KR20220152569A - 프리칼리크레인-연합된 병태를 치료하고 예방하는 조성물 및 방법 - Google Patents
프리칼리크레인-연합된 병태를 치료하고 예방하는 조성물 및 방법 Download PDFInfo
- Publication number
- KR20220152569A KR20220152569A KR1020227035309A KR20227035309A KR20220152569A KR 20220152569 A KR20220152569 A KR 20220152569A KR 1020227035309 A KR1020227035309 A KR 1020227035309A KR 20227035309 A KR20227035309 A KR 20227035309A KR 20220152569 A KR20220152569 A KR 20220152569A
- Authority
- KR
- South Korea
- Prior art keywords
- less
- weeks
- once
- human subject
- oligomeric compound
- 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
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/10—Antioedematous agents; Diuretics
-
- 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
- C12N15/1137—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 enzymes
-
- 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
- C12N2310/322—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
- C12N2310/334—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
- C12N2310/345—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
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21034—Plasma kallikrein (3.4.21.34)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Ophthalmology & Optometry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062989427P | 2020-03-13 | 2020-03-13 | |
| US62/989,427 | 2020-03-13 | ||
| PCT/US2021/022069 WO2021183857A1 (en) | 2020-03-13 | 2021-03-12 | Compositions and methods for treating and preventing prekallikrein-associated conditions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20220152569A true KR20220152569A (ko) | 2022-11-16 |
Family
ID=77672313
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227035309A Pending KR20220152569A (ko) | 2020-03-13 | 2021-03-12 | 프리칼리크레인-연합된 병태를 치료하고 예방하는 조성물 및 방법 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230149438A1 (https=) |
| EP (1) | EP4118208A4 (https=) |
| JP (2) | JP7795467B2 (https=) |
| KR (1) | KR20220152569A (https=) |
| CN (1) | CN115244179A (https=) |
| BR (1) | BR112022016167A2 (https=) |
| IL (1) | IL295631A (https=) |
| MX (1) | MX2022011205A (https=) |
| WO (1) | WO2021183857A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102365486B1 (ko) | 2013-08-28 | 2022-02-18 | 아이오니스 파마수티컬즈, 인코포레이티드 | 프리칼리크레인 (pkk) 발현의 조절 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102365486B1 (ko) * | 2013-08-28 | 2022-02-18 | 아이오니스 파마수티컬즈, 인코포레이티드 | 프리칼리크레인 (pkk) 발현의 조절 |
| HUE052709T2 (hu) * | 2014-05-01 | 2021-05-28 | Ionis Pharmaceuticals Inc | Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására |
| ES2982752T3 (es) * | 2015-10-22 | 2024-10-17 | Iltoo Pharma | Composiciones farmacéuticas de il-2 |
| PL4119569T3 (pl) * | 2015-11-06 | 2024-11-18 | Ionis Pharmaceuticals, Inc. | Sprzężone związki antysensowne do zastosowania w terapii |
| TW201828952A (zh) * | 2017-02-08 | 2018-08-16 | 美商Ionis製藥公司 | 調節血管舒緩素b1(klkb1)用於治療頭痛 |
-
2021
- 2021-03-12 WO PCT/US2021/022069 patent/WO2021183857A1/en not_active Ceased
- 2021-03-12 KR KR1020227035309A patent/KR20220152569A/ko active Pending
- 2021-03-12 IL IL295631A patent/IL295631A/en unknown
- 2021-03-12 US US17/908,773 patent/US20230149438A1/en active Pending
- 2021-03-12 EP EP21768905.8A patent/EP4118208A4/en active Pending
- 2021-03-12 JP JP2022552307A patent/JP7795467B2/ja active Active
- 2021-03-12 CN CN202180020558.4A patent/CN115244179A/zh active Pending
- 2021-03-12 BR BR112022016167A patent/BR112022016167A2/pt unknown
- 2021-03-12 MX MX2022011205A patent/MX2022011205A/es unknown
-
2025
- 2025-12-19 JP JP2025266910A patent/JP2026048915A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4118208A4 (en) | 2025-03-12 |
| CN115244179A (zh) | 2022-10-25 |
| JP7795467B2 (ja) | 2026-01-07 |
| IL295631A (en) | 2022-10-01 |
| US20230149438A1 (en) | 2023-05-18 |
| JP2026048915A (ja) | 2026-03-17 |
| MX2022011205A (es) | 2022-09-19 |
| BR112022016167A2 (pt) | 2022-10-04 |
| AU2021233914A1 (en) | 2022-09-08 |
| JP2023519501A (ja) | 2023-05-11 |
| WO2021183857A1 (en) | 2021-09-16 |
| EP4118208A1 (en) | 2023-01-18 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2018236699C1 (en) | Methods and compositions for modulating apolipoprotein(a) expression | |
| KR102391812B1 (ko) | B형 간염 감염을 치료하기 위해 PAPD5 또는 PAPD7 mRNA를 감소시키기 위한 핵산 분자 | |
| KR102315836B1 (ko) | 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법 | |
| KR101749352B1 (ko) | Sirt1에 대한 천연 안티센스 전사체의 억제에 의해 sirt1 관련된 질환의 치료 | |
| US20260001946A1 (en) | Treatment of a disease or condition in a tissue originating from the endoderm | |
| CN101142321A (zh) | 治疗眼病的方法和组合物 | |
| KR20150030205A (ko) | Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법 | |
| KR20230124915A (ko) | 세포 사멸 유도 dffa 유사 이펙터 b(cideb) 억제제를 이용한 간 질환의 치료 | |
| AU2016216597B2 (en) | Antisense modulation of ptp1b expression | |
| TW202424191A (zh) | 用於降低PAPD5及PAPD7 mRNA以治療B型肝炎感染之核酸分子 | |
| AU2012275096B2 (en) | Methods for modulating kallikrein (KLKB1) expression | |
| CN111032093A (zh) | 控制心肌纤维化和重塑的方法和组合物 | |
| KR20220152569A (ko) | 프리칼리크레인-연합된 병태를 치료하고 예방하는 조성물 및 방법 | |
| CN110167563A (zh) | 用于调节心脏细胞功能的方法、相关核苷酸和化合物 | |
| KR20200104339A (ko) | Htra1 rna 길항제에 대한 동반 진단제 | |
| TW202237842A (zh) | 靶向atxn3之反義寡核苷酸 | |
| JP4504682B2 (ja) | 脂肪細胞分化関連遺伝子およびタンパク質 | |
| KR20230151996A (ko) | 미토콘드리아 글리세롤-3-인산염 아실전이효소 (gpam)억제제를 이용한 간 질환의 치료 | |
| TW202605151A (zh) | 用於病症及疾病之治療的opa1反義寡聚物 | |
| NZ715825B2 (en) | Combination therapy for treatment of acromegaly |
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 |
|
| D16 | Fast track examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D16-EXM-PA0302 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| D17 | Fast track examination accepted |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D17-EXM-PA0302 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| E13 | Pre-grant limitation requested |
Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| P13-X000 | Application amended |
St.27 status event code: A-2-2-P10-P13-nap-X000 |
|
| PA0302 | Request for accelerated examination |
St.27 status event code: A-1-2-D10-D17-exm-PA0302 St.27 status event code: A-1-2-D10-D16-exm-PA0302 |