PE20220941A1 - Metodos para tratar o prevenir la atrofia muscular espinal - Google Patents
Metodos para tratar o prevenir la atrofia muscular espinalInfo
- Publication number
- PE20220941A1 PE20220941A1 PE2021002322A PE2021002322A PE20220941A1 PE 20220941 A1 PE20220941 A1 PE 20220941A1 PE 2021002322 A PE2021002322 A PE 2021002322A PE 2021002322 A PE2021002322 A PE 2021002322A PE 20220941 A1 PE20220941 A1 PE 20220941A1
- Authority
- PE
- Peru
- Prior art keywords
- muscular atrophy
- spinal muscular
- treat
- methods
- pharmaceutical composition
- Prior art date
Links
Classifications
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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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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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
-
- 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
- A61K31/7125—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
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- 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
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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
- 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
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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
- 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
-
- 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
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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
- C12N2310/35—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)
- Biochemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Cell Biology (AREA)
- Dermatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Food Science & Technology (AREA)
- Analytical Chemistry (AREA)
Abstract
La presente invencion se refiere a compuestos antisentidos de formula a), b), c) y d), donde R es OCH2CH2OCH3. Tambien se refiere a un kit que comprende una composicion farmaceutica que comprende 28 mg o 50 mg de dicho compuesto o una sal de este. La composicion farmaceutica tambien comprende una solucion salina tamponada con fosfato (PBS) y liquido cefalorraquideo artificial (aCSF). El compuesto antisentido de la presente invencion es administrado en dos dosis de carga de 50 mg, mediante inyeccion intratecal, y dosis de mantenimiento de 28 mg, siendo util en el tratamiento de pacientes con atrofia muscular espinal (AME).
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 (1)
| Publication Number | Publication Date |
|---|---|
| PE20220941A1 true PE20220941A1 (es) | 2022-05-31 |
Family
ID=71995097
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2021002322A PE20220941A1 (es) | 2019-07-19 | 2020-07-16 | Metodos para tratar o prevenir la atrofia muscular espinal |
Country Status (30)
| Country | Link |
|---|---|
| US (1) | US20220325282A1 (es) |
| EP (2) | EP3999643B1 (es) |
| JP (2) | JP7279253B2 (es) |
| KR (1) | KR20220042113A (es) |
| CN (1) | CN114302727A (es) |
| AR (1) | AR119430A1 (es) |
| AU (1) | AU2020316997A1 (es) |
| BR (1) | BR112022000870A2 (es) |
| CA (1) | CA3149906A1 (es) |
| CL (1) | CL2021003602A1 (es) |
| CO (1) | CO2022001354A2 (es) |
| CR (1) | CR20220071A (es) |
| DK (1) | DK3999643T3 (es) |
| ES (1) | ES2966791T3 (es) |
| FI (1) | FI3999643T3 (es) |
| HR (1) | HRP20231515T1 (es) |
| HU (1) | HUE064071T2 (es) |
| IL (1) | IL289745A (es) |
| JO (1) | JOP20220007A1 (es) |
| LT (1) | LT3999643T (es) |
| MA (1) | MA62935B1 (es) |
| MD (1) | MD3999643T2 (es) |
| MX (1) | MX2022000441A (es) |
| PE (1) | PE20220941A1 (es) |
| PL (1) | PL3999643T3 (es) |
| PT (1) | PT3999643T (es) |
| RS (1) | RS64879B1 (es) |
| SI (1) | SI3999643T1 (es) |
| UY (1) | UY38790A (es) |
| WO (1) | WO2021016032A1 (es) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA39835A (fr) | 2014-04-17 | 2017-02-22 | Biogen Ma Inc | Compositions et procédés de modulation de l'épissage du smn2 chez un patient |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2548560T3 (pl) | 2005-06-23 | 2015-11-30 | Biogen Ma Inc | Kompozycje i sposoby modulowania splicingu SMN2 |
| WO2008157753A1 (en) * | 2007-06-21 | 2008-12-24 | Sma Foundation | Methods of treatment for spinal muscular atrophy |
| 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 |
| 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 |
| MA51678A (fr) | 2018-01-25 | 2020-12-02 | Biogen Ma Inc | Méthodes de traitement de l'amyotrophie musculaire |
| IT201800004359A1 (it) | 2018-04-10 | 2019-10-10 | IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE |
-
2020
- 2020-07-16 PE PE2021002322A patent/PE20220941A1/es unknown
- 2020-07-16 AU AU2020316997A patent/AU2020316997A1/en active Pending
- 2020-07-16 EP EP20753556.8A patent/EP3999643B1/en active Active
- 2020-07-16 JP JP2022500671A patent/JP7279253B2/ja active Active
- 2020-07-16 BR BR112022000870A patent/BR112022000870A2/pt unknown
- 2020-07-16 FI FIEP20753556.8T patent/FI3999643T3/fi active
- 2020-07-16 PL PL20753556.8T patent/PL3999643T3/pl unknown
- 2020-07-16 DK DK20753556.8T patent/DK3999643T3/da active
- 2020-07-16 RS RS20231128A patent/RS64879B1/sr unknown
- 2020-07-16 CN CN202080049461.1A patent/CN114302727A/zh active Pending
- 2020-07-16 KR KR1020227001151A patent/KR20220042113A/ko unknown
- 2020-07-16 HR HRP20231515TT patent/HRP20231515T1/hr unknown
- 2020-07-16 ES ES20753556T patent/ES2966791T3/es active Active
- 2020-07-16 US US17/626,324 patent/US20220325282A1/en active Pending
- 2020-07-16 CR CR20220071A patent/CR20220071A/es unknown
- 2020-07-16 JO JOP/2022/0007A patent/JOP20220007A1/ar unknown
- 2020-07-16 LT LTEPPCT/US2020/042312T patent/LT3999643T/lt unknown
- 2020-07-16 CA CA3149906A patent/CA3149906A1/en active Pending
- 2020-07-16 MD MDE20220574T patent/MD3999643T2/ro unknown
- 2020-07-16 PT PT207535568T patent/PT3999643T/pt unknown
- 2020-07-16 MA MA62935A patent/MA62935B1/fr unknown
- 2020-07-16 SI SI202030323T patent/SI3999643T1/sl unknown
- 2020-07-16 UY UY0001038790A patent/UY38790A/es unknown
- 2020-07-16 EP EP23210526.2A patent/EP4335503A3/en active Pending
- 2020-07-16 WO PCT/US2020/042312 patent/WO2021016032A1/en active Application Filing
- 2020-07-16 MX MX2022000441A patent/MX2022000441A/es unknown
- 2020-07-16 HU HUE20753556A patent/HUE064071T2/hu unknown
- 2020-07-17 AR ARP200102019A patent/AR119430A1/es unknown
-
2021
- 2021-12-31 CL CL2021003602A patent/CL2021003602A1/es unknown
-
2022
- 2022-01-10 IL IL289745A patent/IL289745A/en unknown
- 2022-02-10 CO CONC2022/0001354A patent/CO2022001354A2/es unknown
-
2023
- 2023-05-10 JP JP2023077805A patent/JP2023100911A/ja active Pending
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