SG10202012839TA - Exon skipping oligomer conjugates for muscular dystrophy - Google Patents

Exon skipping oligomer conjugates for muscular dystrophy

Info

Publication number
SG10202012839TA
SG10202012839TA SG10202012839TA SG10202012839TA SG10202012839TA SG 10202012839T A SG10202012839T A SG 10202012839TA SG 10202012839T A SG10202012839T A SG 10202012839TA SG 10202012839T A SG10202012839T A SG 10202012839TA SG 10202012839T A SG10202012839T A SG 10202012839TA
Authority
SG
Singapore
Prior art keywords
muscular dystrophy
exon skipping
oligomer conjugates
skipping oligomer
conjugates
Prior art date
Application number
SG10202012839TA
Inventor
Marco A Passini
Gunnar J Hanson
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 SG10202012839TA publication Critical patent/SG10202012839TA/en

Links

Classifications

    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • 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
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
SG10202012839TA 2016-12-19 2017-12-13 Exon skipping oligomer conjugates for muscular dystrophy SG10202012839TA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662436182P 2016-12-19 2016-12-19
US201762443476P 2017-01-06 2017-01-06
US201762479173P 2017-03-30 2017-03-30
US201762562080P 2017-09-22 2017-09-22

Publications (1)

Publication Number Publication Date
SG10202012839TA true SG10202012839TA (en) 2021-01-28

Family

ID=61006315

Family Applications (1)

Application Number Title Priority Date Filing Date
SG10202012839TA SG10202012839TA (en) 2016-12-19 2017-12-13 Exon skipping oligomer conjugates for muscular dystrophy

Country Status (27)

Country Link
US (4) US10888578B2 (en)
EP (1) EP3554553B1 (en)
JP (3) JP7118063B2 (en)
KR (2) KR20230110811A (en)
CN (1) CN110290812B (en)
AU (1) AU2017382723A1 (en)
BR (1) BR112019012647A2 (en)
CA (1) CA3046793A1 (en)
CO (1) CO2019007397A2 (en)
DK (1) DK3554553T3 (en)
ES (1) ES2927079T3 (en)
HR (1) HRP20220943T1 (en)
HU (1) HUE059843T2 (en)
IL (2) IL267244B (en)
LT (1) LT3554553T (en)
MD (1) MD3554553T2 (en)
MX (1) MX2019006882A (en)
MY (1) MY195801A (en)
NZ (1) NZ755416A (en)
PL (1) PL3554553T3 (en)
PT (1) PT3554553T (en)
RS (1) RS63610B1 (en)
SA (1) SA519402154B1 (en)
SG (1) SG10202012839TA (en)
TW (1) TWI760402B (en)
WO (1) WO2018118599A1 (en)
ZA (1) ZA201904092B (en)

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EP3464306B1 (en) 2016-05-24 2024-03-27 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
CA3025339A1 (en) 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing oligomers
RS63610B1 (en) * 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
NZ755422A (en) 2016-12-19 2023-06-30 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
CN110636866A (en) * 2016-12-19 2019-12-31 萨勒普塔医疗公司 Exon skipping oligomer conjugates for muscular dystrophy
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
EP3955966A1 (en) * 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
JP2022536495A (en) * 2019-06-19 2022-08-17 サレプタ セラピューティクス， インコーポレイテッド How to treat muscular dystrophy
US20230073008A1 (en) 2019-12-26 2023-03-09 Nippon Shinyaku Co., Ltd. Antisense nucleic acid that induces skipping of exon 50
CA3173049A1 (en) 2020-02-28 2021-09-02 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
CA3216839A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
BR112023027298A2 (en) 2021-06-23 2024-03-12 Nat Center Neurology & Psychiatry COMBINATION OF ANTISENSE OLIGOMERS
JPWO2023282344A1 (en) 2021-07-08 2023-01-12
IL310003A (en) 2021-07-08 2024-03-01 Nippon Shinyaku Co Ltd Nephrotoxicity reducing agent
WO2023282346A1 (en) 2021-07-08 2023-01-12 日本新薬株式会社 Precipitation inhibitor
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