SG11201810143PA - Exon skipping oligomers for muscular dystrophy - Google Patents

Exon skipping oligomers for muscular dystrophy

Info

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
Application number
SG11201810143PA
Other languages
English (en)
Inventor
Diane Frank
Richard Bestwick
Original Assignee
Sarepta Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of SG11201810143PA publication Critical patent/SG11201810143PA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic System
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558Heterocyclic 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
    • C07F9/65583Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic System
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561Heterocyclic 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
    • C07F9/65616Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
SG11201810143PA 2016-06-30 2017-06-29 Exon skipping oligomers for muscular dystrophy SG11201810143PA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662356923P 2016-06-30 2016-06-30
US201662357072P 2016-06-30 2016-06-30
PCT/US2017/040017 WO2018005805A1 (fr) 2016-06-30 2017-06-29 Sauts d'exons oligomères pour la dystrophie musculaire

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 (fr)
EP (1) EP3478697A1 (fr)
JP (1) JP2019525742A (fr)
KR (1) KR20190024977A (fr)
CN (1) CN109311919A (fr)
AU (1) AU2017290231A1 (fr)
BR (1) BR112019000006A2 (fr)
CA (1) CA3025575A1 (fr)
CO (1) CO2018014029A2 (fr)
IL (1) IL263892A (fr)
MA (1) MA45618A (fr)
MX (1) MX2018016052A (fr)
SG (1) SG11201810143PA (fr)
TW (1) TW201811807A (fr)
WO (1) WO2018005805A1 (fr)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011057350A1 (fr) 2009-11-12 2011-05-19 The University Of Western Australia Molécules antisens et procédés de traitement de pathologies
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA3024153A1 (fr) * 2016-06-30 2017-11-30 Sarepta Therapeutics, Inc. Procedes de preparation d'oligomeres morpholino de phosphorodiamidate
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
US10765760B2 (en) * 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20210220386A1 (en) * 2018-06-14 2021-07-22 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
US20220152086A1 (en) * 2019-03-28 2022-05-19 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy with casimersen
WO2021025899A1 (fr) * 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Compositions pharmaceutiques à base d'oligomères morpholino phosphorodiamidate
EP4361269A1 (fr) * 2021-06-23 2024-05-01 Nippon Shinyaku Co., Ltd. Combinaison d'oligomères antisens

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Also Published As

Publication number Publication date
MX2018016052A (es) 2019-05-02
US20190262375A1 (en) 2019-08-29
WO2018005805A1 (fr) 2018-01-04
CN109311919A (zh) 2019-02-05
CA3025575A1 (fr) 2018-01-04
KR20190024977A (ko) 2019-03-08
JP2019525742A (ja) 2019-09-12
IL263892A (en) 2019-01-31
US20210187003A1 (en) 2021-06-24
EP3478697A1 (fr) 2019-05-08
BR112019000006A2 (pt) 2019-04-16
MA45618A (fr) 2019-05-08
CO2018014029A2 (es) 2019-03-18
TW201811807A (zh) 2018-04-01
AU2017290231A1 (en) 2019-02-07

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