SG11202100934PA - Muscle targeting complexes and uses thereof for treating dystrophinopathies - Google Patents

Muscle targeting complexes and uses thereof for treating dystrophinopathies

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Publication number
SG11202100934PA
SG11202100934PA SG11202100934PA SG11202100934PA SG11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA SG 11202100934P A SG11202100934P A SG 11202100934PA
Authority
SG
Singapore
Prior art keywords
muscle targeting
targeting complexes
dystrophinopathies
treating
treating dystrophinopathies
Prior art date
Application number
SG11202100934PA
Inventor
Romesh Subramanian
Mohammed Qatanani
Timothy Weeden
Original Assignee
Dyne 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 Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc
Publication of SG11202100934PA publication Critical patent/SG11202100934PA/en

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    • 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
    • A61K47/68Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
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    • 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
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    • 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
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    • A61K47/6849Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
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    • C12N15/1137Non-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 against enzymes
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    • C12Y204/02Pentosyltransferases (2.4.2)
    • C12Y204/02008Hypoxanthine phosphoribosyltransferase (2.4.2.8)
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    • C07K2317/55Fab or Fab'
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    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
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SG11202100934PA 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies SG11202100934PA (en)

Applications Claiming Priority (3)

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US201862714031P 2018-08-02 2018-08-02
US201962855766P 2019-05-31 2019-05-31
PCT/US2019/044949 WO2020028832A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies

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EP (1) EP3829595A4 (en)
JP (1) JP2021532831A (en)
KR (1) KR20210081322A (en)
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AU (1) AU2019312692A1 (en)
CA (1) CA3108282A1 (en)
IL (1) IL280527A (en)
MX (1) MX2021001281A (en)
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* Cited by examiner, † Cited by third party
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US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
KR20210081322A (en) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. Muscle targeting complexes and their use for treating dystrophinopathy
CA3108289A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202106593UA (en) 2018-12-21 2021-07-29 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
IL293997A (en) * 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antisense nucleic acid enabling exon skipping
JP2023537798A (en) * 2020-03-19 2023-09-06 アビディティー バイオサイエンシーズ,インク. Compositions and methods for treating facioscapulohumeral muscular dystrophy
BR112023001125A2 (en) * 2020-07-23 2023-02-14 Dyne Therapeutics Inc MUSCLE TARGETING COMPLEXES AND THEIR USES TO TREAT DYSTROPHINOPATHIES
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