BR112023004012A2 - Métodos para reduzir a expressão de apociii - Google Patents
Métodos para reduzir a expressão de apociiiInfo
- Publication number
- BR112023004012A2 BR112023004012A2 BR112023004012A BR112023004012A BR112023004012A2 BR 112023004012 A2 BR112023004012 A2 BR 112023004012A2 BR 112023004012 A BR112023004012 A BR 112023004012A BR 112023004012 A BR112023004012 A BR 112023004012A BR 112023004012 A2 BR112023004012 A2 BR 112023004012A2
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- reducing
- apociii
- shtg
- sqf
- Prior art date
Links
- 102100030970 Apolipoprotein C-III Human genes 0.000 title abstract 4
- 101000793223 Homo sapiens Apolipoprotein C-III Proteins 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 4
- 208000003929 Familial Partial Lipodystrophy Diseases 0.000 abstract 2
- 208000024891 symptom Diseases 0.000 abstract 2
- ABEXEQSGABRUHS-UHFFFAOYSA-N 16-methylheptadecyl 16-methylheptadecanoate Chemical compound CC(C)CCCCCCCCCCCCCCCOC(=O)CCCCCCCCCCCCCCC(C)C ABEXEQSGABRUHS-UHFFFAOYSA-N 0.000 abstract 1
- 208000004998 Abdominal Pain Diseases 0.000 abstract 1
- 108010004103 Chylomicrons Proteins 0.000 abstract 1
- 206010012735 Diarrhoea Diseases 0.000 abstract 1
- 208000016667 Familial chylomicronemia syndrome Diseases 0.000 abstract 1
- 241000764238 Isis Species 0.000 abstract 1
- 206010048215 Xanthomatosis Diseases 0.000 abstract 1
- 206010019847 hepatosplenomegaly Diseases 0.000 abstract 1
- 208000006575 hypertriglyceridemia Diseases 0.000 abstract 1
- 238000005417 image-selected in vivo spectroscopy Methods 0.000 abstract 1
- 230000001771 impaired effect Effects 0.000 abstract 1
- 238000012739 integrated shape imaging system Methods 0.000 abstract 1
- 208000024866 recurrent acute pancreatitis Diseases 0.000 abstract 1
- 230000000630 rising effect Effects 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/05—Dipeptides
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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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- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/34—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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063087095P | 2020-10-02 | 2020-10-02 | |
| PCT/US2021/052001 WO2022072244A1 (en) | 2020-10-02 | 2021-09-24 | Methods for reducing apociii expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023004012A2 true BR112023004012A2 (pt) | 2023-04-25 |
Family
ID=80950871
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023004012A BR112023004012A2 (pt) | 2020-10-02 | 2021-09-24 | Métodos para reduzir a expressão de apociii |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240033280A1 (https=) |
| EP (1) | EP4221720A4 (https=) |
| JP (1) | JP2023543898A (https=) |
| KR (1) | KR20230079394A (https=) |
| CN (1) | CN116348125A (https=) |
| AU (1) | AU2021353849A1 (https=) |
| BR (1) | BR112023004012A2 (https=) |
| IL (1) | IL300882A (https=) |
| MX (1) | MX2023003855A (https=) |
| WO (1) | WO2022072244A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL277889B2 (en) | 2018-04-12 | 2025-01-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods of using them |
| EP3790596A4 (en) | 2018-05-11 | 2022-04-06 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE |
| AU2023372971A1 (en) * | 2022-11-02 | 2025-05-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating complement factor b expression |
| WO2024194686A2 (en) | 2023-03-17 | 2024-09-26 | Oxitope Pharma B.V. | Anti-phosphocholine antibodies and methods of use thereof |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7598227B2 (en) * | 2003-04-16 | 2009-10-06 | Isis Pharmaceuticals Inc. | Modulation of apolipoprotein C-III expression |
| AU2014216137B2 (en) * | 2013-02-14 | 2018-05-10 | Ionis Pharmaceuticals, Inc. | Modulation of Apolipoprotein C-III (ApoCIII) expression in lipoprotein lipase deficient (LPLD) populations |
| JP2018511555A (ja) * | 2015-02-27 | 2018-04-26 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | リポジストロフィー集団におけるアポリポタンパク質Ｃ−ＩＩＩ（ＡｐｏＣＩＩＩ）発現の調節 |
| US20190046555A1 (en) * | 2015-11-06 | 2019-02-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds for use in therapy |
-
2021
- 2021-09-24 IL IL300882A patent/IL300882A/en unknown
- 2021-09-24 BR BR112023004012A patent/BR112023004012A2/pt unknown
- 2021-09-24 US US18/247,567 patent/US20240033280A1/en active Pending
- 2021-09-24 MX MX2023003855A patent/MX2023003855A/es unknown
- 2021-09-24 EP EP21876250.8A patent/EP4221720A4/en active Pending
- 2021-09-24 CN CN202180066569.6A patent/CN116348125A/zh active Pending
- 2021-09-24 KR KR1020237013301A patent/KR20230079394A/ko active Pending
- 2021-09-24 JP JP2023520059A patent/JP2023543898A/ja active Pending
- 2021-09-24 AU AU2021353849A patent/AU2021353849A1/en active Pending
- 2021-09-24 WO PCT/US2021/052001 patent/WO2022072244A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230079394A (ko) | 2023-06-07 |
| WO2022072244A1 (en) | 2022-04-07 |
| AU2021353849A1 (en) | 2023-03-23 |
| EP4221720A4 (en) | 2024-11-13 |
| US20240033280A1 (en) | 2024-02-01 |
| EP4221720A1 (en) | 2023-08-09 |
| MX2023003855A (es) | 2023-04-14 |
| CN116348125A (zh) | 2023-06-27 |
| IL300882A (en) | 2023-04-01 |
| AU2021353849A8 (en) | 2023-03-30 |
| JP2023543898A (ja) | 2023-10-18 |
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