CA3250848A1 - Compositions for modulating sod-1 expression - Google Patents
Compositions for modulating sod-1 expressionInfo
- Publication number
- CA3250848A1 CA3250848A1 CA3250848A CA3250848A CA3250848A1 CA 3250848 A1 CA3250848 A1 CA 3250848A1 CA 3250848 A CA3250848 A CA 3250848A CA 3250848 A CA3250848 A CA 3250848A CA 3250848 A1 CA3250848 A1 CA 3250848A1
- Authority
- CA
- Canada
- Prior art keywords
- pat
- math
- sub
- semantics
- annotation
- 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.)
- Pending
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
-
- 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
-
- 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)
-
- 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)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461973803P | 2014-04-01 | 2014-04-01 | |
| US61/973,803 | 2014-04-01 | ||
| CA2942394A CA2942394A1 (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2942394A Division CA2942394A1 (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3250848A1 true CA3250848A1 (en) | 2026-03-02 |
Family
ID=54241435
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3250848A Pending CA3250848A1 (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
| CA2942394A Pending CA2942394A1 (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2942394A Pending CA2942394A1 (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) | KR20210097225A (enExample) |
| CN (2) | CN106459972B (enExample) |
| AU (4) | AU2015240761B2 (enExample) |
| BR (1) | BR112016021848B1 (enExample) |
| CA (2) | CA3250848A1 (enExample) |
| CL (3) | CL2016002509A1 (enExample) |
| CY (3) | CY1123288T1 (enExample) |
| DK (2) | DK3126499T3 (enExample) |
| ES (2) | ES2926869T3 (enExample) |
| FI (1) | FIC20240035I1 (enExample) |
| FR (1) | FR24C1039I2 (enExample) |
| HR (2) | HRP20220798T1 (enExample) |
| HU (3) | HUE050704T2 (enExample) |
| IL (2) | IL247714B (enExample) |
| LT (3) | LT3757214T (enExample) |
| LU (1) | LUC00359I2 (enExample) |
| MX (2) | MX373908B (enExample) |
| MY (2) | MY175665A (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) | SG10201906520RA (enExample) |
| SI (2) | SI3126499T1 (enExample) |
| SM (2) | SMT202000456T1 (enExample) |
| WO (1) | WO2015153800A2 (enExample) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HUE050704T2 (hu) | 2014-04-01 | 2020-12-28 | Biogen Ma Inc | Összetételek a SOD-1 expressziójának modulálására |
| EP3174981B1 (en) * | 2014-07-31 | 2020-03-25 | Association Institut de Myologie | Treatment of amyotrophic lateral sclerosis |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| KR102461234B1 (ko) | 2016-06-14 | 2022-10-28 | 바이오젠 엠에이 인코포레이티드 | 올리고뉴클레오타이드의 정제를 위한 소수성 상호 작용 크로마토그래피 |
| KR102722183B1 (ko) | 2016-06-24 | 2024-10-24 | 바이오젠 엠에이 인코포레이티드 | 캡핑 단계 없이 티올화된 올리고뉴클레오타이드의 합성 |
| EP3618839A4 (en) * | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| WO2018218219A1 (en) * | 2017-05-26 | 2018-11-29 | University Of Miami | Determining onset of amyotrophic lateral sclerosis |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| JP7506606B2 (ja) | 2018-01-25 | 2024-06-26 | バイオジェン・エムエイ・インコーポレイテッド | 脊髄性筋萎縮症を治療する方法 |
| 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 |
| MA54383A (fr) * | 2018-12-06 | 2021-10-13 | Biogen Ma Inc | Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique |
| 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 |
| EP3924739A1 (en) | 2019-02-12 | 2021-12-22 | Biogen MA Inc. | Biomarkers of progressive multifocal leukoencephalopathy |
| JP2022520885A (ja) * | 2019-03-26 | 2022-04-01 | ラトガース,ザ ステート ユニバーシティ オブ ニュー ジャージー | 神経変性疾患を処置するための組成物および方法 |
| US20220315930A1 (en) * | 2019-05-01 | 2022-10-06 | Black Swan Pharmaceuticals, Inc. | Treatment for sod1 associated disease |
| EP3966328A4 (en) * | 2019-05-06 | 2023-10-18 | University Of Massachusetts | Anti-c9orf72 oligonucleotides and related methods |
| CN114206897B (zh) | 2019-05-08 | 2024-09-20 | 比奥根Ma公司 | 寡核苷酸的汇聚液相合成 |
| WO2020243292A1 (en) * | 2019-05-28 | 2020-12-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing fus expression |
| WO2021155067A1 (en) * | 2020-01-28 | 2021-08-05 | 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 |
| KR20230172502A (ko) | 2021-03-31 | 2023-12-22 | 바이오젠 엠에이 인코포레이티드 | 근위축성 측삭경화증의 치료 |
| 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 |
| WO2023081908A2 (en) * | 2021-11-08 | 2023-05-11 | 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 |
| JP2025522433A (ja) | 2022-06-15 | 2025-07-15 | アローヘッド ファーマシューティカルズ インコーポレイテッド | スーパーオキシドジスムターゼ1(SOD1)の発現を阻害するためのRNAi剤、その組成物、及び使用の方法 |
| IL318437A (en) * | 2022-07-21 | 2025-03-01 | Amylyx Pharmaceuticals Inc | Compositions containing oligonucleotides, and methods of using them |
| KR20240038469A (ko) | 2022-09-16 | 2024-03-25 | 김정훈 | 사용자 콘텐츠 기반 홍보 서비스 제공 장치 및 방법 |
| WO2025045130A1 (zh) * | 2023-08-29 | 2025-03-06 | 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 |
| IL328914A (en) * | 2023-12-15 | 2026-08-01 | 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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