CA3108282A1 - Muscle targeting complexes and uses thereof for treating dystrophinopathies - Google Patents

Muscle targeting complexes and uses thereof for treating dystrophinopathies Download PDF

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Publication number
CA3108282A1
CA3108282A1 CA3108282A CA3108282A CA3108282A1 CA 3108282 A1 CA3108282 A1 CA 3108282A1 CA 3108282 A CA3108282 A CA 3108282A CA 3108282 A CA3108282 A CA 3108282A CA 3108282 A1 CA3108282 A1 CA 3108282A1
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CA
Canada
Prior art keywords
complex
antibody
muscle
oligonucleotide
transferrin receptor
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3108282A
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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
Publication date
Application filed by Dyne Therapeutics Inc filed Critical Dyne Therapeutics Inc
Publication of CA3108282A1 publication Critical patent/CA3108282A1/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
    • 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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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic 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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    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • C12N15/1137—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 against enzymes
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    • C12Y204/02—Pentosyltransferases (2.4.2)
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    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
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    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/40—Immunoglobulins specific features characterized by post-translational modification
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    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
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    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
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    • C—CHEMISTRY; METALLURGY
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CA3108282A 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies Pending CA3108282A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862714031P 2018-08-02 2018-08-02
US62/714,031 2018-08-02
US201962855766P 2019-05-31 2019-05-31
US62/855,766 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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CA3108282A1 true CA3108282A1 (en) 2020-02-06

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CA3108282A Pending CA3108282A1 (en) 2018-08-02 2019-08-02 Muscle targeting complexes and uses thereof for treating dystrophinopathies

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US (9) US20210308273A1 (enExample)
EP (1) EP3829595A4 (enExample)
JP (2) JP7611825B2 (enExample)
KR (1) KR20210081322A (enExample)
CN (1) CN112955153A (enExample)
AU (1) AU2019312692A1 (enExample)
CA (1) CA3108282A1 (enExample)
IL (1) IL280527A (enExample)
MX (1) MX2021001281A (enExample)
SG (1) SG11202100934PA (enExample)
WO (1) WO2020028832A1 (enExample)

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US20250297024A1 (en) 2025-09-25
US11833217B2 (en) 2023-12-05
US12005124B2 (en) 2024-06-11
KR20210081322A (ko) 2021-07-01
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US20220143206A1 (en) 2022-05-12
AU2019312692A1 (en) 2021-03-11
JP2024160045A (ja) 2024-11-11
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US20240293568A1 (en) 2024-09-05
CN112955153A (zh) 2021-06-11
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US20220288220A1 (en) 2022-09-15
US12478687B2 (en) 2025-11-25
US20210308273A1 (en) 2021-10-07
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