MX2018009853A - Ligandos de direccion. - Google Patents

Ligandos de direccion.

Info

Publication number
MX2018009853A
MX2018009853A MX2018009853A MX2018009853A MX2018009853A MX 2018009853 A MX2018009853 A MX 2018009853A MX 2018009853 A MX2018009853 A MX 2018009853A MX 2018009853 A MX2018009853 A MX 2018009853A MX 2018009853 A MX2018009853 A MX 2018009853A
Authority
MX
Mexico
Prior art keywords
targeting ligands
compounds
expression
target
therapeutic
Prior art date
Application number
MX2018009853A
Other languages
English (en)
Inventor
Glebocka Agnieszka
Kumar Pankaj
Pei Tao
Li Zhen
Lawler Michael
Fleitz Fred
Altenhofer Erich
Original Assignee
Arrowhead Pharmaceuticals Inc
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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of MX2018009853A publication Critical patent/MX2018009853A/es

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K47/555—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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H5/00—Compounds containing saccharide radicals in which the hetero bonds to oxygen have been replaced by the same number of hetero bonds to halogen, nitrogen, sulfur, selenium, or tellurium
    • C07H5/04—Compounds containing saccharide radicals in which the hetero bonds to oxygen have been replaced by the same number of hetero bonds to halogen, nitrogen, sulfur, selenium, or tellurium to nitrogen
    • C07H5/06—Aminosugars
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3517—Marker; Tag
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

La presente invención se relaciona con nuevos ligandos de dirección que pueden enlazarse a compuestos, tales como compuestos terapéuticos que son útiles en la dirección de compuestos al blanco in vivo. Los ligandos de dirección descritos en la presente pueden servir para los compuestos oligoméricos de inhibición de la expresión blanco, tales como agente de ARNi, a células hepáticas para modular la expresión génica. Los ligandos de dirección descritos en la presente, cuando se conjugan a un compuesto terapéutico, pueden usarse en una variedad de aplicaciones, que incluyen el uso en la validación terapéutica, diagnóstico, blanco y aplicaciones de descubrimiento genómico. Las composiciones, que incluyen los ligandos de dirección descritos en la presente, cuando se enlazan a compuestos oligoméricos que inhiben la expresión son capaces de mediar la expresión de secuencias de ácidos nucleicos blanco en las células hepáticas, tales como hepatocitos, que podrían ser útiles en el tratamiento de enfermedades o afecciones que responden a la inhibición de la expresión o actividad génica en una célula, tejido u organismo.
MX2018009853A 2016-09-02 2017-03-07 Ligandos de direccion. MX2018009853A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662383221P 2016-09-02 2016-09-02
US201762456339P 2017-02-08 2017-02-08
PCT/US2017/021147 WO2018044350A1 (en) 2016-09-02 2017-03-07 Targeting ligands

Publications (1)

Publication Number Publication Date
MX2018009853A true MX2018009853A (es) 2018-11-09

Family

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MX2018009853A MX2018009853A (es) 2016-09-02 2017-03-07 Ligandos de direccion.
MX2023006109A MX2023006109A (es) 2016-09-02 2018-08-14 Ligandos de direccion.
MX2023006105A MX2023006105A (es) 2016-09-02 2018-08-14 Ligandos de direccion.

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MX2023006109A MX2023006109A (es) 2016-09-02 2018-08-14 Ligandos de direccion.
MX2023006105A MX2023006105A (es) 2016-09-02 2018-08-14 Ligandos de direccion.

Country Status (14)

Country Link
US (3) US10294474B2 (es)
EP (1) EP3506913A4 (es)
JP (3) JP6989521B2 (es)
KR (3) KR102856522B1 (es)
CN (3) CN109462981B (es)
AU (2) AU2017320582B2 (es)
CA (1) CA3011668A1 (es)
IL (2) IL300869A (es)
JO (2) JOP20170056B1 (es)
MX (3) MX2018009853A (es)
SG (2) SG10201912835QA (es)
TW (2) TWI775743B (es)
UY (1) UY37145A (es)
WO (1) WO2018044350A1 (es)

Families Citing this family (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI659040B (zh) 2011-06-30 2019-05-11 美商艾羅海德製藥公司 用於抑制ｂ型肝炎病毒基因表現之組合物及方法
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
CN115957337A (zh) 2015-08-07 2023-04-14 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
NZ744357A (en) 2016-03-07 2025-09-26 Arrowhead Pharmaceuticals Inc Targeting ligands for therapeutic compounds
TW202423483A (zh) 2016-06-06 2024-06-16 美商愛羅海德製藥公司 5’-環-磷酸修飾之核苷酸
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
KR102856522B1 (ko) 2016-09-02 2025-09-08 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
TWI826365B (zh) 2017-01-10 2023-12-21 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
US10597657B2 (en) * 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
WO2019055633A1 (en) 2017-09-14 2019-03-21 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE
EP3697909A4 (en) 2017-10-17 2021-10-13 Arrowhead Pharmaceuticals, Inc. RNAI-BASED AGENTS AND COMPOSITIONS INTENDED TO INHIBIT ASIALOGLYCOPROTEIN RECEPTOR 1 EXPRESSION
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
AU2018374219C1 (en) 2017-12-01 2023-05-11 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
EP3718572B1 (en) 2017-12-01 2024-07-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use
EP3732185B1 (en) 2017-12-29 2025-02-26 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
EP3842534A4 (en) 2018-08-21 2022-07-06 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID AND METHOD OF USE THEREOF
WO2020061177A1 (en) * 2018-09-19 2020-03-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
KR20210110839A (ko) 2018-12-28 2021-09-09 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 핵산을 함유하는 조성물 및 접합체, 이의 제조 방법 및 용도
CN118562796A (zh) * 2019-01-18 2024-08-30 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
EP3923989A1 (en) * 2019-03-21 2021-12-22 Mitotherapeutix LLC Multivalent ligand clusters for targeted delivery of therapeutic agents
CA3139195A1 (en) 2019-05-22 2020-11-26 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
JP7697889B2 (ja) 2019-06-06 2025-06-24 アローヘッド ファーマシューティカルズ インコーポレイテッド アルファ－１アンチトリプシン欠乏症（ａａｔｄ）の治療法
AU2020295796A1 (en) 2019-06-18 2022-02-10 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting rnai
KR20220058578A (ko) * 2019-09-03 2022-05-09 아크투루스 쎄라퓨틱스, 인크. 치료 활성 접합체의 아시알로당단백질 수용체 매개 전달
TW202208625A (zh) 2020-03-06 2022-03-01 美商艾利格斯醫療公司 經修飾之短干擾核酸(siNA)分子及其用途
CN115397436B (zh) * 2020-03-26 2025-07-08 箭头药业股份有限公司 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
WO2021198958A1 (en) 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
EP4133080A1 (en) 2020-04-10 2023-02-15 Aligos Therapeutics, Inc. Short interfering nucleic acid (sina) molecules and uses thereof for coronavirus diseases
TW202214301A (zh) * 2020-06-16 2022-04-16 大陸商上海拓界生物醫藥科技有限公司 一種碳水化合物分子簇及其製備方法和醫藥用途
EP4196610A1 (en) * 2020-08-12 2023-06-21 Cambridge Enterprise Limited Method for targeted nucleic acid cleavage
KR20230067648A (ko) 2020-09-15 2023-05-16 애로우헤드 파마슈티컬스 인코포레이티드 Z-aat 단백질 수준을 감소시키기 위한 방법
CA3199678A1 (en) * 2020-11-05 2022-05-12 Amgen Inc. Methods for treating atherosclerotic cardiovascular disease with lpa-targeted rnai constructs
WO2022189861A1 (en) 2021-03-08 2022-09-15 Tollys Carbohydrate conjugates of tlr3 ligands and uses thereof
KR20240028335A (ko) 2021-04-23 2024-03-05 간엔에이 바이오, 인크. 글리칸 변형 핵산, 제조 방법 및 치료 용도
JP2024523237A (ja) 2021-06-18 2024-06-28 ホンジーン バイオテック コーポレイション 官能化されたｎ－アセチルガラクトサミンヌクレオシド
US11549112B1 (en) 2021-06-21 2023-01-10 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
MX2024000085A (es) * 2021-06-24 2024-01-18 Lilly Co Eli Terapeutica con arn novedosos y usos de estos.
US20250289843A1 (en) * 2021-07-23 2025-09-18 Arrowhead Pharmaceuticals, Inc. Process for preparation of targeting ligands
WO2023034719A1 (en) 2021-08-30 2023-03-09 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
MX2024002782A (es) 2021-09-08 2024-04-16 Aligos Therapeutics Inc Moleculas modificadas de acido nucleico de interferencia corto (anic) y usos de las mismas.
KR20240099159A (ko) 2021-09-08 2024-06-28 알리고스 테라퓨틱스 인코포레이티드 변형된 짧은 간섭 핵산(sina) 분자 및 이의 용도
CA3238865A1 (en) * 2021-11-16 2023-05-25 Shanghai Argo Biopharmaceutical Co., Ltd. Composition and methods for inhibiting expression of angiotensinogen (agt) protein
EP4448540A1 (en) 2021-12-15 2024-10-23 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
CA3249102A1 (en) * 2022-01-20 2025-06-17 Chengdu Xinzhenghe Pharmaceutical Technology Co. Ltd Targeting Ligand and its Use
EP4471140A1 (en) 2022-01-30 2024-12-04 Rona Bioscience, Limited Targeting ligand containing n-acetylgalactosamine
IL315546A (en) 2022-03-16 2024-11-01 Empirico Inc GALNAC compositions to improve siRNA bioavailability
CA3247602A1 (en) 2022-04-08 2023-10-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
EP4541893A1 (en) 2022-06-14 2025-04-23 Rona Bioscience, Limited Sirna molecule for regulating pcsk9 gene activity
WO2024002093A1 (zh) 2022-06-27 2024-01-04 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
WO2024098061A2 (en) 2022-11-04 2024-05-10 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
WO2024108217A1 (en) 2022-11-18 2024-05-23 Genkardia Inc. Methods and compositions for preventing, treating, or reversing cardiac diastolic dysfunction
WO2024118503A1 (en) 2022-11-28 2024-06-06 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
AU2024240837A1 (en) 2023-03-21 2025-09-11 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use
WO2024227059A2 (en) 2023-04-27 2024-10-31 Aligos Therapeutics, Inc. Short interfering nucleic acid (sina) molecules targeting angptl 3 and angptl 8 and methods of using the same
WO2025038661A1 (en) 2023-08-14 2025-02-20 Arrowhead Pharmaceuticals, Inc. Oligonucleotide synthesis methods
US12503696B2 (en) 2023-08-30 2025-12-23 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of inhibin subunit beta E (INHBE), pharmaceutical compositions thereof, and methods of use
WO2025074157A1 (en) 2023-10-06 2025-04-10 Glaxosmithkline Intellectual Property (No.3) Limited Treatment of a non-alcoholic fatty liver disease
WO2025137384A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Methods and reagents for improved oligonucleotide synthesis
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease
WO2025242321A1 (en) 2024-05-24 2025-11-27 Glaxosmithkline Intellectual Property (No.3) Limited Novel use

Family Cites Families (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US4751219A (en) 1985-02-05 1988-06-14 Nederlandse Centrale Organisatie Voor Toegepast-Natuur-Wetenschappelijk Onderzoek Synthetic glycolipides, a process for the preparation thereof and several uses for these synthetic glycolipides
NL9201440A (nl) * 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US6172045B1 (en) 1994-12-07 2001-01-09 Neorx Corporation Cluster clearing agents
US6908903B1 (en) 1994-12-07 2005-06-21 Aletheon Pharmaceuticals, Inc. Cluster clearing agents
JP2000501414A (ja) 1995-11-22 2000-02-08 ザ・ジョンズ・ホプキンス・ユニバーシティー 生体分子の細胞取り込みを高めるリガンド
US20030119724A1 (en) 1995-11-22 2003-06-26 Ts`O Paul O.P. Ligands to enhance cellular uptake of biomolecules
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6620916B1 (en) 1996-09-26 2003-09-16 Ajinomoto Co., Inc. Modified physiologically active proteins and medicinal compositions containing the same
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
EP1155702A4 (en) 1998-10-30 2004-12-15 Daiichi Seiyaku Co DDS CONNECTIONS AND TEST METHODS
US6383812B1 (en) 1999-05-28 2002-05-07 Academia Sinica Anti liver disease drug R-YEEE and method of synthesizing branched galactose-terminal glycoproteins
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US8541548B2 (en) 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
US6906182B2 (en) 2000-12-01 2005-06-14 Cell Works Therapeutics, Inc. Conjugates of glycosylated/galactosylated peptide, bifunctional linker, and nucleotidic monomers/polymers, and related compositions and method of use
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
EP1389209B1 (en) 2001-04-24 2009-04-08 Purdue Research Foundation Folate mimetics and folate-receptor binding conjugates thereof
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
EP3231445A1 (en) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030148928A1 (en) 2001-07-20 2003-08-07 Leonid Beigelman Enzymatic nucleic acid peptide conjugates
US7439043B2 (en) 2001-10-10 2008-10-21 Neose Technologies, Inc. Galactosyl nucleotide sugars
US20100240730A1 (en) 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
EP1543019A2 (en) 2002-09-11 2005-06-22 Santaris Pharma A/S Modified pna molecules
EP2216407B1 (en) 2003-03-07 2016-01-13 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
CA2488224A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals, Inc. Irna conjugates
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
AU2004233092C9 (en) 2003-04-17 2010-10-28 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
WO2004101619A1 (ja) 2003-05-15 2004-11-25 Shionogi Co., Ltd. 機能的糖ペプチドの合理的設計および合成
US20100267778A1 (en) * 2003-08-04 2010-10-21 Shinya Kusuda Diphenyl ether compound, process for producing the same, and use
JP5192234B2 (ja) 2004-08-10 2013-05-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド 化学修飾オリゴヌクレオチド
US20090203132A1 (en) 2004-09-09 2009-08-13 Swayze Eric E Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1841867A1 (en) 2005-01-24 2007-10-10 Avaris AB COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
ES2395929T3 (es) * 2005-02-25 2013-02-18 Ono Pharmaceutical Co., Ltd. Compuestos de indol para tratar trastornos respiratorios
US8658211B2 (en) 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
EP2129402A2 (en) 2007-02-16 2009-12-09 KTB Tumorforschungsgesellschaft mbH Receptor and antigen targeted prodrug
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
CA2708171C (en) 2007-12-04 2018-02-27 Alnylam Pharmaceuticals, Inc. Folate conjugates
JP2011511004A (ja) 2008-01-31 2011-04-07 アルナイラム ファーマシューティカルズ インコーポレイテッド ＰＣＳＫ９遺伝子を標的とするｄｓＲＮＡを送達するための最適化された方法
WO2009142822A2 (en) 2008-03-26 2009-11-26 Alnylam Pharmaceuticals, Inc. 2-f modified rna interference agents
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
JP2012513953A (ja) 2008-09-23 2012-06-21 アルニラム ファーマスーティカルズ インコーポレイテッド 付加環化を用いたモノマーおよびオリゴヌクレオチドの化学修飾
PT2937418T (pt) 2008-10-20 2018-01-23 Alnylam Pharmaceuticals Inc Composições e métodos de inibição da expressão de transtirretina
EP2358398A2 (en) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
EA037404B1 (ru) 2008-11-10 2021-03-24 Арбутус Биофарма Корпорэйшн Липиды и композиции для доставки лекарственных средств
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
HRP20211619T1 (hr) 2009-06-10 2022-02-04 Arbutus Biopharma Corporation Poboljšana formulacija lipida
WO2011038356A2 (en) 2009-09-25 2011-03-31 Johns Hopkins University Novel liver-targeting agents and their synthesis
TWI388338B (zh) 2009-10-26 2013-03-11 Iner Aec Executive Yuan 對聚合醣鏈進行放射標誌以作為肝受體造影劑之方法
TWI391144B (zh) 2009-10-26 2013-04-01 Iner Aec Executive Yuan 一種定量肝殘餘功能的檢驗方法與其新穎肝受體造影檢驗藥劑
WO2011072290A2 (en) 2009-12-11 2011-06-16 The Regents Of The University Of Michigan Targeted dendrimer-drug conjugates
NZ601737A (en) * 2010-02-24 2013-06-28 Arrowhead Res Corp Compositions for targeted delivery of sirna
US8349308B2 (en) 2010-03-26 2013-01-08 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
US8865675B2 (en) * 2010-05-12 2014-10-21 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
US20130236968A1 (en) * 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
JP2013541334A (ja) 2010-09-15 2013-11-14 アルニラム ファーマスーティカルズ インコーポレイテッド 修飾されたｉＲＮＡ剤
EP3192800A1 (en) * 2010-12-17 2017-07-19 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
BR122020024394B1 (pt) 2010-12-29 2021-05-11 F. Hoffmann-La Roche Ag conjugado e composição farmacêutica
ES2856266T3 (es) 2011-04-21 2021-09-27 Glaxo Group Ltd Modulación de la expresión del virus de la hepatitis B (VHB)
JP2014531476A (ja) 2011-08-26 2014-11-27 アローヘッド リサーチ コーポレイション インビボ核酸送達のためのポリ（ビニルエステル）ポリマー
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
TW201808342A (zh) 2012-05-02 2018-03-16 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
EP3358013B1 (en) 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
CA2879693A1 (en) 2012-08-06 2014-02-13 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated rna agents and process for their preparation
US9228184B2 (en) * 2012-09-29 2016-01-05 Dynavax Technologies Corporation Human toll-like receptor inhibitors and methods of use thereof
RU2015119411A (ru) 2012-11-15 2017-01-10 Рош Инновейшен Сентер Копенгаген А/С Конъюгаты антисмысловых соединений, направленные на аполипопротеин в
WO2014118267A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Lna oligonucleotide carbohydrate conjugates
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
NZ630890A (en) 2013-05-01 2017-09-29 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
EP3019200B1 (en) 2013-07-11 2022-03-23 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
EP3065758B1 (en) 2013-11-06 2019-09-25 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
MX373283B (es) 2014-05-01 2020-05-20 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del receptor de la hormona del crecimiento.
US20170145424A1 (en) 2014-06-06 2017-05-25 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
CN104087574B (zh) * 2014-06-23 2019-08-02 中国人民解放军海军医学研究所 一种用于制备肝细胞靶向转递小干扰rna的方法
WO2016011123A1 (en) * 2014-07-16 2016-01-21 Arrowhead Research Corporation Organic compositions to treat apoc3-related diseases
WO2016044267A1 (en) * 2014-09-15 2016-03-24 Qubole, Inc. Systems and methods for providing metadata-aware background caching in data analysis
SG11201702877TA (en) 2014-10-10 2017-05-30 Hoffmann La Roche Galnac phosphoramidites, nucleic acid conjugates thereof and their use
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
EP3271482A4 (en) 2015-03-17 2019-02-13 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR INHIBITING THE GENE EXPRESSION OF FACTOR XII
CN115957337A (zh) 2015-08-07 2023-04-14 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
NZ744357A (en) * 2016-03-07 2025-09-26 Arrowhead Pharmaceuticals Inc Targeting ligands for therapeutic compounds
JOP20170161A1 (ar) * 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
KR102856522B1 (ko) 2016-09-02 2025-09-08 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
TWI826365B (zh) * 2017-01-10 2023-12-21 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
CN119548520A (zh) * 2017-01-30 2025-03-04 箭头药业股份有限公司 用于抑制因子xii基因表达的组合物和方法
US10597657B2 (en) * 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
WO2019055633A1 (en) * 2017-09-14 2019-03-21 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE
EP3697909A4 (en) * 2017-10-17 2021-10-13 Arrowhead Pharmaceuticals, Inc. RNAI-BASED AGENTS AND COMPOSITIONS INTENDED TO INHIBIT ASIALOGLYCOPROTEIN RECEPTOR 1 EXPRESSION
US12065458B2 (en) * 2018-02-17 2024-08-20 Arrowhead Pharmaceuticals, Inc. Trialkyne linking agents and methods of use
WO2020061177A1 (en) * 2018-09-19 2020-03-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use
MX2022001460A (es) * 2019-08-05 2022-02-22 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de enfermedades y trastornos relacionados con apoc3.
CN115397436B (zh) * 2020-03-26 2025-07-08 箭头药业股份有限公司 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
US11549112B1 (en) * 2021-06-21 2023-01-10 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use

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