JP2019516679A - 生物活性剤に関する方法および組成物 - Google Patents

生物活性剤に関する方法および組成物 Download PDF

Info

Publication number
JP2019516679A
JP2019516679A JP2018557083A JP2018557083A JP2019516679A JP 2019516679 A JP2019516679 A JP 2019516679A JP 2018557083 A JP2018557083 A JP 2018557083A JP 2018557083 A JP2018557083 A JP 2018557083A JP 2019516679 A JP2019516679 A JP 2019516679A
Authority
JP
Japan
Prior art keywords
hydrochloride
muscle
composition
oligonucleotide
lipid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2018557083A
Other languages
English (en)
Japanese (ja)
Inventor
チャンドラ ヴァージェシー
チャンドラ ヴァージェシー
ジェイソン ジンシン チャン
ジェイソン ジンシン チャン
セスマダバン ディバカラメノン
セスマダバン ディバカラメノン
デビット チャールズ ドネル バトラー
デビット チャールズ ドネル バトラー
ゲンリャン ル
ゲンリャン ル
ナオキ イワモト
ナオキ イワモト
ハイリン ヤン
ハイリン ヤン
マリア デイビッド フランク−カメネスキー
マリア デイビッド フランク−カメネスキー
スブラマニアン マラッパン
スブラマニアン マラッパン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Wave Life Sciences Pte Ltd
Original Assignee
Wave Life Sciences Pte 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 Pte Ltd filed Critical Wave Life Sciences Pte Ltd
Publication of JP2019516679A publication Critical patent/JP2019516679A/ja
Priority to JP2023041621A priority Critical patent/JP2023078293A/ja
Priority to JP2025064806A priority patent/JP2025108550A/ja
Pending legal-status Critical Current

Links

Images

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Pain & Pain Management (AREA)
  • Obesity (AREA)
  • Rheumatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2018557083A 2016-05-04 2017-05-03 生物活性剤に関する方法および組成物 Pending JP2019516679A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023041621A JP2023078293A (ja) 2016-05-04 2023-03-16 生物活性剤に関する方法および組成物
JP2025064806A JP2025108550A (ja) 2016-05-04 2025-04-10 生物活性剤に関する方法および組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662331961P 2016-05-04 2016-05-04
US62/331,961 2016-05-04
US201662405810P 2016-10-07 2016-10-07
US62/405,810 2016-10-07
PCT/US2017/030777 WO2017192679A1 (en) 2016-05-04 2017-05-03 Methods and compositions of biologically active agents

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023041621A Division JP2023078293A (ja) 2016-05-04 2023-03-16 生物活性剤に関する方法および組成物

Publications (1)

Publication Number Publication Date
JP2019516679A true JP2019516679A (ja) 2019-06-20

Family

ID=60203379

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018557083A Pending JP2019516679A (ja) 2016-05-04 2017-05-03 生物活性剤に関する方法および組成物
JP2023041621A Pending JP2023078293A (ja) 2016-05-04 2023-03-16 生物活性剤に関する方法および組成物
JP2025064806A Pending JP2025108550A (ja) 2016-05-04 2025-04-10 生物活性剤に関する方法および組成物

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2023041621A Pending JP2023078293A (ja) 2016-05-04 2023-03-16 生物活性剤に関する方法および組成物
JP2025064806A Pending JP2025108550A (ja) 2016-05-04 2025-04-10 生物活性剤に関する方法および組成物

Country Status (6)

Country Link
US (2) US20190249173A1 (OSRAM)
EP (1) EP3452597A4 (OSRAM)
JP (3) JP2019516679A (OSRAM)
CN (1) CN109462995A (OSRAM)
MA (1) MA45290A (OSRAM)
WO (1) WO2017192679A1 (OSRAM)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023503644A (ja) * 2019-11-27 2023-01-31 ディー・ティー・エックス・ファーマ・インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための化合物および方法
JP2023545041A (ja) * 2020-10-05 2023-10-26 エスキュイグレック・テラピュティク Ｄｍｄ患者におけるエクソン５１スキッピングによるジストロフィンレスキューのためのオリゴマー化合物

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
BR112014001244A2 (pt) 2011-07-19 2017-02-21 Wave Life Sciences Pte Ltd métodos para a síntese de ácidos nucléicos funcionalizados
ES2862073T3 (es) 2012-07-13 2021-10-06 Wave Life Sciences Ltd Grupo auxiliar asimétrico
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
WO2016097212A1 (en) 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
NZ781029A (en) 2015-10-09 2025-07-25 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
US11407775B2 (en) 2016-03-13 2022-08-09 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45188A (fr) 2016-06-03 2019-04-10 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
ES2837076T3 (es) 2016-09-01 2021-06-29 Proqr Therapeutics Ii Bv Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
SG10202012839TA (en) 2016-12-19 2021-01-28 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
MD3554554T2 (ro) 2016-12-19 2022-12-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
BR112019012664A2 (pt) 2016-12-19 2020-01-21 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP7557941B2 (ja) 2017-06-02 2024-09-30 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
WO2018237194A1 (en) 2017-06-21 2018-12-27 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN110996968A (zh) 2017-08-08 2020-04-10 波涛生命科学有限公司 寡核苷酸组合物及其方法
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
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
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
JP2020536057A (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 (en) 2017-10-12 2019-04-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
KR20250148679A (ko) 2017-12-06 2025-10-14 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
WO2019126179A1 (en) 2017-12-20 2019-06-27 Lunella Biotech, Inc. Targeting mitochondrial fission through mdivi-1 derivatives
AU2019266207B2 (en) 2018-05-07 2025-04-24 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
AU2019265904A1 (en) 2018-05-11 2020-11-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR102889572B1 (ko) * 2018-05-30 2025-11-24 노파르티스 아게 지질-변형된 핵산 화합물 및 방법
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
TW202020153A (zh) 2018-07-27 2020-06-01 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
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020117706A1 (en) 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with mlh3 activity
IL318474A (en) 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
SG11202106531VA (en) * 2018-12-20 2021-07-29 Aegis Therapeutics Llc Compositions, devices, and methods for the treatment of overdose and reward-based disorders
CN109799216B (zh) * 2018-12-29 2021-11-12 佛山科学技术学院 一种基于吲哚箐绿纳米的荧光oct双模成像方法和装置
JPWO2020158636A1 (ja) * 2019-01-29 2021-12-02 国立大学法人 東京大学 核酸等を細胞内へ導入する方法
CN109870583B (zh) * 2019-04-15 2020-03-06 德阳市人民医院 急性胰腺炎相关的代谢物及其应用
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
JP7186442B2 (ja) * 2019-04-22 2022-12-09 国立研究開発法人産業技術総合研究所 新規スクアレン誘導体および抗炎症剤
EP3959319A4 (en) 2019-04-25 2023-06-07 Avidity Biosciences, Inc. NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING
CA3141488A1 (en) * 2019-05-22 2020-11-26 Biovie Inc. Formulations of terlipressin
JP7627495B2 (ja) 2019-07-12 2025-02-06 学校法人東海大学 ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド
CN110527698B (zh) * 2019-08-23 2021-10-01 温氏食品集团股份有限公司 一种利用小分子化合物提高基因组定点插入效率的方法
CN111063870B (zh) * 2019-11-27 2021-09-24 深圳先进技术研究院 纳米材料及其制备方法、电极和二次电池
CN111012788B (zh) * 2019-12-12 2021-01-15 武汉职业技术学院 硝唑尼特和替唑尼特在制备抗猪繁殖与呼吸综合征病毒药物中的应用
CN111055603B (zh) * 2019-12-31 2021-11-05 安徽省粤隆印刷科技有限公司 一种防止残剩油墨过度乳化和形成残渣的包装盒印刷工艺
TW202146650A (zh) * 2020-03-01 2021-12-16 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其方法
IL296387B2 (en) 2020-03-19 2024-08-01 Avidity Biosciences Inc Preparations and methods for the treatment of facial, back and arm muscle atrophy
CN111358955B (zh) * 2020-04-01 2023-05-02 重庆理工大学 一种用于治疗脂质代谢疾病的炎症靶向的宾达利纳米粒、制备方法及其应用
CN111249298B (zh) * 2020-04-08 2021-03-12 曲阜师范大学 一种含有马杜霉素和顺铂的抗癌药物组合物
JP2023526533A (ja) 2020-05-22 2023-06-21 ウェイブ ライフ サイエンシズ リミテッド 二本鎖オリゴヌクレオチド組成物及びそれに関連する方法
IT202000012577A1 (it) * 2020-05-27 2021-11-27 Univ Degli Studi Roma La Sapienza Composizione farmaceutica per l’inibizione chimica di tgs1 come trattamento terapeutico per le telomeropatie
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
CN111789859A (zh) * 2020-07-20 2020-10-20 中国人民解放军海军军医大学 地塞米松在制备预防或缓解水母蜇伤药物中的应用
WO2022069511A1 (en) * 2020-09-30 2022-04-07 Biomarin Technologies B.V. Antisense oligonucleotides targeting the exon 51 of dystrophin gene
CN115216474B (zh) * 2021-04-15 2025-09-30 武汉大学 促进pd-l1外显子3跳跃的反义寡核苷酸及其应用
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN113520995B (zh) * 2021-08-16 2023-03-10 海南鑫开源医药科技有限公司 一种离子敏感型眼用原位凝胶、其制备方法及应用
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
WO2023044398A1 (en) * 2021-09-16 2023-03-23 Dyne Therapeutics, Inc. Dosing of muscle targeting complexes for treating dystrophinopathies
JP2024537711A (ja) 2021-09-30 2024-10-16 サレプタ セラピューティクス， インコーポレイテッド １つ以上の脱塩基ユニットを有するアンチセンスオリゴヌクレオチド
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
AU2023254846A1 (en) 2022-04-15 2024-10-10 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
EP4555085A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Chemically modified oligonucleotides for adar-mediated rna editing
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
KR20250113455A (ko) 2022-11-24 2025-07-25 프로큐알 테라퓨틱스 Ⅱ 비.브이. 유전성 hfe-혈색소침착증의 치료를 위한 안티센스 올리고뉴클레오티드
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
KR20250115388A (ko) 2022-12-09 2025-07-30 프로큐알 테라퓨틱스 Ⅱ 비.브이. 심혈관 질환 치료용 안티센스 올리고뉴클레오타이드
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
US12396981B2 (en) 2023-03-09 2025-08-26 William Shulman Methods of using DMT
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
CN121039282A (zh) 2023-03-24 2025-11-28 ProQR治疗上市公司Ⅱ 用于治疗神经系统病症的反义寡核苷酸
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
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
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
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CN116754689A (zh) * 2023-07-27 2023-09-15 苏州和合医学检验有限公司 一种高效液相色谱串联质谱法检测5种药物的方法及应用
CN117099681B (zh) * 2023-08-28 2024-07-05 南京农业大学 一种利用紫杉醇加倍玉米单倍体的方法
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025213142A2 (en) * 2024-04-05 2025-10-09 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
CN119015397B (zh) * 2024-08-20 2025-04-18 广州医科大学附属中医医院(广州市中医中药研究所、广州市中医医院、广州中医药大学附属广州中医医院、广州市针灸医院) 一种抑制人前列腺癌细胞增殖的白僵菌素组合物及其制备方法

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04503957A (ja) * 1989-03-07 1992-07-16 ジェネンテク，インコーポレイテッド 脂質とオリゴヌクレオチドの共有結合コンジュゲート
JP2002510319A (ja) * 1997-07-01 2002-04-02 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 オリゴヌクレオチドの消化管を介したデリバリーのための組成物及び方法
JP2007504830A (ja) * 2003-09-09 2007-03-08 ジェロン・コーポレーション テロメラーゼ阻害のための改変オリゴヌクレオチド
JP2010507680A (ja) * 2006-10-24 2010-03-11 サーナ セラピューティクス インコーポレーテッド 生物学的に活性な分子を送達するための脂質ナノ粒子系組成物及び方法
JP2012506697A (ja) * 2008-10-27 2012-03-22 プロセンサ テクノロジーズ ベー．フェー． Ｄｕｃｈｅｎｎｅ型筋ジストロフィーｍＲＮＡ前駆体のエクソン４５の効率的なスキッピングのための方法及び手段
WO2015107425A2 (en) * 2014-01-16 2015-07-23 Wave Life Sciences Pte. Ltd. Chiral design

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6676963B1 (en) * 2000-10-27 2004-01-13 Barnes-Jewish Hospital Ligand-targeted emulsions carrying bioactive agents
US7511131B2 (en) * 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
US20100008937A1 (en) * 2006-04-25 2010-01-14 Immune Disease Institute, Inc. Targeted delivery to leukocytes using non-protein carriers
US8097596B2 (en) * 2006-06-30 2012-01-17 Lakewood-Amedex, Inc. Compositions and methods for the treatment of muscle wasting
CN101896186A (zh) * 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
MX348474B (es) * 2009-12-23 2017-06-14 Novartis Ag * Lipidos, composiciones de lipido, y metodos de uso de los mismos.
EP2582397A4 (en) * 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04503957A (ja) * 1989-03-07 1992-07-16 ジェネンテク，インコーポレイテッド 脂質とオリゴヌクレオチドの共有結合コンジュゲート
JP2002510319A (ja) * 1997-07-01 2002-04-02 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 オリゴヌクレオチドの消化管を介したデリバリーのための組成物及び方法
JP2007504830A (ja) * 2003-09-09 2007-03-08 ジェロン・コーポレーション テロメラーゼ阻害のための改変オリゴヌクレオチド
JP2010507680A (ja) * 2006-10-24 2010-03-11 サーナ セラピューティクス インコーポレーテッド 生物学的に活性な分子を送達するための脂質ナノ粒子系組成物及び方法
JP2012506697A (ja) * 2008-10-27 2012-03-22 プロセンサ テクノロジーズ ベー．フェー． Ｄｕｃｈｅｎｎｅ型筋ジストロフィーｍＲＮＡ前駆体のエクソン４５の効率的なスキッピングのための方法及び手段
WO2015107425A2 (en) * 2014-01-16 2015-07-23 Wave Life Sciences Pte. Ltd. Chiral design

Non-Patent Citations (9)

* Cited by examiner, † Cited by third party
Title
ADVANCED DRUG DELIVERY REVIEWS, vol. 87, JPN6021017231, 2015, pages 104 - 107, ISSN: 0004922445 *
DRUG DELIVERY SYSTEM, vol. 22-2, JPN6021017230, 2007, pages 140 - 147, ISSN: 0004922443 *
JOURNAL OF DRUG DISCOVERY, DEVELOPMENT AND DELIVERY, vol. 3, no. 2, JPN6021017234, 3 May 2016 (2016-05-03), pages 1022 - 1, ISSN: 0004922444 *
NUCLEIC ACIDS RES., 18[13] (1990) P.3777-3783, JPN6010035990, ISSN: 0004922448 *
NUCLEIC ACIDS RES., 20[13] (1992) P.3411-3417, JPN6010035989, ISSN: 0004922447 *
NUCLEIC ACIDS RESEARCH, vol. 40, no. 21, JPN6021017233, 2012, pages 10585 - 10595, ISSN: 0004922446 *
YAKUGAKU ZASSHI, vol. 132, no. 12, JPN6021017229, 2012, pages 1383 - 1388, ISSN: 0004922442 *
ファルマシア, vol. 51, no. 5, JPN6022013965, 2015, pages 429 - 433, ISSN: 0004922449 *
核酸医薬の最前線＜＜普及版＞＞, vol. 普及版第１刷, JPN6021017228, 2014, pages 14 - 17, ISSN: 0004922441 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023503644A (ja) * 2019-11-27 2023-01-31 ディー・ティー・エックス・ファーマ・インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための化合物および方法
JP2023545041A (ja) * 2020-10-05 2023-10-26 エスキュイグレック・テラピュティク Ｄｍｄ患者におけるエクソン５１スキッピングによるジストロフィンレスキューのためのオリゴマー化合物

Also Published As

Publication number Publication date
EP3452597A1 (en) 2019-03-13
MA45290A (fr) 2019-03-13
WO2017192679A1 (en) 2017-11-09
JP2025108550A (ja) 2025-07-23
US20190249173A1 (en) 2019-08-15
US20230295617A1 (en) 2023-09-21
EP3452597A4 (en) 2020-05-06
CN109462995A (zh) 2019-03-12
JP2023078293A (ja) 2023-06-06

Similar Documents

Publication Publication Date Title
JP2023078293A (ja) 生物活性剤に関する方法および組成物
JP7625544B2 (ja) キラル制御
JP7606954B2 (ja) オリゴヌクレオチド組成物およびその方法
US10724035B2 (en) Oligonucleotide compositions and methods thereof
RU2571218C2 (ru) Конъюгат мирнк и способ его получения
JP2024147677A (ja) オリゴヌクレオチド、その組成物および方法
CN108135923B (zh) 靶向超氧化物歧化酶1（sod1）的核酸分子
KR20180056766A (ko) 뉴클레오티드 조성물 및 이의 방법
EA024960B1 (ru) Улучшенная липидная композиция
JP2024525713A (ja) 糖のアノマー部位におけるクリックケミストリーを介したターゲティングリガンドの多重化
JP2024525552A (ja) アミノオキシクリックケミストリー（ａｏｃｃ）：多用途バイオコンジュゲーションアプローチ
CN119799705B (zh) 靶向调控HBV基因表达的siRNA及其应用
RU2830607C2 (ru) Композиции олигонуклеотидов и способы с ними
RU2797833C1 (ru) Композиции олигонуклеотидов и способы с ними
JP2025534336A (ja) 修飾オリゴヌクレオチドおよび二本鎖ｒｎａ
JP2024526730A (ja) ２，６－ジアミノプリン及び２－アミノアデニンのコンジュゲートをオリゴヌクレオチドに導入するための簡便な化学的アプローチ
AU2015200143A1 (en) Sirna conjugate and preparation method thereof

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200501

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210511

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210721

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211111

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220412

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220712

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221006

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20221116