JP6386461B2 - 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法 - Google Patents
特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法 Download PDFInfo
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- JP6386461B2 JP6386461B2 JP2015538467A JP2015538467A JP6386461B2 JP 6386461 B2 JP6386461 B2 JP 6386461B2 JP 2015538467 A JP2015538467 A JP 2015538467A JP 2015538467 A JP2015538467 A JP 2015538467A JP 6386461 B2 JP6386461 B2 JP 6386461B2
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- 0 C*C*C(N*C)=O Chemical compound C*C*C(N*C)=O 0.000 description 4
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- 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
-
- 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
- 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
- A61K47/54—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 compound
- A61K47/55—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 compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- 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]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261719284P | 2012-10-26 | 2012-10-26 | |
| US61/719,284 | 2012-10-26 | ||
| EP12382414 | 2012-10-26 | ||
| EP12382414.6 | 2012-10-26 | ||
| PCT/EP2013/072410 WO2014064257A1 (en) | 2012-10-26 | 2013-10-25 | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015536927A JP2015536927A (ja) | 2015-12-24 |
| JP2015536927A5 JP2015536927A5 (enExample) | 2016-12-15 |
| JP6386461B2 true JP6386461B2 (ja) | 2018-09-05 |
Family
ID=47148690
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015538467A Active JP6386461B2 (ja) | 2012-10-26 | 2013-10-25 | 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US9084825B2 (enExample) |
| EP (1) | EP2911695B1 (enExample) |
| JP (1) | JP6386461B2 (enExample) |
| CN (1) | CN104884094A (enExample) |
| AU (1) | AU2013336581A1 (enExample) |
| CA (1) | CA2890112A1 (enExample) |
| ES (1) | ES2967530T3 (enExample) |
| HK (1) | HK1214179A1 (enExample) |
| MX (1) | MX2015005352A (enExample) |
| WO (1) | WO2014064257A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12398176B2 (en) | 2018-08-27 | 2025-08-26 | Regeneron Pharmaceuticals, Inc. | Use of Raman spectroscopy in downstream purification |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2796722C (en) | 2010-04-19 | 2021-06-01 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| EP3521451A1 (en) | 2010-11-17 | 2019-08-07 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| EP3208338B1 (en) | 2011-08-04 | 2019-04-10 | Yeda Research and Development Co., Ltd. | Micro-rna mir-15 and compositions comprising same for the treatment of corticotropin-releasing hormone- associated medical conditions |
| ES2967530T3 (es) | 2012-10-26 | 2024-04-30 | Palomo Ltd | Composiciones y métodos para el tratamiento de la enfermedad de Parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas |
| CN106715695B (zh) | 2014-02-05 | 2020-07-31 | 耶达研究及发展有限公司 | 用于治疗和诊断的微rna和包含所述微rna的组合物 |
| WO2016186968A1 (en) | 2015-05-15 | 2016-11-24 | The Arizona Board Of Regents On Behalf Of The University Of Arizona | Compositions and methods for treating motor disorders |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US10733023B1 (en) * | 2015-08-06 | 2020-08-04 | D2Iq, Inc. | Oversubscription scheduling |
| EP3415524A4 (en) * | 2016-01-07 | 2019-09-04 | Osaka University | INHIBITOR OF α-SYNNUCLEINE EXPRESSION |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| WO2019009298A1 (ja) * | 2017-07-05 | 2019-01-10 | 国立大学法人大阪大学 | α-シヌクレイン発現抑制剤 |
| US20200172903A1 (en) * | 2017-07-05 | 2020-06-04 | Osaka University | ENA ANTISENSE OLIGONUCLEOTIDE FOR INHIBITION OF alpha-SYNUCLEIN EXPRESSION |
| KR101890925B1 (ko) | 2017-08-08 | 2018-08-24 | 주식회사 이레산업 | 캠핑용 그릴 세트 |
| AU2018346507B2 (en) * | 2017-10-04 | 2025-03-13 | Avidity Biosciences, Inc. | Nucleic acid-polypeptide compositions and uses thereof |
| WO2019079887A1 (en) * | 2017-10-23 | 2019-05-02 | Carleton University | APTAMERS AS A THERAPEUTIC TO PREVENT AGGREGATION OF PROTEINS IN NEURODEGENERATIVE DISEASE |
| TWI809004B (zh) | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| KR20200109338A (ko) | 2018-01-12 | 2020-09-22 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | 알파-시누클레인 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2020106859A1 (en) * | 2018-11-21 | 2020-05-28 | University Georgia Research Foundation, Inc., | Bimodal nanoparticle conjugates for non-invasive central nervous system tissue imaging |
| EP3888663A1 (en) * | 2018-11-30 | 2021-10-06 | Kyowa Kirin Co., Ltd. | Nucleic acid complex |
| CN114364404A (zh) | 2019-04-11 | 2022-04-15 | 东北大学 | 寡核苷酸-聚合物多手臂偶联物以及使用方法 |
| CA3142337A1 (en) | 2019-06-06 | 2020-12-10 | Avidity Biosciences, Inc. | Una amidites and uses thereof |
| WO2021094750A1 (en) * | 2019-11-11 | 2021-05-20 | Oxford University Innovation Limited | Method of isolating a selected population of exosomes |
| CN115168823B (zh) * | 2022-08-05 | 2025-07-18 | 聊城大学 | 基于组平均的多任务功能性近红外光谱信号的身份识别 |
| WO2024166019A1 (en) * | 2023-02-08 | 2024-08-15 | Biorchestra Co., Ltd. | Sirna targeting alpha-synuclein and uses thereof |
Family Cites Families (22)
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| IL66831A0 (en) | 1981-10-05 | 1982-12-31 | Kefalas As | Indane derivatives |
| US4902505A (en) | 1986-07-30 | 1990-02-20 | Alkermes | Chimeric peptides for neuropeptide delivery through the blood-brain barrier |
| US4904582A (en) | 1987-06-11 | 1990-02-27 | Synthetic Genetics | Novel amphiphilic nucleic acid conjugates |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
| US5264564A (en) | 1989-10-24 | 1993-11-23 | Gilead Sciences | Oligonucleotide analogs with novel linkages |
| US6455736B1 (en) | 1994-12-16 | 2002-09-24 | Uop Llc | Process for preparation of pharmaceutically desired sertraline and sertraline analogs |
| EP1108724B1 (en) | 1996-01-16 | 2007-09-19 | Sirna Therpeutics, Inc. | Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules |
| WO2006006948A2 (en) * | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| CA2542232A1 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals, Inc. | Method for treating neurodegenerative disease by inhibiting alpha-synuclein |
| WO2005067391A2 (en) * | 2004-01-19 | 2005-07-28 | Technion Research & Development Foundation Ltd. | Diagnostic test for parkinson's disease |
| WO2007050789A2 (en) * | 2005-10-25 | 2007-05-03 | Nëdken Corporation | Glycosyl-oligonucleotide conjugates and methods |
| GB0605337D0 (en) | 2006-03-17 | 2006-04-26 | Genomica Sau | Treatment of CNS conditions |
| US20090176729A1 (en) * | 2007-12-14 | 2009-07-09 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| CN101946001A (zh) | 2007-12-20 | 2011-01-12 | 安吉奥开米公司 | 多肽-核酸结合物及其应用 |
| US9255266B2 (en) * | 2009-05-06 | 2016-02-09 | Rutgers, The State University Of New Jersey | RNA targeting in alpha-synucleinopathies |
| WO2011087804A2 (en) | 2009-12-21 | 2011-07-21 | Medtronic, Inc. | Peptide-polynucleotide compositions, and methods for transfecting a cell with dna and treatment of neurodegenerative disease |
| CA2796722C (en) | 2010-04-19 | 2021-06-01 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| WO2012027713A2 (en) | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
| EP3521451A1 (en) * | 2010-11-17 | 2019-08-07 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| ES2967530T3 (es) | 2012-10-26 | 2024-04-30 | Palomo Ltd | Composiciones y métodos para el tratamiento de la enfermedad de Parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas |
| AU2013336582A1 (en) | 2012-10-26 | 2015-06-11 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to cell types |
-
2013
- 2013-10-25 ES ES13783061T patent/ES2967530T3/es active Active
- 2013-10-25 WO PCT/EP2013/072410 patent/WO2014064257A1/en not_active Ceased
- 2013-10-25 MX MX2015005352A patent/MX2015005352A/es unknown
- 2013-10-25 HK HK16102420.8A patent/HK1214179A1/zh unknown
- 2013-10-25 JP JP2015538467A patent/JP6386461B2/ja active Active
- 2013-10-25 CN CN201380068146.3A patent/CN104884094A/zh active Pending
- 2013-10-25 CA CA2890112A patent/CA2890112A1/en not_active Abandoned
- 2013-10-25 EP EP13783061.8A patent/EP2911695B1/en active Active
- 2013-10-25 AU AU2013336581A patent/AU2013336581A1/en not_active Abandoned
- 2013-10-28 US US14/065,024 patent/US9084825B2/en active Active - Reinstated
-
2015
- 2015-07-20 US US14/803,811 patent/US10125363B2/en active Active
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12398176B2 (en) | 2018-08-27 | 2025-08-26 | Regeneron Pharmaceuticals, Inc. | Use of Raman spectroscopy in downstream purification |
Also Published As
| Publication number | Publication date |
|---|---|
| CN104884094A (zh) | 2015-09-02 |
| WO2014064257A1 (en) | 2014-05-01 |
| HK1214179A1 (zh) | 2016-07-22 |
| EP2911695B1 (en) | 2023-11-29 |
| MX2015005352A (es) | 2015-10-29 |
| AU2013336581A1 (en) | 2015-06-11 |
| EP2911695A1 (en) | 2015-09-02 |
| ES2967530T3 (es) | 2024-04-30 |
| CA2890112A1 (en) | 2014-05-01 |
| US9084825B2 (en) | 2015-07-21 |
| US10125363B2 (en) | 2018-11-13 |
| US20140120158A1 (en) | 2014-05-01 |
| JP2015536927A (ja) | 2015-12-24 |
| US20160083726A1 (en) | 2016-03-24 |
| EP2911695C0 (en) | 2023-11-29 |
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