AR128558A1 - Oligonucleótido antisentido - Google Patents
Oligonucleótido antisentidoInfo
- Publication number
- AR128558A1 AR128558A1 ARP230100385A ARP230100385A AR128558A1 AR 128558 A1 AR128558 A1 AR 128558A1 AR P230100385 A ARP230100385 A AR P230100385A AR P230100385 A ARP230100385 A AR P230100385A AR 128558 A1 AR128558 A1 AR 128558A1
- Authority
- AR
- Argentina
- Prior art keywords
- seq
- positions
- antisense oligonucleotide
- a1cf
- sequence
- Prior art date
Links
- 239000000074 antisense oligonucleotide Substances 0.000 title abstract 8
- 238000012230 antisense oligonucleotides Methods 0.000 title abstract 8
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 6
- 239000002773 nucleotide Substances 0.000 abstract 5
- 125000003729 nucleotide group Chemical group 0.000 abstract 5
- 238000000034 method Methods 0.000 abstract 4
- 239000008194 pharmaceutical composition Substances 0.000 abstract 3
- 102000012758 APOBEC-1 Deaminase Human genes 0.000 abstract 2
- 108010079649 APOBEC-1 Deaminase Proteins 0.000 abstract 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 2
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 abstract 2
- 150000003839 salts Chemical class 0.000 abstract 2
- 241000700721 Hepatitis B virus Species 0.000 abstract 1
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 abstract 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 abstract 1
- 230000003197 catalytic effect Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 208000015181 infectious disease Diseases 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
Classifications
-
- 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
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
-
- 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
- C12N2310/11—Antisense
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Communicable Diseases (AREA)
- Physics & Mathematics (AREA)
- Oncology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La presente invención se refiere a oligonucleótidos antisentido que reducen la expresión de A1CF, así como conjugados, sales y composiciones farmacéuticas de estos. La invención también se refiere a usos de tales oligonucleótidos antisentido, conjugados, sales y composiciones farmacéuticas en métodos para reducir la expresión de A1CF y en usos médicos y métodos para tratar enfermedades, particularmente para tratar una infección por el virus de la hepatitis B (HBV). Reivindicación 1: Un oligonucleótido antisentido con una longitud de 12 a 30 nucleótidos, que comprende una secuencia de nucleótidos contiguos con una longitud de 12 a 30 nucleótidos, en donde la secuencia de nucleótidos contiguos es capaz de unirse a una secuencia diana en un mARN de factor de complementación (A1CF) de la subunidad catalítica 1 de la enzima de edición de mARN de la apolipoproteína B (APOBEC1), en donde la secuencia diana es una secuencia de al menos 17 nucleótidos contiguos dentro de cualquiera de las siguientes secuencias: GCCUAUCUGAGAAACUUUU (SEQ ID Nº 32) (posiciones 2181 - 2199 de SEQ ID Nº 45), GAGAAAAACCUAUAAUGCCU (SEQ ID Nº 42) (posiciones 6951 - 6970 de SEQ ID Nº 45), AAGUAAAAUUAACAUGUCCA (SEQ ID Nº 43) (posiciones 16970 - 16989 de SEQ ID Nº 45), AAACACCACAAUCUUAAAAC (SEQ ID Nº 39) (posiciones 26358 - 26377 de SEQ ID Nº 45), CAGGUAUAUAACAAGUUCA (SEQ ID Nº 34) (posiciones 38053 - 38071 de SEQ ID Nº 45), y AGACACACAAAACUCUAU (SEQ ID Nº 44) (posiciones 78973 - 78990 de SEQ ID Nº 45), y en donde el oligonucleótido antisentido es capaz de reducir la expresión de A1CF. Reivindicación 23: El conjugado de oligonucleótido antisentido de acuerdo con la reivindicación 22, en donde la porción de conjugado es una porción de conjugado de N-acetilgalactosamina (GalNAc). Reivindicación 42: Un método in vitro o in vivo para reducir la expresión de A1CF en una célula diana, en donde el método comprende administrar una cantidad eficaz del oligonucleótido antisentido o el conjugado de oligonucleótido antisentido de acuerdo con cualquiera de las reivindicaciones 1 a 40 o la composición farmacéutica de acuerdo con la reivindicación 41 a la célula diana.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP22157822 | 2022-02-21 | ||
| EP22185488 | 2022-07-18 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR128558A1 true AR128558A1 (es) | 2024-05-22 |
Family
ID=85238940
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP230100385A AR128558A1 (es) | 2022-02-21 | 2023-02-17 | Oligonucleótido antisentido |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20240167040A1 (es) |
| EP (1) | EP4482959A1 (es) |
| AR (1) | AR128558A1 (es) |
| TW (1) | TW202346587A (es) |
| WO (1) | WO2023156652A1 (es) |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| DE69829760T3 (de) | 1997-09-12 | 2016-04-14 | Exiqon A/S | Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga |
| KR100573231B1 (ko) | 1999-02-12 | 2006-04-24 | 상꾜 가부시키가이샤 | 신규 뉴클레오시드 및 올리고뉴클레오티드 유사체 |
| NZ514348A (en) | 1999-05-04 | 2004-05-28 | Exiqon As | L-ribo-LNA analogues |
| US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| WO2003070910A2 (en) * | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| CA2994089A1 (en) | 2002-11-18 | 2004-06-03 | Roche Innovation Center Copenhagen A/S | Antisense gapmer oligonucleotides |
| US7339051B2 (en) * | 2003-04-28 | 2008-03-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for the treatment of severe acute respiratory syndrome (SARS) |
| CA2640171C (en) | 2006-01-27 | 2014-10-28 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| CA2651453C (en) | 2006-05-11 | 2014-10-14 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| EP2170917B1 (en) | 2007-05-30 | 2012-06-27 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| EP2173760B2 (en) | 2007-06-08 | 2015-11-04 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| ES2376507T5 (es) | 2007-07-05 | 2015-08-31 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos 6-disustituidos |
| US8546556B2 (en) | 2007-11-21 | 2013-10-01 | Isis Pharmaceuticals, Inc | Carbocyclic alpha-L-bicyclic nucleic acid analogs |
| DK2356129T3 (da) | 2008-09-24 | 2013-05-13 | Isis Pharmaceuticals Inc | Substituerede alpha-L-bicykliske nukleosider |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| WO2011156202A1 (en) | 2010-06-08 | 2011-12-15 | Isis Pharmaceuticals, Inc. | Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| WO2013154798A1 (en) | 2012-04-09 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| DK2920304T3 (da) | 2012-11-15 | 2019-05-13 | Roche Innovation Ct Copenhagen As | Oligonukleotidkonjugater |
| MY178929A (en) | 2013-05-01 | 2020-10-23 | Glaxo Group Ltd | Compositions and methods for modulating hbv and ttr expression |
| EP3591054A1 (en) | 2013-06-27 | 2020-01-08 | Roche Innovation Center Copenhagen A/S | Antisense oligomers and conjugates targeting pcsk9 |
| US10358643B2 (en) | 2014-01-30 | 2019-07-23 | Hoffmann-La Roche, Inc. | Poly oligomer compound with biocleavable conjugates |
| US10781445B2 (en) | 2015-03-11 | 2020-09-22 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Decoy oligonucleotides for the treatment of diseases |
| EP3394258B1 (en) | 2015-10-22 | 2021-09-22 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| CN116157522A (zh) * | 2020-08-21 | 2023-05-23 | 豪夫迈·罗氏有限公司 | A1cf抑制剂用于治疗乙型肝炎病毒感染的用途 |
-
2023
- 2023-02-17 US US18/170,685 patent/US20240167040A1/en active Pending
- 2023-02-17 AR ARP230100385A patent/AR128558A1/es unknown
- 2023-02-20 EP EP23705297.2A patent/EP4482959A1/en active Pending
- 2023-02-20 TW TW112106127A patent/TW202346587A/zh unknown
- 2023-02-20 WO PCT/EP2023/054174 patent/WO2023156652A1/en active Application Filing
Also Published As
| Publication number | Publication date |
|---|---|
| TW202346587A (zh) | 2023-12-01 |
| EP4482959A1 (en) | 2025-01-01 |
| WO2023156652A1 (en) | 2023-08-24 |
| US20240167040A1 (en) | 2024-05-23 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6471901B2 (ja) | 特定遺伝子の発現を抑制するための方法および薬剤 | |
| AR128558A1 (es) | Oligonucleótido antisentido | |
| CN104995300B (zh) | Rna活性和血管通透性的调节 | |
| PE20230613A1 (es) | Oligonucleotidos mejorados para modular la expresion de fubp1 | |
| KR101783444B1 (ko) | miR-33-5p 를 이용한 뇌신경세포 보호 물질 스크리닝 방법 | |
| WO2024110191A1 (en) | Codon-optimized oligonucleotide for induction of elastin de-novo synthesis in mammals | |
| KR20240024194A (ko) | 생성물 및 조성물 | |
| AR129312A1 (es) | Composiciones y métodos para inhibir la expresión de snca | |
| AU2008202208C1 (en) | Method and medicament for inhibiting the expression of a defined gene | |
| CN117957321A (zh) | 产品和组合物 | |
| AU2017276342A1 (en) | Method and medicament for inhibiting the expression of a defined gene | |
| MX2007003667A (es) | Metodo para tratar una emfermedad inflamatoria mediante acido ribonucleico bicatenario |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |