UY38562A - Compuestos y métodos para reducir la expresión de app - Google Patents
Compuestos y métodos para reducir la expresión de appInfo
- Publication number
- UY38562A UY38562A UY0001038562A UY38562A UY38562A UY 38562 A UY38562 A UY 38562A UY 0001038562 A UY0001038562 A UY 0001038562A UY 38562 A UY38562 A UY 38562A UY 38562 A UY38562 A UY 38562A
- Authority
- UY
- Uruguay
- Prior art keywords
- methods
- compounds
- reduce
- animal
- cell
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 3
- 208000024891 symptom Diseases 0.000 abstract 3
- 206010002942 Apathy Diseases 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 208000037259 Amyloid Plaque Diseases 0.000 abstract 1
- 208000028698 Cognitive impairment Diseases 0.000 abstract 1
- 206010010904 Convulsion Diseases 0.000 abstract 1
- 206010012289 Dementia Diseases 0.000 abstract 1
- 206010017577 Gait disturbance Diseases 0.000 abstract 1
- 230000002159 abnormal effect Effects 0.000 abstract 1
- 230000003542 behavioural effect Effects 0.000 abstract 1
- 208000010877 cognitive disease Diseases 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 230000001771 impaired effect Effects 0.000 abstract 1
- 230000004770 neurodegeneration Effects 0.000 abstract 1
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 1
- 230000000750 progressive effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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
- C12N15/1138—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 against receptors or cell surface proteins
-
- 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
- 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
- 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]
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/32—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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- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962798353P | 2019-01-29 | 2019-01-29 | |
| US201962841169P | 2019-04-30 | 2019-04-30 | |
| US201962915764P | 2019-10-16 | 2019-10-16 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| UY38562A true UY38562A (es) | 2020-08-31 |
Family
ID=71840111
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| UY0001038562A UY38562A (es) | 2019-01-29 | 2020-01-29 | Compuestos y métodos para reducir la expresión de app |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20220380773A1 (https=) |
| EP (1) | EP3918073A4 (https=) |
| JP (2) | JP7557469B2 (https=) |
| TW (1) | TW202043472A (https=) |
| UY (1) | UY38562A (https=) |
| WO (1) | WO2020160163A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| US11732260B2 (en) | 2018-03-02 | 2023-08-22 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of amyloid-β precursor protein |
| AU2019405783B2 (en) * | 2018-12-19 | 2026-03-26 | Alnylam Pharmaceuticals, Inc. | Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof |
| EP3947680A2 (en) * | 2019-04-03 | 2022-02-09 | Bristol-Myers Squibb Company | Angptl2 antisense oligonucleotides and uses thereof |
| KR20220062517A (ko) | 2019-08-15 | 2022-05-17 | 아이오니스 파마수티컬즈, 인코포레이티드 | 결합 변형된 올리고머 화합물 및 이의 용도 |
| CA3189922A1 (en) | 2020-07-28 | 2022-02-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing app expression |
| US20240117349A1 (en) * | 2021-01-29 | 2024-04-11 | Alnylam Pharmaceuticals, Inc. | iRNA COMPOSITIONS AND METHODS FOR SILENCING AMYLOID PRECURSOR PROTEIN (APP) |
| CN118369427A (zh) * | 2021-10-15 | 2024-07-19 | 阿尔尼拉姆医药品有限公司 | 肝外递送irna组合物及其使用方法 |
| WO2025045195A1 (zh) * | 2023-08-31 | 2025-03-06 | 上海拓界生物医药科技有限公司 | 靶向APP的RNAi剂及其医药用途 |
| WO2025228429A1 (zh) * | 2024-04-30 | 2025-11-06 | 北京安龙生物医药有限公司 | 靶向淀粉样前体蛋白基因的寡核苷酸及其用途 |
| WO2025261396A1 (zh) * | 2024-06-18 | 2025-12-26 | 艾码生物科技(南京)有限公司 | 靶向app的rna干扰方法、核酸及其应用 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1993013114A1 (en) * | 1991-12-24 | 1993-07-08 | Isis Pharmaceuticals, Inc. | COMPOSITIONS AND METHODS FOR MODULATING β-AMYLOID |
| US20030232435A1 (en) * | 2002-06-14 | 2003-12-18 | Isis Pharmaceuticals Inc. | Antisense modulation of amyloid beta protein precursor expression |
| GB0324854D0 (en) * | 2003-10-24 | 2003-11-26 | Expresson Biosystems Ltd | App/ena antisense |
| WO2009105572A2 (en) | 2008-02-19 | 2009-08-27 | Edunn Biotechonology Inc. | Antisense modulation of amyloid beta protein expression |
| US20100190807A1 (en) * | 2008-08-26 | 2010-07-29 | Senex Biotechnology, Inc. | CDKI Pathway inhibitors and uses thereof to regulate expression to TAU protein |
| WO2011130742A2 (en) * | 2010-04-16 | 2011-10-20 | Wisconsin Alumni Research Foundation | Diagnostic evaluation of antibody responses to commonly recognized prostate cancer-associated antigens |
| JP2016521556A (ja) | 2013-06-07 | 2016-07-25 | ラナ セラピューティクス インコーポレイテッド | Ｆｏｘｐ３発現を調節するための組成物及び方法 |
| JP2016531570A (ja) | 2013-08-16 | 2016-10-13 | ラナ セラピューティクス インコーポレイテッド | ユークロマチン領域を標的とするオリゴヌクレオチド |
| US10221414B2 (en) | 2013-10-11 | 2019-03-05 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C9ORF72 expression |
| WO2015168172A1 (en) | 2014-04-28 | 2015-11-05 | Isis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| US11732260B2 (en) * | 2018-03-02 | 2023-08-22 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of amyloid-β precursor protein |
-
2020
- 2020-01-29 WO PCT/US2020/015701 patent/WO2020160163A1/en not_active Ceased
- 2020-01-29 US US17/424,672 patent/US20220380773A1/en active Pending
- 2020-01-29 UY UY0001038562A patent/UY38562A/es not_active Application Discontinuation
- 2020-01-29 EP EP20749114.3A patent/EP3918073A4/en active Pending
- 2020-01-29 JP JP2021544144A patent/JP7557469B2/ja active Active
- 2020-01-30 TW TW109102899A patent/TW202043472A/zh unknown
-
2024
- 2024-09-13 JP JP2024158813A patent/JP2025000693A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3918073A1 (en) | 2021-12-08 |
| JP7557469B2 (ja) | 2024-09-27 |
| JP2022518929A (ja) | 2022-03-17 |
| TW202043472A (zh) | 2020-12-01 |
| WO2020160163A1 (en) | 2020-08-06 |
| US20220380773A1 (en) | 2022-12-01 |
| JP2025000693A (ja) | 2025-01-07 |
| EP3918073A4 (en) | 2023-11-22 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| 109 | Application deemed to be withdrawn |
Effective date: 20241111 |