CO2022008105A2 - Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos de uso - Google Patents
Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos de usoInfo
- Publication number
- CO2022008105A2 CO2022008105A2 CONC2022/0008105A CO2022008105A CO2022008105A2 CO 2022008105 A2 CO2022008105 A2 CO 2022008105A2 CO 2022008105 A CO2022008105 A CO 2022008105A CO 2022008105 A2 CO2022008105 A2 CO 2022008105A2
- Authority
- CO
- Colombia
- Prior art keywords
- pnpla3
- methods
- patatin
- phospholipase
- domain
- Prior art date
Links
Classifications
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/08—Solutions
-
- 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
- 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
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3183—Diol linkers, e.g. glycols or propanediols
-
- 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
- C12N2310/321—2'-O-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
- C12N2310/32—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
-
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Inorganic Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Abstract
Agentes de ARNi, por ejemplo, agentes de ARN de hebra doble (ARNhd), dirigidos al gen que contiene un dominio fosfolipasa tipo patatina 3 (PNPLA3). La invención también se relaciona con métodos de uso de dichos agentes de ARNi para inhibir la expresión de un gen PNPLA3 y con métodos para prevenir y tratar un tastorno asociado a PNPLA3, por ejemplo, la enfermedad de hígado graso no alcohólico (NAFLD).
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962948445P | 2019-12-16 | 2019-12-16 | |
US202063040602P | 2020-06-18 | 2020-06-18 | |
PCT/US2020/064776 WO2021126734A1 (en) | 2019-12-16 | 2020-12-14 | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2022008105A2 true CO2022008105A2 (es) | 2022-07-08 |
Family
ID=76437193
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2022/0008105A CO2022008105A2 (es) | 2019-12-16 | 2022-06-07 | Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos de uso |
Country Status (11)
Country | Link |
---|---|
US (2) | US11149276B2 (es) |
EP (1) | EP4077666A1 (es) |
JP (1) | JP2023506258A (es) |
KR (1) | KR20220116520A (es) |
CN (1) | CN115176007A (es) |
AU (1) | AU2020404905A1 (es) |
BR (1) | BR112022011412A2 (es) |
CA (1) | CA3164628A1 (es) |
CO (1) | CO2022008105A2 (es) |
IL (1) | IL293860A (es) |
MX (1) | MX2022007144A (es) |
Family Cites Families (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US7790867B2 (en) | 2002-12-05 | 2010-09-07 | Rosetta Genomics Inc. | Vaccinia virus-related nucleic acids and microRNA |
KR20220045091A (ko) | 2011-11-18 | 2022-04-12 | 알닐람 파마슈티칼스 인코포레이티드 | 트랜스티레틴(TTR) 관련 질병을 치료하기 위한 RNAi 제제, 조성 및 그의 사용방법 |
EA201490993A1 (ru) | 2011-11-18 | 2014-09-30 | Элнилэм Фармасьютикалз, Инк. | МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи |
JP2018510621A (ja) | 2015-02-13 | 2018-04-19 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | パタチン様ホスホリパーゼドメイン含有3(PNPLA3)iRNA組成物およびその使用方法 |
WO2017048620A1 (en) | 2015-09-14 | 2017-03-23 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof |
-
2020
- 2020-12-14 IL IL293860A patent/IL293860A/en unknown
- 2020-12-14 MX MX2022007144A patent/MX2022007144A/es unknown
- 2020-12-14 AU AU2020404905A patent/AU2020404905A1/en active Pending
- 2020-12-14 JP JP2022536726A patent/JP2023506258A/ja active Pending
- 2020-12-14 KR KR1020227024693A patent/KR20220116520A/ko unknown
- 2020-12-14 CN CN202080096559.2A patent/CN115176007A/zh active Pending
- 2020-12-14 EP EP20829496.7A patent/EP4077666A1/en active Pending
- 2020-12-14 BR BR112022011412A patent/BR112022011412A2/pt unknown
- 2020-12-14 CA CA3164628A patent/CA3164628A1/en active Pending
-
2021
- 2021-02-23 US US17/182,348 patent/US11149276B2/en active Active
- 2021-04-27 US US17/241,227 patent/US20220348926A1/en active Pending
-
2022
- 2022-06-07 CO CONC2022/0008105A patent/CO2022008105A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
JP2023506258A (ja) | 2023-02-15 |
CN115176007A (zh) | 2022-10-11 |
BR112022011412A2 (pt) | 2022-08-30 |
AU2020404905A1 (en) | 2022-06-23 |
IL293860A (en) | 2022-08-01 |
EP4077666A1 (en) | 2022-10-26 |
US11149276B2 (en) | 2021-10-19 |
US20210189400A1 (en) | 2021-06-24 |
CA3164628A1 (en) | 2021-06-24 |
US20220348926A1 (en) | 2022-11-03 |
MX2022007144A (es) | 2022-07-11 |
KR20220116520A (ko) | 2022-08-23 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CL2022001590A1 (es) | Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos | |
DOP2019000132A (es) | COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS. | |
CL2021002326A1 (es) | Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso (divisional de solicitud n° 202002865) | |
WO2016130806A3 (en) | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof | |
PE20160030A1 (es) | COMPOSICIONES DE ARNi DE SERPINA 1 Y SUS METODOS DE USO | |
DOP2014000107A (es) | AGENTES DE iARN, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR) | |
UY36357A (es) | Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa)) | |
CO2021000447A2 (es) | Inhibidores de proteína tirosina fosfatasa y sus métodos de uso | |
CL2020001543A1 (es) | Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de los mismos. | |
AR117297A1 (es) | Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de estos | |
CL2022003369A1 (es) | Constructos de arni para inhibir la expresión de hsd17b13 y métodos de uso de los mismos | |
BR112021024080A2 (pt) | Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos | |
AR120341A1 (es) | COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO | |
CO2024001510A2 (es) | Composiciones de arni de beta-catenina (ctnnb1) y métodos de uso de estas | |
CO2022008105A2 (es) | Composiciones de arni que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus métodos de uso | |
AR115960A1 (es) | Composiciones y métodos para inhibir la expresión del gen lect2 | |
AR103676A1 (es) | COMPOSICIONES DE ARNi QUE CONTIENEN UN DOMINIO FOSFOLIPASA TIPO PATATINA 3 (PNPLA3) Y SUS MÉTODOS DE USO | |
AR120979A1 (es) | COMPOSICIONES DE ARNi QUE CONTIENEN UN DOMINIO FOSFOLIPASA TIPO PATATINA 3 (PNPLA3) Y SUS MÉTODOS DE USO | |
AR110225A1 (es) | COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO | |
CL2017002646A1 (es) | Composiciones de arni de serpina1 y sus métodos de uso (divisional de solicitud n° 3441-2015). | |
AR115505A1 (es) | Guías modificadas de arn para edición génica | |
CR20150644A (es) | Composiciones de arni de serapina 1 y sus métodos de uso | |
AR117458A1 (es) | COMPOSICIONES DE AGENTES DE ARNi PARA LA PROTEÍNA PRECURSORA AMILOIDE (APP) Y SUS MÉTODOS DE USO | |
CL2017000872A1 (es) | Composiciones y métodos para la inhibición de la expresión génica de hao1 (hidroxiácido oxidasa 1 (glicolato oxidasa) | |
AR096390A1 (es) | COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO |