JP2009530257A5 - - Google Patents
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- Publication number
- JP2009530257A5 JP2009530257A5 JP2008558913A JP2008558913A JP2009530257A5 JP 2009530257 A5 JP2009530257 A5 JP 2009530257A5 JP 2008558913 A JP2008558913 A JP 2008558913A JP 2008558913 A JP2008558913 A JP 2008558913A JP 2009530257 A5 JP2009530257 A5 JP 2009530257A5
- Authority
- JP
- Japan
- Prior art keywords
- use according
- cns
- compound
- sina
- gene
- 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.)
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- 210000003169 Central Nervous System Anatomy 0.000 claims 8
- 240000003247 Erythrina poeppigiana Species 0.000 claims 6
- 235000009776 Rathbunia alamosensis Nutrition 0.000 claims 6
- 101700060380 SINA Proteins 0.000 claims 6
- 150000001875 compounds Chemical class 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 241000894007 species Species 0.000 claims 3
- IQFYYKKMVGJFEH-XLPZGREQSA-N DEOXYTHYMIDINE Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 2
- 108020004999 Messenger RNA Proteins 0.000 claims 2
- 230000025458 RNA interference Effects 0.000 claims 2
- 229920002106 messenger RNA Polymers 0.000 claims 2
- 230000002987 rna-interference Effects 0.000 claims 2
- 229920000160 (ribonucleotides)n+m Polymers 0.000 claims 1
- 102000012440 Acetylcholinesterase Human genes 0.000 claims 1
- 229940022698 Acetylcholinesterase Drugs 0.000 claims 1
- 108010022752 Acetylcholinesterase Proteins 0.000 claims 1
- 206010001897 Alzheimer's disease Diseases 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 201000001971 Huntington's disease Diseases 0.000 claims 1
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 1
- 229920000272 Oligonucleotide Polymers 0.000 claims 1
- 206010061536 Parkinson's disease Diseases 0.000 claims 1
- 108020004459 Small Interfering RNA Proteins 0.000 claims 1
- 229920001891 Small hairpin RNA Polymers 0.000 claims 1
- 229940104230 Thymidine Drugs 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000001105 regulatory Effects 0.000 claims 1
- 239000002924 silencing RNA Substances 0.000 claims 1
- 239000004055 small Interfering RNA Substances 0.000 claims 1
Claims (16)
- 中枢神経系（ＣＮＳ）で発現される遺伝子にＲＮＡ干渉を引き起こす化合物の、ＣＮＳの障害の治療のための医薬の調製における使用であって、前記医薬が鼻腔内投与用に処方される、使用。
- 前記障害が、認知症、アルツハイマー病、ハンチントン病および／またはパーキンソン病、ならびにＣＮＳの遺伝子の変異に関連する先天性疾患から選択される、請求項１に記載の使用。
- 前記標的遺伝子が、タウ、ハンチンチン、アセチルコリンエステラーゼまたはこれらの遺伝子の変異対立遺伝子から選択される、請求項２に記載の使用。
- 前記標的遺伝子の発現が、ＣＮＳの細胞内で調節される、請求項３に記載の使用。
- 前記化合物がｓｉＲＮＡ、ｄｓＲＮＡ及びｓｈＲＮＡから選択されるｓｉＮＡである、請求項１から４のいずれか一項に記載の使用。
- 前記化合物が修飾されたオリゴヌクレオチドを含む、請求項１から５のいずれか一項に記載の使用。
- 前記ｓｉＮＡが４０塩基対以下の長さである、請求項６に記載の使用。
- 前記ｓｉＮＡが３’突出を有する、請求項６に記載の使用。
- 前記３’突出がチミジンジヌクレオチドである、請求項８に記載の使用。
- 複数の種の化合物が用いられる、請求項１から９のいずれか一項に記載の使用。
- 前記複数の種が、同じｍＲＮＡ種または異なるｍＲＮＡ種を標的にする、請求項１０に記載の使用。
- 前記標的遺伝子がタウであり、前記ｓｉＮＡが、配列番号１〜１６０から選択されるヌクレオチド配列を標的にする、請求項１から１１のいずれか一項に記載の使用。
- 前記ｓｉＮＡが、配列番号１６１〜３１８から選択される配列を有する、請求項１２に記載の使用。
- 鼻腔内投与用に処方される、ＣＮＳで発現される遺伝子を標的にする１つまたは複数のｓｉＮＡを含む医薬組成物。
- 前記遺伝子が、ＣＮＳで特異的に発現される、請求項１４に記載の組成物。
- ＲＮＡ干渉を引き起こす化合物の、ＣＮＳの障害を治療するための医薬の調製における使用であって、前記化合物が患者に鼻腔内投与される、使用。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB0605337.5A GB0605337D0 (en) | 2006-03-17 | 2006-03-17 | Treatment of CNS conditions |
| GB0605337.5 | 2006-03-17 | ||
| PCT/GB2007/050128 WO2007107789A2 (en) | 2006-03-17 | 2007-03-16 | Treatment of cns conditions |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013161765A Division JP5923466B2 (ja) | 2006-03-17 | 2013-08-02 | Ｃｎｓ状態の治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2009530257A JP2009530257A (ja) | 2009-08-27 |
| JP2009530257A5 true JP2009530257A5 (ja) | 2010-03-11 |
| JP5781722B2 JP5781722B2 (ja) | 2015-09-24 |
Family
ID=36292900
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2008558913A Active JP5781722B2 (ja) | 2006-03-17 | 2007-03-16 | Ｃｎｓ状態の治療 |
| JP2013161765A Active JP5923466B2 (ja) | 2006-03-17 | 2013-08-02 | Ｃｎｓ状態の治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013161765A Active JP5923466B2 (ja) | 2006-03-17 | 2013-08-02 | Ｃｎｓ状態の治療 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US8871729B2 (ja) |
| EP (1) | EP2004823B1 (ja) |
| JP (2) | JP5781722B2 (ja) |
| KR (2) | KR101605144B1 (ja) |
| CN (1) | CN101448944B (ja) |
| AU (1) | AU2007228570B2 (ja) |
| CA (1) | CA2645120C (ja) |
| ES (1) | ES2582649T3 (ja) |
| GB (1) | GB0605337D0 (ja) |
| HK (1) | HK1126812A1 (ja) |
| IL (1) | IL194114A (ja) |
| MX (1) | MX2008011731A (ja) |
| RU (1) | RU2426544C2 (ja) |
| WO (1) | WO2007107789A2 (ja) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20080249058A1 (en) * | 2007-04-05 | 2008-10-09 | Erik Roberson | Agents that reduce neuronal overexcitation |
| WO2010080452A2 (en) | 2008-12-18 | 2010-07-15 | Quark Pharmaceuticals, Inc. | siRNA COMPOUNDS AND METHODS OF USE THEREOF |
| CN102625809B (zh) * | 2009-09-11 | 2015-06-24 | Isis制药公司 | 亨廷顿表达的调节 |
| JP2013511990A (ja) | 2009-11-26 | 2013-04-11 | クォーク ファーマシューティカルズ インコーポレーティッド | 末端置換を含むｓｉＲＮＡ化合物 |
| US20110135613A1 (en) * | 2009-12-03 | 2011-06-09 | The J. David Gladstone Institutes | Methods for treating apolipoprotein e4-associated disorders |
| WO2011072091A1 (en) | 2009-12-09 | 2011-06-16 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the cns |
| JP6081798B2 (ja) * | 2010-02-26 | 2017-02-15 | メモリアル スローン−ケタリング キャンサー センター | ｍｉＲＮＡに関連する癌を検出および処置するための方法および組成物およびｍｉＲＮＡインヒビターおよび標的 |
| ES2638309T3 (es) | 2010-04-19 | 2017-10-19 | Nlife Therapeutics S.L. | Composiciones y métodos para la distribución selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas |
| EP2380595A1 (en) | 2010-04-19 | 2011-10-26 | Nlife Therapeutics S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| ES2663009T3 (es) | 2010-10-29 | 2018-04-10 | Sirna Therapeutics, Inc. | Inhibición de la expresión génica mediada por interferencia por ARN utilizando ácidos nucleicos de interferencia cortos (ANic) |
| WO2012078536A2 (en) | 2010-12-06 | 2012-06-14 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
| US9949923B2 (en) * | 2011-03-15 | 2018-04-24 | Optinose As | Nasal delivery |
| EP2766495B1 (en) * | 2011-10-14 | 2018-04-04 | Centre National De La Recherche Scientifique | Method of diagnosis, prognostic or treatment of neurodegenerative diseases |
| WO2013134777A1 (en) * | 2012-03-09 | 2013-09-12 | Northeastern University | Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders |
| CA2866392C (en) | 2012-03-30 | 2023-10-24 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| US9611473B2 (en) | 2012-09-12 | 2017-04-04 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| CA2890112A1 (en) | 2012-10-26 | 2014-05-01 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| RU2745324C2 (ru) | 2013-03-14 | 2021-03-23 | Ионис Фармасьютикалз, Инк. | Композиции и способы модулирования экспрессии tau |
| JP2016523980A (ja) | 2013-07-11 | 2016-08-12 | ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク | タウ発現を抑制するマイクロｒｎａ |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| EP3027223A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| WO2015089375A1 (en) * | 2013-12-13 | 2015-06-18 | The General Hospital Corporation | Soluble high molecular weight (hmw) tau species and applications thereof |
| WO2016151523A1 (en) * | 2015-03-25 | 2016-09-29 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| JOP20200228A1 (ar) * | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| WO2019070894A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMIAL DISORDERS |
| EP3692151A4 (en) | 2017-10-03 | 2021-07-14 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
| MX2020009532A (es) * | 2018-03-13 | 2020-10-05 | Janssen Pharmaceutica Nv | Oligonucleotidos modificados para uso en el tratamiento de tauopatias. |
| CN115552006A (zh) * | 2020-03-18 | 2022-12-30 | 马萨诸塞大学 | 用于mapt调节的寡核苷酸 |
| CN113817728A (zh) * | 2020-06-19 | 2021-12-21 | 四川大学华西医院 | 一种有效干扰Tau蛋白表达的重组慢病毒及其应用 |
| TW202400792A (zh) * | 2022-05-12 | 2024-01-01 | 美商戴瑟納製藥股份有限公司 | 用於抑制ｍａｐｔ表現之組合物及方法 |
| WO2024145474A2 (en) * | 2022-12-29 | 2024-07-04 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2116460A1 (en) | 1993-03-02 | 1994-09-03 | Mitsubishi Chemical Corporation | Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being |
| US6410046B1 (en) * | 1996-11-19 | 2002-06-25 | Intrabrain International Nv | Administering pharmaceuticals to the mammalian central nervous system |
| WO2005003350A2 (en) | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| WO2002086105A1 (en) * | 2001-04-20 | 2002-10-31 | Chiron Corporation | Delivery of polynucleotide agents to the central nervous sysstem |
| US20050159376A1 (en) | 2002-02-20 | 2005-07-21 | Slrna Therapeutics, Inc. | RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA) |
| IL143379A (en) | 2001-05-24 | 2013-11-28 | Yissum Res Dev Co | Oligonucleotide against human ache isoform r and its uses |
| EP1432724A4 (en) | 2002-02-20 | 2006-02-01 | Sirna Therapeutics Inc | RNA inhibition mediated inhibition of MAP KINASE GENES |
| GB2406568B (en) | 2002-02-20 | 2005-09-28 | Sirna Therapeutics Inc | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| AU2003224087B2 (en) | 2002-04-18 | 2009-03-05 | Opko Pharmaceuticals, Llc. | Means and methods for the specific inhibition of genes in cells and tissue of the CNS and/or eye |
| US20040254146A1 (en) | 2002-05-21 | 2004-12-16 | Nastech Pharmaceutical Company Inc. | Carboxylate salts of galantamine and their pharmaceutical use |
| US20030225031A1 (en) | 2002-05-21 | 2003-12-04 | Quay Steven C. | Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid |
| US20050106731A1 (en) | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
| AU2003282877B9 (en) | 2002-09-25 | 2011-05-12 | University Of Massachusetts | In Vivo gene silencing by chemically modified and stable siRNA |
| WO2004028471A2 (en) | 2002-09-28 | 2004-04-08 | Massachusetts Institute Of Technology | Influenza therapeutic |
| ES2334125T3 (es) * | 2002-11-04 | 2010-03-05 | University Of Massachusetts | Interferencia de arn especifico de alelos. |
| US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2005004794A2 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| WO2005044976A2 (en) | 2003-06-20 | 2005-05-19 | Isis Pharmaceuticals, Inc. | Oligomeric compounds for use in gene modulation |
| WO2005014815A1 (en) * | 2003-08-08 | 2005-02-17 | President And Fellows Of Harvard College | siRNA BASED METHODS FOR TREATING ALZHEIMER’S DISEASE |
| WO2005045037A2 (en) | 2003-10-23 | 2005-05-19 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| WO2005105995A2 (en) | 2004-04-14 | 2005-11-10 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US20050265927A1 (en) | 2004-05-17 | 2005-12-01 | Yale University | Intranasal delivery of nucleic acid molecules |
| WO2006041922A2 (en) | 2004-10-08 | 2006-04-20 | Dara Biosciences, Inc. | Agents and methods for administration to the central nervous system |
-
2006
- 2006-03-17 GB GBGB0605337.5A patent/GB0605337D0/en not_active Ceased
-
2007
- 2007-03-16 EP EP07733553.7A patent/EP2004823B1/en active Active
- 2007-03-16 AU AU2007228570A patent/AU2007228570B2/en active Active
- 2007-03-16 CA CA2645120A patent/CA2645120C/en active Active
- 2007-03-16 US US12/293,230 patent/US8871729B2/en active Active
- 2007-03-16 WO PCT/GB2007/050128 patent/WO2007107789A2/en active Application Filing
- 2007-03-16 CN CN200780017978.7A patent/CN101448944B/zh active Active
- 2007-03-16 RU RU2008141162/15A patent/RU2426544C2/ru active
- 2007-03-16 KR KR1020147013792A patent/KR101605144B1/ko active IP Right Grant
- 2007-03-16 JP JP2008558913A patent/JP5781722B2/ja active Active
- 2007-03-16 KR KR1020087025174A patent/KR101669944B1/ko active IP Right Grant
- 2007-03-16 ES ES07733553.7T patent/ES2582649T3/es active Active
- 2007-03-16 MX MX2008011731A patent/MX2008011731A/es active IP Right Grant
-
2008
- 2008-09-15 IL IL194114A patent/IL194114A/en active IP Right Grant
-
2009
- 2009-06-23 HK HK09105618.2A patent/HK1126812A1/zh unknown
-
2013
- 2013-08-02 JP JP2013161765A patent/JP5923466B2/ja active Active
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