SG11201810143PA - Exon skipping oligomers for muscular dystrophy - Google Patents
Exon skipping oligomers for muscular dystrophyInfo
- Publication number
- SG11201810143PA SG11201810143PA SG11201810143PA SG11201810143PA SG11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA SG 11201810143P A SG11201810143P A SG 11201810143PA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- june
- pct
- cambridge
- suite
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
- 208000035342 Isolated congenital onychodysplasia Diseases 0.000 abstract 1
- 241001635911 Sarepta Species 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000003387 muscular Effects 0.000 abstract 1
- 201000010158 nonsyndromic congenital nail disorder 7 Diseases 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 102220240796 rs553605556 Human genes 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
-
- 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
- C07F9/6558—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
-
- 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
-
- 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
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Polymers With Sulfur, Phosphorus Or Metals In The Main Chain (AREA)
- Management, Administration, Business Operations System, And Electronic Commerce (AREA)
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 |
---|---|
SG11201810143PA true SG11201810143PA (en) | 2019-01-30 |
Family
ID=59315756
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201810143PA SG11201810143PA (en) | 2016-06-30 | 2017-06-29 | Exon skipping oligomers for muscular dystrophy |
Country Status (15)
Country | Link |
---|---|
US (2) | US20190262375A1 (zh) |
EP (1) | EP3478697A1 (zh) |
JP (1) | JP2019525742A (zh) |
KR (1) | KR20190024977A (zh) |
CN (1) | CN109311919A (zh) |
AU (1) | AU2017290231A1 (zh) |
BR (1) | BR112019000006A2 (zh) |
CA (1) | CA3025575A1 (zh) |
CO (1) | CO2018014029A2 (zh) |
IL (1) | IL263892A (zh) |
MA (1) | MA45618A (zh) |
MX (1) | MX2018016052A (zh) |
SG (1) | SG11201810143PA (zh) |
TW (1) | TW201811807A (zh) |
WO (1) | WO2018005805A1 (zh) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DK2499249T3 (en) | 2009-11-12 | 2018-12-03 | Univ Western Australia | ANTISENCE MOLECULES AND PROCEDURES FOR TREATING PATHOLOGIES |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
SG11201809502YA (en) * | 2016-05-24 | 2018-12-28 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
JP2021521794A (ja) * | 2018-04-26 | 2021-08-30 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
JP2021526807A (ja) * | 2018-06-14 | 2021-10-11 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
AU2020244803A1 (en) * | 2019-03-28 | 2021-11-18 | 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 |
CN117940564A (zh) * | 2021-06-23 | 2024-04-26 | 日本新药株式会社 | 反义低聚物的组合 |
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EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
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 |
US10160969B2 (en) | 2014-01-16 | 2018-12-25 | Wave Life Sciences Ltd. | Chiral design |
SI3118311T1 (sl) | 2014-03-12 | 2019-05-31 | Nippon Shinyaku Co., Ltd. | Protismiselna nukleinska kislina |
PL3159409T3 (pl) | 2014-06-17 | 2020-05-18 | Nippon Shinyaku Co., Ltd. | Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a |
CN108699555A (zh) * | 2015-10-09 | 2018-10-23 | 萨勒普塔医疗公司 | 用于治疗杜兴肌营养不良和相关病症的组合物和方法 |
-
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 AU AU2017290231A patent/AU2017290231A1/en not_active Abandoned
- 2017-06-29 CA CA3025575A patent/CA3025575A1/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
- 2017-06-29 WO PCT/US2017/040017 patent/WO2018005805A1/en unknown
- 2017-06-29 MA MA045618A patent/MA45618A/fr unknown
- 2017-06-29 KR KR1020197002453A patent/KR20190024977A/ko not_active Application Discontinuation
- 2017-06-29 SG SG11201810143PA patent/SG11201810143PA/en unknown
- 2017-06-29 CN CN201780038876.7A patent/CN109311919A/zh active Pending
- 2017-06-29 JP JP2018567259A patent/JP2019525742A/ja active Pending
- 2017-06-29 EP EP17737999.7A patent/EP3478697A1/en not_active Withdrawn
-
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 |
---|---|
AU2017290231A1 (en) | 2019-02-07 |
TW201811807A (zh) | 2018-04-01 |
CN109311919A (zh) | 2019-02-05 |
BR112019000006A2 (pt) | 2019-04-16 |
EP3478697A1 (en) | 2019-05-08 |
IL263892A (en) | 2019-01-31 |
CO2018014029A2 (es) | 2019-03-18 |
WO2018005805A1 (en) | 2018-01-04 |
KR20190024977A (ko) | 2019-03-08 |
CA3025575A1 (en) | 2018-01-04 |
MX2018016052A (es) | 2019-05-02 |
US20190262375A1 (en) | 2019-08-29 |
MA45618A (fr) | 2019-05-08 |
JP2019525742A (ja) | 2019-09-12 |
US20210187003A1 (en) | 2021-06-24 |
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