MA45290A - Procédés et compositions d'agents biologiquement actifs - Google Patents
Procédés et compositions d'agents biologiquement actifsInfo
- Publication number
- MA45290A MA45290A MA045290A MA45290A MA45290A MA 45290 A MA45290 A MA 45290A MA 045290 A MA045290 A MA 045290A MA 45290 A MA45290 A MA 45290A MA 45290 A MA45290 A MA 45290A
- Authority
- MA
- Morocco
- Prior art keywords
- compositions
- processes
- biologically active
- active agents
- agents
- Prior art date
Links
Classifications
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- 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
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- 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
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- 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
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- 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 N.A.
-
- 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 N.A.
- 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 [CRISPRs]
-
- 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
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662331961P | 2016-05-04 | 2016-05-04 | |
US201662405810P | 2016-10-07 | 2016-10-07 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA45290A true MA45290A (fr) | 2019-03-13 |
Family
ID=60203379
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA045290A MA45290A (fr) | 2016-05-04 | Procédés et compositions d'agents biologiquement actifs |
Country Status (6)
Country | Link |
---|---|
US (2) | US20190249173A1 (fr) |
EP (1) | EP3452597A4 (fr) |
JP (2) | JP2019516679A (fr) |
CN (1) | CN109462995A (fr) |
MA (1) | MA45290A (fr) |
WO (1) | WO2017192679A1 (fr) |
Families Citing this family (77)
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 |
EP2451461A4 (fr) | 2009-07-06 | 2013-05-29 | Ontorii Inc | Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation |
JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
EP2734208B1 (fr) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Procédés pour la synthèse d'acides nucléiques fonctionnalisés |
DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
CA2878945A1 (fr) | 2012-07-13 | 2014-01-16 | Wave Life Sciences Pte. Ltd. | Controle chiral |
EP3095461A4 (fr) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité |
EP3095459A4 (fr) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral |
US10160969B2 (en) | 2014-01-16 | 2018-12-25 | Wave Life Sciences Ltd. | Chiral design |
EP3234134B1 (fr) | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Édition ciblée d'arn |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
CA2999772A1 (fr) | 2015-10-09 | 2017-04-13 | Wave Life Sciences Ltd. | Compositions d'oligonucleotides et procedes associes |
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 |
JP2019520339A (ja) | 2016-06-03 | 2019-07-18 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | オリゴヌクレオチド、その組成物および方法 |
EP3475424A1 (fr) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Oligonucléotides d'édition d'arn monocaténaire |
CN110088113A (zh) | 2016-11-23 | 2019-08-02 | 波涛生命科学有限公司 | 用于亚磷酰胺和寡核苷酸合成的组合物和方法 |
SG10202012839TA (en) | 2016-12-19 | 2021-01-28 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
JP7160807B2 (ja) | 2016-12-19 | 2022-10-25 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
LT3554554T (lt) | 2016-12-19 | 2022-11-25 | Sarepta Therapeutics, Inc. | Egzoną praleidžiantys oligomero konjugatai nuo raumenų distrofijos |
KR20190104381A (ko) | 2017-01-06 | 2019-09-09 | 어비디티 바이오사이언시스 엘엘씨 | 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법 |
JP2020522510A (ja) | 2017-06-02 | 2020-07-30 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | オリゴヌクレオチド組成物及びその使用方法 |
TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
WO2018237194A1 (fr) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Composés, compositions et procédés de synthèse |
GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
EP3664815A4 (fr) | 2017-08-08 | 2021-08-11 | Wave Life Sciences Ltd. | Compositions oligonucléotidiques et méthodes associées |
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 |
WO2019067979A1 (fr) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | Polythérapies pour traiter une dystrophie musculaire |
US20200248178A1 (en) | 2017-09-28 | 2020-08-06 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
US20210145852A1 (en) | 2017-09-28 | 2021-05-20 | Sarepta Therapeutics, Inc. | Combination Therapies for Treating Muscular Dystrophy |
US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
MX2020005860A (es) | 2017-12-06 | 2020-09-09 | Avidity Biosciences Inc | Composiciones y metodos de tratamiento de atrofia muscular y distrofia miotonica. |
WO2019126179A1 (fr) | 2017-12-20 | 2019-06-27 | Lunella Biotech, Inc. | Ciblage de la fission mitochondriale par l'intermédiaire de dérivés de mdivi-1 |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
EP4219717A3 (fr) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Oligomères à saut d'exon pour la dystrophie musculaire |
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 |
US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
CA3108282A1 (fr) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
BR112021011018A2 (pt) | 2018-12-13 | 2021-08-31 | Sarepta Therapeutics, Inc. | Conjugados de oligômero para exon skipping para distrofia muscular |
KR20210131996A (ko) * | 2018-12-20 | 2021-11-03 | 이지스 쎄라퓨틱스, 엘엘씨 | 과다복용 및 보상-기반 장애의 치료를 위한 조성물, 장치 및 방법 |
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 |
WO2020158636A1 (fr) * | 2019-01-29 | 2020-08-06 | 国立大学法人東京大学 | Procédé pour introduire un acide nucléique, etc., dans une cellule |
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 (fr) | 2019-04-18 | 2022-02-23 | Sarepta Therapeutics, Inc. | Compositions pour le traitement de la dystrophie musculaire |
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 |
AU2020313255A1 (en) | 2019-07-12 | 2022-01-06 | Daiichi Sankyo Company, Limited | Antisense oligonucleotide capable of altering splicing of DUX4 pre-mRNA |
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 |
KR20220147575A (ko) * | 2019-11-27 | 2022-11-03 | 디티엑스 파마, 인크. | 뒤시엔느 근위축증의 치료를 위한 화합물 및 방법 |
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 |
CA3169252A1 (fr) * | 2020-03-01 | 2021-09-10 | Abbie Madeline MAGUIRE | Compositions oligonucleotidiques et methodes associees |
US11525137B2 (en) | 2020-03-19 | 2022-12-13 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
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 |
MX2022014606A (es) | 2020-05-22 | 2023-03-08 | Wave Life Sciences Ltd | Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas. |
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 |
TW202229550A (zh) * | 2020-09-30 | 2022-08-01 | 荷蘭商比奧馬林技術公司 | 寡核苷酸 |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
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 |
AU2022345098A1 (en) | 2021-09-16 | 2024-04-04 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
CA3230451A1 (fr) * | 2021-09-16 | 2023-03-23 | Cody A. Desjardins | Dosage de complexes de ciblage de muscle pour traiter des dystrophinopathies |
WO2023055774A1 (fr) | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Oligonucléotides antisens ayant une ou plusieurs unités abasiques |
WO2023152371A1 (fr) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie |
US11931421B2 (en) | 2022-04-15 | 2024-03-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
WO2024013360A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar |
WO2024013361A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides pour édition d'arn médiée par adar et leur utilisation |
WO2024064237A2 (fr) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Efficacité du saut d'exon médié par un oligonucléotide antisens dans le traitement de la dmd |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DE69008521T2 (de) * | 1989-03-07 | 1994-10-20 | Genentech Inc | Kovalente konjugate von lipiden und oligonukleotiden. |
DE69834038D1 (de) * | 1997-07-01 | 2006-05-18 | Isis Pharmaceutical Inc | Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre |
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 |
BRPI0414222B8 (pt) * | 2003-09-09 | 2021-05-25 | Geron Corp | compostos oligonucleotídeos modificados para inibição da telomerase, métodos, composição farmacêutica e usos relacionados |
US7404969B2 (en) * | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
US20130129752A1 (en) * | 2006-04-25 | 2013-05-23 | Immune Disease Institute, Inc. | Targeted delivery to leukocytes using protein carriers |
US8097596B2 (en) * | 2006-06-30 | 2012-01-17 | Lakewood-Amedex, Inc. | Compositions and methods for the treatment of muscle wasting |
PT2203173E (pt) * | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
EP2607484B1 (fr) * | 2008-10-27 | 2016-01-06 | BioMarin Technologies B.V. | Procédés et moyens d'induction du saut de l exon 45 dans l arn pré-messager du gène de la dystrophie musculaire de duchenne |
WO2011076807A2 (fr) * | 2009-12-23 | 2011-06-30 | Novartis Ag | Lipides, compositions lipidiques, et procédés d'utilisation associés |
US9518259B2 (en) * | 2010-06-15 | 2016-12-13 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating interaction between proteins and target nucleic acids |
US10160969B2 (en) * | 2014-01-16 | 2018-12-25 | Wave Life Sciences Ltd. | Chiral design |
-
0
- MA MA045290A patent/MA45290A/fr unknown
-
2017
- 2017-05-03 WO PCT/US2017/030777 patent/WO2017192679A1/fr unknown
- 2017-05-03 EP EP17793227.4A patent/EP3452597A4/fr active Pending
- 2017-05-03 JP JP2018557083A patent/JP2019516679A/ja active Pending
- 2017-05-03 US US16/098,658 patent/US20190249173A1/en not_active Abandoned
- 2017-05-03 CN CN201780040364.4A patent/CN109462995A/zh active Pending
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2022
- 2022-11-30 US US18/072,296 patent/US20230295617A1/en active Pending
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2023
- 2023-03-16 JP JP2023041621A patent/JP2023078293A/ja active Pending
Also Published As
Publication number | Publication date |
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JP2023078293A (ja) | 2023-06-06 |
WO2017192679A1 (fr) | 2017-11-09 |
US20190249173A1 (en) | 2019-08-15 |
US20230295617A1 (en) | 2023-09-21 |
EP3452597A4 (fr) | 2020-05-06 |
EP3452597A1 (fr) | 2019-03-13 |
CN109462995A (zh) | 2019-03-12 |
JP2019516679A (ja) | 2019-06-20 |
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