CN109462995A - 生物活性剂的方法和组合物 - Google Patents
生物活性剂的方法和组合物 Download PDFInfo
- Publication number
- CN109462995A CN109462995A CN201780040364.4A CN201780040364A CN109462995A CN 109462995 A CN109462995 A CN 109462995A CN 201780040364 A CN201780040364 A CN 201780040364A CN 109462995 A CN109462995 A CN 109462995A
- Authority
- CN
- China
- Prior art keywords
- oligonucleotides
- acid
- composition
- lipid
- hydrochloride
- 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
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
- C12N15/111—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)
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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN109462995A true CN109462995A (zh) | 2019-03-12 |
Family
ID=60203379
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201780040364.4A Pending CN109462995A (zh) | 2016-05-04 | 2017-05-03 | 生物活性剂的方法和组合物 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US20190249173A1 (enExample) |
| EP (1) | EP3452597A4 (enExample) |
| JP (3) | JP2019516679A (enExample) |
| CN (1) | CN109462995A (enExample) |
| MA (1) | MA45290A (enExample) |
| WO (1) | WO2017192679A1 (enExample) |
Cited By (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109870583A (zh) * | 2019-04-15 | 2019-06-11 | 德阳市人民医院 | 急性胰腺炎相关的代谢物及其应用 |
| CN111012788A (zh) * | 2019-12-12 | 2020-04-17 | 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 |
| CN111063870A (zh) * | 2019-11-27 | 2020-04-24 | 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 |
| CN111055603A (zh) * | 2019-12-31 | 2020-04-24 | 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 |
| CN111249298A (zh) * | 2020-04-08 | 2020-06-09 | 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 |
| 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 |
| CN113520995A (zh) * | 2021-08-16 | 2021-10-22 | 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 |
| CN114096267A (zh) * | 2019-05-22 | 2022-02-25 | 柏欧韦股份有限公司 | 特利加压素制剂 |
| CN115348883A (zh) * | 2019-11-27 | 2022-11-15 | Dtx医药有限公司 | 用于治疗杜兴氏肌营养不良症的化合物和方法 |
| CN115697351A (zh) * | 2020-05-27 | 2023-02-03 | 罗马大学 | 作为端粒病的治疗方法的用于化学抑制tgs1的药物组合物 |
| 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 |
| CN119015397A (zh) * | 2024-08-20 | 2024-11-26 | 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值的白僵菌素组合物及其制备方法 |
Families Citing this family (106)
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| WO2022069511A1 (en) * | 2020-09-30 | 2022-04-07 | Biomarin Technologies B.V. | Antisense oligonucleotides targeting the exon 51 of dystrophin gene |
| EP3978608A1 (en) * | 2020-10-05 | 2022-04-06 | SQY Therapeutics | Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51 |
| 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 |
| 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 |
| 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 |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040115192A1 (en) * | 2000-10-27 | 2004-06-17 | Lanza Gregory M. | Ligand-targeted emulsions carrying bioactive agents |
| WO2009054725A2 (en) * | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| CN102905763A (zh) * | 2009-12-23 | 2013-01-30 | 诺瓦提斯公司 | 脂质、脂质组合物和使用它们的方法 |
| US20130289092A1 (en) * | 2010-06-15 | 2013-10-31 | Cold Spring Harbor Laboratory | Compounds and methods for modulating interaction between proteins and target nucleic acids |
| WO2015107425A2 (en) * | 2014-01-16 | 2015-07-23 | Wave Life Sciences Pte. Ltd. | Chiral design |
Family Cites Families (8)
| 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 |
| US7511131B2 (en) * | 2002-11-13 | 2009-03-31 | Genzyme Corporation | Antisense modulation of apolipoprotein B expression |
| AU2004271215B2 (en) * | 2003-09-09 | 2009-07-16 | Geron Corporation | Modified oligonucleotides for telomerase inhibition |
| US7404969B2 (en) * | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| 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 |
| DK2607484T3 (en) * | 2008-10-27 | 2016-03-07 | Biomarin Technologies B V | Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-MRNA |
-
0
- MA MA045290A patent/MA45290A/fr unknown
-
2017
- 2017-05-03 CN CN201780040364.4A patent/CN109462995A/zh active Pending
- 2017-05-03 EP EP17793227.4A patent/EP3452597A4/en active Pending
- 2017-05-03 WO PCT/US2017/030777 patent/WO2017192679A1/en not_active Ceased
- 2017-05-03 JP JP2018557083A patent/JP2019516679A/ja active Pending
- 2017-05-03 US US16/098,658 patent/US20190249173A1/en not_active Abandoned
-
2022
- 2022-11-30 US US18/072,296 patent/US20230295617A1/en active Pending
-
2023
- 2023-03-16 JP JP2023041621A patent/JP2023078293A/ja active Pending
-
2025
- 2025-04-10 JP JP2025064806A patent/JP2025108550A/ja active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040115192A1 (en) * | 2000-10-27 | 2004-06-17 | Lanza Gregory M. | Ligand-targeted emulsions carrying bioactive agents |
| WO2009054725A2 (en) * | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| CN102905763A (zh) * | 2009-12-23 | 2013-01-30 | 诺瓦提斯公司 | 脂质、脂质组合物和使用它们的方法 |
| US20130289092A1 (en) * | 2010-06-15 | 2013-10-31 | Cold Spring Harbor Laboratory | Compounds and methods for modulating interaction between proteins and target nucleic acids |
| WO2015107425A2 (en) * | 2014-01-16 | 2015-07-23 | Wave Life Sciences Pte. Ltd. | Chiral design |
Non-Patent Citations (1)
| Title |
|---|
| ANNEMIEKE AARTSMA-RUS等: "Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy", NEUROMUSCUL DISORD, pages 1 * |
Cited By (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109870583A (zh) * | 2019-04-15 | 2019-06-11 | 德阳市人民医院 | 急性胰腺炎相关的代谢物及其应用 |
| CN114096267A (zh) * | 2019-05-22 | 2022-02-25 | 柏欧韦股份有限公司 | 特利加压素制剂 |
| CN111063870A (zh) * | 2019-11-27 | 2020-04-24 | 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 |
| CN115348883B (zh) * | 2019-11-27 | 2024-11-15 | 诺华股份有限公司 | 用于治疗杜兴氏肌营养不良症的化合物和方法 |
| CN115348883A (zh) * | 2019-11-27 | 2022-11-15 | Dtx医药有限公司 | 用于治疗杜兴氏肌营养不良症的化合物和方法 |
| CN111012788A (zh) * | 2019-12-12 | 2020-04-17 | 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 |
| CN111055603A (zh) * | 2019-12-31 | 2020-04-24 | 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 |
| 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 |
| CN111249298A (zh) * | 2020-04-08 | 2020-06-09 | 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 |
| CN115697351A (zh) * | 2020-05-27 | 2023-02-03 | 罗马大学 | 作为端粒病的治疗方法的用于化学抑制tgs1的药物组合物 |
| 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 |
| CN113520995A (zh) * | 2021-08-16 | 2021-10-22 | 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 |
| 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 |
| 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 |
| CN119015397A (zh) * | 2024-08-20 | 2024-11-26 | 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值的白僵菌素组合物及其制备方法 |
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 |
| JP2019516679A (ja) | 2019-06-20 |
| JP2023078293A (ja) | 2023-06-06 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20230295617A1 (en) | Methods and compositions of biologically active agents | |
| US20210290771A1 (en) | Engineered receptor/ligand system for delivery of therapeutic agents | |
| US5795909A (en) | DHA-pharmaceutical agent conjugates of taxanes | |
| US8314077B2 (en) | Fatty acid-pharmaceutical agent conjugates | |
| US20240050317A1 (en) | System and method for making personalized individual unit doses containing pharmaceutical actives | |
| US9308181B2 (en) | Topical formulations, systems and methods | |
| US20030059471A1 (en) | Oral delivery formulation | |
| CA3110997A1 (en) | Oral film compositions and dosage forms having precise active dissolution profiles | |
| CN103271083A (zh) | 多功能离子液体组合物 | |
| KR20220126286A (ko) | 전구약물 조성물 및 치료 방법 | |
| CN104812777A (zh) | 多糖酯微球以及与之相关的方法与制品 | |
| US9642912B2 (en) | Topical formulations for treating skin conditions | |
| US20210145731A1 (en) | Multimodal compositions and methods of treatment | |
| US20220233471A1 (en) | Prodrug compositions and methods of treatment | |
| AU770519B2 (en) | DHA-pharmaceutical agent conjugates | |
| CA3210706A1 (en) | Dosage forms having equivalent biocomparable profiles |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination |