IL295625A - Methods for reducing htt expression - Google Patents
Methods for reducing htt expressionInfo
- Publication number
- IL295625A IL295625A IL295625A IL29562522A IL295625A IL 295625 A IL295625 A IL 295625A IL 295625 A IL295625 A IL 295625A IL 29562522 A IL29562522 A IL 29562522A IL 295625 A IL295625 A IL 295625A
- Authority
- IL
- Israel
- Prior art keywords
- less
- weeks
- administering
- once
- modified oligonucleotide
- Prior art date
Links
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Classifications
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- 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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- 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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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/315—Phosphorothioates
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- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
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- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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- Neurology (AREA)
- Neurosurgery (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Steroid Compounds (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
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| US202062980010P | 2020-02-21 | 2020-02-21 | |
| PCT/US2021/018681 WO2021168183A1 (en) | 2020-02-21 | 2021-02-19 | Methods for reducing htt expression |
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| IL295625A IL295625A (en) | 2020-02-21 | 2021-02-19 | Methods for reducing htt expression |
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| CN (1) | CN115279903A (enExample) |
| AU (1) | AU2021224729A1 (enExample) |
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| WO2025265013A2 (en) | 2024-06-20 | 2025-12-26 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use |
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| EP1991677A2 (en) * | 2006-01-26 | 2008-11-19 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to huntingtin |
| NZ714887A (en) * | 2009-09-11 | 2019-07-26 | Ionis Pharmaceuticals Inc | Modulation of huntingtin expression |
| LT3052628T (lt) * | 2013-10-04 | 2020-09-10 | Alnylam Pharmaceuticals, Inc. | Kompozicijos ir būdai alas1 geno raiškai nuslopinti |
| EP3797780B1 (en) * | 2014-04-17 | 2022-09-14 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| IL320434A (en) * | 2015-07-22 | 2025-06-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods |
| US11268093B2 (en) * | 2017-01-03 | 2022-03-08 | Rula Zain-Luqman | Therapeutic method for huntington's disease |
| CN114286710A (zh) * | 2019-06-28 | 2022-04-05 | 宾州研究基金会 | 治疗亨廷顿病的方法和材料 |
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- 2021-02-19 WO PCT/US2021/018681 patent/WO2021168183A1/en not_active Ceased
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| CA3172336A1 (en) | 2021-08-26 |
| BR112022016370A2 (pt) | 2022-10-04 |
| AU2021224729A1 (en) | 2022-09-08 |
| EP4107269A1 (en) | 2022-12-28 |
| MX2022010204A (es) | 2022-09-19 |
| JP2023515466A (ja) | 2023-04-13 |
| US20230108340A1 (en) | 2023-04-06 |
| KR20220144837A (ko) | 2022-10-27 |
| EP4107269A4 (en) | 2024-11-20 |
| CN115279903A (zh) | 2022-11-01 |
| ZA202209112B (en) | 2024-10-30 |
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