HK1214179A1 - 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 - Google Patents
通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 Download PDFInfo
- Publication number
- HK1214179A1 HK1214179A1 HK16102420.8A HK16102420A HK1214179A1 HK 1214179 A1 HK1214179 A1 HK 1214179A1 HK 16102420 A HK16102420 A HK 16102420A HK 1214179 A1 HK1214179 A1 HK 1214179A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- conjugate
- group
- synuclein
- nucleic acid
- seq
- Prior art date
Links
Classifications
-
- 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
-
- 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)
- Neurology (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (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 | ||
| EP12382414.6 | 2012-10-26 | ||
| EP12382414 | 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 (1)
| Publication Number | Publication Date |
|---|---|
| HK1214179A1 true HK1214179A1 (zh) | 2016-07-22 |
Family
ID=47148690
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HK16102420.8A HK1214179A1 (zh) | 2012-10-26 | 2013-10-25 | 通过选择性递送寡核苷酸分子至特定神经元类型治疗帕金森病的组合物和方法 |
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) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2560687T3 (en) | 2010-04-19 | 2017-09-18 | Nlife Therapeutics S L | Preparations 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 |
| WO2013018060A2 (en) | 2011-08-04 | 2013-02-07 | Yeda Research And Development Co. Ltd. | Micro-rnas and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions |
| MX2015005352A (es) | 2012-10-26 | 2015-10-29 | Nlife Therapeutics S L | 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 |
| JP6569920B2 (ja) * | 2016-01-07 | 2019-09-04 | 国立大学法人大阪大学 | α−シヌクレイン発現抑制剤 |
| 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 |
| EP3628737A4 (en) * | 2017-07-05 | 2021-01-20 | Osaka University | ENA-ANTISENSE-OLIGONUCLEOTIDE TO INHIBIT ALPHA-SYNUCLEIN EXPRESSION |
| KR101890925B1 (ko) | 2017-08-08 | 2018-08-24 | 주식회사 이레산업 | 캠핑용 그릴 세트 |
| MX2020003596A (es) * | 2017-10-04 | 2020-07-22 | Avidity Biosciences Inc | Composiciones de acido nucleico-polipeptido y usos de las mismos. |
| US11814625B2 (en) | 2017-10-23 | 2023-11-14 | Carleton University | Aptamers as a therapeutic tool to prevent protein aggregation 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 |
| AU2019208011B2 (en) | 2018-01-12 | 2025-06-05 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| AU2019207859A1 (en) | 2018-01-12 | 2020-07-02 | Roche Innovation Center Copenhagen A/S | Alpha-synuclein antisense oligonucleotides and uses thereof |
| CA3100035A1 (en) | 2018-08-27 | 2020-03-05 | Regeneron Pharmaceuticals, Inc. | Use of raman spectroscopy in downstream purification |
| US20220096665A1 (en) * | 2018-11-21 | 2022-03-31 | University Of Georgia Research Foundation, Inc. | Bimodal nanoparticle conjugates for non-invasive central nervous system tissue imaging |
| AU2019390097A1 (en) * | 2018-11-30 | 2021-07-15 | Kyowa Kirin Co., Ltd. | Nucleic acid conjugate |
| US12478684B2 (en) | 2019-04-11 | 2025-11-25 | Northeastern University | Oligonucleotide-polymer miktoarm conjugates and methods of use |
| WO2020247818A1 (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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| AU1430097A (en) | 1996-01-16 | 1997-08-11 | Ribozyme Pharmaceuticals, 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 |
| WO2005004794A2 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| 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 |
| WO2009079399A2 (en) * | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| EP2235175A4 (en) | 2007-12-20 | 2012-04-25 | Angiochem Inc | POLYPEPTIDE-NUCLEIC ACID CONJUGATES AND USES THEREOF |
| WO2010129791A1 (en) * | 2009-05-06 | 2010-11-11 | University Of Medicine And Dentistry 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 |
| DK2560687T3 (en) | 2010-04-19 | 2017-09-18 | Nlife Therapeutics S L | Preparations 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 |
| JP2016503394A (ja) | 2012-10-26 | 2016-02-04 | エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． | 細胞型へのオリゴヌクレオチド分子の選択的送達のための組成物および方法 |
| MX2015005352A (es) | 2012-10-26 | 2015-10-29 | Nlife Therapeutics S L | Composiciones y métodos para el tratamiento de la enfermedad de parkinson mediante la administración selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas. |
-
2013
- 2013-10-25 MX MX2015005352A patent/MX2015005352A/es unknown
- 2013-10-25 ES ES13783061T patent/ES2967530T3/es active Active
- 2013-10-25 EP EP13783061.8A patent/EP2911695B1/en active Active
- 2013-10-25 JP JP2015538467A patent/JP6386461B2/ja active Active
- 2013-10-25 CN CN201380068146.3A patent/CN104884094A/zh active Pending
- 2013-10-25 WO PCT/EP2013/072410 patent/WO2014064257A1/en not_active Ceased
- 2013-10-25 AU AU2013336581A patent/AU2013336581A1/en not_active Abandoned
- 2013-10-25 CA CA2890112A patent/CA2890112A1/en not_active Abandoned
- 2013-10-25 HK HK16102420.8A patent/HK1214179A1/zh unknown
- 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
Also Published As
| Publication number | Publication date |
|---|---|
| CA2890112A1 (en) | 2014-05-01 |
| CN104884094A (zh) | 2015-09-02 |
| US9084825B2 (en) | 2015-07-21 |
| WO2014064257A1 (en) | 2014-05-01 |
| ES2967530T3 (es) | 2024-04-30 |
| EP2911695C0 (en) | 2023-11-29 |
| MX2015005352A (es) | 2015-10-29 |
| US20140120158A1 (en) | 2014-05-01 |
| AU2013336581A1 (en) | 2015-06-11 |
| JP2015536927A (ja) | 2015-12-24 |
| US20160083726A1 (en) | 2016-03-24 |
| EP2911695B1 (en) | 2023-11-29 |
| US10125363B2 (en) | 2018-11-13 |
| EP2911695A1 (en) | 2015-09-02 |
| JP6386461B2 (ja) | 2018-09-05 |
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