MX2014001971A - Polimeros de poli(vinilester) para suministro de acido nucleico in vivo. - Google Patents

Polimeros de poli(vinilester) para suministro de acido nucleico in vivo.

Info

Publication number
MX2014001971A
MX2014001971A MX2014001971A MX2014001971A MX2014001971A MX 2014001971 A MX2014001971 A MX 2014001971A MX 2014001971 A MX2014001971 A MX 2014001971A MX 2014001971 A MX2014001971 A MX 2014001971A MX 2014001971 A MX2014001971 A MX 2014001971A
Authority
MX
Mexico
Prior art keywords
polymers
poly
vinyl ester
nucleic acid
acid delivery
Prior art date
Application number
MX2014001971A
Other languages
English (en)
Spanish (es)
Inventor
Darren H Wakefield
Nicholas Rossi
Dan Sheik
Original Assignee
Arrowhead Res Corp
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 Res Corp filed Critical Arrowhead Res Corp
Publication of MX2014001971A publication Critical patent/MX2014001971A/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
    • 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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (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)
MX2014001971A 2011-08-26 2012-08-23 Polimeros de poli(vinilester) para suministro de acido nucleico in vivo. MX2014001971A (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
MX2014001971A true MX2014001971A (es) 2014-03-31

Family

ID=47756738

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2014001971A MX2014001971A (es) 2011-08-26 2012-08-23 Polimeros de poli(vinilester) para suministro de acido nucleico in vivo.

Country Status (12)

Country Link
US (3) US8932572B2 (OSRAM)
JP (1) JP2014531476A (OSRAM)
KR (1) KR20140051357A (OSRAM)
CN (1) CN104024328A (OSRAM)
AU (1) AU2012300476B2 (OSRAM)
BR (1) BR112014004585A2 (OSRAM)
CA (1) CA2842039A1 (OSRAM)
HK (1) HK1199736A1 (OSRAM)
IL (1) IL231146A0 (OSRAM)
MX (1) MX2014001971A (OSRAM)
PH (1) PH12014500214A1 (OSRAM)
WO (1) WO2013032829A1 (OSRAM)

Families Citing this family (104)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
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
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US8796405B2 (en) 2012-01-18 2014-08-05 Wisconsin Alumni Research Foundation Degradable polycations derived from amino acid vinyl esters
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
CA2842041A1 (en) * 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
CA2902393C (en) 2013-02-28 2022-11-01 Arrowhead Research Corporation Organic compositions to treat epas1-related diseases
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
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
SG11201600379TA (en) 2013-08-07 2016-02-26 Arrowhead Res Corp Polyconjugates for delivery of rnai triggers to tumor cells in vivo
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
DK3137476T3 (da) 2014-04-28 2019-11-18 Ionis Pharmaceuticals Inc Linker-modificerede oligomerforbindelser
PH12016502062B1 (en) 2014-05-01 2023-01-27 Ionis Pharmaceuticals Inc Compositions and methods for modulating complement factor b expression
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.
EP3137605B1 (en) 2014-05-01 2020-10-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
EP4534092A3 (en) 2014-05-01 2025-07-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis 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
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
BR112017025698A2 (pt) 2015-05-29 2018-08-14 Arrowhead Pharmaceuticals Inc composições e métodos para inibir a expressão do gene de hif2alfa
BR112017028194B1 (pt) 2015-07-10 2023-03-14 Ionis Pharmaceuticals, Inc Composto oligomérico modulador de diaciglicerol aciltransferase 2 (dgat2), composição, e seus usos
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
AR106135A1 (es) 2015-09-24 2017-12-13 Ionis Pharmaceuticals Inc Moduladores de la expresión del sarcoma de la rata de kirsten (kras)
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
CA3002822A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Herpes simplex virus vaccine
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
EP3718565B1 (en) 2015-10-22 2022-04-27 ModernaTX, Inc. Respiratory virus vaccines
US11643441B1 (en) 2015-10-22 2023-05-09 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (VZV)
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
KR20180096592A (ko) 2015-10-22 2018-08-29 모더나티엑스, 인크. 호흡기 세포융합 바이러스 백신
KR20250078597A (ko) 2015-11-06 2025-06-02 아이오니스 파마수티컬즈, 인코포레이티드 아포리포프로테인 (a) 발현 조정
SMT202400375T1 (it) 2015-11-06 2024-11-15 Ionis Pharmaceuticals Inc Composti antisenso coniugati per uso in terapia
SI3394093T1 (sl) 2015-12-23 2022-05-31 Modernatx, Inc. Metode uporabe liganda OX40, ki kodira polinukleotid
MA43587A (fr) 2016-01-10 2018-11-14 Modernatx Inc Arnm thérapeutiques codant pour des anticorps anti-ctla-4
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
EP4206213A1 (en) 2016-07-15 2023-07-05 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
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
CN109661233A (zh) 2016-10-06 2019-04-19 Ionis 制药公司 缀合低聚化合物的方法
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
JP2020526192A (ja) 2017-07-06 2020-08-31 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 アルファ−ＥＮａＣの発現を阻害するためのＲＮＡｉ剤、および使用方法
CN111315359A (zh) 2017-08-31 2020-06-19 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
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
CA3088522A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US11332733B2 (en) 2018-02-12 2022-05-17 lonis Pharmaceuticals, Inc. Modified compounds and uses thereof
CU20200082A7 (es) 2018-05-09 2021-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para la reducción de la expresión de fxi
US12496311B2 (en) 2018-07-17 2025-12-16 Aronora, Inc. Methods for safely reducing thrombopoietin
EP3833397A4 (en) 2018-08-08 2023-06-14 Arcturus Therapeutics, Inc. COMPOSITIONS AND AGENTS AGAINST NON-ALCOHOLIC 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
IL283967B2 (en) 2018-12-21 2025-03-01 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
MX2021008261A (es) 2019-01-09 2021-08-16 Arrowhead Pharmaceuticals Inc Agentes de rnai para inhibir la expresion de hif-2 a (epas1), composiciones de estos y metodos de uso.
KR20210130736A (ko) 2019-02-07 2021-11-01 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스 감염에 대한 RNAi 작용제
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
AU2020295796A1 (en) 2019-06-18 2022-02-10 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting rnai
EP4045062A1 (en) 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
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
CA3176196A1 (en) 2020-04-21 2021-10-28 Flagship Pioneering, Inc. Bifunctional molecules and methods of using thereof
AU2021340710A1 (en) 2020-09-11 2023-04-06 Arrowhead Pharmaceuticals, Inc. Skeletal muscle delivery platforms and methods of use
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
WO2022056266A2 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
WO2022109139A1 (en) 2020-11-18 2022-05-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
KR20230167082A (ko) 2021-04-08 2023-12-07 애로우헤드 파마슈티컬스 인코포레이티드 진행성 당화 최종 산물에 대한 수용체의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
KR20240028335A (ko) 2021-04-23 2024-03-05 간엔에이 바이오, 인크. 글리칸 변형 핵산, 제조 방법 및 치료 용도
AU2022280863A1 (en) 2021-05-28 2023-11-30 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of mucin 5ac (muc5ac), 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
MX2024004011A (es) 2021-10-01 2024-07-01 Adarx Pharmaceuticals Inc Composiciones moduladoras de precalicreína y métodos de uso de estas.
US20250327041A1 (en) 2022-02-24 2025-10-23 Io Biotech Aps Nucleotide delivery of cancer therapy
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
WO2024220930A2 (en) 2023-04-20 2024-10-24 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
TW202530408A (zh) 2023-09-29 2025-08-01 美商英西特羅公司 用於治療非酒精性脂肪肝病的組合物與方法
WO2025137384A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Methods and reagents for improved oligonucleotide synthesis
US20250297260A1 (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

Family Cites Families (24)

* 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
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
TW316266B (OSRAM) 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
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
DE69709110T2 (de) 1996-07-10 2002-04-25 Commonwealth Scientific And Industrial Research Organisation, Clayton Polymerisation mit "living" kennzeichen
WO1998047927A1 (en) 1997-04-23 1998-10-29 E.I. Du Pont De Nemours And Company Method of macromonomer synthesis
EP1149075B1 (en) 1997-07-21 2008-12-31 Commonwealth Scientific and Industrial Research Organisation Synthesis of dithioester chain transfer agents
IL136389A0 (en) 1997-12-18 2001-06-14 Du Pont Polymerization process with living characteristics and polymers made therefrom
WO2000075162A1 (en) * 1999-06-07 2000-12-14 Mirus Corporation A compound containing a labile disulfide bond
CN1406140A (zh) * 2000-02-28 2003-03-26 吉倪塞思公司 纳米胶囊包封系统与方法
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
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
EP2500015A1 (en) * 2006-12-05 2012-09-19 Landec Corporation Delivery of drugs
CA2706515C (en) * 2007-11-23 2018-04-17 Robert Liska A polymerization-curable composition for the preparation of biodegradable, biocompatible, cross-linked polymers on a polyvinyl alcohol basis
EP2620161A1 (en) 2008-05-13 2013-07-31 University of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
MX2011004242A (es) 2008-11-06 2011-07-20 Univ Washington Copolimeros multibloque.
AU2010206500B2 (en) 2009-01-23 2014-10-09 Commonwealth Scientific And Industrial Research Organisation Raft polymerisation
NZ601737A (en) 2010-02-24 2013-06-28 Arrowhead Res Corp Compositions for targeted delivery of sirna
US20110224377A1 (en) 2010-03-11 2011-09-15 Mahesh Kalyana Mahanthappa Poly(vinyl ester) block copolymers
US9376523B2 (en) 2010-03-19 2016-06-28 Wisconsin Alumni Research Foundation Poly(vinyl alcohol)-poly(vinyl ester) block copolymers
US20130236968A1 (en) 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
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

Also Published As

Publication number Publication date
US8932572B2 (en) 2015-01-13
CA2842039A1 (en) 2013-03-07
BR112014004585A2 (pt) 2017-06-13
US9089611B2 (en) 2015-07-28
US20130121954A1 (en) 2013-05-16
US20150283256A1 (en) 2015-10-08
IL231146A0 (en) 2014-04-30
PH12014500214A1 (en) 2019-03-22
US9474804B2 (en) 2016-10-25
JP2014531476A (ja) 2014-11-27
CN104024328A (zh) 2014-09-03
HK1199736A1 (en) 2015-07-17
WO2013032829A1 (en) 2013-03-07
US20150110732A1 (en) 2015-04-23
AU2012300476B2 (en) 2016-03-24
KR20140051357A (ko) 2014-04-30
AU2012300476A1 (en) 2014-01-23

Similar Documents

Publication Publication Date Title
PH12014500214A1 (en) Poly (vinyl ester) polymers for in vivo nucleic acid delivery
PH12014500213A1 (en) Poly (acrylate) polymers for in vivo nucleic acid delivery
PH12012501596A1 (en) Compositions for targeted delivery of sirna
WO2012083185A8 (en) Peptide-based in vivo sirna delivery system
WO2012083046A3 (en) Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
MX2016001006A (es) Policonjugados para suministro de desencadenantes de acido ribonucleico de interferencia (arni) en celulas tumorales en vivo.
WO2011113030A3 (en) Human cancer micro-rna expression profiles predictive of chemo-response
MX341118B (es) Cojugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.
AR081868A1 (es) Vectores y secuencias de sulfamidasa humana para el tratamiento de mucopolisacaridosis
WO2012075040A3 (en) mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
MX356625B (es) Oligonucleotidos para elaborar un cambio en la secuencia de una molécula de ácido ribonucleico objetivo presente en una célula viva.
WO2013158309A3 (en) Non-disruptive gene targeting
EP4269584A3 (en) Selective antisense compounds and uses thereof
WO2013096958A8 (en) Compositions and methods for the delivery of biologically active rnas
NZ601247A (en) Targeted genomic alteration
NZ627373A (en) Genes involved in noscapine production
PT3679934T (pt) Indução do gene trail de pequenas moléculas por células normais e tumorais enquanto terapia anticancro
WO2011116152A3 (en) Delivery of agents using interfering nanoparticles
WO2012145729A3 (en) Methods and compositions for modulating gene expression using components that self assemble in cells and produce rnai activity
WO2012006169A3 (en) Compositions and methods for enhancement of nucleic acid delivery
WO2005079532A3 (en) Methods and compositions for enhancing risc activity in vitro and in vivo
MX2013004044A (es) Composiciones de pared de celula de acido ribonucleico bacterial y metodos para hacerlas y usarlas.
WO2009137136A3 (en) In vivo gene sensors
PL390563A1 (pl) Geosiatki komórkowe o obniżonym pełzaniu i wysokiej odporności na wpływy mikrobiologiczne oraz hydrolizę
WO2014039330A8 (en) Transcriptional gene silencing of endogenes in plants