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SG10202100491QA - Exon skipping oligomer conjugates for muscular dystrophy - Google Patents

Exon skipping oligomer conjugates for muscular dystrophy

Info

Publication number
SG10202100491QA
SG10202100491QA SG10202100491QA SG10202100491QA SG10202100491QA SG 10202100491Q A SG10202100491Q A SG 10202100491QA SG 10202100491Q A SG10202100491Q A SG 10202100491QA SG 10202100491Q A SG10202100491Q A SG 10202100491QA SG 10202100491Q A SG10202100491Q A SG 10202100491QA
Authority
SG
Singapore
Prior art keywords
muscular dystrophy
exon skipping
oligomer conjugates
skipping oligomer
conjugates
Prior art date
Application number
SG10202100491QA
Inventor
Marco Passini
Gunnar 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 SG10202100491QA publication Critical patent/SG10202100491QA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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 an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
    • 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
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
SG10202100491QA 2016-12-19 2017-12-14 Exon skipping oligomer conjugates for muscular dystrophy SG10202100491QA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662436199P 2016-12-19 2016-12-19
US201762443481P 2017-01-06 2017-01-06
US201762479177P 2017-03-30 2017-03-30
US201762562119P 2017-09-22 2017-09-22

Publications (1)

Publication Number Publication Date
SG10202100491QA true SG10202100491QA (en) 2021-02-25

Family

ID=61006319

Family Applications (1)

Application Number Title Priority Date Filing Date
SG10202100491QA SG10202100491QA (en) 2016-12-19 2017-12-14 Exon skipping oligomer conjugates for muscular dystrophy

Country Status (28)

Country Link
US (2) US11382981B2 (en)
EP (3) EP3554554B1 (en)
JP (3) JP7125940B2 (en)
KR (2) KR102639633B1 (en)
CN (2) CN117298290A (en)
AU (1) AU2017382741A1 (en)
BR (1) BR112019012651A2 (en)
CA (1) CA3046801A1 (en)
CO (1) CO2019007398A2 (en)
DK (2) DK3554554T3 (en)
ES (2) ES2980686T3 (en)
FI (1) FI4122497T3 (en)
HR (2) HRP20221316T1 (en)
HU (2) HUE066576T2 (en)
IL (2) IL304284B1 (en)
LT (2) LT4122497T (en)
MD (2) MD4122497T2 (en)
MX (1) MX2019006989A (en)
NZ (1) NZ755422A (en)
PL (2) PL4122497T3 (en)
PT (2) PT3554554T (en)
RS (2) RS63705B1 (en)
SA (1) SA519402143B1 (en)
SG (1) SG10202100491QA (en)
SI (2) SI3554554T1 (en)
TW (1) TWI780095B (en)
WO (1) WO2018118627A1 (en)
ZA (1) ZA201903940B (en)

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DK3464305T3 (en) 2016-05-24 2024-10-28 Sarepta Therapeutics Inc PROCESSES FOR THE PRODUCTION OF OLIGOMERS
MA45362A (en) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc PROCESSES FOR THE PREPARATION OF MORPHOLINO OLIGOMERS OF PHOSPHORODIAMIDATE
PT3554554T (en) * 2016-12-19 2022-11-08 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
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BR112021018801A2 (en) * 2019-03-28 2021-11-23 Sarepta Therapeutics Inc Methods for treating muscular dystrophy with casimersen
WO2020214763A1 (en) * 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US20220296633A1 (en) * 2019-06-19 2022-09-22 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
US20230073008A1 (en) 2019-12-26 2023-03-09 Nippon Shinyaku Co., Ltd. Antisense nucleic acid that induces skipping of exon 50
US20230140736A1 (en) 2020-02-28 2023-05-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
CN115916208A (en) * 2020-06-17 2023-04-04 康德生物医疗有限公司 Methods and compositions for treating muscular dystrophy
MX2023012559A (en) * 2021-04-30 2023-12-15 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy.
TW202307208A (en) 2021-06-23 2023-02-16 日商日本新藥股份有限公司 Combination of antisense oligomers
KR20240035502A (en) 2021-07-08 2024-03-15 니뽄 신야쿠 가부시키가이샤 Nephrotoxicity reliever
CA3225573A1 (en) 2021-07-08 2023-01-12 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
TW202308664A (en) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 Nephrotoxicity-reducing agent
TW202346589A (en) 2022-03-10 2023-12-01 日商日本新藥股份有限公司 Antiviral antisense oligomer

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