CA3132388A1 - Compositions and methods for treating huntington's disease - Google Patents
Compositions and methods for treating huntington's disease Download PDFInfo
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- CA3132388A1 CA3132388A1 CA3132388A CA3132388A CA3132388A1 CA 3132388 A1 CA3132388 A1 CA 3132388A1 CA 3132388 A CA3132388 A CA 3132388A CA 3132388 A CA3132388 A CA 3132388A CA 3132388 A1 CA3132388 A1 CA 3132388A1
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Classifications
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- 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
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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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- 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
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- 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.
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
- C12N2310/3519—Fusion with another nucleic acid
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Psychology (AREA)
- Neurology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
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| US201962815647P | 2019-03-08 | 2019-03-08 | |
| US62/815,647 | 2019-03-08 | ||
| PCT/US2020/021652 WO2020185651A2 (en) | 2019-03-08 | 2020-03-09 | Compositions and methods for treating huntington's disease |
Publications (1)
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| CA3132388A1 true CA3132388A1 (en) | 2020-09-17 |
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| EP (1) | EP3935167A2 (zh) |
| JP (1) | JP2022524383A (zh) |
| KR (1) | KR20220002882A (zh) |
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| AU (1) | AU2020238872A1 (zh) |
| BR (1) | BR112021017635A2 (zh) |
| CA (1) | CA3132388A1 (zh) |
| IL (1) | IL286083A (zh) |
| SG (1) | SG11202109180RA (zh) |
| WO (1) | WO2020185651A2 (zh) |
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| WO2024035952A1 (en) * | 2022-08-12 | 2024-02-15 | Remix Therapeutics Inc. | Methods and compositions for modulating splicing at alternative splice sites |
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| US6900016B1 (en) * | 2000-09-08 | 2005-05-31 | Applera Corporation | Polymorphisms in known genes associated with inflammatory autoimmune disease, methods of detection and uses thereof |
| US7214786B2 (en) * | 2000-12-14 | 2007-05-08 | Kovalic David K | Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement |
| US20080274989A1 (en) * | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US9441221B2 (en) * | 2007-03-30 | 2016-09-13 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
| CA2713458A1 (en) * | 2008-01-29 | 2009-08-06 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions capable of causing post-transcriptional silencing of gene expression in a synergic manner |
| AU2015252117A1 (en) * | 2008-04-11 | 2015-11-26 | Tekmira Pharmaceuticals Corporation | Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components |
| EP3277814B1 (en) * | 2015-04-03 | 2020-06-03 | University of Massachusetts | Oligonucleotide compounds for targeting huntingtin mrna |
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2020
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- 2020-03-09 SG SG11202109180RA patent/SG11202109180RA/en unknown
- 2020-03-09 EP EP20770560.9A patent/EP3935167A2/en not_active Withdrawn
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- 2020-03-09 AU AU2020238872A patent/AU2020238872A1/en not_active Abandoned
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| IL286083A (en) | 2021-10-31 |
| CN113924364A (zh) | 2022-01-11 |
| EP3935167A2 (en) | 2022-01-12 |
| JP2022524383A (ja) | 2022-05-02 |
| WO2020185651A3 (en) | 2020-10-29 |
| WO2020185651A2 (en) | 2020-09-17 |
| AU2020238872A1 (en) | 2021-09-16 |
| KR20220002882A (ko) | 2022-01-07 |
| SG11202109180RA (en) | 2021-09-29 |
| BR112021017635A2 (pt) | 2021-11-09 |
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