WO2015017675A3 - Methods and compounds useful in conditions related to repeat expansion - Google Patents
Methods and compounds useful in conditions related to repeat expansion Download PDFInfo
- Publication number
- WO2015017675A3 WO2015017675A3 PCT/US2014/049197 US2014049197W WO2015017675A3 WO 2015017675 A3 WO2015017675 A3 WO 2015017675A3 US 2014049197 W US2014049197 W US 2014049197W WO 2015017675 A3 WO2015017675 A3 WO 2015017675A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- methods
- compounds useful
- conditions related
- repeat expansion
- compounds
- 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.)
- Ceased
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
-
- 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
- C12N2310/32—Chemical structure of the sugar
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Abstract
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14832900.6A EP3027617B1 (en) | 2013-07-31 | 2014-07-31 | Methods and compounds useful in conditions related to repeat expansion |
| US14/908,429 US10435430B2 (en) | 2013-07-31 | 2014-07-31 | Methods and compounds useful in conditions related to repeat expansion |
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361860823P | 2013-07-31 | 2013-07-31 | |
| US61/860,823 | 2013-07-31 | ||
| US201462019820P | 2014-07-01 | 2014-07-01 | |
| US62/019,820 | 2014-07-01 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| WO2015017675A2 WO2015017675A2 (en) | 2015-02-05 |
| WO2015017675A3 true WO2015017675A3 (en) | 2015-04-02 |
Family
ID=52432576
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/US2014/049197 Ceased WO2015017675A2 (en) | 2013-07-31 | 2014-07-31 | Methods and compounds useful in conditions related to repeat expansion |
Country Status (3)
| Country | Link |
|---|---|
| US (1) | US10435430B2 (en) |
| EP (1) | EP3027617B1 (en) |
| WO (1) | WO2015017675A2 (en) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102282155B (en) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | The synthetic method of the nucleic acid of phosphorus atoms modification |
| RU2612521C2 (en) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Novel prodrugs of nucleic acids and their application methods |
| JP5868324B2 (en) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN103796657B (en) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | Methods of Synthesizing Functionalized Nucleic Acids |
| PL2872485T3 (en) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| BR112016011770B1 (en) * | 2013-11-27 | 2022-10-25 | Sigma-Aldrich Co. Llc | METHOD AND KIT FOR MICRORNA ISOLATION FROM A BIOLOGICAL SAMPLE |
| BR112016016400A2 (en) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | COMPOSITIONS OF CHIRALLY CONTROLLED OLIGONUCLEOTIDES, THEIR USE, THEIR PHARMACEUTICAL COMPOSITION, AND METHODS |
| EP4285912A3 (en) | 2015-09-25 | 2024-07-10 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating ataxin 3 expression |
| WO2018051762A1 (en) * | 2016-09-14 | 2018-03-22 | レナセラピューティクス株式会社 | Antisense oligonucleotide with reduced side effects |
| JOP20190104A1 (en) * | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | Compounds and methods for reducing atxn3 expression |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| EP4663758A3 (en) | 2018-05-09 | 2026-04-29 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing atxn3 expression |
| EP3965777A4 (en) * | 2019-05-06 | 2024-08-07 | The Children's Hospital of Philadelphia | MICRORNA SITE-BLOCKING OLIGONUCLEOTIDES FOR THE TREATMENT OF EPILEPTIC ENCEPHALOPATHY AND NEURODEVELOPMENTAL DISORDERS |
| CN113950529A (en) | 2019-06-06 | 2022-01-18 | 豪夫迈·罗氏有限公司 | Antisense oligonucleotides targeting ATXN3 |
| KR20220133925A (en) | 2020-01-31 | 2022-10-05 | 가부시키가이샤산와카가쿠켄큐쇼 | Antisense oligonucleotides of ATN1 |
| AR124229A1 (en) | 2020-12-03 | 2023-03-01 | Hoffmann La Roche | ANTISENSE OLIGONUCLOTIDES ACTING ON ATXN3 |
| US20230060373A1 (en) | 2020-12-03 | 2023-03-02 | Hoffmann-La Roche Inc. | Antisense Oligonucleotides Targeting ATXN3 |
| JP2024536132A (en) | 2021-09-29 | 2024-10-04 | エフ. ホフマン-ラ ロシュ アーゲー | RNA editing method |
| EP4594498A2 (en) * | 2022-09-28 | 2025-08-06 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of spinocerebellar ataxias |
| WO2025252669A1 (en) | 2024-06-03 | 2025-12-11 | Evotec International Gmbh | Modified oligonucleotides for reducing atxn3 expression |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20090253583A1 (en) * | 2004-09-27 | 2009-10-08 | Med Biogene Inc. | Hematological Cancer Profiling System |
| WO2013033223A1 (en) * | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| US20130059902A1 (en) * | 2010-02-08 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| WO2013096679A1 (en) * | 2011-12-22 | 2013-06-27 | Alios Biopharma, Inc. | Substituted nucleosides, nucleotides and analogs thereof |
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| FR2575751B1 (en) | 1985-01-08 | 1987-04-03 | Pasteur Institut | NOVEL ADENOSINE DERIVATIVE NUCLEOSIDES, THEIR PREPARATION AND THEIR BIOLOGICAL APPLICATIONS |
| EP0366685B1 (en) | 1987-06-24 | 1994-10-19 | Howard Florey Institute Of Experimental Physiology And Medicine | 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 |
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-
2014
- 2014-07-31 US US14/908,429 patent/US10435430B2/en active Active
- 2014-07-31 WO PCT/US2014/049197 patent/WO2015017675A2/en not_active Ceased
- 2014-07-31 EP EP14832900.6A patent/EP3027617B1/en active Active
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20090253583A1 (en) * | 2004-09-27 | 2009-10-08 | Med Biogene Inc. | Hematological Cancer Profiling System |
| US20130059902A1 (en) * | 2010-02-08 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| WO2013033223A1 (en) * | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| WO2013096679A1 (en) * | 2011-12-22 | 2013-06-27 | Alios Biopharma, Inc. | Substituted nucleosides, nucleotides and analogs thereof |
Also Published As
| Publication number | Publication date |
|---|---|
| US10435430B2 (en) | 2019-10-08 |
| EP3027617C0 (en) | 2026-02-18 |
| EP3027617B1 (en) | 2026-02-18 |
| EP3027617A4 (en) | 2017-04-12 |
| EP3027617A2 (en) | 2016-06-08 |
| US20160159846A1 (en) | 2016-06-09 |
| WO2015017675A2 (en) | 2015-02-05 |
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