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

    • 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
    • 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
    • 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
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type 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

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)
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
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
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 (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 (105)

* 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
HUE069586T2 (hu) 2010-10-01 2025-03-28 Modernatx Inc Módosított nukleozidok, nukleotidok és nukleinsavak, valamint ezek felhasználása
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
HK1199736A1 (en) * 2011-08-26 2015-07-17 箭头研究公司 Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EP3453761A1 (en) 2011-08-29 2019-03-13 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
DK3682905T3 (da) 2011-10-03 2022-02-28 Modernatx Inc Modificerede nukleosider, nukleotider og nukleinsyrer og anvendelser deraf
HRP20220717T1 (hr) 2011-12-16 2022-07-22 Modernatx, Inc. Modificirani pripravci mrna
US8796405B2 (en) * 2012-01-18 2014-08-05 Wisconsin Alumni Research Foundation Degradable polycations derived from amino acid vinyl esters
JP6189415B2 (ja) 2012-04-02 2017-08-30 モデルナティエックス インコーポレイテッドModernaTX,Inc. 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
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
JP2015515530A (ja) * 2012-04-18 2015-05-28 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(アクリラート)ポリマー
EP2922554B1 (en) 2012-11-26 2022-02-23 ModernaTX, Inc. Terminally modified rna
SG11201506805QA (en) 2013-02-28 2015-09-29 Arrowhead Res Corp Organic compositions to treat epas1-related diseases
KR20160014584A (ko) 2013-03-13 2016-02-11 코어 파마슈티칼스 디벨롭먼트 컴퍼니 염증 치료용 면역-변형된 입자
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
IL296543B2 (en) 2013-05-01 2025-02-01 Ionis Pharmaceuticals Inc Compositions and methods for modulating HBV and TTR expression
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
JP2016530294A (ja) 2013-09-03 2016-09-29 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. キメラポリヌクレオチド
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
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
EP3137476B1 (en) 2014-04-28 2019-10-09 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
UA121656C2 (uk) 2014-05-01 2020-07-10 Айоніс Фармасьютикалз, Інк. Олігомерна сполука та спосіб лікування захворювання, пов'язаного з порушенням регуляції альтернативного шляху комплементу
EP4534092A3 (en) 2014-05-01 2025-07-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
BR122020024443B1 (pt) 2014-05-01 2022-02-22 Ionis Pharmaceuticals, Inc Composto e composição farmacêutica para modulação da expressão de angptl3
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
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
CA2984498C (en) 2015-05-29 2024-01-02 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of hif2alpha
BR112017028194B1 (pt) 2015-07-10 2023-03-14 Ionis Pharmaceuticals, Inc Composto oligomérico modulador de diaciglicerol aciltransferase 2 (dgat2), composição, e seus usos
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
CN108513588A (zh) 2015-09-24 2018-09-07 Ionis制药公司 Kras表达的调节剂
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
BR112018008102A2 (pt) 2015-10-22 2018-11-06 Modernatx Inc vacina de vírus sincicial respiratório
EA201890999A1 (ru) 2015-10-22 2018-12-28 МОДЕРНАТиЭкс, ИНК. Вакцина против вируса простого герпеса
EA201891001A1 (ru) 2015-10-22 2018-11-30 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот против вируса ветряной оспы (vzv)
WO2017070613A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Human cytomegalovirus vaccine
KR20180096591A (ko) 2015-10-22 2018-08-29 모더나티엑스, 인크. 광범위 인플루엔자 바이러스 백신
MD3718565T2 (ro) 2015-10-22 2022-09-30 Modernatx Inc Vaccinuri împotriva virusului respirator
JP7486920B2 (ja) 2015-11-06 2024-05-20 アイオーニス ファーマシューティカルズ, インコーポレーテッド アポリポタンパク質(a)発現の調節
LT4119569T (lt) 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje
PT3394093T (pt) 2015-12-23 2022-05-30 Modernatx Inc Métodos de utilização de polinucleotídeos que codificam ligandos ox40
US20190241658A1 (en) 2016-01-10 2019-08-08 Modernatx, Inc. Therapeutic mRNAs encoding anti CTLA-4 antibodies
IL296377A (en) 2016-03-07 2022-11-01 Arrowhead Pharmaceuticals Inc Targeting ligands for therapeutic compounds and uses of same
CA3023764A1 (en) 2016-06-06 2017-12-14 Arrowhead Pharmaceuticals, Inc. 5'-cyclo-phosphonate modified nucleotides
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
US10450565B2 (en) 2017-01-10 2019-10-22 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
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
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
JP7275111B2 (ja) 2017-08-31 2023-05-17 モデルナティエックス インコーポレイテッド 脂質ナノ粒子の生成方法
SG11201912178VA (en) 2017-09-11 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
KR20250004009A (ko) 2017-09-14 2025-01-07 애로우헤드 파마슈티컬스 인코포레이티드 안지오포이에틴-유사 3 (ANGPTL3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물, 및 사용 방법
CA3079413A1 (en) 2017-10-17 2019-04-25 Arrowhead Pharmaceuticals, Inc. Rnai agents and compositions for inhibiting expression of asialoglycoprotein receptor 1
MX2020007369A (es) 2018-01-15 2020-10-28 Ionis Pharmaceuticals Inc Moduladores de la expresion de dnm2.
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
JOP20200280A1 (ar) 2018-05-09 2020-11-05 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير الوراثي عن fxi
EP3823725A4 (en) 2018-07-17 2023-05-10 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
JP7394137B2 (ja) 2018-12-21 2023-12-07 アイオニス・ファーマシューティカルズ・インコーポレイテッド Hsd17b13発現のモジュレータ
CA3120580A1 (en) 2019-01-09 2020-07-16 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use
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
JP2022536945A (ja) 2019-06-18 2022-08-22 ヤンセン・サイエンシズ・アイルランド・アンリミテッド・カンパニー B型肝炎ウイルス(HBV)ワクチンおよびHBVを標的化するRNAiの組合せ
EP4045062A1 (en) 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
TW202517787A (zh) 2020-02-28 2025-05-01 美商Ionis製藥公司 用於調節smn2之化合物及方法
AU2021242321B2 (en) 2020-03-26 2025-09-04 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use
CN115916219A (zh) 2020-04-21 2023-04-04 旗舰创业股份有限公司 双官能分子及其使用方法
MX2023002883A (es) 2020-09-11 2023-03-31 Arrowhead Pharmaceuticals Inc Conjugados lipidicos para el transporte de agentes terapeuticos.
CA3189077A1 (en) 2020-09-11 2022-03-17 Xiaokai Li Skeletal muscle delivery platforms and methods of use
AU2021342155A1 (en) 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
EP4488371A3 (en) 2020-11-18 2025-04-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
EP4319765A1 (en) 2021-04-08 2024-02-14 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
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
WO2023056440A1 (en) 2021-10-01 2023-04-06 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
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
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
CN121285630A (zh) 2023-04-20 2026-01-06 阿达尔克斯制药有限公司 Mapt调节组合物及其使用方法
AU2024273075A1 (en) 2023-05-12 2025-11-20 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
WO2024263694A1 (en) 2023-06-20 2024-12-26 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
WO2025049773A1 (en) 2023-08-30 2025-03-06 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
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

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
CN100473646C (zh) 1996-07-10 2009-04-01 联邦科学及工业研究组织 具有活性特征的聚合作用
AU752670B2 (en) 1997-04-23 2002-09-26 Commonwealth Scientific And Industrial Research Organisation Method of macromonomer synthesis
AU770550B2 (en) 1997-07-21 2004-02-26 Commonwealth Scientific And Industrial Research Organisation Synthesis of dithioester chain transfer agents and use of bis(thioacyl) disulfides or dithioesters as chain transfer agents
NZ505654A (en) 1997-12-18 2002-03-28 John Chiefair Living polymerisation process whereby photo-initiators of polymerisation utilises a thermal process resulting in polymers of controlled molecular weight and low polydispersity
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
JP4751556B2 (ja) * 2000-02-28 2011-08-17 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
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
KR101133799B1 (ko) * 2006-08-18 2012-04-24 에프. 호프만-라 로슈 아게 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체
EP2101745A4 (en) * 2006-12-05 2009-12-30 Landec Corp ADMINISTRATION OF MEDICINES
US8999323B2 (en) * 2007-11-23 2015-04-07 Technische Universität Wien Composition that can be cured by polymerisation for the production of biodegradable, biocompatible, cross-linkable polymers on the basis of polyvinyl alcohol
CN103342789B (zh) 2008-05-13 2016-11-23 华盛顿大学 用于向细胞内递送的二嵌段共聚物和其多核苷酸复合物
EP2364330B1 (en) 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
US9340498B2 (en) 2009-01-23 2016-05-17 Commonwealth Scientific And Industrial Reserach 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
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

Also Published As

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

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
MX347298B (es) Conjugados 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
EP2663336A1 (en) Methods and reagents for efficient and targeted delivery of therapeutic molecules to cxcr4 cells
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
IL226666B (en) Methods of generating corneal cells and cell populations comprising same
WO2012145729A3 (en) Methods and compositions for modulating gene expression using components that self assemble in cells and produce rnai activity
WO2014066915A3 (en) Methods and compositions to produce ss-rnai activity with enhanced potency
WO2012006169A3 (en) Compositions and methods for enhancement of nucleic acid delivery
WO2011146143A3 (en) Enzymatic activity of psa as diagnostic marker for prostate cancer
WO2005079532A3 (en) Methods and compositions for enhancing risc activity in vitro and in vivo