DK3976791T3 - Forbindelser og fremgangsmåder til at reducere fus-ekspression - Google Patents
Forbindelser og fremgangsmåder til at reducere fus-ekspression Download PDFInfo
- Publication number
- DK3976791T3 DK3976791T3 DK20815459.1T DK20815459T DK3976791T3 DK 3976791 T3 DK3976791 T3 DK 3976791T3 DK 20815459 T DK20815459 T DK 20815459T DK 3976791 T3 DK3976791 T3 DK 3976791T3
- Authority
- DK
- Denmark
- Prior art keywords
- compounds
- methods
- reducing
- fus expression
- expression
- Prior art date
Links
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
-
- 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
- C12N2310/31—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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/341—Gapmers, i.e. of the type ===---===
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962853616P | 2019-05-28 | 2019-05-28 | |
| PCT/US2020/034907 WO2020243292A1 (en) | 2019-05-28 | 2020-05-28 | Compounds and methods for reducing fus expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3976791T3 true DK3976791T3 (da) | 2025-05-19 |
Family
ID=73553907
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK20815459.1T DK3976791T3 (da) | 2019-05-28 | 2020-05-28 | Forbindelser og fremgangsmåder til at reducere fus-ekspression |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220243203A1 (da) |
| EP (2) | EP3976791B1 (da) |
| DK (1) | DK3976791T3 (da) |
| ES (1) | ES3033104T3 (da) |
| FI (1) | FI3976791T3 (da) |
| HR (1) | HRP20250468T1 (da) |
| HU (1) | HUE071962T2 (da) |
| LT (1) | LT3976791T (da) |
| PL (1) | PL3976791T3 (da) |
| PT (1) | PT3976791T (da) |
| SI (1) | SI3976791T1 (da) |
| TW (1) | TW202111123A (da) |
| WO (1) | WO2020243292A1 (da) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4473111A1 (en) * | 2022-02-02 | 2024-12-11 | Perron Institute for Neurological and Translational Science Limited | Compositions and methods for treating diseases associated with pathogenic fus variants |
| AU2023214509A1 (en) * | 2022-02-02 | 2024-08-15 | Perron Institute for Neurological and Translational Science Limited | Compositions and methods for treating fus associated diseases |
| EP4519441A2 (en) * | 2022-05-06 | 2025-03-12 | Ionis Pharmaceuticals, Inc. | Rnai agents for modulating snca |
Family Cites Families (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| WO1988010264A1 (en) | 1987-06-24 | 1988-12-29 | Howard Florey Institute Of Experimental Physiology | Nucleoside derivatives |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US6005087A (en) | 1995-06-06 | 1999-12-21 | Isis Pharmaceuticals, Inc. | 2'-modified oligonucleotides |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5859221A (en) | 1990-01-11 | 1999-01-12 | Isis Pharmaceuticals, Inc. | 2'-modified oligonucleotides |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5457191A (en) | 1990-01-11 | 1995-10-10 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| DE69126530T2 (de) | 1990-07-27 | 1998-02-05 | Isis Pharmaceutical, Inc., Carlsbad, Calif. | Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| US5948903A (en) | 1991-01-11 | 1999-09-07 | Isis Pharmaceuticals, Inc. | Synthesis of 3-deazapurines |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| TW393513B (en) | 1991-11-26 | 2000-06-11 | Isis Pharmaceuticals Inc | Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines |
| ATE226093T1 (de) | 1991-11-26 | 2002-11-15 | Isis Pharmaceuticals Inc | Gesteigerte bildung von triple- und doppelhelices aus oligomeren mit modifizierten pyrimidinen |
| US5434257A (en) | 1992-06-01 | 1995-07-18 | Gilead Sciences, Inc. | Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US7875733B2 (en) | 2003-09-18 | 2011-01-25 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| USRE44779E1 (en) | 1997-03-07 | 2014-02-25 | Santaris Pharma A/S | Bicyclonucleoside and oligonucleotide analogues |
| 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 |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
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| WO1999014226A2 (en) | 1997-09-12 | 1999-03-25 | Exiqon A/S | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues |
| HK1048322A1 (zh) | 1999-05-04 | 2003-03-28 | 埃克西库恩公司 | L-核糖-lna类似物 |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| US20030175906A1 (en) | 2001-07-03 | 2003-09-18 | Muthiah Manoharan | Nuclease resistant chimeric oligonucleotides |
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| WO2004106356A1 (en) | 2003-05-27 | 2004-12-09 | Syddansk Universitet | Functionalized nucleotide derivatives |
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| DK2314594T3 (da) | 2006-01-27 | 2014-10-27 | Isis Pharmaceuticals Inc | 6-modificerede bicykliske nukleinsyreanaloger |
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| ES2633565T3 (es) * | 2010-11-12 | 2017-09-22 | The General Hospital Corporation | ARN no codificantes asociados a polycomb |
| EP2655621B1 (en) * | 2010-12-20 | 2018-05-23 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| EP2943225A4 (en) * | 2013-01-09 | 2016-07-13 | Ionis Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT |
| EA031393B1 (ru) | 2013-05-01 | 2018-12-28 | Глэксо Груп Лимитед | Композиции и способы модулирования экспрессии hbv и ttr |
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| KR102285629B1 (ko) * | 2014-04-01 | 2021-08-06 | 바이오젠 엠에이 인코포레이티드 | Sod-1 발현을 조절하기 위한 조성물 |
| WO2015191780A2 (en) * | 2014-06-10 | 2015-12-17 | The General Hospital Corporation | Ccctc-binding factor (ctcf) rna interactome |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
-
2020
- 2020-05-28 HR HRP20250468TT patent/HRP20250468T1/hr unknown
- 2020-05-28 WO PCT/US2020/034907 patent/WO2020243292A1/en not_active Ceased
- 2020-05-28 DK DK20815459.1T patent/DK3976791T3/da active
- 2020-05-28 PL PL20815459.1T patent/PL3976791T3/pl unknown
- 2020-05-28 US US17/613,183 patent/US20220243203A1/en active Pending
- 2020-05-28 HU HUE20815459A patent/HUE071962T2/hu unknown
- 2020-05-28 PT PT208154591T patent/PT3976791T/pt unknown
- 2020-05-28 EP EP20815459.1A patent/EP3976791B1/en active Active
- 2020-05-28 SI SI202030612T patent/SI3976791T1/sl unknown
- 2020-05-28 FI FIEP20815459.1T patent/FI3976791T3/fi active
- 2020-05-28 ES ES20815459T patent/ES3033104T3/es active Active
- 2020-05-28 LT LTEPPCT/US2020/034907T patent/LT3976791T/lt unknown
- 2020-05-28 TW TW109117922A patent/TW202111123A/zh unknown
- 2020-05-28 EP EP25162118.1A patent/EP4556565A2/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| FI3976791T3 (fi) | 2025-06-10 |
| HRP20250468T1 (hr) | 2025-07-18 |
| PT3976791T (pt) | 2025-06-26 |
| PL3976791T3 (pl) | 2025-08-04 |
| EP4556565A2 (en) | 2025-05-21 |
| HUE071962T2 (hu) | 2025-10-28 |
| TW202111123A (zh) | 2021-03-16 |
| WO2020243292A1 (en) | 2020-12-03 |
| SI3976791T1 (sl) | 2025-06-30 |
| US20220243203A1 (en) | 2022-08-04 |
| EP3976791B1 (en) | 2025-04-09 |
| LT3976791T (lt) | 2025-07-25 |
| EP3976791A1 (en) | 2022-04-06 |
| ES3033104T3 (en) | 2025-07-30 |
| EP3976791A4 (en) | 2023-10-11 |
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