JP7846095B2 - Ｚ－ａａｔタンパク質のレベルを低下させる方法 - Google Patents
Ｚ－ａａｔタンパク質のレベルを低下させる方法Info
- Publication number
- JP7846095B2 JP7846095B2 JP2023516545A JP2023516545A JP7846095B2 JP 7846095 B2 JP7846095 B2 JP 7846095B2 JP 2023516545 A JP2023516545 A JP 2023516545A JP 2023516545 A JP2023516545 A JP 2023516545A JP 7846095 B2 JP7846095 B2 JP 7846095B2
- Authority
- JP
- Japan
- Prior art keywords
- aat
- approximately
- pharmaceutical composition
- dose
- subject
- 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.)
- Active
Links
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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- 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
- 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)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063078658P | 2020-09-15 | 2020-09-15 | |
| US63/078,658 | 2020-09-15 | ||
| US202163180487P | 2021-04-27 | 2021-04-27 | |
| US63/180,487 | 2021-04-27 | ||
| PCT/US2021/050247 WO2022060721A2 (en) | 2020-09-15 | 2021-09-14 | Methods for the reduction of z-aat protein levels |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2023541170A JP2023541170A (ja) | 2023-09-28 |
| JPWO2022060721A5 JPWO2022060721A5 (https=) | 2024-09-24 |
| JP2023541170A5 JP2023541170A5 (https=) | 2024-09-24 |
| JP7846095B2 true JP7846095B2 (ja) | 2026-04-14 |
Family
ID=78080560
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023516545A Active JP7846095B2 (ja) | 2020-09-15 | 2021-09-14 | Ｚ－ａａｔタンパク質のレベルを低下させる方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230348905A1 (https=) |
| EP (1) | EP4214316A2 (https=) |
| JP (1) | JP7846095B2 (https=) |
| KR (1) | KR20230067648A (https=) |
| AU (1) | AU2021345026A1 (https=) |
| BR (1) | BR112023004756A2 (https=) |
| CA (1) | CA3192372A1 (https=) |
| CO (1) | CO2023003958A2 (https=) |
| MX (1) | MX2023003066A (https=) |
| TW (1) | TW202220675A (https=) |
| WO (1) | WO2022060721A2 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW201620526A (zh) | 2014-06-17 | 2016-06-16 | 愛羅海德研究公司 | 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法 |
| MX2021015003A (es) | 2019-06-06 | 2022-01-24 | Arrowhead Pharmaceuticals Inc | Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd). |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008016391A2 (en) | 2006-01-31 | 2008-02-07 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| WO2012178033A2 (en) | 2011-06-23 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Serpina1 sirnas: compositions of matter and methods of treatment |
| JP2016528887A (ja) | 2013-07-03 | 2016-09-23 | ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 |
| WO2020247774A1 (en) | 2019-06-06 | 2020-12-10 | Arrowhead Pharmaceuticals, Inc. | Methods for the treatment of alpha-1 antitrypsin deficiency (aatd) |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
| KR102515329B1 (ko) | 2016-03-07 | 2023-03-29 | 애로우헤드 파마슈티컬스 인코포레이티드 | 치료용 화합물을 위한 표적화 리간드 |
| MX2018009853A (es) | 2016-09-02 | 2018-11-09 | Arrowhead Pharmaceuticals Inc | Ligandos de direccion. |
| BR112019014282A2 (pt) | 2017-01-10 | 2020-03-03 | Arrowhead Pharmaceuticals, Inc. | Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso |
-
2021
- 2021-09-14 AU AU2021345026A patent/AU2021345026A1/en active Pending
- 2021-09-14 US US18/026,340 patent/US20230348905A1/en active Pending
- 2021-09-14 JP JP2023516545A patent/JP7846095B2/ja active Active
- 2021-09-14 MX MX2023003066A patent/MX2023003066A/es unknown
- 2021-09-14 WO PCT/US2021/050247 patent/WO2022060721A2/en not_active Ceased
- 2021-09-14 EP EP21787228.2A patent/EP4214316A2/en active Pending
- 2021-09-14 KR KR1020237012414A patent/KR20230067648A/ko active Pending
- 2021-09-14 BR BR112023004756A patent/BR112023004756A2/pt unknown
- 2021-09-14 CA CA3192372A patent/CA3192372A1/en active Pending
- 2021-09-15 TW TW110134359A patent/TW202220675A/zh unknown
-
2023
- 2023-03-28 CO CONC2023/0003958A patent/CO2023003958A2/es unknown
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008016391A2 (en) | 2006-01-31 | 2008-02-07 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
| WO2012178033A2 (en) | 2011-06-23 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Serpina1 sirnas: compositions of matter and methods of treatment |
| JP2016528887A (ja) | 2013-07-03 | 2016-09-23 | ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 |
| WO2020247774A1 (en) | 2019-06-06 | 2020-12-10 | Arrowhead Pharmaceuticals, Inc. | Methods for the treatment of alpha-1 antitrypsin deficiency (aatd) |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022060721A3 (en) | 2022-04-28 |
| JP2023541170A (ja) | 2023-09-28 |
| TW202220675A (zh) | 2022-06-01 |
| WO2022060721A2 (en) | 2022-03-24 |
| CA3192372A1 (en) | 2022-03-24 |
| EP4214316A2 (en) | 2023-07-26 |
| CO2023003958A2 (es) | 2023-04-17 |
| AU2021345026A1 (en) | 2023-05-11 |
| AU2021345026A9 (en) | 2024-06-13 |
| US20230348905A1 (en) | 2023-11-02 |
| BR112023004756A2 (pt) | 2024-02-06 |
| MX2023003066A (es) | 2023-04-10 |
| KR20230067648A (ko) | 2023-05-16 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7488254B2 (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 | |
| JP7697889B2 (ja) | アルファ－１アンチトリプシン欠乏症（ａａｔｄ）の治療法 | |
| CN110268060B (zh) | α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法 | |
| JP7829478B2 (ja) | Ａｐｏｃ３関連疾患および障害の処置のための方法 | |
| JP7846095B2 (ja) | Ｚ－ａａｔタンパク質のレベルを低下させる方法 | |
| JP2025511854A (ja) | 非アルコール性脂肪肝疾患の治療 | |
| CN120112639A (zh) | Angptl3相关疾病和病症的治疗方法 | |
| CN116670278A (zh) | 用于降低z-aat蛋白水平的方法 | |
| RU2851259C1 (ru) | Способы снижения уровней белка z-aat | |
| RU2847618C2 (ru) | Способы лечения дефицита альфа-1-антитрипсина (aatd) | |
| TW202430635A (zh) | 用於抑制補體成分C3(C3)表現之RNAi藥劑、其醫藥組合物及使用方法 | |
| HK40075141A (en) | Methods for the treatment of apoc3-related diseases and disorders |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20240912 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20240912 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20251028 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20260126 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20260303 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20260402 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 7846095 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |