BR112023004756A2 - Métodos para a redução de níveis de proteína z-aat - Google Patents
Métodos para a redução de níveis de proteína z-aatInfo
- Publication number
- BR112023004756A2 BR112023004756A2 BR112023004756A BR112023004756A BR112023004756A2 BR 112023004756 A2 BR112023004756 A2 BR 112023004756A2 BR 112023004756 A BR112023004756 A BR 112023004756A BR 112023004756 A BR112023004756 A BR 112023004756A BR 112023004756 A2 BR112023004756 A2 BR 112023004756A2
- Authority
- BR
- Brazil
- Prior art keywords
- aat
- liver
- methods
- reducing
- aat protein
- Prior art date
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
-
- 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
-
- 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
- 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)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063078658P | 2020-09-15 | 2020-09-15 | |
| US202163180487P | 2021-04-27 | 2021-04-27 | |
| PCT/US2021/050247 WO2022060721A2 (en) | 2020-09-15 | 2021-09-14 | Methods for the reduction of z-aat protein levels |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023004756A2 true BR112023004756A2 (pt) | 2024-02-06 |
Family
ID=78080560
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023004756A BR112023004756A2 (pt) | 2020-09-15 | 2021-09-14 | Métodos para a redução de níveis de proteína z-aat |
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). |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
| 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 |
| US9458457B2 (en) | 2013-07-03 | 2016-10-04 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA |
| CA3011946A1 (en) | 2016-03-07 | 2017-09-14 | Arrowhead Pharmaceuticals, Inc. | Targeting ligands for therapeutic compounds |
| SG11201901841TA (en) | 2016-09-02 | 2019-03-28 | Arrowhead Pharmaceuticals Inc | Targeting ligands |
| CN110268060B (zh) | 2017-01-10 | 2024-07-26 | 箭头药业股份有限公司 | 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 |
| MX2021015003A (es) | 2019-06-06 | 2022-01-24 | Arrowhead Pharmaceuticals Inc | Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd). |
-
2021
- 2021-09-14 JP JP2023516545A patent/JP7846095B2/ja active Active
- 2021-09-14 US US18/026,340 patent/US20230348905A1/en active Pending
- 2021-09-14 BR BR112023004756A patent/BR112023004756A2/pt unknown
- 2021-09-14 EP EP21787228.2A patent/EP4214316A2/en active Pending
- 2021-09-14 KR KR1020237012414A patent/KR20230067648A/ko active Pending
- 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 CA CA3192372A patent/CA3192372A1/en active Pending
- 2021-09-14 AU AU2021345026A patent/AU2021345026A1/en active Pending
- 2021-09-15 TW TW110134359A patent/TW202220675A/zh unknown
-
2023
- 2023-03-28 CO CONC2023/0003958A patent/CO2023003958A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3192372A1 (en) | 2022-03-24 |
| JP7846095B2 (ja) | 2026-04-14 |
| CO2023003958A2 (es) | 2023-04-17 |
| AU2021345026A9 (en) | 2024-06-13 |
| WO2022060721A3 (en) | 2022-04-28 |
| JP2023541170A (ja) | 2023-09-28 |
| TW202220675A (zh) | 2022-06-01 |
| AU2021345026A1 (en) | 2023-05-11 |
| EP4214316A2 (en) | 2023-07-26 |
| WO2022060721A2 (en) | 2022-03-24 |
| MX2023003066A (es) | 2023-04-10 |
| US20230348905A1 (en) | 2023-11-02 |
| KR20230067648A (ko) | 2023-05-16 |
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