JOP20210142A1 - تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها - Google Patents
تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامهاInfo
- Publication number
- JOP20210142A1 JOP20210142A1 JOP/2021/0142A JOP20210142A JOP20210142A1 JO P20210142 A1 JOP20210142 A1 JO P20210142A1 JO P20210142 A JOP20210142 A JO P20210142A JO P20210142 A1 JOP20210142 A1 JO P20210142A1
- Authority
- JO
- Jordan
- Prior art keywords
- rnai constructs
- pnpla3 expression
- inhibiting pnpla3
- inhibiting
- liver disease
- Prior art date
Links
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- 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
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- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- 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
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1025—Acyltransferases (2.3)
- C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
- C12N9/20—Triglyceride splitting, e.g. by means of lipase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y203/00—Acyltransferases (2.3)
- C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
- C12Y203/01051—1-Acylglycerol-3-phosphate O-acyltransferase (2.3.1.51)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01003—Triacylglycerol lipase (3.1.1.3)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
يتعلق الاختراع الحالي بتركيبات RNAi لتقليل التعبير الوراثي عن جين PNPLA3. تم أيضاً وصف طرق استخدام هذه التركيبات RNAi لعلاج أو منع أمراض الكبد، مرض الكبد الدهني غير الكحولي (NAFLD).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862777714P | 2018-12-10 | 2018-12-10 | |
PCT/US2019/065481 WO2020123508A2 (en) | 2018-12-10 | 2019-12-10 | Rnai constructs for inhibiting pnpla3 expression and methods of use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
JOP20210142A1 true JOP20210142A1 (ar) | 2023-01-30 |
Family
ID=69024749
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JOP/2021/0142A JOP20210142A1 (ar) | 2018-12-10 | 2019-12-10 | تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها |
Country Status (21)
Country | Link |
---|---|
US (1) | US20220017906A1 (ar) |
EP (1) | EP3894555A2 (ar) |
JP (1) | JP2022511550A (ar) |
KR (1) | KR20210102932A (ar) |
CN (1) | CN113166761A (ar) |
AR (1) | AR117297A1 (ar) |
AU (1) | AU2019396419A1 (ar) |
BR (1) | BR112021011061A2 (ar) |
CA (1) | CA3121510A1 (ar) |
CL (1) | CL2021001489A1 (ar) |
CO (1) | CO2021008997A2 (ar) |
CR (1) | CR20210371A (ar) |
EA (1) | EA202191629A1 (ar) |
IL (1) | IL283473A (ar) |
JO (1) | JOP20210142A1 (ar) |
MX (1) | MX2021006784A (ar) |
PE (1) | PE20211225A1 (ar) |
SG (1) | SG11202105711SA (ar) |
TW (1) | TW202039843A (ar) |
UY (1) | UY38503A (ar) |
WO (1) | WO2020123508A2 (ar) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
TW202300645A (zh) * | 2021-04-14 | 2023-01-01 | 美商戴瑟納製藥股份有限公司 | 用於調節pnpla3表現之組合物及方法 |
EP4347823A1 (en) * | 2021-06-02 | 2024-04-10 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
CA3230222A1 (en) * | 2021-09-01 | 2023-03-09 | Leonid Beigelman | Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof |
WO2024023256A1 (en) * | 2022-07-27 | 2024-02-01 | E-Therapeutics Plc | Nucleic acid compounds |
WO2024120412A1 (en) * | 2022-12-07 | 2024-06-13 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of patatin-like phospholipase domain-containing 3 (pnpla3) |
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EP0972564B1 (en) | 1991-11-22 | 2003-05-28 | Affymetrix, Inc. (a Delaware Corporation) | Method of forming arrays of polymers |
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2019
- 2019-12-10 MX MX2021006784A patent/MX2021006784A/es unknown
- 2019-12-10 UY UY0001038503A patent/UY38503A/es unknown
- 2019-12-10 TW TW108145175A patent/TW202039843A/zh unknown
- 2019-12-10 US US17/312,721 patent/US20220017906A1/en active Pending
- 2019-12-10 EA EA202191629A patent/EA202191629A1/ru unknown
- 2019-12-10 JP JP2021532216A patent/JP2022511550A/ja active Pending
- 2019-12-10 SG SG11202105711SA patent/SG11202105711SA/en unknown
- 2019-12-10 EP EP19828524.9A patent/EP3894555A2/en active Pending
- 2019-12-10 CR CR20210371A patent/CR20210371A/es unknown
- 2019-12-10 WO PCT/US2019/065481 patent/WO2020123508A2/en unknown
- 2019-12-10 AU AU2019396419A patent/AU2019396419A1/en active Pending
- 2019-12-10 BR BR112021011061-5A patent/BR112021011061A2/pt unknown
- 2019-12-10 KR KR1020217021155A patent/KR20210102932A/ko unknown
- 2019-12-10 PE PE2021000850A patent/PE20211225A1/es unknown
- 2019-12-10 JO JOP/2021/0142A patent/JOP20210142A1/ar unknown
- 2019-12-10 AR ARP190103607A patent/AR117297A1/es unknown
- 2019-12-10 CA CA3121510A patent/CA3121510A1/en active Pending
- 2019-12-10 CN CN201980081364.8A patent/CN113166761A/zh active Pending
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2021
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KR20210102932A (ko) | 2021-08-20 |
CA3121510A1 (en) | 2020-06-18 |
AR117297A1 (es) | 2021-07-28 |
IL283473A (en) | 2021-07-29 |
CO2021008997A2 (es) | 2021-07-30 |
WO2020123508A3 (en) | 2020-08-13 |
JP2022511550A (ja) | 2022-01-31 |
PE20211225A1 (es) | 2021-07-06 |
CR20210371A (es) | 2021-08-27 |
CN113166761A (zh) | 2021-07-23 |
CL2021001489A1 (es) | 2022-01-07 |
WO2020123508A2 (en) | 2020-06-18 |
MX2021006784A (es) | 2021-07-15 |
TW202039843A (zh) | 2020-11-01 |
UY38503A (es) | 2020-06-30 |
BR112021011061A2 (pt) | 2021-08-31 |
EA202191629A1 (ru) | 2021-09-03 |
EP3894555A2 (en) | 2021-10-20 |
SG11202105711SA (en) | 2021-06-29 |
AU2019396419A1 (en) | 2021-06-17 |
US20220017906A1 (en) | 2022-01-20 |
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