JP2012521358A5 - - Google Patents
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- JP2012521358A5 JP2012521358A5 JP2012500998A JP2012500998A JP2012521358A5 JP 2012521358 A5 JP2012521358 A5 JP 2012521358A5 JP 2012500998 A JP2012500998 A JP 2012500998A JP 2012500998 A JP2012500998 A JP 2012500998A JP 2012521358 A5 JP2012521358 A5 JP 2012521358A5
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- 239000003446 ligand Substances 0.000 claims description 10
- 230000008685 targeting Effects 0.000 claims description 8
- 229910052757 nitrogen Inorganic materials 0.000 claims description 7
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims description 6
- 229920000642 polymer Polymers 0.000 claims description 5
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 4
- 239000001257 hydrogen Substances 0.000 claims description 4
- 229910052739 hydrogen Inorganic materials 0.000 claims description 4
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 3
- 125000001183 hydrocarbyl group Chemical group 0.000 claims description 3
- 229910052760 oxygen Inorganic materials 0.000 claims description 3
- 239000001301 oxygen Substances 0.000 claims description 3
- 150000001875 compounds Chemical class 0.000 claims 11
- 238000009472 formulation Methods 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 210000004027 cell Anatomy 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 108091023037 Aptamer Proteins 0.000 claims 2
- RTZKZFJDLAIYFH-UHFFFAOYSA-N Diethyl ether Chemical compound CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims 2
- QUSNBJAOOMFDIB-UHFFFAOYSA-N Ethylamine Chemical compound CCN QUSNBJAOOMFDIB-UHFFFAOYSA-N 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 235000018102 proteins Nutrition 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 150000003384 small molecules Chemical class 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- PYSRRFNXTXNWCD-UHFFFAOYSA-N 3-(2-phenylethenyl)furan-2,5-dione Chemical compound O=C1OC(=O)C(C=CC=2C=CC=CC=2)=C1 PYSRRFNXTXNWCD-UHFFFAOYSA-N 0.000 claims 1
- WHNPOQXWAMXPTA-UHFFFAOYSA-N 3-methylbut-2-enamide Chemical compound CC(C)=CC(N)=O WHNPOQXWAMXPTA-UHFFFAOYSA-N 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 229920002307 Dextran Polymers 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 239000004372 Polyvinyl alcohol Substances 0.000 claims 1
- 108091027967 Small hairpin RNA Proteins 0.000 claims 1
- 229920000147 Styrene maleic anhydride Polymers 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000002981 blocking agent Substances 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 230000010261 cell growth Effects 0.000 claims 1
- 210000000170 cell membrane Anatomy 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 239000002299 complementary DNA Substances 0.000 claims 1
- 210000001163 endosome Anatomy 0.000 claims 1
- 230000000799 fusogenic effect Effects 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 229940088597 hormone Drugs 0.000 claims 1
- 238000001727 in vivo Methods 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 230000002934 lysing effect Effects 0.000 claims 1
- FPYJFEHAWHCUMM-UHFFFAOYSA-N maleic anhydride Chemical compound O=C1OC(=O)C=C1 FPYJFEHAWHCUMM-UHFFFAOYSA-N 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 108091070501 miRNA Proteins 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 108020004707 nucleic acids Proteins 0.000 claims 1
- 102000039446 nucleic acids Human genes 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- 125000002811 oleoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])/C([H])=C([H])\C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
- 239000008191 permeabilizing agent Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 229920001562 poly(N-(2-hydroxypropyl)methacrylamide) Polymers 0.000 claims 1
- 229920002401 polyacrylamide Polymers 0.000 claims 1
- 229920002643 polyglutamic acid Polymers 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 229920002451 polyvinyl alcohol Polymers 0.000 claims 1
- 229920000036 polyvinylpyrrolidone Polymers 0.000 claims 1
- 239000001267 polyvinylpyrrolidone Substances 0.000 claims 1
- 235000013855 polyvinylpyrrolidone Nutrition 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108020004418 ribosomal RNA Proteins 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 239000011782 vitamin Substances 0.000 claims 1
- 229940088594 vitamin Drugs 0.000 claims 1
- 229930003231 vitamin Natural products 0.000 claims 1
- 235000013343 vitamin Nutrition 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US16182809P | 2009-03-20 | 2009-03-20 | |
| US61/161,828 | 2009-03-20 | ||
| PCT/US2010/028000 WO2010108108A2 (en) | 2009-03-20 | 2010-03-19 | Polyamine derivatives |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015018143A Division JP6190401B2 (ja) | 2009-03-20 | 2015-02-02 | ポリアミン誘導体 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012521358A JP2012521358A (ja) | 2012-09-13 |
| JP2012521358A5 true JP2012521358A5 (https=) | 2014-10-16 |
| JP5782602B2 JP5782602B2 (ja) | 2015-09-24 |
Family
ID=42359538
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012500998A Active JP5782602B2 (ja) | 2009-03-20 | 2010-03-19 | ポリアミン誘導体 |
| JP2015018143A Active JP6190401B2 (ja) | 2009-03-20 | 2015-02-02 | ポリアミン誘導体 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015018143A Active JP6190401B2 (ja) | 2009-03-20 | 2015-02-02 | ポリアミン誘導体 |
Country Status (10)
| Country | Link |
|---|---|
| US (4) | US8460696B2 (https=) |
| EP (1) | EP2414322B1 (https=) |
| JP (2) | JP5782602B2 (https=) |
| KR (2) | KR101800833B1 (https=) |
| CN (2) | CN102438978B (https=) |
| AU (1) | AU2010226434A1 (https=) |
| CA (1) | CA2755963C (https=) |
| ES (1) | ES2931174T3 (https=) |
| PT (1) | PT2414322T (https=) |
| WO (1) | WO2010108108A2 (https=) |
Families Citing this family (102)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2941152B1 (fr) * | 2009-01-20 | 2013-10-18 | Centre Nat Rech Scient | Vecteurs comprenant une macromolecule anionique et un lipide cationique pour l'administration de petits acides nucleiques. |
| PT2414322T (pt) | 2009-03-20 | 2022-12-07 | Egen Inc | Derivados de poliamina |
| 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 |
| US20120136073A1 (en) | 2010-11-15 | 2012-05-31 | Life Technologies Corporation | Amine-Containing Transfection Reagents and methods for making and using same |
| US20120202890A1 (en) * | 2011-02-08 | 2012-08-09 | Nian Wu | Polymer-carbohydrate-lipid conjugates |
| EP2691101A2 (en) | 2011-03-31 | 2014-02-05 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| AU2012242455A1 (en) | 2011-04-15 | 2013-11-28 | Molecular Transfer, Inc. | Agents for improved delivery of nucleic acids to eukaryotic cells |
| US8883177B2 (en) * | 2011-06-28 | 2014-11-11 | Nian Wu | Pharmaceutical compositions for parenteral administration |
| 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 |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| CA2868391A1 (en) | 2012-04-02 | 2013-10-10 | Stephane Bancel | Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal 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 |
| WO2014081507A1 (en) | 2012-11-26 | 2014-05-30 | Moderna Therapeutics, Inc. | Terminally modified rna |
| US20140200261A1 (en) | 2013-01-17 | 2014-07-17 | Moderna Therapeutics, Inc. | Signal-sensor polynucleotides for the alteration of cellular phenotypes |
| EP2968391A1 (en) | 2013-03-13 | 2016-01-20 | Moderna Therapeutics, Inc. | Long-lived polynucleotide molecules |
| 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 |
| JP6358661B2 (ja) * | 2013-03-19 | 2018-07-18 | 公立大学法人首都大学東京 | 界面活性剤様化合物 |
| WO2015002513A2 (ko) * | 2013-07-05 | 2015-01-08 | (주)바이오니아 | 호흡기 질환 연관 유전자 특이적 siRNA, 그러한 siRNA를 포함하는 이중나선 올리고 RNA 구조체 및 이를 포함하는 호흡기 질환 예방 또는 치료용 조성물 |
| US9695421B2 (en) | 2013-07-05 | 2017-07-04 | Bioneer Corporation | Dengue virus-specific siRNA, double helix oligo-RNA structure comprising siRNA, and composition for suppressing proliferation of dengue virus comprising RNA structure |
| CN105705638B (zh) | 2013-07-05 | 2019-11-26 | 柏业公司 | 改善的具有高效能的纳米颗粒型寡核苷酸结构及其制备方法 |
| SMT202100691T1 (it) | 2013-07-11 | 2022-01-10 | Modernatx Inc | Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso |
| 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 |
| KR101647178B1 (ko) * | 2013-11-28 | 2016-08-23 | 충남대학교산학협력단 | 효소 절단성 링커 또는 올리고 라이신을 포함하는 폴리에틸렌글리콜-리피드를 이용한 안정화된 플라스미드-지질 입자 |
| CN106061466A (zh) | 2013-12-19 | 2016-10-26 | 诺华股份有限公司 | 瘦蛋白mRNA组合物和制剂 |
| CN106459974A (zh) | 2014-04-04 | 2017-02-22 | 柏业公司 | 新颖的双链寡rna和包含它的用于预防或治疗纤维化或呼吸系统疾病的药物组合物 |
| SG10201912038TA (en) | 2014-04-23 | 2020-02-27 | Modernatx Inc | Nucleic acid vaccines |
| EP3160959B1 (en) * | 2014-06-24 | 2023-08-30 | Translate Bio, Inc. | Stereochemically enriched compositions for delivery of nucleic acids |
| 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 |
| CA2975447C (en) * | 2015-01-30 | 2021-02-23 | The Regents Of The University Of California | Spinal subpial gene delivery system |
| EP4286012A3 (en) | 2015-09-17 | 2024-05-29 | ModernaTX, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| HRP20220652T1 (hr) | 2015-12-10 | 2022-06-24 | Modernatx, Inc. | Pripravci i postupci unosa terapijskih sredstava |
| HUE057877T2 (hu) | 2015-12-22 | 2022-06-28 | Modernatx Inc | Vegyületek és készítmények terápiás szerek intracelluláris bejuttatására |
| US11583504B2 (en) | 2016-11-08 | 2023-02-21 | Modernatx, Inc. | Stabilized formulations of lipid nanoparticles |
| WO2018170322A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Crystal forms of amino lipids |
| SMT202300097T1 (it) | 2017-03-15 | 2023-05-12 | Modernatx Inc | Composto e composizioni per il rilascio intracellulare di agenti terapeutici |
| US11969506B2 (en) | 2017-03-15 | 2024-04-30 | Modernatx, Inc. | Lipid nanoparticle formulation |
| JP7285220B2 (ja) | 2017-05-18 | 2023-06-01 | モデルナティエックス インコーポレイテッド | 連結したインターロイキン-12(il12)ポリペプチドをコードするポリヌクレオチドを含む脂質ナノ粒子 |
| WO2018213789A1 (en) | 2017-05-18 | 2018-11-22 | Modernatx, Inc. | Modified messenger rna comprising functional rna elements |
| EP3638292A1 (en) | 2017-06-14 | 2020-04-22 | ModernaTX, Inc. | Polynucleotides encoding coagulation factor viii |
| US12077501B2 (en) | 2017-06-14 | 2024-09-03 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
| US11786607B2 (en) | 2017-06-15 | 2023-10-17 | Modernatx, Inc. | RNA formulations |
| WO2019046809A1 (en) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | METHODS OF MANUFACTURING LIPID NANOPARTICLES |
| WO2019048645A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE |
| JP7423521B2 (ja) | 2017-11-22 | 2024-01-29 | モダーナティエックス・インコーポレイテッド | フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド |
| US20220265856A1 (en) | 2017-11-22 | 2022-08-25 | Modernatx, Inc. | Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia |
| EP3714048B1 (en) | 2017-11-22 | 2025-04-09 | ModernaTX, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| MA51523A (fr) | 2018-01-05 | 2020-11-11 | Modernatx Inc | Polynucléotides codant pour des anticorps anti-virus du chikungunya |
| KR102056144B1 (ko) | 2018-03-08 | 2019-12-16 | 주식회사 파마리서치프로덕트 | Dna 단편 혼합물이 고농도로 함유된 유동성을 갖는 액제 조성물 및 이의 제조방법 |
| EP3775211B1 (en) | 2018-04-12 | 2023-04-05 | MiNA Therapeutics Limited | Sirt1-sarna compositions and methods of use |
| WO2019226650A1 (en) | 2018-05-23 | 2019-11-28 | Modernatx, Inc. | Delivery of dna |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| EP3833762A4 (en) | 2018-08-09 | 2022-09-28 | Verseau Therapeutics, Inc. | Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof |
| WO2020047201A1 (en) | 2018-09-02 | 2020-03-05 | Modernatx, Inc. | Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency |
| WO2020056147A2 (en) | 2018-09-13 | 2020-03-19 | Modernatx, Inc. | Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease |
| US20220243182A1 (en) | 2018-09-13 | 2022-08-04 | Modernatx, Inc. | Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease |
| WO2020056239A1 (en) | 2018-09-14 | 2020-03-19 | Modernatx, Inc. | Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome |
| US12263248B2 (en) | 2018-09-19 | 2025-04-01 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| AU2019345067A1 (en) | 2018-09-19 | 2021-04-08 | Modernatx, Inc. | High-purity peg lipids and uses thereof |
| MA53650A (fr) | 2018-09-19 | 2021-07-28 | Modernatx Inc | Lipides peg et leurs utilisations |
| 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 |
| EP3856233A1 (en) | 2018-09-27 | 2021-08-04 | Modernatx, Inc. | Polynucleotides encoding arginase 1 for the treatment of arginase deficiency |
| EP3953473A1 (en) | 2019-04-12 | 2022-02-16 | MiNA Therapeutics Limited | Sirt1-sarna compositions and methods of use |
| JP2022532078A (ja) | 2019-05-08 | 2022-07-13 | アストラゼネカ アクチボラグ | 皮膚及び創傷のための組成物並びにその使用の方法 |
| AU2020350759A1 (en) | 2019-09-19 | 2022-03-31 | Modernatx, Inc. | Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents |
| BR112022004936A2 (pt) | 2019-09-19 | 2022-06-14 | Modernatx Inc | Compostos lipídicos contendo carbonato e composições para distribuição intracelular de agentes terapêuticos |
| US20230235298A1 (en) | 2020-06-01 | 2023-07-27 | Modernatx, Inc. | Phenylalanine hydroxylase variants and uses thereof |
| US20230365966A1 (en) * | 2020-10-09 | 2023-11-16 | Napajen Pharma, Inc. | Homogeneous beta-1,3-glucan/nucleic acid complexes and use thereof |
| AU2021377895A1 (en) | 2020-11-13 | 2023-06-15 | Modernatx, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| US11524023B2 (en) | 2021-02-19 | 2022-12-13 | Modernatx, Inc. | Lipid nanoparticle compositions and methods of formulating the same |
| WO2022204371A1 (en) | 2021-03-24 | 2022-09-29 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof |
| WO2022204369A1 (en) | 2021-03-24 | 2022-09-29 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| US20240216288A1 (en) | 2021-03-24 | 2024-07-04 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof |
| WO2022204370A1 (en) | 2021-03-24 | 2022-09-29 | Modernatx, Inc. | Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency |
| US20240207444A1 (en) | 2021-03-24 | 2024-06-27 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof |
| EP4314292A1 (en) | 2021-03-26 | 2024-02-07 | MiNA Therapeutics Limited | Tmem173 sarna compositions and methods of use |
| EP4355882A2 (en) | 2021-06-15 | 2024-04-24 | Modernatx, Inc. | Engineered polynucleotides for cell-type or microenvironment-specific expression |
| WO2022271776A1 (en) | 2021-06-22 | 2022-12-29 | Modernatx, Inc. | Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome |
| US20250017867A1 (en) | 2021-10-01 | 2025-01-16 | Modernatx, Inc. | Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease |
| WO2023099884A1 (en) | 2021-12-01 | 2023-06-08 | Mina Therapeutics Limited | Pax6 sarna compositions and methods of use |
| KR102927154B1 (ko) * | 2021-12-23 | 2026-02-12 | (주)삼양바이오팜 | 약물의 폐 전달용 나노입자 조성물 |
| KR102852074B1 (ko) * | 2021-12-23 | 2025-08-28 | 주식회사 삼양홀딩스 | 약물 전달용 지질 및 이를 포함하는 나노입자, 및 이 나노입자를 포함하는 약물 전달용 조성물 |
| WO2023170435A1 (en) | 2022-03-07 | 2023-09-14 | Mina Therapeutics Limited | Il10 sarna compositions and methods of use |
| WO2023183909A2 (en) | 2022-03-25 | 2023-09-28 | Modernatx, Inc. | Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia |
| US20260035707A1 (en) | 2022-07-26 | 2026-02-05 | Modernatx, Inc. | Engineered polynucleotides for temporal control of expression |
| WO2024044728A1 (en) * | 2022-08-26 | 2024-02-29 | Renagade Therapeutics Management Inc. | Pegylated lipid compounds and methods of use thereof |
| WO2024125597A1 (en) | 2022-12-14 | 2024-06-20 | Providence Therapeutics Holdings Inc. | Compositions and methods for infectious diseases |
| WO2024134199A1 (en) | 2022-12-22 | 2024-06-27 | Mina Therapeutics Limited | Chemically modified sarna compositions and methods of use |
| WO2024197033A1 (en) | 2023-03-21 | 2024-09-26 | Modernatx, Inc. | Polynucleotides encoding relaxin for the treatment of heart failure |
| EP4704816A1 (en) | 2023-05-03 | 2026-03-11 | ModernaTX, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| WO2025072482A1 (en) | 2023-09-27 | 2025-04-03 | Modernatx, Inc. | Immunoglobulin a protease polypeptides, polynucleotides, and uses thereof |
| WO2025255199A1 (en) | 2024-06-05 | 2025-12-11 | Modernatx, Inc. | Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof |
| WO2026027887A2 (en) | 2024-08-02 | 2026-02-05 | Mina Therapeutics Limited | Hbg1/2-sarna compositions and methods of use |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2580880A (en) | 1942-03-27 | 1952-01-01 | Nat Aluminate Corp | Prevention of foaming in steam generation |
| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| AU3203489A (en) | 1988-02-19 | 1989-09-22 | Upjohn Company, The | Lipophilic polyamines useful for treating hypercholesterolemia |
| JP2610752B2 (ja) * | 1992-07-02 | 1997-05-14 | 花王株式会社 | 新規ジアミドアミン及びその製造法 |
| JPH06345704A (ja) | 1993-06-04 | 1994-12-20 | Lion Corp | 新規なジアミドアミン化合物、その中間体及びその製造方法と柔軟剤 |
| CA2164088C (en) | 1993-06-07 | 2005-06-14 | Gary J. Nabel | Plasmids suitable for gene therapy |
| JPH0718571A (ja) * | 1993-06-29 | 1995-01-20 | Lion Corp | 柔軟剤組成物 |
| JPH0718569A (ja) * | 1993-06-29 | 1995-01-20 | Lion Corp | 柔軟剤組成物 |
| DE4425857A1 (de) * | 1994-07-07 | 1996-01-11 | Schering Ag | Kaskaden-Polymer-Komplexe, Verfahren zur ihrer Herstellung und diese enthaltende pharmazeutische Mittel |
| US5962428A (en) | 1995-03-30 | 1999-10-05 | Apollon, Inc. | Compositions and methods for delivery of genetic material |
| JPH101869A (ja) | 1996-06-12 | 1998-01-06 | Lion Corp | 液体柔軟剤組成物 |
| DE69735292T2 (de) | 1996-10-25 | 2006-10-19 | Gilead Sciences, Inc., Foster City | Gefaesszellen wachstumsfaktor (vegf) nukleinsäureligand-komplexe |
| AR014621A1 (es) * | 1997-12-12 | 2001-03-28 | Macromed Inc | Un poli (etilenglicol) con forma de estrella, heterofuncional, biocompatible, metodo para su obtencion y metodo para conjugarlo con una proteina |
| AUPP249298A0 (en) | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
| US6656498B1 (en) * | 1998-11-25 | 2003-12-02 | Vanderbilt University | Cationic liposomes for gene transfer |
| JP2005529959A (ja) * | 2002-06-14 | 2005-10-06 | マイラス コーポレイション | 細胞へのポリヌクレオチドの伝達をするための新規な方法 |
| ES2559828T3 (es) * | 2003-07-16 | 2016-02-16 | Protiva Biotherapeutics Inc. | ARN de interferencia encapsulado en lípidos |
| NZ592917A (en) * | 2003-09-15 | 2012-12-21 | Protiva Biotherapeutics Inc | Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates |
| US20050095280A1 (en) * | 2003-10-15 | 2005-05-05 | Michalakis Savva | Single-component pH-sensitive liposomes of reduced solid-to-liquid phase transition temperatures |
| WO2006049157A1 (ja) * | 2004-11-02 | 2006-05-11 | The New Industry Research Organization | コネキシン26阻害活性を有する多量体型オレアミド誘導体と、その癌治療等への利用 |
| DE502006008083D1 (de) * | 2005-04-15 | 2010-11-25 | Basf Se | Amphiphile wasserlösliche alkoxylierte polyalkylenimine mit einem inneren polyethylenoxidblock und einem äusseren polypropylenoxidblock |
| ATE536854T1 (de) * | 2005-10-20 | 2011-12-15 | Dentsply Detrey Gmbh | Dentales haftmittel |
| EP3192788A1 (en) * | 2006-10-03 | 2017-07-19 | Arbutus Biopharma Corporation | Lipid containing formulations |
| EP1955712A1 (en) * | 2007-02-09 | 2008-08-13 | Scil proteins GmbH | Multimeric conjugate |
| PT2414322T (pt) | 2009-03-20 | 2022-12-07 | Egen Inc | Derivados de poliamina |
-
2010
- 2010-03-19 PT PT107099475T patent/PT2414322T/pt unknown
- 2010-03-19 AU AU2010226434A patent/AU2010226434A1/en not_active Abandoned
- 2010-03-19 JP JP2012500998A patent/JP5782602B2/ja active Active
- 2010-03-19 ES ES10709947T patent/ES2931174T3/es active Active
- 2010-03-19 CN CN201080022046.3A patent/CN102438978B/zh active Active
- 2010-03-19 KR KR1020117024770A patent/KR101800833B1/ko not_active Expired - Fee Related
- 2010-03-19 WO PCT/US2010/028000 patent/WO2010108108A2/en not_active Ceased
- 2010-03-19 US US12/727,987 patent/US8460696B2/en active Active
- 2010-03-19 EP EP10709947.5A patent/EP2414322B1/en active Active
- 2010-03-19 CA CA2755963A patent/CA2755963C/en active Active
- 2010-03-19 KR KR1020177033391A patent/KR101906392B1/ko active Active
- 2010-03-19 CN CN201510146197.4A patent/CN104922676B/zh active Active
-
2013
- 2013-03-08 US US13/791,102 patent/US8932638B2/en active Active
- 2013-03-08 US US13/790,980 patent/US8900621B2/en active Active
-
2014
- 2014-12-18 US US14/575,555 patent/US9254334B2/en active Active
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2015
- 2015-02-02 JP JP2015018143A patent/JP6190401B2/ja active Active
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