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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    • A61K47/68—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
    • C—CHEMISTRY; METALLURGY
    • 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
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    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
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    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
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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 (https=)
EP (1) EP3830259A4 (https=)
JP (2) JP2021533200A (https=)
KR (1) KR20210081324A (https=)
CN (1) CN112912499A (https=)
AU (1) AU2019316103B2 (https=)
CA (1) CA3108289A1 (https=)
EA (1) EA202190416A1 (https=)
IL (1) IL280426A (https=)
MX (1) MX2021001284A (https=)
SG (1) SG11202100928QA (https=)
WO (1) WO2020028864A1 (https=)

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