NZ724100A - Compositions for modulating sod-1 expression - Google Patents
Compositions for modulating sod-1 expressionInfo
- Publication number
- NZ724100A NZ724100A NZ724100A NZ72410015A NZ724100A NZ 724100 A NZ724100 A NZ 724100A NZ 724100 A NZ724100 A NZ 724100A NZ 72410015 A NZ72410015 A NZ 72410015A NZ 724100 A NZ724100 A NZ 724100A
- Authority
- NZ
- New Zealand
- Prior art keywords
- pharmaceutically acceptable
- acceptable salt
- antisense compound
- sod
- salt
- Prior art date
Links
Classifications
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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
- 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
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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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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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
-
- 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
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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
- C12N2310/31—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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
- C12N2310/33—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 ===---===
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461973803P | 2014-04-01 | 2014-04-01 | |
| PCT/US2015/023934 WO2015153800A2 (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ724100A true NZ724100A (en) | 2022-12-23 |
Family
ID=54241435
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ724100A NZ724100A (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
Country Status (31)
| Country | Link |
|---|---|
| US (3) | US10385341B2 (enExample) |
| EP (3) | EP3126499B1 (enExample) |
| JP (6) | JP6622214B2 (enExample) |
| KR (6) | KR20240111010A (enExample) |
| CN (2) | CN111440798A (enExample) |
| AU (4) | AU2015240761B2 (enExample) |
| BR (1) | BR112016021848B1 (enExample) |
| CA (2) | CA2942394A1 (enExample) |
| CL (3) | CL2016002509A1 (enExample) |
| CY (3) | CY1123288T1 (enExample) |
| DK (2) | DK3757214T3 (enExample) |
| ES (2) | ES2818236T3 (enExample) |
| FI (1) | FIC20240035I1 (enExample) |
| FR (1) | FR24C1039I2 (enExample) |
| HR (2) | HRP20220798T1 (enExample) |
| HU (3) | HUE059109T2 (enExample) |
| IL (2) | IL247714B (enExample) |
| LT (3) | LT3757214T (enExample) |
| LU (1) | LUC00359I2 (enExample) |
| MX (2) | MX373908B (enExample) |
| MY (2) | MY192634A (enExample) |
| NL (1) | NL301293I2 (enExample) |
| NZ (1) | NZ724100A (enExample) |
| PL (2) | PL3757214T3 (enExample) |
| PT (2) | PT3126499T (enExample) |
| RS (2) | RS60707B1 (enExample) |
| RU (1) | RU2704619C2 (enExample) |
| SG (3) | SG11201608109TA (enExample) |
| SI (2) | SI3757214T1 (enExample) |
| SM (2) | SMT202200479T1 (enExample) |
| WO (1) | WO2015153800A2 (enExample) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MY192634A (en) * | 2014-04-01 | 2022-08-29 | Biogen Ma Inc | Compositions for modulating sod-1 expression |
| US10590420B2 (en) * | 2014-07-31 | 2020-03-17 | Association Institut De Myologie | Treatment of amyotrophic lateral sclerosis |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| JP7075359B2 (ja) * | 2016-06-14 | 2022-05-25 | バイオジェン・エムエイ・インコーポレイテッド | オリゴヌクレオチドの精製のための疎水性相互作用クロマトグラフィー |
| WO2017223258A1 (en) | 2016-06-24 | 2017-12-28 | Biogen Ma Inc. | Synthesis of thiolated oligonucleotides without a capping step |
| SG11201909870SA (en) * | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP3631470B1 (en) * | 2017-05-26 | 2025-12-31 | University of Miami | DETERMINATION OF THE ONSET OF AMYOTROPHIC LATERAL SCLEROSIS |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| WO2019147960A1 (en) | 2018-01-25 | 2019-08-01 | Biogen Ma Inc. | Methods of treating spinal muscular atrophy |
| TWI833770B (zh) | 2018-06-27 | 2024-03-01 | 美商Ionis製藥公司 | 用於減少 lrrk2 表現之化合物及方法 |
| US12281305B2 (en) * | 2018-11-21 | 2025-04-22 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing prion expression |
| KR20210102294A (ko) * | 2018-12-06 | 2021-08-19 | 바이오젠 엠에이 인코포레이티드 | 근위축성 측삭 경화증에서 치료요법적 중재를 안내하기 위한 신경필라멘트 단백질 |
| TWI906204B (zh) * | 2018-12-14 | 2025-12-01 | 美商百健Ma公司 | 用於治療及預防肌肉萎縮性脊髓側索硬化症之組合物及方法 |
| AU2020248392A1 (en) * | 2019-03-26 | 2021-11-11 | Rutgers, The State University Of New Jersey | Compositions and methods for treating neurodegenerative disorders |
| KR102956323B1 (ko) * | 2019-05-01 | 2026-04-24 | 페론 인스티튜트 포 뉴롤로지컬 앤드 트랜스레이셔널 사이언스 리미티드 | Sod1 관련 질환의 치료 |
| WO2020227395A2 (en) * | 2019-05-06 | 2020-11-12 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| MA55891A (fr) | 2019-05-08 | 2022-03-16 | Ajinomoto Kk | Synthèses en phase liquide convergente d'oligonucléotides |
| SI3976791T1 (sl) * | 2019-05-28 | 2025-06-30 | Ionis Pharmaceuticals, Inc. | Spojine in postopki za zmanjševanje izražanja fus |
| US20230098111A1 (en) * | 2020-01-28 | 2023-03-30 | University Of Southern California | Compositions and methods to treat neurological diseases |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| BR112023019877A2 (pt) | 2021-03-31 | 2023-11-07 | Biogen Ma Inc | Tratamento de esclerose lateral amiotrófica |
| JP2024513403A (ja) | 2021-04-01 | 2024-03-25 | バイオジェン・エムエイ・インコーポレイテッド | 中枢神経系への核酸送達 |
| US20230193281A1 (en) * | 2021-11-08 | 2023-06-22 | University Of Massachusetts | Oligonucleotides for sod1 modulation |
| CN114369130B (zh) * | 2021-12-28 | 2023-10-03 | 杭州天龙药业有限公司 | 修饰的硫代寡核苷酸及其应用 |
| WO2023245060A2 (en) * | 2022-06-15 | 2023-12-21 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of superoxide dismutase 1 (sod1), compositions thereof, and methods of use |
| KR20240038469A (ko) | 2022-09-16 | 2024-03-25 | 김정훈 | 사용자 콘텐츠 기반 홍보 서비스 제공 장치 및 방법 |
| CN120202298A (zh) * | 2023-08-29 | 2025-06-24 | 石药集团中奇制药技术(石家庄)有限公司 | 一种抑制sod1基因表达的dsRNA分子及其应用 |
| WO2025126158A2 (en) * | 2023-12-15 | 2025-06-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) targeting sod1 |
| WO2026062390A1 (en) | 2024-09-20 | 2026-03-26 | Crucible Therapeutics Ltd | Srsf1 antagonist |
| KR102893298B1 (ko) * | 2024-12-19 | 2025-12-04 | 주식회사 에이비파트너스 | 항산화 효능을 가지는 리포좀 기반의 mRNA 전달 시스템 및 이의 용도 |
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