CA3108289A1 - Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy - Google Patents

Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy Download PDF

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Publication number
CA3108289A1
CA3108289A1 CA3108289A CA3108289A CA3108289A1 CA 3108289 A1 CA3108289 A1 CA 3108289A1 CA 3108289 A CA3108289 A CA 3108289A CA 3108289 A CA3108289 A CA 3108289A CA 3108289 A1 CA3108289 A1 CA 3108289A1
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CA
Canada
Prior art keywords
complex
antibody
muscle
oligonucleotide
transferrin receptor
Prior art date
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Pending
Application number
CA3108289A
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English (en)
French (fr)
Inventor
Romesh R. Subramanian
Mohammed T. QATANANI
Timothy Weeden
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Dyne Therapeutics Inc
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Dyne Therapeutics Inc
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Filing date
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Application filed by Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc
Publication of CA3108289A1 publication Critical patent/CA3108289A1/en
Pending legal-status Critical Current

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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/68—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs 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
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    • 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
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    • A61K47/68—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C12Y—ENZYMES
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    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
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CA3108289A 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy Pending CA3108289A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862713933P 2018-08-02 2018-08-02
US62/713,933 2018-08-02
PCT/US2019/044990 WO2020028864A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

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CA3108289A1 true CA3108289A1 (en) 2020-02-06

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CA3108289A Pending CA3108289A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

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US (12) US20220193250A1 (enExample)
EP (1) EP3830259A4 (enExample)
JP (2) JP2021533200A (enExample)
KR (1) KR20210081324A (enExample)
CN (1) CN112912499A (enExample)
AU (1) AU2019316103A1 (enExample)
CA (1) CA3108289A1 (enExample)
EA (1) EA202190416A1 (enExample)
IL (1) IL280426A (enExample)
MX (1) MX2021001284A (enExample)
SG (1) SG11202100928QA (enExample)
WO (1) WO2020028864A1 (enExample)

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