CA3192372A1 - Methods for the reduction of z-aat protein levels - Google Patents
Methods for the reduction of z-aat protein levelsInfo
- Publication number
- CA3192372A1 CA3192372A1 CA3192372A CA3192372A CA3192372A1 CA 3192372 A1 CA3192372 A1 CA 3192372A1 CA 3192372 A CA3192372 A CA 3192372A CA 3192372 A CA3192372 A CA 3192372A CA 3192372 A1 CA3192372 A1 CA 3192372A1
- Authority
- CA
- Canada
- Prior art keywords
- aat
- reduction
- liver
- subject
- drug substance
- 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
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)
- 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 (1)
| Publication Number | Publication Date |
|---|---|
| CA3192372A1 true CA3192372A1 (en) | 2022-03-24 |
Family
ID=78080560
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3192372A Pending CA3192372A1 (en) | 2020-09-15 | 2021-09-14 | Methods for the reduction of z-aat protein levels |
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 |
| EP3366312A1 (en) * | 2011-06-23 | 2018-08-29 | Alnylam Pharmaceuticals, Inc. | Serpina 1 sirnas: compositions of matter and methods of treatment |
| DK3444350T3 (da) * | 2013-07-03 | 2022-02-07 | Dicerna Pharmaceuticals Inc | Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna |
| 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 |
| 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 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
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 |
| 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 |
| JP7846095B2 (ja) | 2026-04-14 |
| MX2023003066A (es) | 2023-04-10 |
| KR20230067648A (ko) | 2023-05-16 |
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 | |
| US12582668B2 (en) | Methods for the treatment of alpha-1 antitrypsin deficiency (AATD) | |
| 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 | |
| US20230139166A1 (en) | RNAi Agents for Inhibiting Expression of Xanthine Dehydrogenase (XDH), Pharmaceutical Compositions Thereof, and Methods of Use | |
| AU2021242321A1 (en) | RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use | |
| US20220152079A1 (en) | Methods For The Treatment Of APOC3-Related Diseases And Disorders | |
| CA3192372A1 (en) | Methods for the reduction of z-aat protein levels | |
| CN120112639A (zh) | Angptl3相关疾病和病症的治疗方法 | |
| JP2025511854A (ja) | 非アルコール性脂肪肝疾患の治療 | |
| 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) | |
| AU2020324974B2 (en) | Methods for the treatment of APOC3-related diseases and disorders | |
| HK40075141A (en) | Methods for the treatment of apoc3-related diseases and disorders | |
| 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 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD Year of fee payment: 3 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20240826 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20240826 Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20240826 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD Year of fee payment: 4 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250821 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250905 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED Effective date: 20250911 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT Effective date: 20250911 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250911 |