CL2020003457A1 - Oligonucleótidos para modular la expresión de tau - Google Patents
Oligonucleótidos para modular la expresión de tauInfo
- Publication number
- CL2020003457A1 CL2020003457A1 CL2020003457A CL2020003457A CL2020003457A1 CL 2020003457 A1 CL2020003457 A1 CL 2020003457A1 CL 2020003457 A CL2020003457 A CL 2020003457A CL 2020003457 A CL2020003457 A CL 2020003457A CL 2020003457 A1 CL2020003457 A1 CL 2020003457A1
- Authority
- CL
- Chile
- Prior art keywords
- oligonucleotides
- tau
- modulate
- expression
- present
- 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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- 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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- 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
- A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
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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
- A61P25/08—Antiepileptics; Anticonvulsants
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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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- 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
- A61P25/24—Antidepressants
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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
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/26—Endoribonucleases producing 5'-phosphomonoesters (3.1.26)
- C12Y301/26004—Ribonuclease H (3.1.26.4)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C12N2310/00—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
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- C12N2310/32—Chemical structure of the sugar
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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
- C12N2310/322—2'-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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- C12N2310/00—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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
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- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
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- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
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- Plant Pathology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Psychology (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente invención se refiere a oligonucleótidos antisentido que pueden modular la expresión de Tau en una célula diana. Los oligonucleótidos se hibridan a mARN de MAPT. La presente invención, además, se refiere a conjugados del oligonucleótido y composiciones farmacéuticas y métodos para el tratamiento de taupatías, enfermedad de Alzheimer, demencia frontotemporal (FTD), FTDP-17, parálisis supranuclear progresiva (PSP), encefalopatía traumática crónica (CTE), degeneración ganglionar corticobasal (CBD), epilepsia, síndrome de Dravet, depresión, trastornos convulsivos y trastornos motores.
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CL2021001072A CL2021001072A1 (es) | 2018-07-03 | 2021-04-26 | Oligonucleótidos para modular la expresión de tau (divisional solicitud no. 202003457) |
| CL2021001071A CL2021001071A1 (es) | 2018-07-03 | 2021-04-26 | Oligonucleótidos para modular la expresión de tau (divisional solicitud no. 202003457) |
| CL2024003932A CL2024003932A1 (es) | 2018-07-03 | 2024-12-18 | Oligonucleótido antisentido de 19 a 30 nucleótidos de longitud; conjugado; composición farmacéutica; y su uso. |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862693851P | 2018-07-03 | 2018-07-03 | |
| US201862726005P | 2018-08-31 | 2018-08-31 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2020003457A1 true CL2020003457A1 (es) | 2021-06-04 |
Family
ID=67439176
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2020003457A CL2020003457A1 (es) | 2018-07-03 | 2020-12-31 | Oligonucleótidos para modular la expresión de tau |
| CL2021001071A CL2021001071A1 (es) | 2018-07-03 | 2021-04-26 | Oligonucleótidos para modular la expresión de tau (divisional solicitud no. 202003457) |
| CL2021001072A CL2021001072A1 (es) | 2018-07-03 | 2021-04-26 | Oligonucleótidos para modular la expresión de tau (divisional solicitud no. 202003457) |
| CL2024001779A CL2024001779A1 (es) | 2018-07-03 | 2024-06-13 | Oligonucleótidos para modular la expresión de tau |
| CL2024001778A CL2024001778A1 (es) | 2018-07-03 | 2024-06-13 | Oligonucleótidos para modular la expresión de tau |
| CL2024003932A CL2024003932A1 (es) | 2018-07-03 | 2024-12-18 | Oligonucleótido antisentido de 19 a 30 nucleótidos de longitud; conjugado; composición farmacéutica; y su uso. |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021001071A CL2021001071A1 (es) | 2018-07-03 | 2021-04-26 | Oligonucleótidos para modular la expresión de tau (divisional solicitud no. 202003457) |
| CL2021001072A CL2021001072A1 (es) | 2018-07-03 | 2021-04-26 | Oligonucleótidos para modular la expresión de tau (divisional solicitud no. 202003457) |
| CL2024001779A CL2024001779A1 (es) | 2018-07-03 | 2024-06-13 | Oligonucleótidos para modular la expresión de tau |
| CL2024001778A CL2024001778A1 (es) | 2018-07-03 | 2024-06-13 | Oligonucleótidos para modular la expresión de tau |
| CL2024003932A CL2024003932A1 (es) | 2018-07-03 | 2024-12-18 | Oligonucleótido antisentido de 19 a 30 nucleótidos de longitud; conjugado; composición farmacéutica; y su uso. |
Country Status (19)
| Country | Link |
|---|---|
| US (5) | US11279929B2 (es) |
| EP (1) | EP3818159A1 (es) |
| JP (3) | JP7102557B2 (es) |
| KR (3) | KR102723469B1 (es) |
| CN (2) | CN112567033A (es) |
| AU (2) | AU2019297391B2 (es) |
| BR (1) | BR112020026973A2 (es) |
| CA (2) | CA3247235A1 (es) |
| CL (6) | CL2020003457A1 (es) |
| CR (3) | CR20210179A (es) |
| IL (1) | IL279056A (es) |
| MX (3) | MX2020013366A (es) |
| MY (1) | MY206038A (es) |
| PE (2) | PE20210346A1 (es) |
| PH (1) | PH12020552276A1 (es) |
| RU (1) | RU2768285C1 (es) |
| SG (1) | SG11202012759XA (es) |
| TW (2) | TW202519658A (es) |
| WO (1) | WO2020007892A1 (es) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12163131B2 (en) | 2018-07-03 | 2024-12-10 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating Tau expression |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2831232A4 (en) | 2012-03-30 | 2015-11-04 | Univ Washington | METHOD FOR MODULATING TAU EXPRESSION TO REDUCE PROBLEMS AND MODIFY A NEURODEGENERATIVE SYNDROME |
| CN203618139U (zh) * | 2012-08-21 | 2014-05-28 | 特电株式会社 | 流体加热装置 |
| TW202246503A (zh) | 2013-07-19 | 2022-12-01 | 美商百健Ma公司 | 用於調節τ蛋白表現之組合物 |
| WO2019157531A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| JP2023515862A (ja) * | 2020-03-01 | 2023-04-14 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| TW202310854A (zh) * | 2021-07-23 | 2023-03-16 | 美商百健Ma公司 | 用於減少tau表現之方法 |
| US20250283084A1 (en) * | 2024-03-08 | 2025-09-11 | Eli Lilly And Company | Mapt antisense oligonucleotide |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12163131B2 (en) | 2018-07-03 | 2024-12-10 | Hoffmann-La Roche Inc. | Oligonucleotides for modulating Tau expression |
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