EA201791417A1 - Супрессия гена гентингтина, индуцированная рнк-интерференцией - Google Patents
Супрессия гена гентингтина, индуцированная рнк-интерференциейInfo
- Publication number
- EA201791417A1 EA201791417A1 EA201791417A EA201791417A EA201791417A1 EA 201791417 A1 EA201791417 A1 EA 201791417A1 EA 201791417 A EA201791417 A EA 201791417A EA 201791417 A EA201791417 A EA 201791417A EA 201791417 A1 EA201791417 A1 EA 201791417A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- rna
- induced
- sequence
- gentingin
- rnc
- Prior art date
Links
Classifications
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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
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- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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
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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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Настоящее изобретение обеспечивает двухцепочечную РНК, содержащую первую последовательность РНК и вторую последовательность РНК, в которой первая и вторая последовательности РНК являются, по существу, комплементарными, причем первая последовательность РНК имеет длину последовательности, равную по меньшей мере 19 нуклеотидам, и является, по существу, комплементарной последовательности SEQ ID NO: 1. Двухцепочечная РНК предназначена для применения в индукции РНК-интерференции против последовательностей экзона 1 гена белка гентингтина. Двухцепочечная РНК по настоящему изобретению была способна снижать гибель нейрональных клеток и образование агрегатов белка гентингтина в экспериментальной модели на животных.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP14200308 | 2014-12-24 | ||
PCT/EP2015/081157 WO2016102664A1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (2)
Publication Number | Publication Date |
---|---|
EA201791417A1 true EA201791417A1 (ru) | 2017-12-29 |
EA037696B1 EA037696B1 (ru) | 2021-05-12 |
Family
ID=52282540
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201791417A EA037696B1 (ru) | 2014-12-24 | 2015-12-23 | Супрессия гена гентингтина, индуцированная рнк-интерференцией |
Country Status (24)
Country | Link |
---|---|
US (4) | US10174321B2 (ru) |
EP (2) | EP3540063A1 (ru) |
JP (2) | JP6839094B2 (ru) |
KR (1) | KR102636633B1 (ru) |
CN (1) | CN108064292B (ru) |
AU (1) | AU2015370903B2 (ru) |
CA (1) | CA2971920C (ru) |
CO (1) | CO2017007335A2 (ru) |
CY (1) | CY1121733T1 (ru) |
DK (1) | DK3237618T3 (ru) |
EA (1) | EA037696B1 (ru) |
ES (1) | ES2732023T3 (ru) |
HK (1) | HK1246344B (ru) |
HR (1) | HRP20190992T1 (ru) |
HU (1) | HUE043842T2 (ru) |
IL (1) | IL252990B (ru) |
LT (1) | LT3237618T (ru) |
MX (1) | MX2017008500A (ru) |
PL (1) | PL3237618T3 (ru) |
PT (1) | PT3237618T (ru) |
RS (1) | RS58862B1 (ru) |
SI (1) | SI3237618T1 (ru) |
TR (1) | TR201908969T4 (ru) |
WO (1) | WO2016102664A1 (ru) |
Families Citing this family (58)
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JP5645840B2 (ja) | 2008-12-02 | 2014-12-24 | 株式会社Wave Life Sciences Japan | リン原子修飾核酸の合成方法 |
WO2011005761A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Novel nucleic acid prodrugs and methods use thereof |
JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
WO2014012081A2 (en) | 2012-07-13 | 2014-01-16 | Ontorii, Inc. | Chiral control |
RU2677639C2 (ru) | 2012-07-13 | 2019-01-18 | Шин Ниппон Биомедикал Лэбораториз, Лтд. | Хиральный адъювант нуклеиновой кислоты |
EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
CA2975583A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
HUE043842T2 (hu) * | 2014-12-24 | 2019-09-30 | Uniqure Ip Bv | RNAi-indukált huntingtin gén szuppresszió |
EA201791805A1 (ru) | 2015-02-10 | 2018-05-31 | Джензим Корпорейшн | ВАРИАНТ СРЕДСТВА ДЛЯ RNAi |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
CA2995110A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
WO2017100726A1 (en) | 2015-12-10 | 2017-06-15 | Ptc Therapeutics, Inc. | Methods for treatng huntington's disease |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Preparations and methods for treating Huntington's disease |
EP3458588A4 (en) | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
US11407753B2 (en) | 2017-06-05 | 2022-08-09 | Ptc Therapeutics, Inc. | Compounds for treating Huntington's disease |
JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | 2テイル自己デリバリー型siRNAおよび関連方法 |
US11395822B2 (en) | 2017-06-28 | 2022-07-26 | Ptc Therapeutics, Inc. | Methods for treating Huntington's disease |
EP3644996B1 (en) | 2017-06-28 | 2023-07-26 | PTC Therapeutics, Inc. | Methods for treating huntington's disease |
KR102686857B1 (ko) | 2017-09-22 | 2024-07-18 | 젠자임 코포레이션 | 변이체 RNAi |
TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
CN108384803A (zh) * | 2017-12-19 | 2018-08-10 | 上海百力格生物技术有限公司 | 一种用于miRNA干扰载体构建筛选特异对照的质粒及其构建方法 |
WO2019191092A1 (en) | 2018-03-27 | 2019-10-03 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
WO2019222413A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Modified aav constructs and uses thereof |
KR20210010549A (ko) * | 2018-05-15 | 2021-01-27 | 유니버시티 오브 워싱톤 | 스플라이싱이상을 감소시키고 rna 우성 질환을 치료하기 위한 조성물 및 방법 |
LT3814357T (lt) | 2018-06-27 | 2024-06-25 | Ptc Therapeutics, Inc. | Heterocikliniai ir heteroarilų junginiai, skirti hantingtono ligos gydymui |
CN112567035A (zh) | 2018-07-02 | 2021-03-26 | 沃雅戈治疗公司 | 肌萎缩侧索硬化症及脊髓相关病症的治疗 |
EP3850098A1 (en) | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
WO2020104295A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3 |
CA3120177A1 (en) * | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Method and means to deliver mirna to target cells |
CA3119721A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | A companion diagnostic to monitor the effects of gene therapy |
CA3125310A1 (en) * | 2019-01-09 | 2020-07-16 | Universidade De Coimbra | Double stranded rna and uses thereof |
EP3997225A1 (en) | 2019-07-10 | 2022-05-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the treatment of epilepsy |
CA3149835A1 (en) | 2019-08-09 | 2021-02-18 | University Of Massachusetts | Chemically modified oligonucleotides targeting snps |
WO2021053018A1 (en) | 2019-09-16 | 2021-03-25 | Uniqure Ip B.V. | Targeting misspliced transcripts in genetic disorders |
TW202131920A (zh) | 2019-11-01 | 2021-09-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
CN114728018B (zh) * | 2019-11-01 | 2024-07-19 | 阿尔尼拉姆医药品有限公司 | 亨廷顿(HTT)iRNA药剂组合物及其使用方法 |
JP2023521347A (ja) * | 2020-04-07 | 2023-05-24 | ユニキュアー アイピー ビー.ブイ. | アンジオポエチン様3(angptl3)をサイレンシングするための遺伝子構築物及びその使用 |
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WO2022031591A2 (en) * | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
BR112023019981A2 (pt) | 2021-03-29 | 2023-12-12 | Alnylam Pharmaceuticals Inc | Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas |
WO2022206819A1 (zh) * | 2021-03-30 | 2022-10-06 | 南京大学 | 一种用于治疗亨廷顿病的rna递送系统 |
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WO2023198662A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Novel systems for nucleic acid regulation |
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HUE043842T2 (hu) * | 2014-12-24 | 2019-09-30 | Uniqure Ip Bv | RNAi-indukált huntingtin gén szuppresszió |
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2015
- 2015-12-23 HU HUE15817385A patent/HUE043842T2/hu unknown
- 2015-12-23 EP EP19167349.0A patent/EP3540063A1/en active Pending
- 2015-12-23 MX MX2017008500A patent/MX2017008500A/es unknown
- 2015-12-23 CN CN201580071228.2A patent/CN108064292B/zh active Active
- 2015-12-23 US US15/538,964 patent/US10174321B2/en active Active
- 2015-12-23 KR KR1020177020454A patent/KR102636633B1/ko active IP Right Grant
- 2015-12-23 CA CA2971920A patent/CA2971920C/en active Active
- 2015-12-23 PT PT15817385T patent/PT3237618T/pt unknown
- 2015-12-23 DK DK15817385.6T patent/DK3237618T3/da active
- 2015-12-23 EP EP15817385.6A patent/EP3237618B1/en active Active
- 2015-12-23 JP JP2017552234A patent/JP6839094B2/ja active Active
- 2015-12-23 PL PL15817385T patent/PL3237618T3/pl unknown
- 2015-12-23 LT LTEP15817385.6T patent/LT3237618T/lt unknown
- 2015-12-23 ES ES15817385T patent/ES2732023T3/es active Active
- 2015-12-23 RS RS20190744A patent/RS58862B1/sr unknown
- 2015-12-23 EA EA201791417A patent/EA037696B1/ru unknown
- 2015-12-23 WO PCT/EP2015/081157 patent/WO2016102664A1/en active Application Filing
- 2015-12-23 TR TR2019/08969T patent/TR201908969T4/tr unknown
- 2015-12-23 SI SI201530791T patent/SI3237618T1/sl unknown
- 2015-12-23 AU AU2015370903A patent/AU2015370903B2/en active Active
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2017
- 2017-06-18 IL IL252990A patent/IL252990B/en unknown
- 2017-07-24 CO CONC2017/0007335A patent/CO2017007335A2/es unknown
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2018
- 2018-04-27 HK HK18105533.3A patent/HK1246344B/zh unknown
- 2018-11-16 US US16/193,908 patent/US10767180B2/en active Active
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2019
- 2019-05-31 HR HRP20190992TT patent/HRP20190992T1/hr unknown
- 2019-06-13 CY CY20191100616T patent/CY1121733T1/el unknown
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2020
- 2020-07-15 US US16/930,035 patent/US11371044B2/en active Active
- 2020-10-01 JP JP2020167128A patent/JP2021000136A/ja active Pending
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2022
- 2022-05-24 US US17/751,934 patent/US20230119344A1/en active Pending
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