MA45290A - Procédés et compositions d'agents biologiquement actifs - Google Patents

Procédés et compositions d'agents biologiquement actifs

Info

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
Application number
MA045290A
Other languages
English (en)
Inventor
David Charles Donnell Butler
Sethumadhavan Divakaramenon
Maria David Frank-Kamenetsky
Naoki Iwamoto
Genliang Lu
Subramanian Marappan
Chandra Vargeese
Hailin Yang
Jason Jingxin Zhang
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 MA45290A publication Critical patent/MA45290A/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
    • C12N15/111—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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Pain & Pain Management (AREA)
  • Obesity (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
MA045290A 2016-05-04 2017-05-03 Procédés et compositions d'agents biologiquement actifs MA45290A (fr)

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 2017-05-03 Procédés et compositions d'agents biologiquement actifs

Country Status (6)

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

Families Citing this family (131)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
EP2451461A4 (fr) 2009-07-06 2013-05-29 Ontorii Inc Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
WO2014010250A1 (fr) 2012-07-13 2014-01-16 Chiralgen, Ltd. Groupe auxiliaire asymétrique
DK2872147T3 (da) 2012-07-13 2023-02-20 Wave Life Sciences Ltd Fremgangsmåde til fremstilling af kirale oligonukleotider
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
EP3094728B1 (fr) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Conception chirale
AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
CN117965536A (zh) 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
MA46427A (fr) 2015-10-09 2018-08-15 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA43822A (fr) 2016-03-13 2018-11-28 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
EP3452596A4 (fr) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
US11013757B2 (en) 2016-06-03 2021-05-25 Wave Life Sciences Ltd. Oligonucleotides, compositions and methods thereof
EP3475424A1 (fr) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Oligonucléotides d'édition d'arn monocaténaire
WO2018041973A1 (fr) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Oligonucléotides d'édition d'arn simple brin chimiquement modifiés
WO2018098264A1 (fr) 2016-11-23 2018-05-31 Wave Life Sciences Ltd. Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
NZ755416A (en) 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
PT4122497T (pt) 2016-12-19 2024-06-12 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
ES3051834T3 (en) 2016-12-19 2025-12-30 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3065523A1 (fr) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs procedes d'utilisation
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
AU2018316159A1 (en) 2017-08-08 2020-02-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
SG11202002517RA (en) * 2017-09-22 2020-04-29 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
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
JP2020536058A (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
EP3694530A4 (fr) 2017-10-12 2021-06-30 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
US11559527B2 (en) 2017-12-20 2023-01-24 Lunella Biotech, Inc. Targeting mitochondrial fission through mDIVI-1 derivatives
JP2021521140A (ja) 2018-04-12 2021-08-26 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
CA3098623A1 (fr) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Administration extra-hepatique
CA3098624A1 (fr) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Compositions d'oligonucleotides et leurs procedes d'utilisation
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP7583712B2 (ja) * 2018-05-30 2024-11-14 ノバルティス アーゲー 脂質修飾された核酸化合物および方法
EP4219717A3 (fr) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Oligomères à saut d'exon pour la dystrophie musculaire
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
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
EP3830259A4 (fr) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de la dystrophie musculaire facio-scapulo-humérale
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028832A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
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
BR112021011018A2 (pt) 2018-12-13 2021-08-31 Sarepta Therapeutics, Inc. Conjugados de oligômero para exon skipping para distrofia muscular
WO2020132263A1 (fr) * 2018-12-20 2020-06-25 Aegis Therapeutics, Inc. Compositions, dispositifs et procédés de traitement de troubles reposant sur la surdose et la récompense
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
AU2020241559A1 (en) 2019-03-20 2021-11-11 Wave Life Sciences Ltd. Technologies useful for oligonucleotide preparation
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
WO2020214763A1 (fr) 2019-04-18 2020-10-22 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
WO2020219820A1 (fr) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Compositions d'acide nucléique et méthodes de saut multi-exon
MX2021012981A (es) 2019-04-25 2022-01-18 Wave Life Sciences Ltd Composiciones de oligonucleotido y metodos de uso de las mismas.
KR20220011697A (ko) * 2019-05-22 2022-01-28 바이오비에 아이앤씨. 테를리프레신 제형
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
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
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
WO2021154477A1 (fr) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anticorps anti-récepteur de la transferrine (tfr) et utilisations associées
AU2021230473A1 (en) * 2020-03-01 2022-09-22 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
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
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
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
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2022069511A1 (fr) * 2020-09-30 2022-04-07 Biomarin Technologies B.V. Oligonucléotide anti-sens ciblant l'exon 51 du gène de la dystrophine
EP3978608A1 (fr) * 2020-10-05 2022-04-06 SQY Therapeutics Composé oligomère de sauvetage de dystrophine chez des patients atteints de dmd durant des sauts d'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
WO2023283623A1 (fr) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes ciblant le muscle et formulations de traitement de 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
IL311507A (en) * 2021-09-16 2024-05-01 Dyne Therapeutics Inc Dosing of muscle targeting complexes for the treatment of dystrophinopathy
EP4401792A4 (fr) 2021-09-16 2025-12-03 Avidity Biosciences Inc Compositions et méthodes de traitement d'une dystrophie musculaire facio-scapulo-humérale
US20240425864A1 (en) 2021-09-30 2024-12-26 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
EP4479535A1 (fr) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
EP4555085A1 (fr) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar
EP4555086A1 (fr) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucléotides pour édition d'arn médiée par adar et leur utilisation
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
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
WO2024121373A1 (fr) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (fr) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire athérosclérotique
US12396981B2 (en) 2023-03-09 2025-08-26 William Shulman Methods of using DMT
JP2026510408A (ja) 2023-03-24 2026-04-02 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 神経障害の処置のためのアンチセンスオリゴヌクレオチド
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
EP4688143A1 (fr) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Oligonucléotides antisens pour traitement des maladies du foie
EP4702144A1 (fr) 2023-04-27 2026-03-04 Sarepta Therapeutics, Inc. Oligomères antisens pour le traitement d'une néphropathie chronique
WO2024256620A1 (fr) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement des maladies neurodégénératives
AU2024287308A1 (en) 2023-07-13 2025-12-18 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
EP4774393A1 (fr) 2023-09-07 2026-07-15 ProQR Therapeutics II B.V. Oligonucléotides antisens pour le traitement de troubles métaboliques
WO2025085810A2 (fr) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Oligomères antisens pour le traitement de myopathies centronucléaires
WO2025096809A1 (fr) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucléotides comprenant des liaisons internucléotidiques de phosphoramidate
AU2024379264A1 (en) 2023-11-16 2026-05-14 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025128799A1 (fr) 2023-12-12 2025-06-19 Korro Bio, Inc. Oligonucléotides d'édition d'arn double brin et leurs utilisations
AU2024402199A1 (en) 2023-12-20 2026-07-16 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 (fr) * 2024-04-05 2025-10-09 Wave Life Sciences Ltd. Compositions d'oligonucléotides et procédés associés
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
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
WO2026050243A1 (fr) 2024-08-26 2026-03-05 Korro Bio, Inc. Oligonucléotides conjugués à des fractions galnac pour édition d'arn
WO2026060374A2 (fr) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles neurologiques
WO2026068781A1 (fr) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour traitement des maladies hépatiques
WO2026093339A1 (fr) 2024-10-29 2026-05-07 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement de troubles de biogenèse du peroxysome dans le spectre de zellweger (pbd-zsd)
WO2026122478A1 (fr) 2024-12-02 2026-06-11 Korro Bio, Inc. Formulations et méthodes d'administration extrahépatique
WO2026136663A1 (fr) 2024-12-18 2026-06-25 Proqr Therapeutics Ii B.V. Oligonucléotides antisens pour le traitement des maladies neurodégénératives

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1990010448A2 (fr) * 1989-03-07 1990-09-20 Genentech, Inc. Conjugue covalent de lipides et d'oligonucleotides
WO1999001579A1 (fr) * 1997-07-01 1999-01-14 Isis Pharmaceuticals, Inc. Compositions et procedes d'apport d'oligonucleotides par le tube digestif
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
DK3342425T3 (da) * 2003-09-09 2020-03-16 Geron Corp Modificerede oligonukleotider til inhibering af telomerase
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
NZ584793A (en) * 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
SI2607484T1 (sl) * 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
WO2011076807A2 (fr) * 2009-12-23 2011-06-30 Novartis Ag Lipides, compositions lipidiques, et procédés d'utilisation associés
WO2011159836A2 (fr) * 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Composés et procédés pour moduler l'interaction entre des protéines et des acides nucléiques cibles
EP3094728B1 (fr) * 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Conception chirale

Also Published As

Publication number Publication date
JP2025108550A (ja) 2025-07-23
US20190249173A1 (en) 2019-08-15
JP2019516679A (ja) 2019-06-20
US20230295617A1 (en) 2023-09-21
WO2017192679A1 (fr) 2017-11-09
EP3452597A4 (fr) 2020-05-06
CN109462995A (zh) 2019-03-12
JP7825588B2 (ja) 2026-03-06
EP3452597A1 (fr) 2019-03-13
JP2023078293A (ja) 2023-06-06

Similar Documents

Publication Publication Date Title
EP3452597A4 (fr) Procédés et compositions d'agents biologiquement actifs
MA45270A (fr) Compositions d'oligonucléotides et procédés associés
EP3704239A4 (fr) Compositions de casz et procédés d'utilisation
EP3634430A4 (fr) Agents multibiotiques et procédés d'utilisation de ceux-ci
MA45688A (fr) Compositions et procédés de potentialisation d'agents antimicrobiens
MA46427A (fr) Compositions d'oligonucléotides et procédés associés
EP3596213A4 (fr) Compositions et procédés d'amplification d'expression génique
EP3630199A4 (fr) Compositions d'oligonucléotides et leurs procédés d'utilisation
EP3630788A4 (fr) Compositions d'oligonucléotides et leurs procédés d'utilisation
EP3630789A4 (fr) Compositions d'oligonucléotides et leurs procédés d'utilisation
EP3402493A4 (fr) Composition d'organoïde neural et procédés d'utilisation
EP3516627A4 (fr) Création et édition d'avatar
MA47499A (fr) Anticorps anti-tau et leurs procédés d'utilisation
EP3298168A4 (fr) Agents réducteurs stabilisés et leurs procédés d'utilisation
EP3377042A4 (fr) Substances et procédés de traitement de myopathies à base de titine et d'autres titinopathies
EP3352774A4 (fr) Compositions de flavonoïdes et procédés d'utilisation
EP3313404A4 (fr) Compositions thérapeutiques, associations et procédés d'utilisation
EP3435956A4 (fr) Compositions photo-stabilisées et leurs procédés d'utilisation
EP3407976A4 (fr) Compositions et utilisations d'agents alpha-adrénergiques
EP3430140A4 (fr) Procédés améliorés d'amplification et de séquençage
EP3522882A4 (fr) Compositions d'acides aminés et utilisations associées
EP3635000A4 (fr) Manabodies et procédés d'utilisation
EP3706558A4 (fr) Compositions et procédés d'aquaculture
EP3496768A4 (fr) Compositions antimicrobiennes et procédés d'utilisation associés.
EP3606465A4 (fr) Schémas posologiques et compositions et procédés associés