PH12014500214A1 - Poly (vinyl ester) polymers for in vivo nucleic acid delivery - Google Patents

Poly (vinyl ester) polymers for in vivo nucleic acid delivery

Info

Publication number
PH12014500214A1
PH12014500214A1 PH1/2014/500214A PH12014500214A PH12014500214A1 PH 12014500214 A1 PH12014500214 A1 PH 12014500214A1 PH 12014500214 A PH12014500214 A PH 12014500214A PH 12014500214 A1 PH12014500214 A1 PH 12014500214A1
Authority
PH
Philippines
Prior art keywords
polymers
poly
vinyl ester
nucleic acid
acid delivery
Prior art date
Application number
PH1/2014/500214A
Other languages
English (en)
Inventor
Nicholas Rossi
Dan Sheik
Darren H Wakefield
Original Assignee
Arrowhead Res Corporation
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 Corporation filed Critical Arrowhead Res Corporation
Publication of PH12014500214A1 publication Critical patent/PH12014500214A1/en

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    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08FMACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F20/00Homopolymers 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
    • C08F20/02Monocarboxylic acids having less than ten carbon atoms, Derivatives thereof
    • C08F20/10Esters
    • C08F20/34Esters containing nitrogen, e.g. N,N-dimethylaminoethyl (meth)acrylate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/58Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08FMACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F218/00Copolymers 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
    • C08F218/02Esters of monocarboxylic acids
    • C08F218/04Vinyl esters
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08FMACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F218/00Copolymers 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
    • C08F218/02Esters of monocarboxylic acids
    • C08F218/04Vinyl esters
    • C08F218/10Vinyl esters of monocarboxylic acids containing three or more carbon atoms
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08FMACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F220/00Copolymers 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
    • C08F220/02Monocarboxylic acids having less than ten carbon atoms; Derivatives thereof
    • C08F220/10Esters
    • C08F220/34Esters containing nitrogen, e.g. N,N-dimethylaminoethyl (meth)acrylate
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08LCOMPOSITIONS OF MACROMOLECULAR COMPOUNDS
    • C08L33/00Compositions 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
    • C08L33/04Homopolymers or copolymers of esters
    • C08L33/06Homopolymers or copolymers of esters of esters containing only carbon, hydrogen and oxygen, which oxygen atoms are present only as part of the carboxyl radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08FMACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F218/00Copolymers 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
    • C08F218/22Esters containing nitrogen
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

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  • 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)
PH1/2014/500214A 2011-08-26 2012-08-23 Poly (vinyl ester) polymers for in vivo nucleic acid delivery PH12014500214A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161527703P 2011-08-26 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 US8932572B2 (en) 2011-08-26 2012-08-23 Poly(vinyl ester) polymers for in vivo nucleic acid delivery

Publications (1)

Publication Number Publication Date
PH12014500214A1 true PH12014500214A1 (en) 2019-03-22

Family

ID=47756738

Family Applications (1)

Application Number Title Priority Date Filing Date
PH1/2014/500214A PH12014500214A1 (en) 2011-08-26 2012-08-23 Poly (vinyl ester) polymers for in vivo nucleic acid delivery

Country Status (12)

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

Families Citing this family (102)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP3431485B2 (en) 2010-10-01 2024-09-04 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
MX2014001971A (es) * 2011-08-26 2014-03-31 Arrowhead Res Corp Polimeros de poli(vinilester) para suministro de acido nucleico in vivo.
EP2751270B1 (en) 2011-08-29 2018-08-22 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
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
KR20140102759A (ko) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
US8796405B2 (en) 2012-01-18 2014-08-05 Wisconsin Alumni Research Foundation Degradable polycations derived from amino acid vinyl esters
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
MX2014001965A (es) * 2012-04-18 2014-03-31 Arrowhead Res Corp Polimeros de poli(acrilato) para suministro de acido nucleico in vivo.
CA2892529C (en) 2012-11-26 2023-04-25 Moderna Therapeutics, Inc. Terminally modified rna
US9868949B2 (en) 2013-02-28 2018-01-16 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat EPAS1-related diseases
HK1220368A1 (zh) 2013-03-13 2017-05-05 Cour Pharmaceuticals Development Company 用於治疗炎症的免疫修饰性颗粒
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, 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
KR102482890B1 (ko) 2013-05-01 2022-12-30 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법
CN105451743B (zh) 2013-08-07 2019-11-19 箭头研究公司 用于在活体中递送RNAi触发子至肿瘤细胞的聚偶联物
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EA201690675A1 (ru) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
CN110903337A (zh) 2014-05-01 2020-03-24 Ionis制药公司 用于调节生长激素受体表达的组合物和方法
CN106232125B (zh) 2014-05-01 2020-10-16 Ionis制药公司 用于调节pkk表达的组合物和方法
SI3137605T1 (sl) 2014-05-01 2021-02-26 Ionis Pharmaceuticals, Inc. Sestavki in metode za modulacijo ekspresije angiopoetin 3-podobnega
PE20170010A1 (es) 2014-05-01 2017-03-04 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
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
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
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).
PE20180800A1 (es) 2015-07-10 2018-05-09 Ionis Pharmaceuticals Inc Moduladores de diaciglicerol aciltransferasa 2 (dgat2)
KR20180038465A (ko) 2015-08-07 2018-04-16 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스 감염에 대한 rnai 치료법
TW201723176A (zh) 2015-09-24 2017-07-01 Ionis製藥公司 Kras表現之調節劑
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
MA52645B1 (fr) 2015-10-22 2022-06-30 Modernatx Inc Vaccins contre le virus respiratoire
MA46316A (fr) 2015-10-22 2021-03-24 Modernatx Inc Vaccin contre le cytomégalovirus humain
CA3003103A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Broad spectrum influenza virus vaccine
CN117731769A (zh) 2015-10-22 2024-03-22 摩登纳特斯有限公司 用于水痘带状疱疹病毒(vzv)的核酸疫苗
KR20180096592A (ko) 2015-10-22 2018-08-29 모더나티엑스, 인크. 호흡기 세포융합 바이러스 백신
MX2018004918A (es) 2015-10-22 2019-04-01 Modernatx Inc Vacuna del virus herpes simple.
DK4119569T3 (da) 2015-11-06 2024-08-12 Ionis Pharmaceuticals Inc Konjugerede antisense-forbindelser til anvendelse i behandling
CA2999341A1 (en) 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
EP4039699A1 (en) 2015-12-23 2022-08-10 ModernaTX, Inc. Methods of using ox40 ligand encoding polynucleotides
US20190241658A1 (en) 2016-01-10 2019-08-08 Modernatx, Inc. Therapeutic mRNAs encoding anti CTLA-4 antibodies
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
CN114736256B (zh) 2016-06-06 2025-09-02 箭头药业股份有限公司 5’-环膦酸酯修饰核苷酸
EP3484524B1 (en) 2016-07-15 2022-11-09 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
CN109462981B (zh) 2016-09-02 2023-07-07 箭头药业股份有限公司 靶向配体
CN109661233A (zh) 2016-10-06 2019-04-19 Ionis 制药公司 缀合低聚化合物的方法
KR20240096846A (ko) 2017-01-10 2024-06-26 애로우헤드 파마슈티컬스 인코포레이티드 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법
WO2018140920A1 (en) 2017-01-30 2018-08-02 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor xii gene expression
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
US11786607B2 (en) 2017-06-15 2023-10-17 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
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
SG11201912178VA (en) 2017-09-11 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
KR102737464B1 (ko) 2017-09-14 2024-12-02 애로우헤드 파마슈티컬스 인코포레이티드 안지오포이에틴-유사 3 (ANGPTL3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물, 및 사용 방법
CN111212909A (zh) 2017-10-17 2020-05-29 箭头药业股份有限公司 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物
AU2019206731A1 (en) 2018-01-15 2020-07-30 Ionis Pharmaceuticals, Inc. Modulators of DNM2 expression
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
US20210355497A1 (en) 2018-05-09 2021-11-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fxi expression
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
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
UA128236C2 (uk) 2018-12-21 2024-05-15 Айоніс Фармасутікалз, Інк. Модулятори експресії hsd17b13
JP2022518384A (ja) 2019-01-09 2022-03-15 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 HIF-2アルファ(EPAS1)の発現を阻害するためのRNAi剤、その組成物及び使用方法
AU2020219355A1 (en) 2019-02-07 2021-09-30 Arrowhead Pharmaceuticals, Inc. RNAi agents for Hepatitis B virus infection
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
WO2020255007A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combination of hepatitis b virus (hbv) vaccines and hbv-targeting rnai
JP7757277B2 (ja) 2019-10-14 2025-10-21 アストラゼネカ・アクチエボラーグ Pnpla3発現のモジュレーター
AR121446A1 (es) 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
US12054718B2 (en) 2020-03-26 2024-08-06 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use
EP4138858A4 (en) 2020-04-21 2024-07-03 Flagship Pioneering, Inc. BIFUNCTIONAL MOLECULES AND METHODS OF USE THEREOF
IL301186A (en) 2020-09-11 2023-05-01 Arrowhead Pharmaceuticals Inc Skeletal muscle delivery platforms and methods of use
WO2022056266A2 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
UY39420A (es) 2020-09-11 2022-03-31 Arrowhead Pharmaceuticals Inc Conjugados lipídicos para el transporte de agentes terapéuticos
TW202227101A (zh) 2020-11-18 2022-07-16 美商Ionis製藥公司 用於調節血管收縮素原表現之化合物及方法
AU2022254705A1 (en) 2021-04-08 2023-10-05 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use
EP4326334A1 (en) 2021-04-23 2024-02-28 GanNA Bio, Inc. Glycan modified nucleic acids, methods of preparation, and therapeutic uses
KR20240014067A (ko) 2021-05-28 2024-01-31 애로우헤드 파마슈티컬스 인코포레이티드 뮤신 5AC (MUC5AC)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
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
CA3233755A1 (en) 2021-10-01 2023-04-06 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
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
CN119730859A (zh) 2022-06-15 2025-03-28 箭头药业股份有限公司 用于抑制超氧化物歧化酶1(SOD1)表达的RNAi剂、其组合物及使用方法
US12378558B2 (en) 2023-03-21 2025-08-05 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of complement factor B (CFB), pharmaceutical compositions thereof, and methods of use
TW202448484A (zh) 2023-04-20 2024-12-16 美商雅迪克斯製藥公司 Mapt調節組合物及其使用方法
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
WO2024249328A2 (en) 2023-05-26 2024-12-05 Adarx Pharmaceuticals, Inc. Sod1-modulating compositions and methods of use thereof
TW202506137A (zh) 2023-06-20 2025-02-16 美商雅迪克斯製藥公司 Lrrk2調節組合物及其使用方法
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
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

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 (enExample) 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
WO1998001478A1 (en) 1996-07-10 1998-01-15 E.I. Du Pont De Nemours And Company Polymerization with living characteristics
KR100672873B1 (ko) 1997-04-23 2007-01-24 이 아이 듀폰 디 네모아 앤드 캄파니 거대단량체의 합성 방법
IL139666A0 (en) 1997-07-21 2002-02-10 Commw Scient Ind Res Org Synthesis of dithioester chain transfer agents and use of bis (thioacyl) disulfides or dithioesters as chain transfer agents
CA2309279C (en) 1997-12-18 2009-07-14 E.I. Du Pont De Nemours And Company 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
CA2400172C (en) * 2000-02-28 2010-04-20 Genesegues, Inc. Nanocapsule encapsulation system and method
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
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
EP2500015A1 (en) * 2006-12-05 2012-09-19 Landec Corporation Delivery of drugs
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
CA3065577C (en) 2008-05-13 2022-05-31 Phaserx, Inc. Diblock copolymers and polynucleotide complexes thereof for delivery into cells
JP5766611B2 (ja) 2008-11-06 2015-08-19 ユニヴァーシティ オブ ワシントン マルチブロック共重合体
CN102361889B (zh) 2009-01-23 2014-09-17 联邦科学与工业研究组织 Raft聚合
ES2562817T3 (es) * 2010-02-24 2016-03-08 Arrowhead Research Corporation Composiciones para el suministro dirigido de ARNip
US20110224377A1 (en) 2010-03-11 2011-09-15 Mahesh Kalyana Mahanthappa Poly(vinyl ester) block copolymers
WO2011115641A1 (en) 2010-03-19 2011-09-22 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
MX2014001971A (es) * 2011-08-26 2014-03-31 Arrowhead Res Corp Polimeros de poli(vinilester) para suministro de acido nucleico in vivo.

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