JP2022513721A - 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 - Google Patents
筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 Download PDFInfo
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- JP2022513721A JP2022513721A JP2021531983A JP2021531983A JP2022513721A JP 2022513721 A JP2022513721 A JP 2022513721A JP 2021531983 A JP2021531983 A JP 2021531983A JP 2021531983 A JP2021531983 A JP 2021531983A JP 2022513721 A JP2022513721 A JP 2022513721A
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- 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
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- 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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- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/315—Phosphorothioates
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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
- C12N2310/32—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
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- 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
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
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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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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/28—Neurological disorders
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- Bioinformatics & Cheminformatics (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
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- Immunology (AREA)
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- Animal Behavior & Ethology (AREA)
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- Analytical Chemistry (AREA)
- Neurology (AREA)
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- Hematology (AREA)
- Urology & Nephrology (AREA)
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- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024072811A JP2024096258A (ja) | 2018-12-06 | 2024-04-26 | 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862776253P | 2018-12-06 | 2018-12-06 | |
| US62/776,253 | 2018-12-06 | ||
| US201962840431P | 2019-04-30 | 2019-04-30 | |
| US62/840,431 | 2019-04-30 | ||
| US201962842063P | 2019-05-02 | 2019-05-02 | |
| US62/842,063 | 2019-05-02 | ||
| PCT/US2019/064190 WO2020117772A1 (en) | 2018-12-06 | 2019-12-03 | Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024072811A Division JP2024096258A (ja) | 2018-12-06 | 2024-04-26 | 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022513721A true JP2022513721A (ja) | 2022-02-09 |
| JPWO2020117772A5 JPWO2020117772A5 (https=) | 2022-12-08 |
| JP2022513721A5 JP2022513721A5 (https=) | 2022-12-08 |
Family
ID=68966114
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021531983A Withdrawn JP2022513721A (ja) | 2018-12-06 | 2019-12-03 | 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 |
| JP2024072811A Pending JP2024096258A (ja) | 2018-12-06 | 2024-04-26 | 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024072811A Pending JP2024096258A (ja) | 2018-12-06 | 2024-04-26 | 筋萎縮性側索硬化症における治療的介入を導くためのニューロフィラメントタンパク質 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12429487B2 (https=) |
| EP (1) | EP3890752A1 (https=) |
| JP (2) | JP2022513721A (https=) |
| KR (1) | KR20210102294A (https=) |
| CN (1) | CN113365640A (https=) |
| BR (1) | BR112021010660A2 (https=) |
| CA (1) | CA3121749A1 (https=) |
| MA (1) | MA54383A (https=) |
| MX (1) | MX2021006548A (https=) |
| WO (1) | WO2020117772A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202006677XA (en) | 2018-01-25 | 2020-08-28 | Biogen Ma Inc | Methods of treating spinal muscular atrophy |
| MA54383A (fr) | 2018-12-06 | 2021-10-13 | Biogen Ma Inc | Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique |
| CN115004350B (zh) | 2019-06-11 | 2025-10-28 | 库力索法荷兰有限公司 | 利用光学系统特性的调整的分立组件装配体的位置误差补偿 |
| US20230365937A1 (en) * | 2020-10-26 | 2023-11-16 | Hadasit Medical Research Services And Development Ltd. | Mesenchymal stem cells and their culture |
| KR200498472Y1 (ko) | 2024-05-23 | 2024-10-24 | 김민경 | 경고 또는 안내 문구를 교체형으로 구성시킬 수 있는 배수용 그레이팅 |
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| CA2790034A1 (en) | 2001-06-21 | 2003-01-03 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| US20050019915A1 (en) * | 2001-06-21 | 2005-01-27 | Bennett C. Frank | Antisense modulation of superoxide dismutase 1, soluble expression |
| SI2548560T1 (sl) | 2005-06-23 | 2015-12-31 | Isis Pharmaceuticals, Inc. | Sestavki in postopki za moduliranje izrezovanja smn2 |
| WO2008127974A1 (en) | 2007-04-13 | 2008-10-23 | The Trustees Of Columbia University In The City Of New York | Stem cell-based culture system for drug development |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| PT3305302T (pt) | 2009-06-17 | 2018-12-14 | Biogen Ma Inc | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| US8465727B2 (en) | 2009-10-06 | 2013-06-18 | University of Pittsburgh—of the Commonwealth System of Higher Education | Biomarkers for the diagnosis of ALS |
| CA2872190C (en) | 2012-05-09 | 2024-06-25 | Karyopharm Therapeutics, Inc. | Nuclear transport modulators and uses thereof |
| ES2762326T5 (es) | 2012-10-15 | 2023-04-27 | Ionis Pharmaceuticals Inc | Métodos para modular la expresión de C9ORF72 |
| HK1212597A1 (zh) | 2012-10-15 | 2016-06-17 | Isis Pharmaceuticals, Inc. | 用於調節c90rf72表達的組合物 |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| WO2014065341A1 (ja) | 2012-10-24 | 2014-05-01 | 第一三共株式会社 | 筋萎縮性側索硬化症治療剤 |
| EP2943225A4 (en) | 2013-01-09 | 2016-07-13 | Ionis Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR MODULATING SMN2 DISTRIBUTION IN THE BODY OF A PATIENT |
| EP4166667A3 (en) | 2013-10-11 | 2023-08-02 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| US20160230172A1 (en) | 2013-10-14 | 2016-08-11 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| AU2014337506B2 (en) | 2013-10-14 | 2020-10-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating expression of C9ORF72 antisense transcript |
| WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods for modulating ataxin 2 expression |
| CN106103717A (zh) | 2014-03-19 | 2016-11-09 | Ionis制药公司 | 用于调节共济失调蛋白2表达的组合物 |
| RS60707B1 (sr) * | 2014-04-01 | 2020-09-30 | Biogen Ma Inc | Kompozicije za modulaciju ekspresije sod-1 |
| US10024870B2 (en) | 2014-04-02 | 2018-07-17 | Wisconsin Alumni Research Foundation | Therapeutic and diagnostic methods and compositions for neurodegenerative diseases |
| WO2015161170A2 (en) | 2014-04-17 | 2015-10-22 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| KR20230104759A (ko) | 2014-09-30 | 2023-07-10 | 뉴리뮨 홀딩 아게 | 인간-유래의 항-디펩티드 반복체(dpr) 항체 |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016112132A1 (en) | 2015-01-06 | 2016-07-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| RS60230B1 (sr) | 2015-04-16 | 2020-06-30 | Ionis Pharmaceuticals Inc | Kompozicije za moduliranje ekspresije c9orf72 |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| US20190002887A1 (en) | 2015-12-31 | 2019-01-03 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| AU2017248637A1 (en) | 2016-04-13 | 2018-09-27 | Ionis Pharmaceuticals, Inc. | Methods for reducing C9ORF72 expression |
| WO2018218219A1 (en) | 2017-05-26 | 2018-11-29 | University Of Miami | Determining onset of amyotrophic lateral sclerosis |
| TW201920672A (zh) | 2017-08-08 | 2019-06-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| 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 |
| SG11202006677XA (en) | 2018-01-25 | 2020-08-28 | Biogen Ma Inc | Methods of treating spinal muscular atrophy |
| EA201992364A1 (ru) | 2018-04-02 | 2020-03-23 | Кохерус Байосайенсис Инк. | АГОНИСТ PPARγ ДЛЯ ЛЕЧЕНИЯ ПРОГРЕССИРУЮЩЕГО НАДЪЯДЕРНОГО ПАРАЛИЧА |
| BR112020021855A2 (pt) | 2018-04-27 | 2021-02-23 | Biogen Ma Inc. | anticorpo antirrepetição de dipeptídeo (dpr) (poli-ga) derivado de humanos |
| WO2020061355A2 (en) | 2018-09-20 | 2020-03-26 | Biogen Ma Inc. | Serum neurofilament protein for guiding therapeutic intervention in multiple sclerosis |
| 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 |
| MA54949A (fr) | 2019-02-12 | 2021-12-22 | Biogen Ma Inc | Biomarqueurs de la leucoencéphalopathie multifocale progressive |
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2019
- 2019-12-03 MA MA054383A patent/MA54383A/fr unknown
- 2019-12-03 KR KR1020217020744A patent/KR20210102294A/ko active Pending
- 2019-12-03 CN CN201980080522.8A patent/CN113365640A/zh active Pending
- 2019-12-03 BR BR112021010660-0A patent/BR112021010660A2/pt unknown
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- 2019-12-03 EP EP19824179.6A patent/EP3890752A1/en active Pending
- 2019-12-03 MX MX2021006548A patent/MX2021006548A/es unknown
- 2019-12-03 CA CA3121749A patent/CA3121749A1/en active Pending
- 2019-12-03 JP JP2021531983A patent/JP2022513721A/ja not_active Withdrawn
- 2019-12-03 WO PCT/US2019/064190 patent/WO2020117772A1/en not_active Ceased
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2024
- 2024-04-26 JP JP2024072811A patent/JP2024096258A/ja active Pending
Non-Patent Citations (1)
| Title |
|---|
| MCCAMPBELL, A. ET AL., J CLIN INVEST, vol. 128, no. 8, JPN6023044167, August 2018 (2018-08-01), pages 3558 - 3567, ISSN: 0005184008 * |
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| CA3121749A1 (en) | 2020-06-11 |
| JP2024096258A (ja) | 2024-07-12 |
| MA54383A (fr) | 2021-10-13 |
| CN113365640A (zh) | 2021-09-07 |
| BR112021010660A2 (pt) | 2021-08-24 |
| WO2020117772A1 (en) | 2020-06-11 |
| EP3890752A1 (en) | 2021-10-13 |
| US12429487B2 (en) | 2025-09-30 |
| MX2021006548A (es) | 2021-09-21 |
| US20220034907A1 (en) | 2022-02-03 |
| KR20210102294A (ko) | 2021-08-19 |
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