KR20220044551A - Apoc3-관련 질환 및 장애의 치료 방법 - Google Patents
Apoc3-관련 질환 및 장애의 치료 방법 Download PDFInfo
- Publication number
- KR20220044551A KR20220044551A KR1020227007372A KR20227007372A KR20220044551A KR 20220044551 A KR20220044551 A KR 20220044551A KR 1020227007372 A KR1020227007372 A KR 1020227007372A KR 20227007372 A KR20227007372 A KR 20227007372A KR 20220044551 A KR20220044551 A KR 20220044551A
- Authority
- KR
- South Korea
- Prior art keywords
- dose
- pharmaceutical composition
- apoc3
- apoc3 rnai
- administered
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Images
Classifications
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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
-
- 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
- A61K47/02—Inorganic compounds
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
-
- 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
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- 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
-
- 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
- C12N2310/3521—Methyl
-
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
-
- 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)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Dermatology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962883046P | 2019-08-05 | 2019-08-05 | |
| US62/883,046 | 2019-08-05 | ||
| US201962936559P | 2019-11-17 | 2019-11-17 | |
| US62/936,559 | 2019-11-17 | ||
| US202062970613P | 2020-02-05 | 2020-02-05 | |
| US62/970,613 | 2020-02-05 | ||
| PCT/US2020/044879 WO2021026150A1 (en) | 2019-08-05 | 2020-08-04 | Methods for the treatment of apoc3-related diseases and disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20220044551A true KR20220044551A (ko) | 2022-04-08 |
Family
ID=74503223
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227007372A Pending KR20220044551A (ko) | 2019-08-05 | 2020-08-04 | Apoc3-관련 질환 및 장애의 치료 방법 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220152079A1 (https=) |
| EP (1) | EP4010075A4 (https=) |
| JP (2) | JP7829478B2 (https=) |
| KR (1) | KR20220044551A (https=) |
| CN (1) | CN114555188A (https=) |
| CA (1) | CA3147725A1 (https=) |
| MX (1) | MX2022001460A (https=) |
| TW (1) | TW202120104A (https=) |
| WO (1) | WO2021026150A1 (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2026049536A1 (en) * | 2024-08-30 | 2026-03-05 | Olix Pharmaceuticals, Inc. | RNAi AGENT TARGETING APOC3 GENE AND USE THEREOF |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201901841TA (en) * | 2016-09-02 | 2019-03-28 | Arrowhead Pharmaceuticals Inc | Targeting ligands |
| CN114934074A (zh) * | 2022-06-01 | 2022-08-23 | 北京大学 | 一种ApoC3基因敲除仓鼠模型的构建方法 |
| AU2024280172A1 (en) | 2023-06-01 | 2025-12-04 | Sunshine Lake Pharma Co., Ltd. | Novel double-stranded sirna, conjugate thereof, and use thereof |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101383006B1 (ko) * | 2009-02-10 | 2014-04-08 | 아마린 파마, 인크. | 고중성지방혈증 치료 방법 |
| MX390699B (es) * | 2011-06-21 | 2025-03-21 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3). |
| EP2723758B1 (en) * | 2011-06-21 | 2018-06-20 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof |
| JP2017535552A (ja) * | 2014-11-17 | 2017-11-30 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | アポリポタンパク質C3(APOC3)iRNA組成物およびその使用方法 |
| WO2018112320A1 (en) * | 2016-12-16 | 2018-06-21 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions |
| CN120330183A (zh) * | 2017-06-02 | 2025-07-18 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| KR20240161202A (ko) * | 2017-09-11 | 2024-11-12 | 애로우헤드 파마슈티컬스 인코포레이티드 | 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물 |
| EP3719127A4 (en) * | 2017-12-01 | 2021-10-20 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE |
-
2020
- 2020-08-04 MX MX2022001460A patent/MX2022001460A/es unknown
- 2020-08-04 JP JP2022506919A patent/JP7829478B2/ja active Active
- 2020-08-04 EP EP20851025.5A patent/EP4010075A4/en active Pending
- 2020-08-04 WO PCT/US2020/044879 patent/WO2021026150A1/en not_active Ceased
- 2020-08-04 CA CA3147725A patent/CA3147725A1/en active Pending
- 2020-08-04 KR KR1020227007372A patent/KR20220044551A/ko active Pending
- 2020-08-04 CN CN202080069368.7A patent/CN114555188A/zh active Pending
- 2020-08-05 TW TW109126581A patent/TW202120104A/zh unknown
-
2022
- 2022-02-03 US US17/592,182 patent/US20220152079A1/en active Pending
-
2025
- 2025-01-28 JP JP2025012194A patent/JP2025061882A/ja active Pending
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2026049536A1 (en) * | 2024-08-30 | 2026-03-05 | Olix Pharmaceuticals, Inc. | RNAi AGENT TARGETING APOC3 GENE AND USE THEREOF |
Also Published As
| Publication number | Publication date |
|---|---|
| MX2022001460A (es) | 2022-02-22 |
| CN114555188A (zh) | 2022-05-27 |
| WO2021026150A1 (en) | 2021-02-11 |
| AU2020324974A1 (en) | 2022-02-24 |
| CA3147725A1 (en) | 2021-02-11 |
| JP2022543136A (ja) | 2022-10-07 |
| JP7829478B2 (ja) | 2026-03-13 |
| EP4010075A4 (en) | 2023-11-15 |
| TW202120104A (zh) | 2021-06-01 |
| JP2025061882A (ja) | 2025-04-11 |
| US20220152079A1 (en) | 2022-05-19 |
| EP4010075A1 (en) | 2022-06-15 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| TWI811238B (zh) | 用於抑制脂蛋白元C-III(APOC3)表現之RNAi試劑及組合物 | |
| JP2022501040A (ja) | 17β−HSD13型(HSD17B13)の発現を阻害するためのRNAi剤、その組成物、および使用方法 | |
| JP7829478B2 (ja) | Apoc3関連疾患および障害の処置のための方法 | |
| WO2020247774A1 (en) | Methods for the treatment of alpha-1 antitrypsin deficiency (aatd) | |
| US20250230439A1 (en) | Methods For The Treatment Of ANGPTL3-Related Diseases And Disorders | |
| EP4214316A2 (en) | Methods for the reduction of z-aat protein levels | |
| AU2020324974B2 (en) | Methods for the treatment of APOC3-related diseases and disorders | |
| RU2851259C1 (ru) | Способы снижения уровней белка z-aat | |
| HK40075141A (en) | Methods for the treatment of apoc3-related diseases and disorders | |
| HK40128585A (zh) | Angptl3相关疾病和病症的治疗方法 | |
| EA048423B1 (ru) | РНКи-АГЕНТЫ И КОМПОЗИЦИИ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (APOC3) |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
St.27 status event code: A-0-1-A10-A15-nap-PA0105 |
|
| PG1501 | Laying open of application |
St.27 status event code: A-1-1-Q10-Q12-nap-PG1501 |
|
| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
|
| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| P13-X000 | Application amended |
St.27 status event code: A-2-2-P10-P13-nap-X000 |
|
| PA0201 | Request for examination |
St.27 status event code: A-1-2-D10-D11-exm-PA0201 |
|
| D21 | Rejection of application intended |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| PE0902 | Notice of grounds for rejection |
St.27 status event code: A-1-2-D10-D21-exm-PE0902 |