CN104024328A - 用于体内核酸递送的聚(乙烯基酯)聚合物 - Google Patents
用于体内核酸递送的聚(乙烯基酯)聚合物 Download PDFInfo
- Publication number
- CN104024328A CN104024328A CN201280041442.XA CN201280041442A CN104024328A CN 104024328 A CN104024328 A CN 104024328A CN 201280041442 A CN201280041442 A CN 201280041442A CN 104024328 A CN104024328 A CN 104024328A
- Authority
- CN
- China
- Prior art keywords
- vinyl ester
- poly
- polymkeric substance
- polynucleotide
- group
- 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
Links
- VAQFXLQUIXEPQZ-UHFFFAOYSA-N CCSC(CCC(C(O1)=O)=C(C)C1=O)=O Chemical compound CCSC(CCC(C(O1)=O)=C(C)C1=O)=O VAQFXLQUIXEPQZ-UHFFFAOYSA-N 0.000 description 1
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)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161527703P | 2011-08-26 | 2011-08-26 | |
| US61/527,703 | 2011-08-26 | ||
| US13/592,393 US8932572B2 (en) | 2011-08-26 | 2012-08-23 | Poly(vinyl ester) polymers for in vivo nucleic acid delivery |
| US13/592,393 | 2012-08-23 | ||
| 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 |
|---|---|
| CN104024328A true CN104024328A (zh) | 2014-09-03 |
Family
ID=47756738
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201280041442.XA Pending CN104024328A (zh) | 2011-08-26 | 2012-08-23 | 用于体内核酸递送的聚(乙烯基酯)聚合物 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US8932572B2 (https=) |
| JP (1) | JP2014531476A (https=) |
| KR (1) | KR20140051357A (https=) |
| CN (1) | CN104024328A (https=) |
| AU (1) | AU2012300476B2 (https=) |
| BR (1) | BR112014004585A2 (https=) |
| CA (1) | CA2842039A1 (https=) |
| HK (1) | HK1199736A1 (https=) |
| IL (1) | IL231146A0 (https=) |
| MX (1) | MX2014001971A (https=) |
| PH (1) | PH12014500214A1 (https=) |
| WO (1) | WO2013032829A1 (https=) |
Families Citing this family (109)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012019168A2 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| RU2013120302A (ru) | 2010-10-01 | 2014-11-20 | Модерна Терапьютикс, Инк. | Сконструированные нуклеиновые кислоты и способы их применения |
| EP2691101A2 (en) | 2011-03-31 | 2014-02-05 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| WO2013032829A1 (en) * | 2011-08-26 | 2013-03-07 | Arrowhead Research Corporation | 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 |
| RU2648950C2 (ru) | 2011-10-03 | 2018-04-02 | Модерна Терапьютикс, Инк. | Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение |
| CN104114572A (zh) | 2011-12-16 | 2014-10-22 | 现代治疗公司 | 经修饰的核苷、核苷酸和核酸组合物 |
| 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 |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| 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 |
| WO2014081507A1 (en) | 2012-11-26 | 2014-05-30 | Moderna Therapeutics, Inc. | Terminally modified rna |
| SG10201706960TA (en) | 2013-02-28 | 2017-10-30 | Arrowhead Res Corp | Organic compositions to treat epas1-related diseases |
| CN105263476A (zh) * | 2013-03-13 | 2016-01-20 | 库尔制药开发公司 | 用于治疗炎症的免疫修饰性颗粒 |
| 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 |
| DK2991656T3 (da) | 2013-05-01 | 2020-03-23 | Ionis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression |
| WO2015021092A1 (en) | 2013-08-07 | 2015-02-12 | Arrowhead Research Corporation | Polyconjugates for delivery of rnai triggers to tumor cells in vivo |
| US20160194368A1 (en) | 2013-09-03 | 2016-07-07 | Moderna Therapeutics, Inc. | Circular polynucleotides |
| US20160194625A1 (en) | 2013-09-03 | 2016-07-07 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| US10023626B2 (en) | 2013-09-30 | 2018-07-17 | 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 |
| DK3137476T3 (da) | 2014-04-28 | 2019-11-18 | Ionis Pharmaceuticals Inc | Linker-modificerede oligomerforbindelser |
| 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 |
| PT3137605T (pt) | 2014-05-01 | 2020-12-18 | Ionis Pharmaceuticals Inc | Composições e métodos para modulação da expressão de angiopoietina de tipo 3 |
| SG11201608502TA (en) | 2014-05-01 | 2016-11-29 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| EP3862362A3 (en) | 2014-05-01 | 2021-10-27 | Ionis Pharmaceuticals, Inc. | Conjugates of modified antisense oligonucleotides and their use 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 |
| EP3169693B1 (en) | 2014-07-16 | 2022-03-09 | ModernaTX, 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 |
| HK1252852A1 (zh) | 2015-05-29 | 2019-06-06 | Arrowhead Pharmaceuticals, Inc. | 阻止HIF2α基因表达的组合物及方法 |
| CA2991894A1 (en) | 2015-07-10 | 2017-01-19 | Ionis Pharmaceuticals, Inc. | Modulators of diacyglycerol acyltransferase 2 (dgat2) |
| KR20180038465A (ko) | 2015-08-07 | 2018-04-16 | 애로우헤드 파마슈티컬스 인코포레이티드 | B형 간염 바이러스 감염에 대한 rnai 치료법 |
| JP6877414B2 (ja) | 2015-09-24 | 2021-05-26 | 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Ionis Pharmaceuticals,Inc. | Kras発現のモジュレーター |
| JOP20160211B1 (ar) | 2015-10-01 | 2021-08-17 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| AU2016342049B2 (en) | 2015-10-22 | 2023-05-18 | Modernatx, Inc. | Herpes simplex virus vaccine |
| MX2018004917A (es) | 2015-10-22 | 2019-04-01 | Modernatx Inc | Vacuna de virus sincitial respiratorio. |
| TW201729835A (zh) | 2015-10-22 | 2017-09-01 | 現代公司 | 呼吸道病毒疫苗 |
| WO2017070620A2 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Broad spectrum influenza virus vaccine |
| EP3364981A4 (en) | 2015-10-22 | 2019-08-07 | ModernaTX, Inc. | VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS |
| CA3002912A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Nucleic acid vaccines for varicella zoster virus (vzv) |
| AU2016349625B2 (en) | 2015-11-06 | 2022-07-07 | Ionis Pharmaceuticals, Inc. | Modulating apolipoprotein (a) expression |
| US20190046555A1 (en) | 2015-11-06 | 2019-02-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds for use in therapy |
| RS63135B1 (sr) | 2015-12-23 | 2022-05-31 | Modernatx Inc | Postupci upotrebe polinukleotida koji kodiraju ox40 ligand |
| US20190241658A1 (en) | 2016-01-10 | 2019-08-08 | Modernatx, Inc. | Therapeutic mRNAs encoding anti CTLA-4 antibodies |
| KR102515329B1 (ko) | 2016-03-07 | 2023-03-29 | 애로우헤드 파마슈티컬스 인코포레이티드 | 치료용 화합물을 위한 표적화 리간드 |
| WO2017214112A1 (en) | 2016-06-06 | 2017-12-14 | Arrowhead Pharmaceuticals, Inc. | 5'-cyclo-phosphonate modified nucleotides |
| ES2935658T3 (es) | 2016-07-15 | 2023-03-09 | Ionis Pharmaceuticals Inc | Compuestos y métodos para la modulación de SMN2 |
| JOP20190015A1 (ar) | 2016-08-04 | 2019-02-04 | Arrowhead Pharmaceuticals Inc | عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب |
| MX2018009853A (es) | 2016-09-02 | 2018-11-09 | Arrowhead Pharmaceuticals Inc | Ligandos de direccion. |
| EP3522898A4 (en) | 2016-10-06 | 2020-05-27 | Ionis Pharmaceuticals, Inc. | METHOD FOR CONJUGATING OLIGOMERIC COMPOUNDS |
| BR112019014282A2 (pt) | 2017-01-10 | 2020-03-03 | Arrowhead Pharmaceuticals, Inc. | Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso |
| AU2018213379A1 (en) | 2017-01-30 | 2019-07-18 | 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 |
| TN2019000308A1 (en) | 2017-07-06 | 2021-05-07 | Arrowhead Pharmaceuticals Inc | RNAi AGENTS FOR INHIBITING EXPRESSION OF ALPHA-ENaC AND METHODS OF USE |
| WO2019046809A1 (en) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | METHODS OF MANUFACTURING LIPID NANOPARTICLES |
| US10597657B2 (en) | 2017-09-11 | 2020-03-24 | Arrowhead Pharmaceuticals, Inc. | RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3) |
| MX2020001912A (es) | 2017-09-14 | 2020-03-24 | Arrowhead Pharmaceuticals Inc | Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso. |
| US11492624B2 (en) | 2017-10-17 | 2022-11-08 | Arrowheads 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 |
| AU2019218987B2 (en) | 2018-02-12 | 2025-04-24 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| JP7438135B2 (ja) | 2018-05-09 | 2024-02-26 | 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 | Fxiの発現を低下させるための化合物及び方法 |
| EP3823725A4 (en) | 2018-07-17 | 2023-05-10 | Aronora, Inc. | METHODS FOR SAFELY REDUCING THROMBOPOIETIN |
| US20210292768A1 (en) | 2018-08-08 | 2021-09-23 | Arcturus Therapeutics, Inc. | Compositions and agents against nonalcoholic steatohepatitis |
| TWI856973B (zh) | 2018-09-19 | 2024-10-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
| MX2021003077A (es) | 2018-09-19 | 2021-05-27 | Arrowhead Pharmaceuticals Inc | Agentes de arni para inhibir la expresion de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y metodos de uso. |
| CN113271926A (zh) | 2018-09-20 | 2021-08-17 | 摩登纳特斯有限公司 | 脂质纳米颗粒的制备及其施用方法 |
| EP3899024A4 (en) | 2018-12-21 | 2023-05-31 | Ionis Pharmaceuticals, Inc. | Modulators of hsd17b13 expression |
| SG11202105577TA (en) | 2019-01-09 | 2021-06-29 | Arrowhead Pharmaceuticals Inc | Rnai agents for inhibiting expression of hif-2 alpha (epas1), compositions thereof, and methods of use |
| SG11202108395SA (en) | 2019-02-07 | 2021-08-30 | Arrowhead Pharmaceuticals Inc | Rnai agents for hepatitis b virus infection |
| MX2021015003A (es) | 2019-06-06 | 2022-01-24 | Arrowhead Pharmaceuticals Inc | Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd). |
| KR20220042116A (ko) | 2019-06-18 | 2022-04-04 | 얀센 사이언시즈 아일랜드 언리미티드 컴퍼니 | B형 간염 바이러스(HBV) 백신 및 HBV-타케팅 RNAi의 조합 |
| CA3143679A1 (en) | 2019-06-18 | 2020-12-24 | 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 |
| CR20220485A (es) | 2020-02-28 | 2022-11-10 | Ionis Pharmaceuticals Inc | Compuestos y 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 |
| AU2021260934A1 (en) | 2020-04-21 | 2022-11-24 | Flagship Pioneering, Inc. | Bifunctional molecules and methods of using thereof |
| AU2021342158A1 (en) | 2020-09-11 | 2023-04-13 | Arrowhead Pharmaceuticals, Inc. | Lipid conjugates for the delivery of therapeutic agents |
| AU2021340710A1 (en) | 2020-09-11 | 2023-04-06 | Arrowhead Pharmaceuticals, Inc. | Skeletal muscle delivery platforms and methods of use |
| IL301187A (en) | 2020-09-11 | 2023-05-01 | Arrowhead Pharmaceuticals Inc | RNAi agents for suppressing DUX4 expression, preparations containing them and uses thereof |
| EP4136092B1 (en) | 2020-11-18 | 2024-07-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating angiotensinogen expression |
| US12486502B2 (en) | 2021-04-08 | 2025-12-02 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use |
| AU2022262794A1 (en) | 2021-04-23 | 2023-11-02 | Beth Israel Deaconess Medical Center, Inc. | Glycan modified nucleic acids, methods of preparation, and therapeutic uses |
| CN117858949A (zh) | 2021-05-28 | 2024-04-09 | 箭头药业股份有限公司 | 用于抑制粘蛋白5AC(MUC5AC)的表达的RNAi试剂、其组合物及其使用方法 |
| 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 |
| MX2024004011A (es) | 2021-10-01 | 2024-07-01 | Adarx Pharmaceuticals Inc | Composiciones moduladoras de precalicreína y métodos de uso de estas. |
| WO2023161350A1 (en) | 2022-02-24 | 2023-08-31 | 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 |
| EP4540390A2 (en) | 2022-06-15 | 2025-04-23 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of superoxide dismutase 1 (sod1), compositions thereof, and methods of use |
| 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 |
| WO2024220930A2 (en) | 2023-04-20 | 2024-10-24 | Adarx Pharmaceuticals, Inc. | Mapt-modulating compositions and methods of use thereof |
| CN121398851A (zh) | 2023-05-12 | 2026-01-23 | 阿达尔克斯制药有限公司 | Nmda配体缀合的化合物及其用途 |
| IL324720A (en) | 2023-05-26 | 2026-01-01 | Adarx Pharmaceuticals Inc | SOD1 modulators and methods of using them |
| CN121712891A (zh) | 2023-06-20 | 2026-03-20 | 阿达尔克斯制药有限公司 | Lrrk2调节组合物及其使用方法 |
| IL326882A (en) | 2023-08-30 | 2026-04-01 | Arrowhead Pharmaceuticals Inc | RNAi agents for inhibiting expression of inhibitory subunit beta E (INHBE), their pharmaceutical compositions 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 |
| WO2025137390A1 (en) | 2023-12-20 | 2025-06-26 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of activin receptor-like kinase 7 (alk7), compositions thereof, and methods of use |
| TW202602463A (zh) | 2024-03-22 | 2026-01-16 | 日商武田藥品工業股份有限公司 | 用於抑制細胞色素p450家族7次家族a成員1(cyp7a1)表現之組成物及方法 |
| US12551569B2 (en) | 2024-03-31 | 2026-02-17 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use |
| WO2025226980A2 (en) | 2024-04-25 | 2025-10-30 | Insitro, Inc. | Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease |
| US20260021195A1 (en) | 2024-06-20 | 2026-01-22 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use |
| WO2026044017A2 (en) | 2024-08-21 | 2026-02-26 | Insitro, Inc. | Compositions and methods for treating nonalcoholic fatty liver disease |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040162260A1 (en) * | 2003-02-18 | 2004-08-19 | Rozema David B. | Reversible attachment of a membrane active polymer to a polynucleotide |
| WO2008022309A2 (en) * | 2006-08-18 | 2008-02-21 | F. Hoffmann-La Roche Ag | Polyconjugates for in vivo delivery of polynucleotides |
| US20090048410A1 (en) * | 2002-03-11 | 2009-02-19 | Wakefield Darren H | Membrane Active Heteropolymers |
| US20110009571A1 (en) * | 2006-12-05 | 2011-01-13 | David Taft | Systems and methods for delivery of materials |
Family Cites Families (20)
| 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 (https=) | 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 |
| ES2166092T3 (es) | 1996-07-10 | 2002-04-01 | Du Pont | Polimerizacion con caracteristicas vivientes. |
| CA2284026A1 (en) | 1997-04-23 | 1998-10-29 | E.I. Du Pont De Nemours And Company | Method of macromonomer synthesis |
| CA2337339A1 (en) | 1997-07-21 | 1999-02-04 | Commonwealth Scientific And Industrial Research Organisation | 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 |
| EP1102784A4 (en) * | 1999-06-07 | 2009-12-30 | Mirus Bio Corp | A CONNECTION WITH A LABILES DISULFIDE TIE |
| 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 |
| 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 |
| 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 |
| JP5756027B2 (ja) | 2009-01-23 | 2015-07-29 | コモンウェルス サイエンティフィック アンド インダストリアル リサーチ オーガナイゼーション | Raft重合 |
| DK2539451T3 (da) * | 2010-02-24 | 2016-04-04 | Arrowhead Res Corp | Sammensætninger til målrettet tilførsel af siRNA |
| 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 |
| WO2011163121A1 (en) | 2010-06-21 | 2011-12-29 | Alnylam Pharmaceuticals, Inc. | Multifunctional copolymers for nucleic acid delivery |
| WO2013032829A1 (en) * | 2011-08-26 | 2013-03-07 | Arrowhead Research Corporation | Poly(vinyl ester) polymers for in vivo nucleic acid delivery |
-
2012
- 2012-08-23 WO PCT/US2012/051968 patent/WO2013032829A1/en not_active Ceased
- 2012-08-23 PH PH1/2014/500214A patent/PH12014500214A1/en unknown
- 2012-08-23 HK HK15100132.2A patent/HK1199736A1/xx unknown
- 2012-08-23 JP JP2014527280A patent/JP2014531476A/ja active Pending
- 2012-08-23 CA CA 2842039 patent/CA2842039A1/en not_active Abandoned
- 2012-08-23 KR KR20147004852A patent/KR20140051357A/ko not_active Withdrawn
- 2012-08-23 BR BR112014004585A patent/BR112014004585A2/pt unknown
- 2012-08-23 MX MX2014001971A patent/MX2014001971A/es unknown
- 2012-08-23 US US13/592,393 patent/US8932572B2/en active Active
- 2012-08-23 CN CN201280041442.XA patent/CN104024328A/zh active Pending
- 2012-08-23 AU AU2012300476A patent/AU2012300476B2/en not_active Ceased
-
2014
- 2014-02-25 IL IL231146A patent/IL231146A0/en unknown
- 2014-12-05 US US14/561,339 patent/US9089611B2/en not_active Expired - Fee Related
-
2015
- 2015-06-22 US US14/746,070 patent/US9474804B2/en not_active Expired - Fee Related
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20090048410A1 (en) * | 2002-03-11 | 2009-02-19 | Wakefield Darren H | Membrane Active Heteropolymers |
| US20040162260A1 (en) * | 2003-02-18 | 2004-08-19 | Rozema David B. | Reversible attachment of a membrane active polymer to a polynucleotide |
| WO2008022309A2 (en) * | 2006-08-18 | 2008-02-21 | F. Hoffmann-La Roche Ag | Polyconjugates for in vivo delivery of polynucleotides |
| WO2008022309A3 (en) * | 2006-08-18 | 2008-11-20 | Hoffmann La Roche | Polyconjugates for in vivo delivery of polynucleotides |
| US20110009571A1 (en) * | 2006-12-05 | 2011-01-13 | David Taft | Systems and methods for delivery of materials |
Also Published As
| Publication number | Publication date |
|---|---|
| PH12014500214A1 (en) | 2019-03-22 |
| US20130121954A1 (en) | 2013-05-16 |
| JP2014531476A (ja) | 2014-11-27 |
| KR20140051357A (ko) | 2014-04-30 |
| US20150283256A1 (en) | 2015-10-08 |
| IL231146A0 (en) | 2014-04-30 |
| US8932572B2 (en) | 2015-01-13 |
| AU2012300476B2 (en) | 2016-03-24 |
| US9089611B2 (en) | 2015-07-28 |
| AU2012300476A1 (en) | 2014-01-23 |
| US9474804B2 (en) | 2016-10-25 |
| BR112014004585A2 (pt) | 2017-06-13 |
| MX2014001971A (es) | 2014-03-31 |
| US20150110732A1 (en) | 2015-04-23 |
| HK1199736A1 (en) | 2015-07-17 |
| CA2842039A1 (en) | 2013-03-07 |
| WO2013032829A1 (en) | 2013-03-07 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN104024328A (zh) | 用于体内核酸递送的聚(乙烯基酯)聚合物 | |
| CA2972694C (en) | Diblock copolymers and polynucleotide complexes thereof for delivery into cells | |
| US8933047B2 (en) | Poly(acrylate) polymers for in vivo nucleic acid delivery | |
| US9415113B2 (en) | Targeting monomers and polymers having targeting blocks | |
| CA2816041C (en) | In vivo polynucleotide delivery conjugates having enzyme sensitive linkages | |
| JP7021076B2 (ja) | Hif2αの遺伝子発現を阻害する組成物及び方法 | |
| WO2014066912A1 (en) | Polymeric nanoparticles | |
| EP2296708A2 (en) | Polymeric carrier | |
| CN103547272A (zh) | 基于肽的体内siRNA递送系统 | |
| Lin et al. | Grafting from small interfering ribonucleic acid (siRNA) as an alternative synthesis route to siRNA–polymer conjugates | |
| Wang et al. | Acid-triggered polymer backbone degradation and disassembly to achieve release of camptothecin from functional polyphosphoramidate nanoparticles | |
| WO2011060281A1 (en) | Hydrophobic block conjugated therapeutic agents | |
| Xia et al. | Triplex Hybridization of siRNA with Bifacial Glycopolymer Nucleic Acid Enables Hepatocyte-Targeted Silencing | |
| EP2807216A1 (en) | Poly(vinyl ester) polymers for in vivo nucleic acid delivery | |
| HK1194691A (en) | Poly(acrylate) polymers for in vivo nucleic acid delivery | |
| EP2838544A1 (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 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| C06 | Publication | ||
| PB01 | Publication | ||
| C10 | Entry into substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| REG | Reference to a national code |
Ref country code: HK Ref legal event code: DE Ref document number: 1199736 Country of ref document: HK |
|
| RJ01 | Rejection of invention patent application after publication | ||
| RJ01 | Rejection of invention patent application after publication |
Application publication date: 20140903 |
|
| REG | Reference to a national code |
Ref country code: HK Ref legal event code: WD Ref document number: 1199736 Country of ref document: HK |