DK3999643T5 - Fremgangsmåder til behandling eller forebyggelse af spinal muskelatrofi - Google Patents
Fremgangsmåder til behandling eller forebyggelse af spinal muskelatrofi Download PDFInfo
- Publication number
- DK3999643T5 DK3999643T5 DK20753556.8T DK20753556T DK3999643T5 DK 3999643 T5 DK3999643 T5 DK 3999643T5 DK 20753556 T DK20753556 T DK 20753556T DK 3999643 T5 DK3999643 T5 DK 3999643T5
- Authority
- DK
- Denmark
- Prior art keywords
- treating
- methods
- muscular atrophy
- spinal muscular
- preventing spinal
- Prior art date
Links
- 208000002320 spinal muscular atrophy Diseases 0.000 title 1
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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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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
-
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
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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
- 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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- 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
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- 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
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
- G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
-
- 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
- C12N2310/315—Phosphorothioates
-
- 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
-
- 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
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Dermatology (AREA)
- Pathology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962876360P | 2019-07-19 | 2019-07-19 | |
| PCT/US2020/042312 WO2021016032A1 (en) | 2019-07-19 | 2020-07-16 | Methods of treating or preventing spinal muscular atrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DK3999643T3 DK3999643T3 (da) | 2023-12-04 |
| DK3999643T5 true DK3999643T5 (da) | 2024-08-05 |
Family
ID=71995097
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK20753556.8T DK3999643T5 (da) | 2019-07-19 | 2020-07-16 | Fremgangsmåder til behandling eller forebyggelse af spinal muskelatrofi |
Country Status (31)
| Country | Link |
|---|---|
| US (1) | US20220325282A1 (da) |
| EP (2) | EP4335503A3 (da) |
| JP (2) | JP7279253B2 (da) |
| KR (1) | KR20220042113A (da) |
| CN (1) | CN114302727A (da) |
| AR (1) | AR119430A1 (da) |
| AU (1) | AU2020316997A1 (da) |
| BR (1) | BR112022000870A2 (da) |
| CA (1) | CA3149906A1 (da) |
| CL (1) | CL2021003602A1 (da) |
| CO (1) | CO2022001354A2 (da) |
| CR (1) | CR20220071A (da) |
| DK (1) | DK3999643T5 (da) |
| ES (1) | ES2966791T3 (da) |
| FI (1) | FI3999643T3 (da) |
| HR (1) | HRP20231515T1 (da) |
| HU (1) | HUE064071T2 (da) |
| IL (1) | IL289745A (da) |
| JO (1) | JOP20220007A1 (da) |
| LT (1) | LT3999643T (da) |
| MA (1) | MA62935B1 (da) |
| MD (1) | MD3999643T2 (da) |
| MX (1) | MX2022000441A (da) |
| PE (1) | PE20220941A1 (da) |
| PL (1) | PL3999643T3 (da) |
| PT (1) | PT3999643T (da) |
| RS (1) | RS64879B1 (da) |
| SI (1) | SI3999643T1 (da) |
| TW (1) | TWI841762B (da) |
| UY (1) | UY38790A (da) |
| WO (1) | WO2021016032A1 (da) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2024138018A1 (en) * | 2022-12-23 | 2024-06-27 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Treatment of gemin5-mediated disorders |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL3308788T3 (pl) | 2005-06-23 | 2019-05-31 | Biogen Ma Inc | Kompozycje i sposoby modulacji splicingu smn2 |
| WO2008157753A1 (en) * | 2007-06-21 | 2008-12-24 | Sma Foundation | Methods of treatment for spinal muscular atrophy |
| KR20120093138A (ko) * | 2009-06-17 | 2012-08-22 | 콜드스프링하버러보러토리 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| WO2011032109A1 (en) | 2009-09-11 | 2011-03-17 | Sma Foundation | Biomarkers for spinal muscular atrophy |
| EP2714037B1 (en) * | 2011-05-25 | 2016-07-13 | Université Paris Descartes | Erk inhibitors for use in treating spinal muscular atrophy |
| US9926559B2 (en) * | 2013-01-09 | 2018-03-27 | Biogen Ma 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 |
| SG11201900238UA (en) * | 2016-07-15 | 2019-02-27 | Ionis Pharmaceuticals Inc | Compounds and methods for modulation of smn2 |
| TW201927241A (zh) | 2017-12-21 | 2019-07-16 | 瑞士商赫孚孟拉羅股份公司 | 用於肌肉失能之數位生物標記 |
| KR20200112906A (ko) | 2018-01-25 | 2020-10-05 | 바이오젠 엠에이 인코포레이티드 | 척추 근위축증의 치료 방법 |
| IT201800004359A1 (it) | 2018-04-10 | 2019-10-10 | IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE | |
| JP2021534154A (ja) * | 2018-08-15 | 2021-12-09 | バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. | 脊髄性筋萎縮症のための併用療法 |
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2020
- 2020-07-16 DK DK20753556.8T patent/DK3999643T5/da active
- 2020-07-16 UY UY0001038790A patent/UY38790A/es unknown
- 2020-07-16 JO JOP/2022/0007A patent/JOP20220007A1/ar unknown
- 2020-07-16 EP EP23210526.2A patent/EP4335503A3/en active Pending
- 2020-07-16 SI SI202030323T patent/SI3999643T1/sl unknown
- 2020-07-16 MX MX2022000441A patent/MX2022000441A/es unknown
- 2020-07-16 PL PL20753556.8T patent/PL3999643T3/pl unknown
- 2020-07-16 PE PE2021002322A patent/PE20220941A1/es unknown
- 2020-07-16 EP EP20753556.8A patent/EP3999643B1/en active Active
- 2020-07-16 JP JP2022500671A patent/JP7279253B2/ja active Active
- 2020-07-16 WO PCT/US2020/042312 patent/WO2021016032A1/en active Application Filing
- 2020-07-16 US US17/626,324 patent/US20220325282A1/en active Pending
- 2020-07-16 HR HRP20231515TT patent/HRP20231515T1/hr unknown
- 2020-07-16 PT PT207535568T patent/PT3999643T/pt unknown
- 2020-07-16 MA MA62935A patent/MA62935B1/fr unknown
- 2020-07-16 CA CA3149906A patent/CA3149906A1/en active Pending
- 2020-07-16 AU AU2020316997A patent/AU2020316997A1/en active Pending
- 2020-07-16 KR KR1020227001151A patent/KR20220042113A/ko unknown
- 2020-07-16 ES ES20753556T patent/ES2966791T3/es active Active
- 2020-07-16 RS RS20231128A patent/RS64879B1/sr unknown
- 2020-07-16 BR BR112022000870A patent/BR112022000870A2/pt unknown
- 2020-07-16 MD MDE20220574T patent/MD3999643T2/ro unknown
- 2020-07-16 HU HUE20753556A patent/HUE064071T2/hu unknown
- 2020-07-16 CN CN202080049461.1A patent/CN114302727A/zh active Pending
- 2020-07-16 FI FIEP20753556.8T patent/FI3999643T3/fi active
- 2020-07-16 LT LTEPPCT/US2020/042312T patent/LT3999643T/lt unknown
- 2020-07-16 CR CR20220071A patent/CR20220071A/es unknown
- 2020-07-17 AR ARP200102019A patent/AR119430A1/es unknown
- 2020-07-17 TW TW109124224A patent/TWI841762B/zh active
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2021
- 2021-12-31 CL CL2021003602A patent/CL2021003602A1/es unknown
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2022
- 2022-01-10 IL IL289745A patent/IL289745A/en unknown
- 2022-02-10 CO CONC2022/0001354A patent/CO2022001354A2/es unknown
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2023
- 2023-05-10 JP JP2023077805A patent/JP2023100911A/ja active Pending
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