JP2018536689A5 - - Google Patents
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- JP2018536689A5 JP2018536689A5 JP2018530028A JP2018530028A JP2018536689A5 JP 2018536689 A5 JP2018536689 A5 JP 2018536689A5 JP 2018530028 A JP2018530028 A JP 2018530028A JP 2018530028 A JP2018530028 A JP 2018530028A JP 2018536689 A5 JP2018536689 A5 JP 2018536689A5
- Authority
- JP
- Japan
- Prior art keywords
- double
- nucleotides
- composition
- scap
- stranded
- 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
- 229920002477 rna polymer Polymers 0.000 claims 14
- 239000002773 nucleotide Substances 0.000 claims 13
- 125000003729 nucleotide group Chemical group 0.000 claims 13
- 239000003795 chemical substances by application Substances 0.000 claims 11
- 239000000203 mixture Substances 0.000 claims 11
- 108091081021 Sense strand Proteins 0.000 claims 6
- 102000009822 Sterol Regulatory Element Binding Proteins Human genes 0.000 claims 6
- 108010020396 Sterol Regulatory Element Binding Proteins Proteins 0.000 claims 6
- 102100027223 Sterol regulatory element-binding protein cleavage-activating protein Human genes 0.000 claims 6
- 101710184555 Sterol regulatory element-binding protein cleavage-activating protein Proteins 0.000 claims 6
- 230000000692 anti-sense effect Effects 0.000 claims 6
- 238000012986 modification Methods 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 230000014509 gene expression Effects 0.000 claims 5
- 230000002401 inhibitory effect Effects 0.000 claims 4
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 claims 4
- 108010006519 Molecular Chaperones Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 231100000240 steatosis hepatitis Toxicity 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 208000004930 Fatty Liver Diseases 0.000 claims 1
- 206010019708 Hepatic steatosis Diseases 0.000 claims 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical class CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 1
- 101150000107 SCAP gene Proteins 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 208000010706 fatty liver disease Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000007863 steatosis Effects 0.000 claims 1
- 229940124597 therapeutic agent Drugs 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021203915A JP7475323B2 (ja) | 2015-12-10 | 2021-12-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
| JP2024065896A JP2024105280A (ja) | 2015-12-10 | 2024-04-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562265580P | 2015-12-10 | 2015-12-10 | |
| US62/265,580 | 2015-12-10 | ||
| US201662378964P | 2016-08-24 | 2016-08-24 | |
| US62/378,964 | 2016-08-24 | ||
| PCT/US2016/065781 WO2017100542A1 (en) | 2015-12-10 | 2016-12-09 | Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021203915A Division JP7475323B2 (ja) | 2015-12-10 | 2021-12-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2018536689A JP2018536689A (ja) | 2018-12-13 |
| JP2018536689A5 true JP2018536689A5 (OSRAM) | 2020-01-30 |
Family
ID=57750612
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018530028A Pending JP2018536689A (ja) | 2015-12-10 | 2016-12-09 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
| JP2021203915A Active JP7475323B2 (ja) | 2015-12-10 | 2021-12-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
| JP2024065896A Pending JP2024105280A (ja) | 2015-12-10 | 2024-04-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021203915A Active JP7475323B2 (ja) | 2015-12-10 | 2021-12-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
| JP2024065896A Pending JP2024105280A (ja) | 2015-12-10 | 2024-04-16 | ステロール調節エレメント結合タンパク質(SREBP)シャペロン(SCAP)iRNA組成物およびその使用方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (4) | US10767177B2 (OSRAM) |
| EP (1) | EP3387129A1 (OSRAM) |
| JP (3) | JP2018536689A (OSRAM) |
| WO (1) | WO2017100542A1 (OSRAM) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FI3366775T4 (fi) * | 2011-11-18 | 2025-11-14 | Alnylam Pharmaceuticals Inc | Modifioituja rnai-agensseja |
| WO2017100542A1 (en) | 2015-12-10 | 2017-06-15 | Alnylam Pharmaceuticals, Inc. | Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| CA3059348A1 (en) | 2017-04-11 | 2018-10-18 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family |
| KR102694809B1 (ko) | 2017-10-11 | 2024-08-16 | 리제너론 파마슈티칼스 인코포레이티드 | Pnpla3 i148m 변이를 발현하는 환자의 간 질환의 치료에서의 hsd17b13의 저해 |
| JP7261494B2 (ja) | 2017-12-01 | 2023-04-20 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用 |
| EP3719128B1 (en) | 2017-12-01 | 2025-01-15 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof |
| EP3718572B1 (en) | 2017-12-01 | 2024-07-31 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use |
| CN118291456A (zh) | 2017-12-01 | 2024-07-05 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途 |
| JP7360716B2 (ja) | 2017-12-01 | 2023-10-13 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物および複合体ならびに調製方法と使用 |
| JP7436030B2 (ja) | 2017-12-29 | 2024-02-21 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 複合体及びその調製方法と使用 |
| MX2020009812A (es) | 2018-03-21 | 2021-01-08 | Regeneron Pharma | COMPOSICIONES DE ARNI DE 17ß-HIDROXIESTEROIDE DESHIDROGENASA TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS. |
| BR112021001613A2 (pt) | 2018-08-13 | 2021-05-04 | Alnylam Pharmaceuticals, Inc. | agentes de ácido ribonucleico de fita dupla, célula, composições farmacêuticas, métodos de inibição da expressão gênica, de inibição da replicação e de tratar um sujeito, métodos para reduzir o nível de um antígeno e para reduzir a carga viral e uso de um agente de dsrna |
| WO2020038377A1 (zh) | 2018-08-21 | 2020-02-27 | 苏州瑞博生物技术有限公司 | 一种核酸、含有该核酸的药物组合物和缀合物及其用途 |
| US11896674B2 (en) | 2018-09-30 | 2024-02-13 | Suzhou Ribo Life Science Co., Ltd. | SiRNA conjugate, preparation method therefor and use thereof |
| HRP20231338T1 (hr) | 2018-12-20 | 2024-02-16 | Vir Biotechnology, Inc. | Kombinirana terapija hbv |
| JP7507495B2 (ja) | 2018-12-28 | 2024-06-28 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用 |
| CN113825839A (zh) * | 2019-03-20 | 2021-12-21 | 雷杰纳荣制药公司 | 用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高 |
| CA3133000A1 (en) | 2019-03-20 | 2020-09-24 | Regeneron Pharmaceuticals, Inc. | Treatment of increased lipid levels with sterol regulatory element binding transcription factor 1 (srebf1) inhibitors |
| CN118256498A (zh) | 2019-05-22 | 2024-06-28 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与缀合物及制备方法和用途 |
| US20220307022A1 (en) * | 2019-05-30 | 2022-09-29 | Amgen Inc. | Rnai constructs for inhibiting scap expression and methods of use thereof |
| KR20220163960A (ko) * | 2020-03-06 | 2022-12-12 | 알리고스 테라퓨틱스 인코포레이티드 | 변경된 짧은 개재성 핵산(siNA) 분자 및 이의 용도 |
| US20230323357A1 (en) * | 2020-06-09 | 2023-10-12 | Alnylam Pharmaceuticals, Inc. | TUMOR NECROSIS FACTOR RECEPTOR ASSOCIATED FACTOR 6 (TRAF6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| IL315878A (en) * | 2022-04-15 | 2024-11-01 | Dicerna Pharmaceuticals Inc | Compounds and Methods for Modulating SCAP Activity |
| WO2024104416A1 (zh) * | 2022-11-17 | 2024-05-23 | 北京福元医药股份有限公司 | 抑制SCAP基因表达的siRNA、其缀合物和药物组合物及用途 |
| CN116059403B (zh) * | 2023-03-17 | 2024-08-16 | 常州市武进人民医院(常州市第八人民医院) | 一种胆酸修饰的红细胞囊泡及其制备方法和应用 |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US1861608A (en) | 1929-12-21 | 1932-06-07 | Emerson Electric Mfg Co | Fan and means for directing the air current therethrough |
| US1861108A (en) | 1930-01-24 | 1932-05-31 | Eugene O Brace | Integral clutch and transmission control |
| US3974808A (en) | 1975-07-02 | 1976-08-17 | Ford Motor Company | Air intake duct assembly |
| US4708708A (en) | 1982-12-06 | 1987-11-24 | International Paper Company | Method and apparatus for skiving and hemming |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4683202A (en) | 1985-03-28 | 1987-07-28 | Cetus Corporation | Process for amplifying nucleic acid sequences |
| US4920016A (en) | 1986-12-24 | 1990-04-24 | Linear Technology, Inc. | Liposomes with enhanced circulation time |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| FR2645866B1 (fr) | 1989-04-17 | 1991-07-05 | Centre Nat Rech Scient | Nouvelles lipopolyamines, leur preparation et leur emploi |
| NL8901881A (nl) | 1989-07-20 | 1991-02-18 | Rockwool Grodan Bv | Drainagekoppelelement. |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| US5283185A (en) | 1991-08-28 | 1994-02-01 | University Of Tennessee Research Corporation | Method for delivering nucleic acids into cells |
| EP0646178A1 (en) | 1992-06-04 | 1995-04-05 | The Regents Of The University Of California | expression cassette with regularoty regions functional in the mammmlian host |
| WO1994000569A1 (en) | 1992-06-18 | 1994-01-06 | Genpharm International, Inc. | Methods for producing transgenic non-human animals harboring a yeast artificial chromosome |
| US5783701A (en) | 1992-09-08 | 1998-07-21 | Vertex Pharmaceuticals, Incorporated | Sulfonamide inhibitors of aspartyl protease |
| CA2156288C (en) | 1993-02-19 | 2005-10-18 | Junichi Yano | Glycerol derivative, device and pharmaceutical composition |
| US6191105B1 (en) | 1993-05-10 | 2001-02-20 | Protein Delivery, Inc. | Hydrophilic and lipophilic balanced microemulsion formulations of free-form and/or conjugation-stabilized therapeutic agents such as insulin |
| US6054299A (en) | 1994-04-29 | 2000-04-25 | Conrad; Charles A. | Stem-loop cloning vector and method |
| US5543152A (en) | 1994-06-20 | 1996-08-06 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5981501A (en) | 1995-06-07 | 1999-11-09 | Inex Pharmaceuticals Corp. | Methods for encapsulating plasmids in lipid bilayers |
| US7422902B1 (en) | 1995-06-07 | 2008-09-09 | The University Of British Columbia | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
| CA2222328C (en) | 1995-06-07 | 2012-01-10 | Inex Pharmaceuticals Corporation | Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer |
| US5858397A (en) | 1995-10-11 | 1999-01-12 | University Of British Columbia | Liposomal formulations of mitoxantrone |
| US5854033A (en) | 1995-11-21 | 1998-12-29 | Yale University | Rolling circle replication reporter systems |
| US5858401A (en) | 1996-01-22 | 1999-01-12 | Sidmak Laboratories, Inc. | Pharmaceutical composition for cyclosporines |
| US5994316A (en) | 1996-02-21 | 1999-11-30 | The Immune Response Corporation | Method of preparing polynucleotide-carrier complexes for delivery to cells |
| US6034135A (en) | 1997-03-06 | 2000-03-07 | Promega Biosciences, Inc. | Dimeric cationic lipids |
| AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
| PL341762A1 (en) | 1997-12-24 | 2001-05-07 | Vertex Pharma | Precursors of aspartil protease inhibitors |
| US6436989B1 (en) | 1997-12-24 | 2002-08-20 | Vertex Pharmaceuticals, Incorporated | Prodrugs of aspartyl protease inhibitors |
| WO2000003683A2 (en) | 1998-07-20 | 2000-01-27 | Inex Pharmaceuticals Corporation | Liposomal encapsulated nucleic acid-complexes |
| CA2345936A1 (en) | 1998-10-09 | 2000-04-20 | Ingene, Inc. | Production of ssdna in a cell |
| BR9914772A (pt) | 1998-10-09 | 2001-12-11 | Ingene Inc | Conjunto de elementos genéticos, vetor, célulahospedeira, conjunto para a produção de umasequência de ácido nucléico, método para aprodução in vivo ou in vitro de uma sequência deácido nucléico, transcrição de cdna, molécula deácido nucléico inibidor, transcrição de mrna,molécula heteroduplex e composiçãofarmacêutica |
| TNSN00027A1 (fr) | 1999-02-12 | 2005-11-10 | Vertex Pharma | Inhibiteurs de l'aspartyle protease |
| IT1318539B1 (it) | 2000-05-26 | 2003-08-27 | Italfarmaco Spa | Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente |
| US7063860B2 (en) | 2001-08-13 | 2006-06-20 | University Of Pittsburgh | Application of lipid vehicles and use for drug delivery |
| EP2213738B1 (en) * | 2002-11-14 | 2012-10-10 | Dharmacon, Inc. | siRNA molecules targeting Bcl-2 |
| 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 |
| JP2007325525A (ja) * | 2006-06-07 | 2007-12-20 | Ikuo Wada | タンパク質分泌・輸送機構に関与する分泌・輸送機構関連タンパク質、ならびにこれらの同定・機能解析方法 |
| US7737266B2 (en) * | 2006-09-18 | 2010-06-15 | Board Of Regents, The University Of Texas System | RNAi modulation of SCAP and therapeutics uses thereof |
| KR102066189B1 (ko) | 2009-06-10 | 2020-01-14 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| WO2011005861A1 (en) | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide end caps |
| KR102232287B1 (ko) * | 2011-06-21 | 2021-03-29 | 알닐람 파마슈티칼스 인코포레이티드 | 안지오포이에틴-유사 3(ANGPTL3) iRNA 조성물 및 그 사용 방법 |
| WO2012177639A2 (en) * | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
| AU2012340159B2 (en) * | 2011-11-18 | 2017-09-07 | Alnylam Pharmaceuticals, Inc. | RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases |
| WO2017100542A1 (en) | 2015-12-10 | 2017-06-15 | Alnylam Pharmaceuticals, Inc. | Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof |
-
2016
- 2016-12-09 WO PCT/US2016/065781 patent/WO2017100542A1/en not_active Ceased
- 2016-12-09 EP EP16822839.3A patent/EP3387129A1/en active Pending
- 2016-12-09 JP JP2018530028A patent/JP2018536689A/ja active Pending
-
2018
- 2018-06-07 US US16/002,034 patent/US10767177B2/en active Active
-
2020
- 2020-07-27 US US16/939,119 patent/US11434487B2/en active Active
-
2021
- 2021-12-16 JP JP2021203915A patent/JP7475323B2/ja active Active
-
2022
- 2022-07-08 US US17/860,141 patent/US20230093948A1/en not_active Abandoned
-
2024
- 2024-04-16 JP JP2024065896A patent/JP2024105280A/ja active Pending
- 2024-06-10 US US18/738,165 patent/US20250034570A1/en active Pending
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