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
compounds
expression
therapeutic
ligands described
useful
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

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
    • C12N15/113—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
    • 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
    • C07H21/04—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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

ID=61281611

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2018009853A MX2018009853A (es) 2016-09-02 2017-03-07 Ligandos de direccion.
MX2023006105A MX2023006105A (es) 2016-09-02 2018-08-14 Ligandos de direccion.
MX2023006109A MX2023006109A (es) 2016-09-02 2018-08-14 Ligandos de direccion.

Family Applications After (2)

Application Number Title Priority Date Filing Date
MX2023006105A MX2023006105A (es) 2016-09-02 2018-08-14 Ligandos de direccion.
MX2023006109A MX2023006109A (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) CN116832169A (es)
AU (3) AU2017320582B2 (es)
CA (1) CA3011668A1 (es)
IL (2) IL264750B2 (es)
JO (2) JOP20170056B1 (es)
MX (3) MX2018009853A (es)
SG (2) SG11201901841TA (es)
TW (2) TWI775743B (es)
UY (1) UY37145A (es)
WO (1) WO2018044350A1 (es)

Families Citing this family (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG194751A1 (en) 2011-06-30 2013-12-30 Arrowhead Res Corp Compositions and methods for inhibiting gene expression of hepatitis b virus
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
CN108271387B (zh) 2015-08-07 2023-06-27 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
CN109069529B (zh) 2016-03-07 2021-08-20 箭头药业股份有限公司 用于治疗性化合物的靶向配体
KR102639586B1 (ko) 2016-06-06 2024-02-23 애로우헤드 파마슈티컬스 인코포레이티드 5'-시클로-포스포네이트 변형된 뉴클레오티드
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
JOP20170056B1 (ar) * 2016-09-02 2021-08-17 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة
IL310548A (en) 2017-01-10 2024-03-01 Arrowhead Pharmaceuticals Inc Alpha-1 antitrypsin (aat) rnai agents, compositions including aat rnai agents, and methods of use
JOP20200054A1 (ar) 2017-09-11 2020-03-10 Arrowhead Pharmaceuticals Inc عوامل RNAi وتركيبات لتثبيط تعبير صَميمُ البروتينِ الشَّحْمِيّ C-III (APOC3)
US10995335B2 (en) 2017-09-14 2021-05-04 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
US11492624B2 (en) 2017-10-17 2022-11-08 Arrowheads Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of Asialoglycoprotein receptor 1
EP3719128B1 (en) 2017-12-01 2025-01-15 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
CN118291456A (zh) 2017-12-01 2024-07-05 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
JP7261494B2 (ja) 2017-12-01 2023-04-20 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
JP7273417B2 (ja) 2017-12-01 2023-05-15 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および複合体ならびに調製方法と使用
KR20250107294A (ko) 2017-12-01 2025-07-11 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
US11633482B2 (en) 2017-12-29 2023-04-25 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
CN111655849B (zh) 2018-08-21 2024-05-10 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
SG11202101698WA (en) 2018-09-19 2021-04-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use
JP7376952B2 (ja) 2018-09-30 2023-11-09 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 ｓｉＲＮＡ複合体及びその調製方法と使用
JP7507495B2 (ja) 2018-12-28 2024-06-28 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
WO2020147847A1 (zh) * 2019-01-18 2020-07-23 苏州瑞博生物技术有限公司 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
CN114206389A (zh) * 2019-03-21 2022-03-18 有丝分裂疗法有限责任公司 用于靶向递送治疗剂的多价配体簇
CN113227376B (zh) 2019-05-22 2024-04-09 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
US12540323B2 (en) 2019-05-22 2026-02-03 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
KR20220019255A (ko) 2019-06-06 2022-02-16 애로우헤드 파마슈티컬스 인코포레이티드 알파-1 항트립신 결핍증 (aatd)의 치료 방법
CN114340664A (zh) 2019-06-18 2022-04-12 爱尔兰詹森科学公司 乙型肝炎病毒(HBV)疫苗和靶向HBV的RNAi的组合
US12290567B2 (en) * 2019-09-03 2025-05-06 Arcturus Therapeutics, Inc. Asialoglycoprotein Receptor mediated delivery of therapeutically active conjugates
PE20221578A1 (es) 2020-03-06 2022-10-06 Aligos Therapeutics Inc Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas
CA3176418A1 (en) * 2020-03-26 2021-09-30 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of pnpla3, pharmaceutical compositions thereof, and methods of use
WO2021198958A1 (en) 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
AU2021251249A1 (en) 2020-04-10 2022-11-10 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
US20230348905A1 (en) 2020-09-15 2023-11-02 Arrowhead Pharmaceuticals, Inc. Methods for the reduction of z-aat protein levels
AU2021373780A1 (en) * 2020-11-05 2023-06-08 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
IL307885A (en) 2021-04-23 2023-12-01 Ganna Bio Inc Nucleic acids with different glycans, preparation methods and therapeutic uses
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
CA3224145A1 (en) * 2021-06-24 2022-12-29 Melissa Ann Bellinger Novel rna therapeutics and uses thereof
WO2023002452A1 (en) * 2021-07-23 2023-01-26 Janssen Pharmaceuticals, Inc. Process for preparation of targeting ligands
US11692001B2 (en) 2021-08-30 2023-07-04 Hongene Biotech Corporation Functionalized n-acetylgalactosamine analogs
WO2023039076A1 (en) 2021-09-08 2023-03-16 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
KR20240099159A (ko) 2021-09-08 2024-06-28 알리고스 테라퓨틱스 인코포레이티드 변형된 짧은 간섭 핵산(sina) 분자 및 이의 용도
MX2024005862A (es) * 2021-11-16 2024-07-10 Shanghai Argo Biopharmaceutical Co Ltd Composiciones y metodos para inhibir la expresion de la proteina angiotensinogeno (agt).
US11993626B2 (en) 2021-12-15 2024-05-28 Hongene Biotech Corporation Functionalized N-acetylgalactosamine analogs
EP4455155A1 (en) * 2022-01-20 2024-10-30 Chengdu Xinzhenghe Pharmaceutical Technology Co. Ltd Targeting ligand and use thereof
AU2023211407A1 (en) 2022-01-30 2024-08-15 Rona Bioscience, Limited Targeting ligand containing n-acetylgalactosamine
AU2023234536A1 (en) 2022-03-16 2024-09-26 Empirico Inc. Galnac compositions for improving sirna bioavailability
US20250283089A1 (en) 2022-04-08 2025-09-11 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
WO2023241591A1 (zh) 2022-06-14 2023-12-21 大睿生物医药科技(上海)有限公司 调控PCSK9基因活性的siRNA分子
CN119421952A (zh) 2022-06-27 2025-02-11 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
AU2023383571A1 (en) 2022-11-18 2025-07-03 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
WO2024197017A2 (en) 2023-03-21 2024-09-26 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
WO2025137390A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of activin receptor-like kinase 7 (alk7), compositions thereof, and methods of use
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
WO2025226980A2 (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.
US6908903B1 (en) 1994-12-07 2005-06-21 Aletheon Pharmaceuticals, Inc. Cluster clearing agents
US6172045B1 (en) 1994-12-07 2001-01-09 Neorx Corporation Cluster clearing agents
WO1997020563A1 (en) 1995-11-22 1997-06-12 The Johns-Hopkins University Ligands to enhance cellular uptake of biomolecules
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
CN1231675A (zh) 1996-09-26 1999-10-13 味之素株式会社 修饰的生理活性蛋白及含有该蛋白的药物组合物
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
CN1661044A (zh) 1998-10-30 2005-08-31 第一制药株式会社 Dds化合物及其测定方法
US6383812B1 (en) 1999-05-28 2002-05-07 Academia Sinica Anti liver disease drug R-YEEE and method of synthesizing branched galactose-terminal glycoproteins
US8541548B2 (en) 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
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
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
EP1309726B2 (en) 2000-03-30 2018-10-03 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
WO2002043771A2 (en) 2000-12-01 2002-06-06 Cell Works Inc. Conjugates of glycosylated/galactosylated peptide
EP1389209B1 (en) 2001-04-24 2009-04-08 Purdue Research Foundation Folate mimetics and folate-receptor binding conjugates thereof
EP2415486B1 (en) 2001-05-18 2017-02-22 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2003008628A2 (en) 2001-07-20 2003-01-30 Ribozyme Pharmacuticals, Inc. 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
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals 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
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
CA2522637C (en) 2003-04-17 2014-01-21 Alnylam Pharmaceuticals, Inc. Modified irna agents
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
EP1661879A4 (en) * 2003-08-04 2006-11-29 Ono Pharmaceutical Co A DIPHENYL ETHER COMPOUND, PROCESS FOR THE PRODUCTION AND USE THEREOF
CA2576233C (en) 2004-08-10 2016-03-15 Alnylam Pharmaceuticals, Inc. Conjugate comprising an antagomir and a ligand
WO2006031461A2 (en) 2004-09-09 2006-03-23 Isis Pharmaceuticals, Inc. 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
NZ560513A (en) * 2005-02-25 2010-07-30 Ono Pharmaceutical Co Indole compound and use thereof
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
US8658211B2 (en) 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
EP2604284A1 (en) 2007-02-16 2013-06-19 KTB Tumorforschungsgesellschaft mbH Receptor And Antigen Targeted Prodrug
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
CA2930393C (en) 2007-12-04 2022-11-29 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
EP2231194B1 (en) 2007-12-04 2017-02-22 Alnylam Pharmaceuticals Inc. Folate-irna conjugates
AU2009241591A1 (en) 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of DSRNA targeting the PCSK9 gene
WO2009120878A2 (en) 2008-03-26 2009-10-01 Alnylam Pharmaceuticals, Inc. Non-natural ribonucleotides, and methods of use thereof
AU2009234266B2 (en) 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
CA2737661C (en) 2008-09-23 2019-08-20 Alnylam Pharmaceuticals, Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
IL302142B2 (en) 2008-10-20 2024-07-01 Alnylam Pharmaceuticals Inc Compounds and methods for inhibiting transthyretin expression
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
CA3033577A1 (en) 2008-11-10 2010-05-14 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
WO2010129709A1 (en) 2009-05-05 2010-11-11 Alnylam Pharmaceuticals, Inc. Lipid compositions
SMT201800499T1 (it) 2009-06-10 2018-11-09 Arbutus Biopharma Corp Formulazione lipidica migliorata
US8552163B2 (en) 2009-09-25 2013-10-08 Johns Hopkins University Liver-targeting agents and their synthesis
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
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
WO2011072290A2 (en) 2009-12-11 2011-06-16 The Regents Of The University Of Michigan Targeted dendrimer-drug conjugates
AU2011219941C1 (en) * 2010-02-24 2015-05-07 Arrowhead Pharmaceuticals, Inc. 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
WO2011163121A1 (en) * 2010-06-21 2011-12-29 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
AU2011343664B2 (en) * 2010-12-17 2015-10-08 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
BR122020024388B1 (pt) 2010-12-29 2021-09-21 F. Hoffmann-La Roche Ag Oligonucleotídeo e composição farmacêutica
UA118951C2 (uk) 2011-04-21 2019-04-10 Айоніс Фармасьютікалз, Інк. Модулювання експресії вірусу гепатиту b (hbv)
BR112014004585A2 (pt) 2011-08-26 2017-06-13 Arrowhead Res Corp polímeros de poli(éster vinílico) para liberação de ácido nucleico in vivo
EP3453761A1 (en) 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2844261B1 (en) 2012-05-02 2018-10-17 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
TWI595885B (zh) 2012-05-02 2017-08-21 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
ES2947520T3 (es) 2012-08-06 2023-08-10 Alnylam Pharmaceuticals Inc Proceso para la preparación de agentes de ARN conjugados con carbohidratos
US9228184B2 (en) * 2012-09-29 2016-01-05 Dynavax Technologies Corporation Human toll-like receptor inhibitors and methods of use thereof
DK2920307T3 (en) 2012-11-15 2018-07-16 Roche Innovation Ct Copenhagen As ANTI-APOB, ANTISENSE CONJUGATE RELATIONSHIPS
DK2951305T3 (en) 2013-01-30 2018-10-29 Hoffmann La Roche LNA oligonucleotide KULHYDRATKONJUGATER
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
KR20160002977A (ko) 2013-05-01 2016-01-08 아이시스 파마수티컬즈 인코포레이티드 조성물 및 방법
EP2992095B1 (en) 2013-05-01 2019-01-09 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-122
CA3177846A1 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
WO2015069587A2 (en) 2013-11-06 2015-05-14 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
RU2724527C2 (ru) 2014-05-01 2020-06-23 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии рецептора гормона роста
EP3151839A4 (en) 2014-06-06 2018-02-28 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
EP3169784B1 (en) * 2014-07-16 2020-06-10 Arrowhead Pharmaceuticals, Inc. Rnai 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
BR112017005938A2 (pt) 2014-10-10 2017-12-12 Hoffmann La Roche compostos, processo de preparação de composto, processo de preparação de conjugado de galnac e ácido nucleico e usos de composto
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
WO2016149331A2 (en) 2015-03-17 2016-09-22 Arrowhead Research Corporation Compositions and methods for inhibiting gene expression of factor xii
CN108271387B (zh) 2015-08-07 2023-06-27 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
CN109069529B (zh) 2016-03-07 2021-08-20 箭头药业股份有限公司 用于治疗性化合物的靶向配体
JOP20170161A1 (ar) * 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
JOP20170056B1 (ar) 2016-09-02 2021-08-17 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة
IL310548A (en) * 2017-01-10 2024-03-01 Arrowhead Pharmaceuticals Inc Alpha-1 antitrypsin (aat) rnai agents, compositions including aat rnai agents, and methods of use
CA3048661A1 (en) * 2017-01-30 2018-08-02 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor xii gene expression
JOP20200054A1 (ar) * 2017-09-11 2020-03-10 Arrowhead Pharmaceuticals Inc عوامل RNAi وتركيبات لتثبيط تعبير صَميمُ البروتينِ الشَّحْمِيّ C-III (APOC3)
US10995335B2 (en) * 2017-09-14 2021-05-04 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
US11492624B2 (en) * 2017-10-17 2022-11-08 Arrowheads Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of Asialoglycoprotein receptor 1
US12065458B2 (en) * 2018-02-17 2024-08-20 Arrowhead Pharmaceuticals, Inc. Trialkyne linking agents and methods of use
SG11202101698WA (en) * 2018-09-19 2021-04-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use
EP4010075A4 (en) * 2019-08-05 2023-11-15 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of apoc3-related diseases and disorders
CA3176418A1 (en) * 2020-03-26 2021-09-30 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of pnpla3, pharmaceutical compositions thereof, and methods of use
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

Also Published As

Publication number Publication date
TW202320855A (zh) 2023-06-01
CA3011668A1 (en) 2018-03-08
AU2023255025A1 (en) 2023-11-16
US20190256849A1 (en) 2019-08-22
JP2024116406A (ja) 2024-08-27
KR20220077157A (ko) 2022-06-08
AU2017320582A1 (en) 2019-03-21
US10294474B2 (en) 2019-05-21
TW201811375A (zh) 2018-04-01
EP3506913A1 (en) 2019-07-10
SG11201901841TA (en) 2019-03-28
US20180064819A1 (en) 2018-03-08
BR112019004178A2 (pt) 2019-05-28
CN109462981A (zh) 2019-03-12
AU2017320582B2 (en) 2023-11-16
TWI775743B (zh) 2022-09-01
IL300869A (en) 2023-04-01
JP2022028920A (ja) 2022-02-16
US11174481B2 (en) 2021-11-16
KR102856522B1 (ko) 2025-09-08
JP7510403B2 (ja) 2024-07-03
CN109462981B (zh) 2023-07-07
JP2019526527A (ja) 2019-09-19
NZ751214A (en) 2025-05-02
IL300869B1 (en) 2026-01-01
KR20190043132A (ko) 2019-04-25
MX2023006109A (es) 2023-06-08
JOP20170056B1 (ar) 2021-08-17
JOP20210207A1 (ar) 2023-01-30
CN116942841A (zh) 2023-10-27
KR20230115344A (ko) 2023-08-02
AU2023255025B2 (en) 2025-09-25
JP6989521B2 (ja) 2022-01-05
WO2018044350A1 (en) 2018-03-08
SG10201912835QA (en) 2020-02-27
KR102403408B1 (ko) 2022-05-30
CN116832169A (zh) 2023-10-03
IL264750A (es) 2019-03-31
US20220033817A1 (en) 2022-02-03
UY37145A (es) 2019-08-30
IL264750B2 (en) 2023-08-01
MX2023006105A (es) 2023-06-08
AU2025275263A1 (en) 2026-01-15
EP3506913A4 (en) 2020-06-10
KR102557906B1 (ko) 2023-07-20
IL264750B1 (en) 2023-04-01

Similar Documents

Publication Publication Date Title
MX2018009853A (es) Ligandos de direccion.
JOP20210231A1 (ar) مركبات ترابطية مستهدفة كمركبات علاجية
WO2017053431A3 (en) Allele selective gene editing and uses thereof
MX2019005816A (es) Agentes de arn modificados con efecto reducido fuera de la diana.
ECSP18083519A (es) Inhibidores de bromodominios
MX2017002144A (es) Agentes de ácido ribonucleico (arn) de cadena doble modificados.
EA201500394A1 (ru) Ингибиторы деметилаз гистонов
BR112013026494A2 (pt) composto, composição farmacêutica, método para tratar condição
HK1208361A1 (en) Multi-target modulation for treating fibrosis and inflammatory conditions
BR112012023421A2 (pt) composição farmacêutica para tratamento de aumento de pressão intraocular
MX2016001587A (es) Inhibidores de kdm1a para el tratamiento de enfermedades.
NZ732777A (en) Ionizable cationic lipid for rna delivery
BR112015004747A2 (pt) moléculas oligonucleotídicas de fita dupla para p53 e métodos de uso das mesmas
BR112016029781A2 (pt) composições e métodos para inibir a expressão de gene de alfa-1 antitripsina
MX378277B (es) Metodo para tratar cancer usando una combinacion de inhibidores de chk1 y relacionado con atm y rad3 (atr).
EA201792103A1 (ru) Композиции и способы для ингибирования экспрессии генов hif2альфа
PH12013500449A1 (en) Treatment of diseases
MX2023008469A (es) Oligonucleotidos de doble hebra modificados.
EP3804725A3 (en) Nanomaterial compositions, synthesis, and assembly
BR112017009552A8 (pt) Métodos para alvejar o controle transcricional em regiões de super-realçador
MX2021015188A (es) Moduladores de nueva generacion del estimulador de genes de interferon (sting).
MX379585B (es) Composición para usarse en el tratamiento de cáncer que presenta pérdida hemicigota de tp53.
PE20242070A1 (es) Mitocetoscinas: agentes terapeuticos basados en mitocondrias que fijan como objetivo el metabolismo de cetonas en celulas cancerosas
BR112017016883A2 (pt) compostos com atividade antitumoral
BR112015010196A2 (pt) métodos de tratar doença do fígado