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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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) JP7296724B2 (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) HK1255369A1 (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 (157)

* 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
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US10273474B2 (en) 2012-03-30 2019-04-30 Washington University Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
WO2015108048A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
WO2015108047A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
EP3234131B1 (fr) * 2014-12-16 2020-04-22 Roche Innovation Center Copenhagen A/S Procédé de dépistage de la toxicité chirale
CA2968336C (fr) 2014-12-17 2021-11-23 Proqr Therapeutics Ii B.V. Construction pour l'edition en site d'un nucleotide d'adenosine dans l'arn cible
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
WO2016161388A1 (fr) 2015-04-03 2016-10-06 University Of Massachusetts Petit arn interférent asymétrique entièrement stabilisé
CN107980062B (zh) 2015-04-03 2022-11-25 马萨诸塞大学 用于靶向亨廷汀mRNA的寡核苷酸化合物
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
EP3334499A4 (fr) * 2015-08-14 2019-04-17 University of Massachusetts Conjugués bioactifs pour l'administration d'oligonucléotides
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
CN110946865B (zh) 2015-12-10 2024-01-26 Ptc医疗公司 用于治疗亨廷顿病的方法
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
AU2017234150B2 (en) 2016-03-13 2021-09-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
WO2017210647A1 (fr) 2016-06-03 2017-12-07 Wave Life Sciences Ltd. Oligonucléotides, compositions et méthodes associées
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
WO2018031933A2 (fr) 2016-08-12 2018-02-15 University Of Massachusetts Oligonucléotides conjugués
EP3507366B1 (fr) 2016-09-01 2020-10-07 ProQR Therapeutics II B.V. Oligonucléotides d'édition d'arn simple brin chimiquement modifiés
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
JP7296882B2 (ja) 2016-11-23 2023-06-23 ウェイブ ライフ サイエンシズ リミテッド ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
HRP20240705T1 (hr) 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
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
EP3589751A4 (fr) 2017-03-03 2021-11-17 The Regents of The University of California Ciblage arn de mutations par l'intermédiaire d'arnt suppresseurs et de désaminases
WO2018223056A1 (fr) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs procédés d'utilisation
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP3634953B1 (fr) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Composés permettant de traiter la maladie de huntington
WO2018237194A1 (fr) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Composés, compositions et procédés de synthèse
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111163838B (zh) 2017-06-28 2023-03-28 Ptc医疗公司 用于治疗亨廷顿氏病的方法
CA3072076A1 (fr) * 2017-08-08 2019-02-14 Wave Life Sciences Ltd. Compositions oligonucleotidiques et methodes associees
SG11202000276YA (en) 2017-09-18 2020-04-29 Wave Life Sciences Ltd Technologies for oligonucleotide preparation
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3687577A1 (fr) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Polythérapies pour traiter une dystrophie musculaire
EP3687519A1 (fr) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Polythérapies pour traiter une dystrophie musculaire
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
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
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
MX2020007433A (es) 2018-01-12 2020-09-14 Bristol Myers Squibb Co Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos.
MX2020007369A (es) 2018-01-15 2020-10-28 Ionis Pharmaceuticals Inc Moduladores de la expresion de dnm2.
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
BR112020020670A2 (pt) * 2018-04-12 2021-03-02 Wave Life Sciences Ltd. composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
SG11202010012PA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing fxi expression
WO2019217708A1 (fr) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Composés et procédés pour réduire l'expression d'atxn3
TWI844541B (zh) * 2018-05-11 2024-06-11 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其使用方法
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
AU2019287635B2 (en) * 2018-06-14 2026-02-12 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
PL3814357T3 (pl) 2018-06-27 2024-09-16 Ptc Therapeutics, Inc. Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona
KR20210038845A (ko) 2018-06-27 2021-04-08 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료를 위한 헤테로아릴 화합물
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
CA3106986A1 (fr) 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Composes et methodes permettant de reduire l'expression d'atxn2
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
AU2019316640B2 (en) 2018-08-10 2026-03-12 University Of Massachusetts Modified oligonucleotides targeting SNPs
EP3840759A4 (fr) 2018-08-23 2022-06-01 University Of Massachusetts Oligonucléotides complètement stabilisés riches en o-méthyle
TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
US20210380976A1 (en) * 2018-10-05 2021-12-09 Ionis Pharmaceuticals, Inc. Chirally enriched oligomeric compounds
AU2019380940A1 (en) 2018-11-15 2021-06-03 Ionis Pharmaceuticals, Inc. Modulators of IRF5 expression
EP3884053A4 (fr) 2018-11-21 2023-02-01 Ionis Pharmaceuticals, Inc. Composés et méthodes permettant de réduire l'expression de prion
EP3894559A4 (fr) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. Méthodes thérapeutiques pour les maladies par expansion de répétitions trinucléotidiques associés à une activité mlh3
SG11202104960PA (en) 2018-12-13 2021-06-29 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
SG11202106378VA (en) 2018-12-21 2021-07-29 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
AU2020207935A1 (en) 2019-01-18 2021-08-26 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
SG11202107318YA (en) * 2019-02-01 2021-08-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
MX2021010152A (es) 2019-02-27 2021-09-14 Ionis Pharmaceuticals Inc Moduladores de la expresion 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
MD3947684T2 (ro) 2019-03-29 2025-10-31 Ionis Pharmaceuticals Inc Compuși și metode pentru modularea UBE3A-ATS
JP2022528725A (ja) 2019-04-18 2022-06-15 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治療するための組成物
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
US20230145795A1 (en) * 2019-04-25 2023-05-11 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN114245794B (zh) 2019-05-13 2024-09-13 Ptc医疗公司 用于治疗亨廷顿氏病的化合物
US20220243203A1 (en) 2019-05-28 2022-08-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fus expression
EP4660307A2 (fr) 2019-07-26 2025-12-10 Ionis Pharmaceuticals, Inc. Composés et procédés de modulation de gfap
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
JP7757277B2 (ja) 2019-10-14 2025-10-21 アストラゼネカ・アクチエボラーグ Ｐｎｐｌａ３発現のモジュレーター
US20240216369A1 (en) 2019-11-01 2024-07-04 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
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
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
US20230203486A1 (en) * 2020-03-30 2023-06-29 Alnylam Pharmaceuticals, Inc. MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
JP2023522957A (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
IL298406A (en) 2020-05-22 2023-01-01 Wave Life Sciences Ltd Double stranded oligonucleotide compositions and methods relating thereto
US12534724B2 (en) 2020-05-26 2026-01-27 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
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
MX2023005736A (es) 2020-11-18 2023-05-25 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la expresion de angiotensinogeno.
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
PE20240696A1 (es) 2021-03-08 2024-04-10 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina
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
KR20240037226A (ko) 2021-06-22 2024-03-21 아큐라스템 인코포레이티드 Pikfyve 안티센스 올리고뉴클레오티드
IL309334A (en) 2021-06-23 2024-02-01 Univ Massachusetts Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders
KR20240095157A (ko) 2021-07-21 2024-06-25 아큐라스템 인코포레이티드 Unc13a 안티센스 올리고뉴클레오타이드
AU2022358322A1 (en) 2021-09-30 2024-05-16 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
IL311518A (en) 2021-10-01 2024-05-01 Adarx Pharmaceuticals Inc Preparations that modulate perkalkerine and methods of using them
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
KR20250007559A (ko) 2022-04-28 2025-01-14 아큐라스템 인코포레이티드 Syf2 안티센스 올리고뉴클레오타이드
US20260027237A1 (en) 2022-07-15 2026-01-29 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
EP4555086A1 (fr) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucléotides pour édition d'arn médiée par adar et leur utilisation
CN120476205A (zh) * 2022-08-11 2025-08-12 波涛生命科学有限公司 寡核苷酸组合物及其方法
EP4590311A2 (fr) 2022-09-21 2025-07-30 Sarepta Therapeutics, Inc. Efficacité du saut d'exon médié par un oligonucléotide antisens dans le traitement de la dmd
US12152052B2 (en) 2022-09-23 2024-11-26 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing MECP2 expression
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
JP2025536808A (ja) 2022-11-24 2025-11-07 プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップ 遺伝性ｈｆｅヘモクロマトーシスの治療のためのアンチセンスオリゴヌクレオチド
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
JP2025540146A (ja) 2022-12-09 2025-12-11 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 心血管疾患の治療のためのアンチセンスオリゴヌクレオチド
WO2024155986A2 (fr) 2023-01-20 2024-07-25 AcuraStem Incorporated Oligonucléotides antisens unc13a
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
IL322500A (en) 2023-01-31 2025-09-01 AcuraStem Incorporated syf2 molecular-readable oligonucleotides
AU2024217638A1 (en) 2023-02-06 2025-07-31 AcuraStem Incorporated Pikfyve antisense oligonucleotides
EP4669753A1 (fr) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire athérosclérotique
WO2024206175A1 (fr) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles neurologiques
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
CN121002181A (zh) 2023-03-27 2025-11-21 ProQR治疗上市公司Ⅱ 用于治疗肝脏疾病的反义寡核苷酸
EP4698652A2 (fr) 2023-04-20 2026-02-25 Adarx Pharmaceuticals, Inc. Compositions de modulation de mapt et leurs procédés d'utilisation
WO2024227040A1 (fr) 2023-04-27 2024-10-31 Sarepta Therapeutics, Inc. Oligomères antisens pour le traitement d'une néphropathie chronique
KR20260021635A (ko) 2023-05-12 2026-02-13 아다르엑스 파마슈티컬스, 인크. Nmda 리간드 접합 화합물 및 이의 용도
CN121335980A (zh) 2023-05-26 2026-01-13 阿达尔克斯制药有限公司 Sod1调节组合物及其使用方法
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
TW202506137A (zh) 2023-06-20 2025-02-16 美商雅迪克斯製藥公司 Ｌｒｒｋ２調節組合物及其使用方法
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
US20250243487A1 (en) 2023-09-14 2025-07-31 Ionis Pharmaceuticals, Inc. Compounds and Methods for Reducing APOCIII Expression
WO2025064821A2 (fr) 2023-09-21 2025-03-27 Ionis Pharmaceuticals, Inc. Composés et procédés d'inhibition de lpa
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

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
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 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
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
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
EP0366685B1 (fr) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Derives de nucleosides
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
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5262530A (en) 1988-12-21 1993-11-16 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
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
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
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
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
EP0544824B1 (fr) 1990-07-27 1997-06-11 Isis Pharmaceuticals, Inc. Oligonucleotides, a pyrimidine modifiee et resistants a la nuclease, detectant et modulant l'expression de genes
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
EP1044987B1 (fr) 1991-12-24 2006-02-15 Isis Pharmaceuticals, Inc. Oligonucléotides modifiés en 2' à ouverture
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
JPH09507836A (ja) 1993-11-16 1997-08-12 ジンタ・インコーポレイテッド 非ホスホネート・ヌクレオシジル間結合と混合したキラリティー的に純粋なホスホネート・ヌクレオシジル間結合を有する合成オリゴマー
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
ATE327244T1 (de) 1995-03-06 2006-06-15 Isis Pharmaceuticals Inc Verfahren zur synthese von 2'-0-substituierten pyrimidinen und oligomere davon
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
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
CN101298481A (zh) * 1998-02-20 2008-11-05 吉宁特有限公司 补体活化的抑制剂
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6242589B1 (en) * 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US6867294B1 (en) * 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate 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)
WO2003085110A2 (fr) 2002-04-05 2003-10-16 Santaris Pharma A/S Composes oligomeres servant a moduler l'expression du facteur hif-1$g(a)
US8101348B2 (en) 2002-07-10 2012-01-24 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. RNA-interference by single-stranded RNA molecules
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
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
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
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
JP4616175B2 (ja) 2003-09-02 2011-01-19 株式会社キラルジェン ５’−ホスフィチル化モノマーおよびｈ−ホスホネートオリゴヌクレオチド誘導体の製造方法
JP4580870B2 (ja) 2003-09-02 2010-11-17 株式会社キラルジェン リボヌクレオチド又はリボヌクレオチド誘導体の製造方法
ES2485848T3 (es) 2003-09-12 2014-08-14 University Of Massachusetts ARN de interferencia para el tratamiento de trastornos relacionados con la ganancia de función
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
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
EP2397563A3 (fr) 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
WO2006066260A2 (fr) 2004-12-17 2006-06-22 Thiosense, Inc. Compositions de monomeres et oligomeres de phosphore chiraux et procedes de production
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
US20060257912A1 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
EP2316967A1 (fr) 2005-06-28 2011-05-04 Medtronic, Inc. Méthodes et séquences pour supprimer préférentiellement l'expression du gène huntingtin muté.
WO2007028065A2 (fr) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Composes oligomeriques chimeriques pour la modulation de l'epissage
CN101365801B (zh) 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
JP5425474B2 (ja) 2006-01-26 2014-02-26 アイシス ファーマシューティカルズ， インコーポレーテッド ハンチンチン対する、組成物及びその使用
CA2640171C (fr) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. Analogues d'acides nucleiques bicycliques modifies en position 6
CA3044969A1 (fr) 2006-05-05 2007-12-21 Ionis Pharmaceuticals, Inc. Composes et procedes de modulation de l'expression genique
JP5441688B2 (ja) 2006-05-11 2014-03-12 アイシス ファーマシューティカルズ， インコーポレーテッド ５’修飾二環式核酸類似体
ES2389112T3 (es) 2006-05-31 2012-10-23 Toray Industries, Inc. Oligonucleótido inmunoestimulador y aplicación farmacéutica del mismo
DK2092065T4 (da) * 2006-10-18 2019-10-21 Ionis Pharmaceuticals Inc Antisense-forbindelser
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
DE602007005629D1 (de) 2007-06-18 2010-05-12 Commissariat Energie Atomique Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington
JP5737937B2 (ja) 2007-07-09 2015-06-17 イデラ ファーマシューティカルズ インコーポレイテッドＩｄｅｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 安定化免疫調節ｒｎａ（ｓｉｍｒａ）化合物
EP2224912B1 (fr) * 2008-01-02 2016-05-11 TEKMIRA Pharmaceuticals Corporation Compositions et procédés améliorés pour la délivrance d'acides nucléiques
US20110105587A1 (en) 2008-02-04 2011-05-05 David Frederik Fishcher Target sequences and methods to identify the same, useful in treatment of neurodegenerative diseases
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
KR20110044983A (ko) * 2008-07-10 2011-05-03 아카데미쉬 지에켄후이스 비즈 드 유니버시테이트 반 암스테르담 보체 길항제 및 그 용도
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
WO2010091301A1 (fr) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Composés oligomères et 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
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
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
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
US8927553B2 (en) 2009-08-10 2015-01-06 Daljit Singh Dhanoa Deuterium-enriched alkyl sulfonamides and uses thereof
NZ599032A (en) 2009-09-11 2014-09-26 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
CN102791862B (zh) 2009-12-31 2017-04-05 库尔纳公司 通过抑制胰岛素受体底物2(irs2)和转录因子e3(tfe3)的天然反义转录物而治疗irs2相关疾病
WO2011097644A2 (fr) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Réduction sélective de variants alléliques
EP3561060A1 (fr) 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Réduction sélective de variants alléliques
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
JP5847700B2 (ja) 2010-03-05 2016-01-27 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ リボヌクレオシドホスホロチオエートの製造方法
KR20130103662A (ko) 2010-04-19 2013-09-24 엔라이프 떼라퓨틱스, 에스.엘. 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법
EA201390145A1 (ru) * 2010-08-20 2013-11-29 Серулин Фарма Инк. Конъюгаты, частицы, композиции и связанные с ними способы
EP2611927B1 (fr) 2010-08-31 2018-08-01 Sirna Therapeutics, Inc. Nouvelles entités chimiques simples et procédés pour l'administration d'oligonucléotides
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012073857A1 (fr) 2010-11-30 2012-06-07 株式会社キラルジェン Arn modifié par 2'-o
EP3467109A1 (fr) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Composés oligomères comprenant des nucléotides bicycliques et leurs utilisations
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
US20150038549A1 (en) * 2011-04-20 2015-02-05 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Components That Self Assemble in Cells and Produce RNAi Activity
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP2742056B2 (fr) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Composés antisens sélectifs et utilisations de ceux-ci
EP2785729B1 (fr) 2011-11-30 2020-11-04 Sarepta Therapeutics, Inc. Oligonucléotides pour le traitement de maladies à répétition étendue
ES2907250T3 (es) * 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
KR20240148947A (ko) * 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500243WA (en) * 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
CA2887884A1 (fr) 2012-10-12 2014-04-17 Isis Pharmaceuticals, Inc. Composes anti-sens selectifs et leurs utilisations
WO2014064258A1 (fr) 2012-10-26 2014-05-01 Nlife Therapeutics, S.L. Compositions et méthodes d'administration sélective de molécules d'oligonucléotides à des types de cellules
HK1214179A1 (zh) 2012-10-26 2016-07-22 恩莱弗医疗有限公司 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法
DK2951305T3 (en) 2013-01-30 2018-10-29 Hoffmann La Roche LNA oligonucleotide KULHYDRATKONJUGATER
WO2014121287A2 (fr) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Composés antisens sélectifs et leurs utilisations
PE20152002A1 (es) * 2013-05-01 2016-01-21 Isis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de ttr y vhb
EP3052521A1 (fr) * 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucléotides codant pour un récepteur de lipoprotéines de faible densité
NZ719477A (en) 2013-11-11 2022-05-27 Sangamo Therapeutics Inc Methods and compositions for treating huntington’s disease
EP3095460A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral ayant une activité anti-allergique, et agent anti-allergique
WO2015108047A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
WO2015108048A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
EP3647318B1 (fr) 2014-04-28 2021-06-30 Ionis Pharmaceuticals, Inc. Composés oligomères modifiés de liaison
MX378789B (es) 2014-05-08 2025-03-11 Sangamo Biosciences Inc Métodos y composiciones para tratar la enfermedad de huntington.
JP6663859B2 (ja) 2014-05-20 2020-03-13 ユニバーシティー オブ アイオワ リサーチ ファウンデーション ハンチントン病の治療化合物
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
JP2017536119A (ja) 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス キラルホスホロチオエート連結を含むｌｎａギャップマーオリゴヌクレオチド
EP3234131B1 (fr) 2014-12-16 2020-04-22 Roche Innovation Center Copenhagen A/S Procédé de dépistage de la toxicité chirale
SMT201900347T1 (it) 2014-12-24 2019-07-11 Uniqure Ip Bv Soppressione del gene huntingtina indotta da rnai
US11761951B2 (en) 2015-02-04 2023-09-19 Bristol-Myers Squibb Company Methods of selecting therapeutic molecules
EP3253871A1 (fr) 2015-02-04 2017-12-13 Bristol-Myers Squibb Company Oligonucléotides lna à flancs alternés
SG11201706444TA (en) 2015-02-10 2017-09-28 Genzyme Corp VARIANT RNAi
CN107980062B (zh) 2015-04-03 2022-11-25 马萨诸塞大学 用于靶向亨廷汀mRNA的寡核苷酸化合物
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
WO2017067970A1 (fr) 2015-10-22 2017-04-27 Roche Innovation Center Copenhagen A/S Essai de dépistage de la toxicité in vitro
AU2017234150B2 (en) 2016-03-13 2021-09-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
WO2017210647A1 (fr) 2016-06-03 2017-12-07 Wave Life Sciences Ltd. Oligonucléotides, compositions et méthodes associées
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
JP7296882B2 (ja) 2016-11-23 2023-06-23 ウェイブ ライフ サイエンシズ リミテッド ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法
WO2018223056A1 (fr) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs procédés d'utilisation
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN120330183A (zh) 2017-06-02 2025-07-18 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
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
SG11202000276YA (en) 2017-09-18 2020-04-29 Wave Life Sciences Ltd Technologies for oligonucleotide preparation
WO2019075357A1 (fr) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
BR112020020670A2 (pt) 2018-04-12 2021-03-02 Wave Life Sciences Ltd. composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
TWI844541B (zh) 2018-05-11 2024-06-11 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其使用方法
AU2019392928A1 (en) 2018-12-06 2021-06-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
SG11202107318YA (en) 2019-02-01 2021-08-30 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
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
US20230145795A1 (en) 2019-04-25 2023-05-11 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN114502177A (zh) 2019-05-09 2022-05-13 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
CA3156176A1 (fr) 2019-10-06 2021-04-15 Wave Life Sciences Ltd. Compositions oligonucleotidiques et leurs procedes d'utilisation
BR112022006205A2 (pt) 2019-10-06 2022-07-19 Wave Life Sciences Ltd Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método
WO2021178237A2 (fr) 2020-03-01 2021-09-10 Wave Life Sciences Ltd. Compositions oligonucléotidiques et méthodes associées
AU2021277407A1 (en) 2020-05-22 2022-11-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
IL298406A (en) 2020-05-22 2023-01-01 Wave Life Sciences Ltd Double stranded oligonucleotide compositions and methods relating thereto
WO2022046667A1 (fr) 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cellules et animaux non humains modifiés pour exprimer adar1 et leurs utilisations
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
WO2022067023A1 (fr) 2020-09-24 2022-03-31 Mayo Foundation For Medical Education And Research Plates-formes de criblage
CA3197311A1 (fr) 2020-11-08 2022-05-12 Wave Life Sciences Ltd. Compositions d'oligonucleotides et procedes associes
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
WO2023049477A2 (fr) 2021-09-26 2023-03-30 Wave Life Sciences Ltd. Compositions pour l'édition de transcrits mecp2 et procédés associés
KR20240063964A (ko) 2021-09-26 2024-05-10 웨이브 라이프 사이언시스 리미티드 올리고뉴클레오티드 조성물 및 이의 방법
WO2023075766A1 (fr) 2021-10-27 2023-05-04 Wave Life Sciences Ltd. Compositions d'oligonucléotides et leurs méthodes d'utilisation
WO2023076352A2 (fr) 2021-10-27 2023-05-04 Wave Life Sciences Ltd. Compositions oligonucléotidiques et leurs procédés d'utilisation
WO2023154528A1 (fr) 2022-02-11 2023-08-17 Wave Life Sciences Ltd. Technologies stéréosélectives pour composés chiraux
AU2023227808A1 (en) 2022-03-02 2024-09-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof for exon skipping
EP4507700A2 (fr) 2022-04-15 2025-02-19 Wave Life Sciences Ltd. Compositions oligonucléotidiques et procédés associés
WO2023220440A1 (fr) 2022-05-12 2023-11-16 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
CN120476205A (zh) 2022-08-11 2025-08-12 波涛生命科学有限公司 寡核苷酸组合物及其方法
AU2024315761A1 (en) 2023-08-03 2026-02-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

Also Published As

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

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
CR11045A (es) Mezclas activas pesticidas que comprenden compuestos de aminotiazolina
EP3608403A3 (fr) Composition détergente comprenant des variantes de subtilase
MX2011013176A (es) Motivos de modificación química para inhibidores y miméticos del miarn.
PA8634101A1 (es) DERIVADOS DE TETRAAZABENZO[e] AZULENO Y SUS ANALOGOS
AR072521A1 (es) Marcadores asociados a la resistencia a la roya de la soja y metodos para su utilizacion
EA201401193A1 (ru) Триарильные соединения и композиции, их содержащие
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
MX388737B (es) Oligonucleótidos terapéuticos.
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