JP2014531476A - 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 - Google Patents

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 Download PDF

Info

Publication number
JP2014531476A
JP2014531476A JP2014527280A JP2014527280A JP2014531476A JP 2014531476 A JP2014531476 A JP 2014531476A JP 2014527280 A JP2014527280 A JP 2014527280A JP 2014527280 A JP2014527280 A JP 2014527280A JP 2014531476 A JP2014531476 A JP 2014531476A
Authority
JP
Japan
Prior art keywords
polymer
poly
vinyl ester
random copolymer
polynucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2014527280A
Other languages
English (en)
Japanese (ja)
Other versions
JP2014531476A5 (cg-RX-API-DMAC7.html
Inventor
ダレン エイチ． ウェークフィールド
ダレン エイチ． ウェークフィールド
ニコラス ロッシ
ニコラス ロッシ
ダン シーク
ダン シーク
Original Assignee
アローヘッド リサーチ コーポレイション
アローヘッド リサーチ コーポレイション
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 アローヘッド リサーチ コーポレイション, アローヘッド リサーチ コーポレイション filed Critical アローヘッド リサーチ コーポレイション
Publication of JP2014531476A publication Critical patent/JP2014531476A/ja
Publication of JP2014531476A5 publication Critical patent/JP2014531476A5/ja
Pending legal-status Critical Current

Links

Images

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
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F20/00—Homopolymers and copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and only one being terminated by only one carboxyl radical or a salt, anhydride, ester, amide, imide or nitrile thereof
    • C08F20/02—Monocarboxylic acids having less than ten carbon atoms, Derivatives thereof
    • C08F20/10—Esters
    • C08F20/34—Esters containing nitrogen, e.g. N,N-dimethylaminoethyl (meth)acrylate
    • 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
    • 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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/58—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
    • 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
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F218/00—Copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and at least one being terminated by an acyloxy radical of a saturated carboxylic acid, of carbonic acid or of a haloformic acid
    • C08F218/02—Esters of monocarboxylic acids
    • C08F218/04—Vinyl esters
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F218/00—Copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and at least one being terminated by an acyloxy radical of a saturated carboxylic acid, of carbonic acid or of a haloformic acid
    • C08F218/02—Esters of monocarboxylic acids
    • C08F218/04—Vinyl esters
    • C08F218/10—Vinyl esters of monocarboxylic acids containing three or more carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F220/00—Copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and only one being terminated by only one carboxyl radical or a salt, anhydride ester, amide, imide or nitrile thereof
    • C08F220/02—Monocarboxylic acids having less than ten carbon atoms; Derivatives thereof
    • C08F220/10—Esters
    • C08F220/34—Esters containing nitrogen, e.g. N,N-dimethylaminoethyl (meth)acrylate
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08L—COMPOSITIONS OF MACROMOLECULAR COMPOUNDS
    • C08L33/00—Compositions of homopolymers or copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and only one being terminated by only one carboxyl radical, or of salts, anhydrides, esters, amides, imides or nitriles thereof; Compositions of derivatives of such polymers
    • C08L33/04—Homopolymers or copolymers of esters
    • C08L33/06—Homopolymers or copolymers of esters of esters containing only carbon, hydrogen and oxygen, which oxygen atoms are present only as part of the carboxyl radical
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F218/00—Copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and at least one being terminated by an acyloxy radical of a saturated carboxylic acid, of carbonic acid or of a haloformic acid
    • C08F218/22—Esters containing nitrogen
    • 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
    • C12N2310/35—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Polymers & Plastics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Addition Polymer Or Copolymer, Post-Treatments, Or Chemical Modifications (AREA)
JP2014527280A 2011-08-26 2012-08-23 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 Pending JP2014531476A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161527703P 2011-08-26 2011-08-26
US61/527,703 2011-08-26
PCT/US2012/051968 WO2013032829A1 (en) 2011-08-26 2012-08-23 Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US13/592,393 2012-08-23
US13/592,393 US8932572B2 (en) 2011-08-26 2012-08-23 Poly(vinyl ester) polymers for in vivo nucleic acid delivery

Publications (2)

Publication Number Publication Date
JP2014531476A true JP2014531476A (ja) 2014-11-27
JP2014531476A5 JP2014531476A5 (cg-RX-API-DMAC7.html) 2015-09-17

Family

ID=47756738

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2014527280A Pending JP2014531476A (ja) 2011-08-26 2012-08-23 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

Country Status (12)

Country Link
US (3) US8932572B2 (cg-RX-API-DMAC7.html)
JP (1) JP2014531476A (cg-RX-API-DMAC7.html)
KR (1) KR20140051357A (cg-RX-API-DMAC7.html)
CN (1) CN104024328A (cg-RX-API-DMAC7.html)
AU (1) AU2012300476B2 (cg-RX-API-DMAC7.html)
BR (1) BR112014004585A2 (cg-RX-API-DMAC7.html)
CA (1) CA2842039A1 (cg-RX-API-DMAC7.html)
HK (1) HK1199736A1 (cg-RX-API-DMAC7.html)
IL (1) IL231146A0 (cg-RX-API-DMAC7.html)
MX (1) MX2014001971A (cg-RX-API-DMAC7.html)
PH (1) PH12014500214A1 (cg-RX-API-DMAC7.html)
WO (1) WO2013032829A1 (cg-RX-API-DMAC7.html)

Families Citing this family (104)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
MX2013003681A (es) 2010-10-01 2013-11-20 Moderna Therapeutics Inc Ácidos nucleicos manipulados y métodos de uso de los mismos.
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
HK1199736A1 (en) * 2011-08-26 2015-07-17 箭头研究公司 Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US9428535B2 (en) 2011-10-03 2016-08-30 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
SMT202200355T1 (it) 2011-12-16 2022-11-18 Modernatx Inc Composizioni di mrna modificato
US8796405B2 (en) * 2012-01-18 2014-08-05 Wisconsin Alumni Research Foundation Degradable polycations derived from amino acid vinyl esters
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
EP2834260A4 (en) 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US8933047B2 (en) * 2012-04-18 2015-01-13 Arrowhead Madison Inc. Poly(acrylate) polymers for in vivo nucleic acid delivery
EP2922554B1 (en) 2012-11-26 2022-02-23 ModernaTX, Inc. Terminally modified rna
HK1214301A1 (zh) 2013-02-28 2016-07-22 箭头研究公司 治療epas1相關疾病的有機組合物
IL303425A (en) * 2013-03-13 2023-08-01 Cour Pharmaceuticals Dev Company Secondary vaccine particles for the treatment of inflammation
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
HUE050394T2 (hu) 2013-05-01 2020-11-30 Ionis Pharmaceuticals Inc Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények
EP3030244B1 (en) 2013-08-07 2021-04-21 Arrowhead Pharmaceuticals, Inc. Polyconjugates for delivery of rnai triggers to tumor cells in vivo
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低密度リポタンパク質受容体をコードするポリヌクレオチド
US9926556B2 (en) 2014-04-28 2018-03-27 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
KR20200102553A (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
AU2015252895B2 (en) 2014-05-01 2021-07-15 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
CN106232804B (zh) 2014-05-01 2019-10-25 Ionis制药公司 用于调节补体因子b表达的组合物和方法
EP4534092A3 (en) 2014-05-01 2025-07-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
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
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
MX2017014641A (es) 2015-05-29 2018-01-23 Arrowhead Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen de factor inducible por hipoxia alfa (hif2alfa).
AU2016294347B2 (en) 2015-07-10 2022-07-28 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (DGAT2)
JP7336191B2 (ja) 2015-08-07 2023-08-31 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 Ｂ型肝炎ウイルス感染に対するＲＮＡｉ療法
RU2018113709A (ru) 2015-09-24 2019-10-30 Айонис Фармасьютикалз, Инк. Модуляторы экспрессии kras
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
CN118846026A (zh) 2015-10-22 2024-10-29 摩登纳特斯有限公司 广谱流感病毒疫苗
TN2018000152A1 (en) 2015-10-22 2019-10-04 Modernatx Inc Nucleic acid vaccines for varicella zoster virus (vzv)
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス， インコーポレイテッド ヒトサイトメガロウイルスワクチン
AU2016342049B2 (en) 2015-10-22 2023-05-18 Modernatx, Inc. Herpes simplex virus vaccine
JP7687767B2 (ja) 2015-10-22 2025-06-03 モデルナティエックス インコーポレイテッド 呼吸器合胞体ウイルスワクチン
EP4349405A3 (en) 2015-10-22 2024-06-19 ModernaTX, Inc. Respiratory virus vaccines
EP3370708A4 (en) 2015-11-06 2019-06-26 Ionis Pharmaceuticals, Inc. MODULATION OF APOLIPOPROTEIN (A) EXPRESSION
FI4119569T3 (fi) 2015-11-06 2024-08-30 Ionis Pharmaceuticals Inc Konjugoituja antisense-yhdisteitä käytettäviksi hoidossa
LT3394093T (lt) 2015-12-23 2022-04-25 Modernatx, Inc. Ox40 ligandus koduojančių polinukleotidų naudojimo būdai
WO2017120612A1 (en) 2016-01-10 2017-07-13 Modernatx, Inc. Therapeutic mrnas encoding anti ctla-4 antibodies
US10246709B2 (en) 2016-03-07 2019-04-02 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
CN109526222B (zh) 2016-06-06 2022-04-29 箭头药业股份有限公司 5’-环膦酸酯修饰核苷酸
MX2019000619A (es) 2016-07-15 2019-08-01 Ionis Pharmaceuticals Inc Compuestos y metodos para la modulacion de smn2.
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
CN116942841A (zh) 2016-09-02 2023-10-27 箭头药业股份有限公司 靶向配体
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
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
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
CR20190572A (es) 2017-07-06 2020-05-23 Arrowhead Pharmaceuticals Inc AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO
CA3073211A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. Methods of making lipid nanoparticles
PE20201283A1 (es) 2017-09-11 2020-11-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3)
SG11201912188RA (en) 2017-09-14 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
AU2018352379A1 (en) 2017-10-17 2020-06-04 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of Asialoglycoprotein receptor 1
BR112020014425A2 (pt) 2018-01-15 2020-12-29 Ionis Pharmaceuticals, Inc. Moduladores de expressão de dnm2
WO2019157531A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CR20200605A (es) 2018-05-09 2021-01-29 Ionis Pharmaceuticals Inc Compuestos y métodos para la reducción de la expresión de fxi
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
WO2020033748A1 (en) 2018-08-08 2020-02-13 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis
TWI869213B (zh) 2018-09-19 2025-01-01 美商Ionis製藥公司 Pnpla3表現之調節劑
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
AU2019403447B2 (en) 2018-12-21 2023-07-27 Ionis Pharmaceuticals, Inc. Modulators of HSD17B13 expression
KR20210113273A (ko) 2019-01-09 2021-09-15 애로우헤드 파마슈티컬스 인코포레이티드 HIF-2 알파 (EPAS1)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
AU2020219355A1 (en) 2019-02-07 2021-09-30 Arrowhead Pharmaceuticals, Inc. RNAi agents for Hepatitis B virus infection
KR20220042116A (ko) 2019-06-18 2022-04-04 얀센 사이언시즈 아일랜드 언리미티드 컴퍼니 B형 간염 바이러스(HBV) 백신 및 HBV-타케팅 RNAi의 조합
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
CN114728017B (zh) 2019-10-14 2025-10-28 阿斯利康(瑞典)有限公司 Pnpla3表达的调节剂
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
TW202202153A (zh) 2020-03-26 2022-01-16 美商艾羅海德製藥公司 用於抑制ＰＮＰＬＡ３表現之ＲＮＡｉ劑、其醫藥組成物及使用方法
IL297482A (en) 2020-04-21 2022-12-01 Flagship Pioneering Inc Bifunctional molecules and methods of using thereof
JP2023540806A (ja) 2020-09-11 2023-09-26 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 骨格筋への送達プラットフォームおよび使用の方法
TW202227135A (zh) 2020-09-11 2022-07-16 美商愛羅海德製藥公司 用於遞送治療劑之脂質結合物
KR20230064620A (ko) 2020-09-11 2023-05-10 애로우헤드 파마슈티컬스 인코포레이티드 DUX4의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
ES2990026T3 (es) 2020-11-18 2024-11-28 Ionis Pharmaceuticals Inc Compuestos y métodos para modular la expresión del angiotensinógeno
IL307421A (en) 2021-04-08 2023-12-01 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use
IL307885A (en) 2021-04-23 2023-12-01 Ganna Bio Inc Nucleic acids with different glycans, preparation methods and therapeutic uses
IL308883A (en) 2021-05-28 2024-01-01 Arrowhead Pharmaceuticals Inc RNAi AGENTS FOR INHIBITING EXPRESSION OF MUCIN 5AC (MUC5AC), COMPOSITIONS THEREOF, AND METHODS OF USE
US11629349B2 (en) 2021-06-21 2023-04-18 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
US20250115915A1 (en) 2021-10-01 2025-04-10 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
JP2025508467A (ja) 2022-02-24 2025-03-26 アイオー バイオテック エーピーエス 癌治療のヌクレオチド送達
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
JP2025522433A (ja) 2022-06-15 2025-07-15 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 スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法
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
AU2024258561A1 (en) 2023-04-20 2025-10-30 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
AU2024279767A1 (en) 2023-05-26 2025-12-04 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
WO2024263694A1 (en) 2023-06-20 2024-12-26 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
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
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
WO2025072748A2 (en) 2023-09-29 2025-04-03 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
US12442002B2 (en) 2023-12-20 2025-10-14 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of activin receptor-like kinase 7 (ALK7), compositions thereof, and methods of use
WO2025196505A2 (en) 2024-03-22 2025-09-25 Takeda Pharmaceutical Company Limited Compositions and methods for inhibiting cytochrome p450 family 7 subfamily a member 1 (cyp7a1) expression
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0359003A (ja) * 1989-07-27 1991-03-14 Mitsubishi Kasei Corp 酸感受性ポリマー
JPH0931057A (ja) * 1995-07-21 1997-02-04 Eiweiss Kk プロリン骨格を有するビニルエーテル、その製法およびその重合体ならびに共重合体
JP2003501440A (ja) * 1999-06-07 2003-01-14 マイラス コーポレーション 反応活性なジスルフィド結合を含む化合物
JP2003524654A (ja) * 2000-02-28 2003-08-19 ジーンシーグス， インコーポレイテッド ナノカプセルカプセル化システムおよび方法
JP2010511713A (ja) * 2006-12-05 2010-04-15 ランデック コーポレイション 薬物送達
JP2010521133A (ja) * 2006-08-18 2010-06-24 エフ．ホフマン−ラ ロシュ アーゲー ポリヌクレオチドのインビボ送達のためのポリ結合体
JP2011505179A (ja) * 2007-11-23 2011-02-24 テヒニーシェ ウニヴェルジテート ウィーン ポリビニルアルコールベースの生分解性、生体適合性、架橋ポリマーの調製用重合硬化性組成物
US20110207799A1 (en) * 2010-02-24 2011-08-25 Roche Madison Inc. Compositions for Targeted Delivery of siRNA
US20110224377A1 (en) * 2010-03-11 2011-09-15 Mahesh Kalyana Mahanthappa Poly(vinyl ester) block copolymers

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
TW316266B (cg-RX-API-DMAC7.html) 1993-07-30 1997-09-21 Commw Scient Ind Res Org
US6291620B1 (en) 1994-11-09 2001-09-18 E. I. Du Pont De Nemours And Company Polymer synthesis
KR100479628B1 (ko) 1996-07-10 2005-04-06 이.아이,듀우판드네모아앤드캄파니 리빙 특성을 갖는 중합 방법
BR9809294A (pt) 1997-04-23 2000-07-04 Du Pont Processo para a sìntese de polìmeros
NZ508106A (en) 1997-07-21 2002-06-28 A one pot synthesis of CTAs (chain transfer agents) and narrow polydiversity polymer by reacting with free radicals
KR100589073B1 (ko) 1997-12-18 2006-06-13 이 아이 듀폰 디 네모아 앤드 캄파니 리빙 특성의 중합 방법 및 이 방법으로 제조된 중합체
US8138383B2 (en) * 2002-03-11 2012-03-20 Arrowhead Madison Inc. Membrane active heteropolymers
US7816337B2 (en) * 2003-02-18 2010-10-19 Roche Madison Inc. Reversible attachment of a membrane active polymer to a polynucleotide
WO2009140427A2 (en) 2008-05-13 2009-11-19 University Of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
US9464300B2 (en) 2008-11-06 2016-10-11 University Of Washington Multiblock copolymers
CN102361889B (zh) 2009-01-23 2014-09-17 联邦科学与工业研究组织 Raft聚合
WO2011115641A1 (en) 2010-03-19 2011-09-22 Wisconsin Alumni Research Foundation Poly(vinyl alcohol) -poly(vinyl ester) block copolymers
WO2011163121A1 (en) 2010-06-21 2011-12-29 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
HK1199736A1 (en) * 2011-08-26 2015-07-17 箭头研究公司 Poly(vinyl ester) polymers for in vivo nucleic acid delivery

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0359003A (ja) * 1989-07-27 1991-03-14 Mitsubishi Kasei Corp 酸感受性ポリマー
JPH0931057A (ja) * 1995-07-21 1997-02-04 Eiweiss Kk プロリン骨格を有するビニルエーテル、その製法およびその重合体ならびに共重合体
JP2003501440A (ja) * 1999-06-07 2003-01-14 マイラス コーポレーション 反応活性なジスルフィド結合を含む化合物
JP2003524654A (ja) * 2000-02-28 2003-08-19 ジーンシーグス， インコーポレイテッド ナノカプセルカプセル化システムおよび方法
JP2010521133A (ja) * 2006-08-18 2010-06-24 エフ．ホフマン−ラ ロシュ アーゲー ポリヌクレオチドのインビボ送達のためのポリ結合体
JP2010511713A (ja) * 2006-12-05 2010-04-15 ランデック コーポレイション 薬物送達
JP2011505179A (ja) * 2007-11-23 2011-02-24 テヒニーシェ ウニヴェルジテート ウィーン ポリビニルアルコールベースの生分解性、生体適合性、架橋ポリマーの調製用重合硬化性組成物
US20110207799A1 (en) * 2010-02-24 2011-08-25 Roche Madison Inc. Compositions for Targeted Delivery of siRNA
US20110224377A1 (en) * 2010-03-11 2011-09-15 Mahesh Kalyana Mahanthappa Poly(vinyl ester) block copolymers

Also Published As

Publication number Publication date
US20150283256A1 (en) 2015-10-08
CN104024328A (zh) 2014-09-03
AU2012300476B2 (en) 2016-03-24
PH12014500214A1 (en) 2019-03-22
US20150110732A1 (en) 2015-04-23
WO2013032829A1 (en) 2013-03-07
US9089611B2 (en) 2015-07-28
KR20140051357A (ko) 2014-04-30
IL231146A0 (en) 2014-04-30
BR112014004585A2 (pt) 2017-06-13
CA2842039A1 (en) 2013-03-07
HK1199736A1 (en) 2015-07-17
US20130121954A1 (en) 2013-05-16
US8932572B2 (en) 2015-01-13
AU2012300476A1 (en) 2014-01-23
US9474804B2 (en) 2016-10-25
MX2014001971A (es) 2014-03-31

Similar Documents

Publication Publication Date Title
JP2014531476A (ja) 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
US9452221B2 (en) Poly(acrylate) polymers for in vivo nucleic acid delivery
CN103547272B (zh) 基于肽的体内siRNA递送系统
AU2011219941C1 (en) Compositions for targeted delivery of siRNA
CA2816041C (en) In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
EP2807216A1 (en) Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EP2838544A1 (en) Poly(acrylate) polymers for in vivo nucleic acid delivery
HK1194691A (en) Poly(acrylate) polymers for in vivo nucleic acid delivery
HK1191566A (en) Peptide-based in vivo sirna delivery system
HK1191566B (en) Peptide-based in vivo sirna delivery system
HK1188947B (en) In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
HK1188947A (en) In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

Legal Events

Date Code Title Description
RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20150317

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150728

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20150728

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20160720

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20160801

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20161031

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20170330