα
MA65895B1 (fr) Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire
EA201492120A1 (ru) Композиции и способы для модулирования экспрессии atp2a2
EA201492116A1 (ru) Композиции и способы для модулирования экспрессии mecp2
CR10355A (es) Antagonistas de los piridil amida de los canales de calcio de tipo t
EA201492117A1 (ru) Композиции и способы для модулирования экспрессии bdnf
EP3608403A3 (fr) Composition détergente comprenant des variantes de subtilase
MX2011013176A (es) Motivos de modificación química para inhibidores y miméticos del miarn.
EA201401193A1 (ru) Триарильные соединения и композиции, их содержащие
PA8634101A1 (es) DERIVADOS DE TETRAAZABENZO[e] AZULENO Y SUS ANALOGOS
EP1963536A4 (fr) Polymerases permettant d' incorporer des analogues de nucleotides
EA201492118A1 (ru) Композиции и способы для модулирования экспрессии pten
PE20050081A1 (es) Nuevas imidazopirazinas como inhibidores de cinasas dependientes de ciclinas
WO2007147110A3 (fr) Initiation régulée d'extension d'amorce
EA200900260A1 (ru) Гранулы и орально дезинтегрируемые таблетки, содержащие оксикодон
MX388737B (es) Oligonucleótidos terapéuticos.
FR2998580B1 (fr) Marqueurs moleculaires et methodes pour l'identification des genotypes de palmier dattier
CL2017000382A1 (es) Compuestos bicíclicos sustituidos
TW200702382A (en) Use of amphiphilic block copolymers for producing polymer blends
ATE509945T1 (de) Chirale mit phosphonsäureester- oder phosphonsäure- substituierte verbindungen
AR105488A1 (es) Composiciones de oligonucleótidos y métodos de los mismos
ATE522664T1 (de) Bausatz zur erstellung von pflasterflächen