EA202091693A1 - Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения - Google Patents
Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их примененияInfo
- Publication number
- EA202091693A1 EA202091693A1 EA202091693A EA202091693A EA202091693A1 EA 202091693 A1 EA202091693 A1 EA 202091693A1 EA 202091693 A EA202091693 A EA 202091693A EA 202091693 A EA202091693 A EA 202091693A EA 202091693 A1 EA202091693 A1 EA 202091693A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- targetingly
- synucleine
- applications
- sense oligonucleotides
- affecting
- Prior art date
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- 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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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
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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
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
- A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
- A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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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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- 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
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- C—CHEMISTRY; METALLURGY
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- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Abstract
Настоящее изобретение относится к антисмысловым олигонуклеотидам, которые оказывают целенаправленное воздействие на мРНК SNCA (например, в месте соединения интрона и экзона) в клетке, что приводит к пониженной экспрессии белка SNCA. Снижение экспрессии белка SNCA является благоприятным для лечения определенных медицинских нарушений, например неврологического нарушения.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862616994P | 2018-01-12 | 2018-01-12 | |
PCT/US2019/013255 WO2019140236A1 (en) | 2018-01-12 | 2019-01-11 | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
EA202091693A1 true EA202091693A1 (ru) | 2021-04-14 |
Family
ID=65269098
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA202091693A EA202091693A1 (ru) | 2018-01-12 | 2019-01-11 | Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения |
Country Status (15)
Country | Link |
---|---|
US (1) | US20200362347A1 (ru) |
EP (1) | EP3737761A1 (ru) |
JP (1) | JP2021510511A (ru) |
KR (1) | KR20200140240A (ru) |
CN (1) | CN111819283A (ru) |
AU (1) | AU2019208011A1 (ru) |
BR (1) | BR112020014011A2 (ru) |
CA (1) | CA3087966A1 (ru) |
CO (1) | CO2020009715A2 (ru) |
EA (1) | EA202091693A1 (ru) |
IL (1) | IL275903A (ru) |
MX (1) | MX2020007439A (ru) |
PE (1) | PE20211749A1 (ru) |
SG (1) | SG11202006528XA (ru) |
WO (1) | WO2019140236A1 (ru) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN110506057B (zh) | 2017-02-17 | 2023-09-29 | 百时美施贵宝公司 | Alpha突触核蛋白抗体及其应用 |
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 |
EP3737387A1 (en) | 2018-01-12 | 2020-11-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
CA3090901A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
AU2021206182A1 (en) * | 2020-01-06 | 2022-07-28 | AUM LifeTech, Inc. | Antisense oligonucleotides for treatment of neurological disorders |
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EP0724447B1 (en) | 1991-10-24 | 2003-05-07 | Isis Pharmaceuticals, Inc. | Derivatized oligonucleotides having improved uptake |
JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
AU9063398A (en) | 1997-09-12 | 1999-04-05 | Exiqon A/S | Oligonucleotide analogues |
NZ513402A (en) | 1999-02-12 | 2003-06-30 | Sankyo Co | Novel nucleosides and oligonucleotide analogues |
CN102180924A (zh) | 1999-05-04 | 2011-09-14 | 桑塔里斯制药公司 | L-核糖-lna类似物 |
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ES2607471T3 (es) | 2002-11-18 | 2017-03-31 | Roche Innovation Center Copenhagen A/S | Diseño antisentido |
EP1635763B1 (en) * | 2003-06-09 | 2012-08-08 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
EP1931778A2 (en) * | 2005-09-15 | 2008-06-18 | Santaris Pharma A/S | RNA ANTAGONIST COMPOUNDS FOR THE INHIBITION OF APO-Bl00 EXPRESSION |
WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
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WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
EP2640853B1 (en) * | 2010-11-17 | 2018-12-26 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
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EP3737387A1 (en) * | 2018-01-12 | 2020-11-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
-
2019
- 2019-01-11 PE PE2020000954A patent/PE20211749A1/es unknown
- 2019-01-11 US US16/961,578 patent/US20200362347A1/en active Pending
- 2019-01-11 BR BR112020014011-2A patent/BR112020014011A2/pt unknown
- 2019-01-11 KR KR1020207023162A patent/KR20200140240A/ko unknown
- 2019-01-11 MX MX2020007439A patent/MX2020007439A/es unknown
- 2019-01-11 CN CN201980018033.XA patent/CN111819283A/zh active Pending
- 2019-01-11 AU AU2019208011A patent/AU2019208011A1/en active Pending
- 2019-01-11 JP JP2020538629A patent/JP2021510511A/ja active Pending
- 2019-01-11 WO PCT/US2019/013255 patent/WO2019140236A1/en unknown
- 2019-01-11 SG SG11202006528XA patent/SG11202006528XA/en unknown
- 2019-01-11 EP EP19702766.7A patent/EP3737761A1/en active Pending
- 2019-01-11 EA EA202091693A patent/EA202091693A1/ru unknown
- 2019-01-11 CA CA3087966A patent/CA3087966A1/en active Pending
-
2020
- 2020-07-07 IL IL275903A patent/IL275903A/en unknown
- 2020-08-05 CO CONC2020/0009715A patent/CO2020009715A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
BR112020014011A2 (pt) | 2020-12-08 |
PE20211749A1 (es) | 2021-09-06 |
CO2020009715A2 (es) | 2020-08-21 |
SG11202006528XA (en) | 2020-08-28 |
CN111819283A (zh) | 2020-10-23 |
US20200362347A1 (en) | 2020-11-19 |
KR20200140240A (ko) | 2020-12-15 |
WO2019140236A1 (en) | 2019-07-18 |
EP3737761A1 (en) | 2020-11-18 |
AU2019208011A1 (en) | 2020-07-23 |
IL275903A (en) | 2020-08-31 |
MX2020007439A (es) | 2020-09-14 |
CA3087966A1 (en) | 2019-07-18 |
JP2021510511A (ja) | 2021-04-30 |
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