CN115244179A - 用于治疗和预防前激肽释放酶相关疾患的组合物和方法 - Google Patents
用于治疗和预防前激肽释放酶相关疾患的组合物和方法 Download PDFInfo
- Publication number
- CN115244179A CN115244179A CN202180020558.4A CN202180020558A CN115244179A CN 115244179 A CN115244179 A CN 115244179A CN 202180020558 A CN202180020558 A CN 202180020558A CN 115244179 A CN115244179 A CN 115244179A
- Authority
- CN
- China
- 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
-
- 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
-
- 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)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Ophthalmology & Optometry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (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 |
|---|---|
| CN115244179A true CN115244179A (zh) | 2022-10-25 |
Family
ID=77672313
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180020558.4A Pending CN115244179A (zh) | 2020-03-13 | 2021-03-12 | 用于治疗和预防前激肽释放酶相关疾患的组合物和方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20230149438A1 (enExample) |
| EP (1) | EP4118208A4 (enExample) |
| JP (2) | JP7795467B2 (enExample) |
| KR (1) | KR20220152569A (enExample) |
| CN (1) | CN115244179A (enExample) |
| AU (2) | AU2021233914B2 (enExample) |
| BR (1) | BR112022016167A2 (enExample) |
| IL (1) | IL295631B1 (enExample) |
| MX (1) | MX2022011205A (enExample) |
| WO (1) | WO2021183857A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9670492B2 (en) | 2013-08-28 | 2017-06-06 | Ionis Pharmaceuticals, Inc. | Modulation of prekallikrein (PKK) expression |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105517556A (zh) * | 2013-08-28 | 2016-04-20 | Ionis制药公司 | 前激肽释放酶(pkk)表达的调节 |
| US20170183661A1 (en) * | 2014-05-01 | 2017-06-29 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating pkk expression |
| WO2018148449A1 (en) * | 2017-02-08 | 2018-08-16 | Ionis Pharmaceuticals, Inc. | Modulation of kallikrein b1 (klkb1) for treatment of headache |
| CN108602893A (zh) * | 2015-12-11 | 2018-09-28 | 戴埃克斯有限公司 | 血浆激肽释放酶抑制剂及其治疗遗传性血管性水肿发作的用途 |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2982752T3 (es) * | 2015-10-22 | 2024-10-17 | Iltoo Pharma | Composiciones farmacéuticas de il-2 |
| LT4119569T (lt) * | 2015-11-06 | 2024-09-25 | Ionis Pharmaceuticals, Inc. | Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje |
-
2021
- 2021-03-12 US US17/908,773 patent/US20230149438A1/en active Pending
- 2021-03-12 AU AU2021233914A patent/AU2021233914B2/en active Active
- 2021-03-12 KR KR1020227035309A patent/KR20220152569A/ko active Pending
- 2021-03-12 IL IL295631A patent/IL295631B1/en unknown
- 2021-03-12 BR BR112022016167A patent/BR112022016167A2/pt unknown
- 2021-03-12 EP EP21768905.8A patent/EP4118208A4/en active Pending
- 2021-03-12 WO PCT/US2021/022069 patent/WO2021183857A1/en not_active Ceased
- 2021-03-12 JP JP2022552307A patent/JP7795467B2/ja active Active
- 2021-03-12 CN CN202180020558.4A patent/CN115244179A/zh active Pending
- 2021-03-12 MX MX2022011205A patent/MX2022011205A/es unknown
-
2025
- 2025-07-16 US US19/271,374 patent/US20260158051A1/en active Pending
- 2025-12-19 JP JP2025266910A patent/JP2026048915A/ja active Pending
-
2026
- 2026-05-13 AU AU2026203664A patent/AU2026203664A1/en active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105517556A (zh) * | 2013-08-28 | 2016-04-20 | Ionis制药公司 | 前激肽释放酶(pkk)表达的调节 |
| US20170183661A1 (en) * | 2014-05-01 | 2017-06-29 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating pkk expression |
| CN108602893A (zh) * | 2015-12-11 | 2018-09-28 | 戴埃克斯有限公司 | 血浆激肽释放酶抑制剂及其治疗遗传性血管性水肿发作的用途 |
| WO2018148449A1 (en) * | 2017-02-08 | 2018-08-16 | Ionis Pharmaceuticals, Inc. | Modulation of kallikrein b1 (klkb1) for treatment of headache |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112022016167A2 (pt) | 2022-10-04 |
| IL295631B1 (en) | 2026-08-01 |
| AU2026203664A1 (en) | 2026-05-28 |
| WO2021183857A1 (en) | 2021-09-16 |
| EP4118208A1 (en) | 2023-01-18 |
| AU2021233914A1 (en) | 2022-09-08 |
| JP2026048915A (ja) | 2026-03-17 |
| MX2022011205A (es) | 2022-09-19 |
| US20230149438A1 (en) | 2023-05-18 |
| IL295631A (en) | 2022-10-01 |
| JP7795467B2 (ja) | 2026-01-07 |
| AU2021233914B2 (en) | 2026-04-30 |
| US20260158051A1 (en) | 2026-06-11 |
| EP4118208A4 (en) | 2025-03-12 |
| KR20220152569A (ko) | 2022-11-16 |
| JP2023519501A (ja) | 2023-05-11 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR102712656B1 (ko) | Hsd17b13 변종 및 이것의 용도 | |
| RU2748495C2 (ru) | Способы и композиции для модулирования экспрессии аполипопротеина (а) | |
| AU2020233603A1 (en) | Compositions and methods | |
| CN112041446B (zh) | 用于减少fxi表达的化合物和方法 | |
| KR20230124915A (ko) | 세포 사멸 유도 dffa 유사 이펙터 b(cideb) 억제제를 이용한 간 질환의 치료 | |
| KR20230061563A (ko) | Ert-치료경험이 없는 환자 및 있는 환자에서 파브리병의 치료 | |
| CN109797150A (zh) | 因子11表达的调节 | |
| CN101142321A (zh) | 治疗眼病的方法和组合物 | |
| CN110193009A (zh) | 用于治疗或预防眼科病的方法 | |
| 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 | |
| JP2026048915A (ja) | プレカリクレイン関連の状態を治療及び予防するための組成物及び方法 | |
| KR20240017911A (ko) | 인간 염색체 9 개방 해독 프레임 72(C9orf72) iRNA 제제 조성물 및 이의 사용 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 | |
| CN115362255A (zh) | 用无七之子2(sos2)抑制剂治疗眼科疾患 | |
| CN112534265A (zh) | 预选个体的抗癌治疗及鉴定易患病个体的筛选方法 | |
| KR20200104339A (ko) | Htra1 rna 길항제에 대한 동반 진단제 | |
| CN113825839A (zh) | 用固醇调节元件结合蛋白切割激活蛋白(scap)抑制剂治疗脂质水平升高 | |
| CN116867510A (zh) | 用线粒体甘油-3-磷酸酰基转移酶(gpam)抑制剂治疗肝病 | |
| HK40007037A (en) | Treatment of fabry disease in ert-naïve and ert-experienced patients |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PB01 | Publication | ||
| PB01 | Publication | ||
| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination |