MX2018016052A - Oligomeros de omision de exon para distrofia muscular. - Google Patents
Oligomeros de omision de exon para distrofia muscular.Info
- Publication number
- MX2018016052A MX2018016052A MX2018016052A MX2018016052A MX2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A
- Authority
- MX
- Mexico
- Prior art keywords
- muscular dystrophy
- exon skipping
- oligomers
- skipping oligomers
- exon
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
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
- A61K31/711—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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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
- C07F9/65583—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
-
- 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
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
- C07F9/65616—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
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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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- 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
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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
- C12N2310/3233—Morpholino-type ring
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Management, Administration, Business Operations System, And Electronic Commerce (AREA)
- Polymers With Sulfur, Phosphorus Or Metals In The Main Chain (AREA)
Abstract
La presente invencion se refiere a oligomeros antisentido complementarios a un sitio objetivo seleccionado en el gen de distrofina humana para inducir la omision del exon 45.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662357072P | 2016-06-30 | 2016-06-30 | |
US201662356923P | 2016-06-30 | 2016-06-30 | |
PCT/US2017/040017 WO2018005805A1 (en) | 2016-06-30 | 2017-06-29 | Exon skipping oligomers for muscular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2018016052A true MX2018016052A (es) | 2019-05-02 |
Family
ID=59315756
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2018016052A MX2018016052A (es) | 2016-06-30 | 2017-06-29 | Oligomeros de omision de exon para distrofia muscular. |
Country Status (15)
Country | Link |
---|---|
US (2) | US20190262375A1 (es) |
EP (1) | EP3478697A1 (es) |
JP (1) | JP2019525742A (es) |
KR (1) | KR20190024977A (es) |
CN (1) | CN109311919A (es) |
AU (1) | AU2017290231A1 (es) |
BR (1) | BR112019000006A2 (es) |
CA (1) | CA3025575A1 (es) |
CO (1) | CO2018014029A2 (es) |
IL (1) | IL263892A (es) |
MA (1) | MA45618A (es) |
MX (1) | MX2018016052A (es) |
SG (1) | SG11201810143PA (es) |
TW (1) | TW201811807A (es) |
WO (1) | WO2018005805A1 (es) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
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CN105838714B (zh) | 2009-11-12 | 2020-07-17 | 西澳大利亚大学 | 反义分子和治疗疾病的方法 |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
CN109152792B (zh) * | 2016-06-30 | 2021-09-14 | 萨勒普塔医疗公司 | 制备磷酸二酰胺吗啉代寡聚物的方法 |
WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
US10765760B2 (en) * | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
EP3810150A4 (en) * | 2018-06-14 | 2023-01-25 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATE FOR MUSCLE DYSTROPHY |
EP3946376A1 (en) * | 2019-03-28 | 2022-02-09 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy with casimersen |
WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
TW202307208A (zh) * | 2021-06-23 | 2023-02-16 | 日商日本新藥股份有限公司 | 反義寡聚物之組合 |
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JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
CA2936712A1 (en) | 2014-01-16 | 2015-07-23 | Meena | Chiral design |
AU2015227733B2 (en) | 2014-03-12 | 2019-10-31 | National Center Of Neurology And Psychiatry | Antisense nucleic acids |
AU2015277924B2 (en) | 2014-06-17 | 2021-02-25 | National Center Of Neurology And Psychiatry | Antisense nucleic acids |
BR112018007066A2 (pt) * | 2015-10-09 | 2018-10-23 | Sarepta Therapeutics Inc | composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados |
-
2017
- 2017-06-29 MX MX2018016052A patent/MX2018016052A/es unknown
- 2017-06-29 US US16/312,803 patent/US20190262375A1/en not_active Abandoned
- 2017-06-29 JP JP2018567259A patent/JP2019525742A/ja active Pending
- 2017-06-29 KR KR1020197002453A patent/KR20190024977A/ko not_active Application Discontinuation
- 2017-06-29 CN CN201780038876.7A patent/CN109311919A/zh active Pending
- 2017-06-29 CA CA3025575A patent/CA3025575A1/en not_active Abandoned
- 2017-06-29 SG SG11201810143PA patent/SG11201810143PA/en unknown
- 2017-06-29 EP EP17737999.7A patent/EP3478697A1/en not_active Withdrawn
- 2017-06-29 WO PCT/US2017/040017 patent/WO2018005805A1/en unknown
- 2017-06-29 MA MA045618A patent/MA45618A/fr unknown
- 2017-06-29 AU AU2017290231A patent/AU2017290231A1/en not_active Abandoned
- 2017-06-29 BR BR112019000006-2A patent/BR112019000006A2/pt not_active Application Discontinuation
- 2017-06-29 TW TW106121834A patent/TW201811807A/zh unknown
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2018
- 2018-12-21 CO CONC2018/0014029A patent/CO2018014029A2/es unknown
- 2018-12-23 IL IL263892A patent/IL263892A/en unknown
-
2020
- 2020-07-28 US US16/941,318 patent/US20210187003A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
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EP3478697A1 (en) | 2019-05-08 |
CN109311919A (zh) | 2019-02-05 |
AU2017290231A1 (en) | 2019-02-07 |
TW201811807A (zh) | 2018-04-01 |
BR112019000006A2 (pt) | 2019-04-16 |
SG11201810143PA (en) | 2019-01-30 |
WO2018005805A1 (en) | 2018-01-04 |
US20190262375A1 (en) | 2019-08-29 |
US20210187003A1 (en) | 2021-06-24 |
CA3025575A1 (en) | 2018-01-04 |
JP2019525742A (ja) | 2019-09-12 |
CO2018014029A2 (es) | 2019-03-18 |
MA45618A (fr) | 2019-05-08 |
IL263892A (en) | 2019-01-31 |
KR20190024977A (ko) | 2019-03-08 |
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