JP2018511307A5 - - Google Patents
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- JP2018511307A5 JP2018511307A5 JP2017546624A JP2017546624A JP2018511307A5 JP 2018511307 A5 JP2018511307 A5 JP 2018511307A5 JP 2017546624 A JP2017546624 A JP 2017546624A JP 2017546624 A JP2017546624 A JP 2017546624A JP 2018511307 A5 JP2018511307 A5 JP 2018511307A5
- Authority
- JP
- Japan
- Prior art keywords
- compound
- wing segment
- modified oligonucleotide
- linked nucleosides
- modified
- 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.)
- Granted
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- 150000001875 compounds Chemical class 0.000 claims description 69
- 239000002777 nucleoside Substances 0.000 claims description 52
- 108091034117 Oligonucleotide Proteins 0.000 claims description 51
- 125000003835 nucleoside group Chemical group 0.000 claims description 37
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 15
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 13
- XEEYBQQBJWHFJM-UHFFFAOYSA-N Iron Chemical compound [Fe] XEEYBQQBJWHFJM-UHFFFAOYSA-N 0.000 claims description 12
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 11
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 9
- 208000007502 anemia Diseases 0.000 claims description 8
- 230000000295 complement effect Effects 0.000 claims description 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 8
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 8
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical group CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 8
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims description 7
- 239000000203 mixture Substances 0.000 claims description 7
- 241001465754 Metazoa Species 0.000 claims description 6
- 229910052742 iron Inorganic materials 0.000 claims description 6
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims description 5
- 229930024421 Adenine Natural products 0.000 claims description 4
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 4
- 206010053138 Congenital aplastic anaemia Diseases 0.000 claims description 4
- 208000002903 Thalassemia Diseases 0.000 claims description 4
- 102000004338 Transferrin Human genes 0.000 claims description 4
- 108090000901 Transferrin Proteins 0.000 claims description 4
- 238000009825 accumulation Methods 0.000 claims description 4
- 229960000643 adenine Drugs 0.000 claims description 4
- 239000003085 diluting agent Substances 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000035475 disorder Diseases 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims description 4
- 229940113082 thymine Drugs 0.000 claims description 4
- 239000012581 transferrin Substances 0.000 claims description 4
- 150000003839 salts Chemical class 0.000 claims description 3
- XJOTXKZIRSHZQV-RXHOOSIZSA-N (3S)-3-amino-4-[[(2S,3R)-1-[[(2S)-1-[[(2S)-1-[(2S)-2-[[(2S,3S)-1-[[(1R,6R,12R,17R,20S,23S,26R,31R,34R,39R,42S,45S,48S,51S,59S)-51-(4-aminobutyl)-31-[[(2S)-6-amino-1-[[(1S,2R)-1-carboxy-2-hydroxypropyl]amino]-1-oxohexan-2-yl]carbamoyl]-20-benzyl-23-[(2S)-butan-2-yl]-45-(3-carbamimidamidopropyl)-48-(hydroxymethyl)-42-(1H-imidazol-4-ylmethyl)-59-(2-methylsulfanylethyl)-7,10,19,22,25,33,40,43,46,49,52,54,57,60,63,64-hexadecaoxo-3,4,14,15,28,29,36,37-octathia-8,11,18,21,24,32,41,44,47,50,53,55,58,61,62,65-hexadecazatetracyclo[32.19.8.26,17.212,39]pentahexacontan-26-yl]amino]-3-methyl-1-oxopentan-2-yl]carbamoyl]pyrrolidin-1-yl]-1-oxo-3-phenylpropan-2-yl]amino]-3-(1H-imidazol-4-yl)-1-oxopropan-2-yl]amino]-3-hydroxy-1-oxobutan-2-yl]amino]-4-oxobutanoic acid Chemical compound CC[C@H](C)[C@H](NC(=O)[C@@H]1CCCN1C(=O)[C@H](Cc1ccccc1)NC(=O)[C@H](Cc1cnc[nH]1)NC(=O)[C@@H](NC(=O)[C@@H](N)CC(O)=O)[C@@H](C)O)C(=O)N[C@H]1CSSC[C@H](NC(=O)[C@@H]2CSSC[C@@H]3NC(=O)[C@@H]4CSSC[C@H](NC(=O)[C@H](Cc5ccccc5)NC(=O)[C@@H](NC1=O)[C@@H](C)CC)C(=O)N[C@@H](CSSC[C@H](NC(=O)[C@H](CCCCN)NC(=O)[C@H](CO)NC(=O)[C@H](CCCNC(N)=N)NC(=O)[C@H](Cc1cnc[nH]1)NC3=O)C(=O)NCC(=O)N[C@@H](CCSC)C(=O)N2)C(=O)NCC(=O)N4)C(=O)N[C@@H](CCCCN)C(=O)N[C@@H]([C@@H](C)O)C(O)=O XJOTXKZIRSHZQV-RXHOOSIZSA-N 0.000 claims description 2
- 102100033051 40S ribosomal protein S19 Human genes 0.000 claims description 2
- 208000033932 Blackfan-Diamond anemia Diseases 0.000 claims description 2
- 201000004449 Diamond-Blackfan anemia Diseases 0.000 claims description 2
- 201000004939 Fanconi anemia Diseases 0.000 claims description 2
- 208000025499 G6PD deficiency Diseases 0.000 claims description 2
- 206010018444 Glucose-6-phosphate dehydrogenase deficiency Diseases 0.000 claims description 2
- 206010018910 Haemolysis Diseases 0.000 claims description 2
- 208000018565 Hemochromatosis Diseases 0.000 claims description 2
- 208000031953 Hereditary hemorrhagic telangiectasia Diseases 0.000 claims description 2
- 101000798696 Homo sapiens Transmembrane protease serine 6 Proteins 0.000 claims description 2
- 201000003793 Myelodysplastic syndrome Diseases 0.000 claims description 2
- 208000008601 Polycythemia Diseases 0.000 claims description 2
- 102100032452 Transmembrane protease serine 6 Human genes 0.000 claims description 2
- 208000027418 Wounds and injury Diseases 0.000 claims description 2
- 208000005980 beta thalassemia Diseases 0.000 claims description 2
- 125000002619 bicyclic group Chemical group 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims description 2
- 230000001684 chronic effect Effects 0.000 claims description 2
- 230000006378 damage Effects 0.000 claims description 2
- 229910003460 diamond Inorganic materials 0.000 claims description 2
- 239000010432 diamond Substances 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 210000003617 erythrocyte membrane Anatomy 0.000 claims description 2
- 208000008605 glucosephosphate dehydrogenase deficiency Diseases 0.000 claims description 2
- 230000008588 hemolysis Effects 0.000 claims description 2
- 102000018511 hepcidin Human genes 0.000 claims description 2
- 229940066919 hepcidin Drugs 0.000 claims description 2
- 108060003558 hepcidin Proteins 0.000 claims description 2
- 208000014674 injury Diseases 0.000 claims description 2
- 210000000056 organ Anatomy 0.000 claims description 2
- 208000007056 sickle cell anemia Diseases 0.000 claims description 2
- 208000011580 syndromic disease Diseases 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 210000001519 tissue Anatomy 0.000 claims description 2
- 229940126836 transmembrane protease serine 6 synthesis reducer Drugs 0.000 claims description 2
- HWPZZUQOWRWFDB-UHFFFAOYSA-N 1-methylcytosine Chemical compound CN1C=CC(N)=NC1=O HWPZZUQOWRWFDB-UHFFFAOYSA-N 0.000 claims 1
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical group P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims 1
- 239000002953 phosphate buffered saline Substances 0.000 claims 1
- 210000002966 serum Anatomy 0.000 description 2
- 229940104302 cytosine Drugs 0.000 description 1
- 230000003247 decreasing effect Effects 0.000 description 1
- 238000000034 method Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562142986P | 2015-04-03 | 2015-04-03 | |
| US62/142,986 | 2015-04-03 | ||
| PCT/US2016/025883 WO2016161429A1 (en) | 2015-04-03 | 2016-04-04 | Compounds and methods for modulating tmprss6 expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021015452A Division JP7037681B2 (ja) | 2015-04-03 | 2021-02-03 | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018511307A JP2018511307A (ja) | 2018-04-26 |
| JP2018511307A5 true JP2018511307A5 (cg-RX-API-DMAC7.html) | 2019-05-16 |
| JP6833705B2 JP6833705B2 (ja) | 2021-02-24 |
Family
ID=57007353
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017546624A Active JP6833705B2 (ja) | 2015-04-03 | 2016-04-04 | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
| JP2021015452A Active JP7037681B2 (ja) | 2015-04-03 | 2021-02-03 | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
| JP2022033166A Withdrawn JP2022104917A (ja) | 2015-04-03 | 2022-03-04 | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021015452A Active JP7037681B2 (ja) | 2015-04-03 | 2021-02-03 | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
| JP2022033166A Withdrawn JP2022104917A (ja) | 2015-04-03 | 2022-03-04 | Ｔｍｐｒｓｓ６発現を調節するための化合物及び方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (4) | US10415038B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3277817A4 (cg-RX-API-DMAC7.html) |
| JP (3) | JP6833705B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR102539587B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN107429250B (cg-RX-API-DMAC7.html) |
| AU (1) | AU2016244106B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2978100C (cg-RX-API-DMAC7.html) |
| HK (1) | HK1249140A1 (cg-RX-API-DMAC7.html) |
| IL (2) | IL253509B2 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2017012426A (cg-RX-API-DMAC7.html) |
| RU (1) | RU2017137410A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2016161429A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| CN111278991B (zh) | 2017-08-25 | 2022-04-01 | 斯托克制药公司 | 用于治疗病况和疾病的反义寡聚体 |
| WO2019157531A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| TWI840345B (zh) * | 2018-03-02 | 2024-05-01 | 美商Ionis製藥公司 | Irf4表現之調節劑 |
| EP3788065B1 (en) | 2018-04-30 | 2025-08-27 | The Children's Hospital Of Philadelphia | Improving anemias by combining agents |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| EP3598995A1 (en) * | 2018-07-26 | 2020-01-29 | Silence Therapeutics GmbH | Products and compositions |
| AU2019406186A1 (en) * | 2018-12-20 | 2021-07-15 | Praxis Precision Medicines, Inc. | Compositions and methods for the treatment of KCNT1 related disorders |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| JP2023130528A (ja) * | 2020-06-24 | 2023-09-21 | 田辺三菱製薬株式会社 | Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物 |
| US12152243B2 (en) | 2020-09-17 | 2024-11-26 | Quelltx, Inc. | Therapeutic compositions for treating pain via multiple targets |
| AU2022265493A1 (en) * | 2021-04-27 | 2023-09-21 | Silence Therapeutics Gmbh | Sirna targeting tmprss6 for the treatment of myeloproliferative disorders |
| CA3218205A1 (en) * | 2021-04-28 | 2022-11-03 | Q-State Biosciences, Inc. | Therapeutic compositions for treating pain via multiple targets |
| CN115247173B (zh) * | 2021-11-11 | 2025-11-11 | 南京启真基因工程有限公司 | 构建tmprss6基因突变的缺铁性贫血猪核移植供体细胞的基因编辑系统及其应用 |
| TWI868755B (zh) | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 |
| CN117136236B (zh) * | 2022-09-29 | 2024-03-15 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
| WO2024060649A1 (zh) * | 2022-09-29 | 2024-03-28 | 广州必贝特医药股份有限公司 | 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用 |
| WO2024200512A1 (en) | 2023-03-27 | 2024-10-03 | Silence Therapeutics Gmbh | Compounds and compositions for use in stem cell transplantation |
| WO2025148896A1 (en) * | 2024-01-08 | 2025-07-17 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of transmembrane serine protease 6 (tmprss6) |
| WO2025206092A1 (ja) * | 2024-03-27 | 2025-10-02 | 田辺三菱製薬株式会社 | Ｍｙｏｒｅｇｕｌｉｎ発現を調節するための化合物及び医薬組成物 |
Family Cites Families (84)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
| JPH05504552A (ja) | 1989-10-24 | 1993-07-15 | ギリアド サイエンシズ，インコーポレイテッド | ２’位が改変されたオリゴヌクレオチド |
| US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
| US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
| GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
| EP0745689A3 (en) | 1990-05-11 | 1996-12-11 | Microprobe Corporation | A dipstick for a nucleic acid hybridization assay |
| US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
| EP0538194B1 (de) | 1991-10-17 | 1997-06-04 | Novartis AG | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
| US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
| FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
| EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
| EP0691968B1 (en) | 1993-03-30 | 1997-07-16 | Sanofi | Acyclic nucleoside analogs and oligonucleotide sequences containing them |
| US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
| US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
| US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
| US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
| JP2000501414A (ja) | 1995-11-22 | 2000-02-08 | ザ・ジョンズ・ホプキンス・ユニバーシティー | 生体分子の細胞取り込みを高めるリガンド |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| EP2253639A1 (en) | 1997-09-12 | 2010-11-24 | Exiqon A/S | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
| US6300319B1 (en) | 1998-06-16 | 2001-10-09 | Isis Pharmaceuticals, Inc. | Targeted oligonucleotide conjugates |
| US6043352A (en) | 1998-08-07 | 2000-03-28 | Isis Pharmaceuticals, Inc. | 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides |
| IL145778A0 (en) | 1999-04-21 | 2002-07-25 | American Home Prod | Methods and compositions for inhibiting the function of polynucleotide sequences |
| CN102180924A (zh) | 1999-05-04 | 2011-09-14 | 桑塔里斯制药公司 | L-核糖-lna类似物 |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| DE60027203T2 (de) | 1999-12-30 | 2007-01-04 | K.U. Leuven Research & Development | Cyclohexennukleinsäuren |
| US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
| CN100406065C (zh) | 2000-12-01 | 2008-07-30 | 细胞工厂治疗公司 | 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法 |
| WO2003004602A2 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| WO2004044139A2 (en) | 2002-11-05 | 2004-05-27 | Isis Parmaceuticals, Inc. | Modified oligonucleotides for use in rna interference |
| AU2003291753B2 (en) | 2002-11-05 | 2010-07-08 | Isis Pharmaceuticals, Inc. | Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation |
| US8090542B2 (en) | 2002-11-14 | 2012-01-03 | Dharmacon Inc. | Functional and hyperfunctional siRNA |
| US7250496B2 (en) | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| US7217807B2 (en) | 2002-11-26 | 2007-05-15 | Rosetta Genomics Ltd | Bioinformatically detectable group of novel HIV regulatory genes and uses thereof |
| US6673661B1 (en) | 2002-12-20 | 2004-01-06 | Taiwan Semiconductor Manufacturing Co., Ltd. | Self-aligned method for forming dual gate thin film transistor (TFT) device |
| US7723509B2 (en) | 2003-04-17 | 2010-05-25 | Alnylam Pharmaceuticals | IRNA agents with biocleavable tethers |
| WO2004106356A1 (en) | 2003-05-27 | 2004-12-09 | Syddansk Universitet | Functionalized nucleotide derivatives |
| US7888497B2 (en) | 2003-08-13 | 2011-02-15 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof |
| DK1661905T3 (da) | 2003-08-28 | 2012-07-23 | Takeshi Imanishi | Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype |
| US20050054027A1 (en) | 2003-09-09 | 2005-03-10 | Irm Llc | Modulators of transmembrane protease serine 6 |
| WO2005027962A1 (en) | 2003-09-18 | 2005-03-31 | Isis Pharmaceuticals, Inc. | 4’-thionucleosides and oligomeric compounds |
| WO2005032733A1 (en) | 2003-10-06 | 2005-04-14 | Nam Huat Tiling & Panelling Co. Pte Ltd | A tile management system |
| JP2008501694A (ja) | 2004-06-03 | 2008-01-24 | アイシス ファーマシューティカルズ、インク． | 遺伝子調節の使用のために個別に修飾された鎖を有する二本鎖組成物 |
| US20080261905A1 (en) | 2004-11-08 | 2008-10-23 | K.U. Leuven Research And Development | Modified Nucleosides for Rna Interference |
| US20060148740A1 (en) | 2005-01-05 | 2006-07-06 | Prosensa B.V. | Mannose-6-phosphate receptor mediated gene transfer into muscle cells |
| CN102908630B (zh) | 2006-01-27 | 2014-11-19 | Isis制药公司 | 6-修饰的双环核酸类似物 |
| US8178503B2 (en) | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| JP5441688B2 (ja) | 2006-05-11 | 2014-03-12 | アイシス ファーマシューティカルズ， インコーポレーテッド | ５’修飾二環式核酸類似体 |
| WO2008021290A2 (en) | 2006-08-09 | 2008-02-21 | Homestead Clinical Corporation | Organ-specific proteins and methods of their use |
| WO2008024858A2 (en) | 2006-08-22 | 2008-02-28 | University Of Virginia Patent Foundation | Methods and compounds regulating the erythroid response to iron deficiency |
| EP2125852B1 (en) | 2007-02-15 | 2016-04-06 | Ionis Pharmaceuticals, Inc. | 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom |
| DK2170917T3 (da) | 2007-05-30 | 2012-10-08 | Isis Pharmaceuticals Inc | N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro |
| ES2386492T3 (es) | 2007-06-08 | 2012-08-21 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos carbocíclicos |
| ES2376507T5 (es) | 2007-07-05 | 2015-08-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos 6-disustituidos |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| US8530640B2 (en) | 2008-02-07 | 2013-09-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexitol nucleic acid analogs |
| CA3044980A1 (en) | 2008-04-11 | 2009-10-15 | Alnylam Pharmaceuticals, Inc. | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| WO2009143387A2 (en) | 2008-05-22 | 2009-11-26 | Isis Pharmaceuticals, Inc. | Modulation of smrt expression |
| KR101877698B1 (ko) * | 2008-08-25 | 2018-07-12 | 엑스칼리아드 파마슈티컬즈, 인코포레이티드 | 결합 조직 성장 인자에 대한 안티센스 올리고뉴클레오타이드 및 그의 용도 |
| EP2356129B1 (en) | 2008-09-24 | 2013-04-03 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
| WO2010036696A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Cyclohexenyl nucleic acid analogs |
| CA2786071C (en) | 2010-01-08 | 2021-01-12 | Isis Pharmaceuticals, Inc. | Modulation of angiopoietin-like 3 expression |
| US8993738B2 (en) | 2010-04-28 | 2015-03-31 | Isis Pharmaceuticals, Inc. | Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom |
| WO2012037254A1 (en) | 2010-09-15 | 2012-03-22 | Alnylam Pharmaceuticals, Inc. | MODIFIED iRNA AGENTS |
| PH12013501969B1 (en) * | 2011-03-29 | 2018-08-31 | Alnylam Pharmaceuticals Inc | Compositions and methods for inhibiting expression of tmprss6 gene |
| EP3640332A1 (en) | 2011-08-29 | 2020-04-22 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| RU2598709C2 (ru) * | 2011-09-20 | 2016-09-27 | Айсис Фармасьютикалс, Инк. | Антисмысловая регуляция экспрессии gcgr |
| WO2013070786A1 (en) * | 2011-11-07 | 2013-05-16 | Isis Pharmaceuticals, Inc. | Modulation of tmprss6 expression |
| EP2850183A4 (en) | 2012-05-16 | 2016-02-10 | Rana Therapeutics Inc | COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION |
| WO2014076196A1 (en) | 2012-11-15 | 2014-05-22 | Santaris Pharma A/S | Anti apob antisense conjugate compounds |
| HUE050394T2 (hu) | 2013-05-01 | 2020-11-30 | Ionis Pharmaceuticals Inc | Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények |
| WO2014190157A1 (en) * | 2013-05-22 | 2014-11-27 | Alnylam Pharmaceuticals, Inc. | Tmprss6 compositions and methods of use thereof |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| WO2016137923A1 (en) | 2015-02-23 | 2016-09-01 | Ionis Pharmaceuticals, Inc. | Method for solution phase detritylation of oligomeric compounds |
-
2016
- 2016-04-04 EP EP16774409.3A patent/EP3277817A4/en active Pending
- 2016-04-04 CN CN201680015815.4A patent/CN107429250B/zh active Active
- 2016-04-04 US US15/562,843 patent/US10415038B2/en active Active
- 2016-04-04 KR KR1020177030957A patent/KR102539587B1/ko active Active
- 2016-04-04 RU RU2017137410A patent/RU2017137410A/ru not_active Application Discontinuation
- 2016-04-04 MX MX2017012426A patent/MX2017012426A/es unknown
- 2016-04-04 HK HK18108870.8A patent/HK1249140A1/zh unknown
- 2016-04-04 WO PCT/US2016/025883 patent/WO2016161429A1/en not_active Ceased
- 2016-04-04 JP JP2017546624A patent/JP6833705B2/ja active Active
- 2016-04-04 AU AU2016244106A patent/AU2016244106B2/en active Active
- 2016-04-04 CA CA2978100A patent/CA2978100C/en active Active
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- 2019-08-01 US US16/528,925 patent/US20200190522A1/en not_active Abandoned
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2021
- 2021-02-03 JP JP2021015452A patent/JP7037681B2/ja active Active
- 2021-09-24 US US17/484,386 patent/US20220259600A1/en not_active Abandoned
- 2021-09-26 IL IL286669A patent/IL286669A/en unknown
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2022
- 2022-03-04 JP JP2022033166A patent/JP2022104917A/ja not_active Withdrawn
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2023
- 2023-07-25 US US18/358,591 patent/US20240026364A1/en active Pending
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