WO2021142217A8 - Complexes de ciblage musculaire et leurs utilisations pour la modulation de milck1 - Google Patents

Complexes de ciblage musculaire et leurs utilisations pour la modulation de milck1 Download PDF

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Publication number
WO2021142217A8
WO2021142217A8 PCT/US2021/012637 US2021012637W WO2021142217A8 WO 2021142217 A8 WO2021142217 A8 WO 2021142217A8 US 2021012637 W US2021012637 W US 2021012637W WO 2021142217 A8 WO2021142217 A8 WO 2021142217A8
Authority
WO
WIPO (PCT)
Prior art keywords
muscle
milck1
modulation
oligonucleotide
muscle targeting
Prior art date
Application number
PCT/US2021/012637
Other languages
English (en)
Other versions
WO2021142217A1 (fr
Inventor
Romesh R. Subramanian
Mohammed T. QATANANI
Timothy Weeden
Cody A. Desjardins
Brendan QUINN
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 WO2021142217A1 publication Critical patent/WO2021142217A1/fr
Publication of WO2021142217A8 publication Critical patent/WO2021142217A8/fr
Priority to US17/811,332 priority Critical patent/US20230203180A1/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • 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
    • 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
    • A61K47/6835—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—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 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Des aspects de l'invention concernent des charges utiles moléculaires qui modulent l'expression ou l'activité de MLCK1 et des complexes comprenant un agent de ciblage musculaire lié de manière covalente aux charges utiles moléculaires. Dans certains modes de réalisation, l'agent de ciblage musculaire se lie de manière spécifique à un récepteur de surface cellulaire d'internalisation sur une cellule musculaire (par exemple, une cellule de muscle lisse). Dans certains modes de réalisation, la charge utile moléculaire est un oligonucléotide, tel qu'un oligonucléotide antisens ou un oligonucléotide d'ARNi.
PCT/US2021/012637 2020-01-10 2021-01-08 Complexes de ciblage musculaire et leurs utilisations pour la modulation de milck1 WO2021142217A1 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
US17/811,332 US20230203180A1 (en) 2020-01-10 2022-07-08 Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202062959334P 2020-01-10 2020-01-10
US62/959,334 2020-01-10

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2021/012650 Continuation-In-Part WO2021142227A1 (fr) 2020-01-10 2021-01-08 Complexes de ciblage musculaire et leurs utilisations pour la modulation de gènes associés à la santé musculaire

Related Child Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2022/073362 Continuation-In-Part WO2023283531A2 (fr) 2020-01-10 2022-07-01 Complexes de ciblage musculaire et leurs utilisations pour la modulation de gènes associés à la santé musculaire

Publications (2)

Publication Number Publication Date
WO2021142217A1 WO2021142217A1 (fr) 2021-07-15
WO2021142217A8 true WO2021142217A8 (fr) 2022-05-12

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2021/012637 WO2021142217A1 (fr) 2020-01-10 2021-01-08 Complexes de ciblage musculaire et leurs utilisations pour la modulation de milck1

Country Status (1)

Country Link
WO (1) WO2021142217A1 (fr)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3108282A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11648318B2 (en) 2021-07-09 2023-05-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8309614B2 (en) * 2008-04-11 2012-11-13 Cedars-Sinai Medical Center Poly(beta malic acid) with pendant leu-leu-leu tripeptide for effective cytoplasmic drug delivery
GB0904214D0 (en) * 2009-03-11 2009-04-22 Ucb Pharma Sa Biological products
CA3083526A1 (fr) * 2017-12-06 2019-06-13 Andrew John Geall Compositions et procedes de traitement de l'atrophie musculaire et de la dystrophie myotonique
US20210324101A1 (en) * 2018-08-02 2021-10-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy
JP2021533199A (ja) * 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 筋強直症ジストロフィーを処置するための筋標的化複合体およびそれらの使用

Also Published As

Publication number Publication date
WO2021142217A1 (fr) 2021-07-15

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