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MX2024003266A - Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral. - Google Patents

Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.

Info

Publication number
MX2024003266A
MX2024003266A MX2024003266A MX2024003266A MX2024003266A MX 2024003266 A MX2024003266 A MX 2024003266A MX 2024003266 A MX2024003266 A MX 2024003266A MX 2024003266 A MX2024003266 A MX 2024003266A MX 2024003266 A MX2024003266 A MX 2024003266A
Authority
MX
Mexico
Prior art keywords
methods
muscular dystrophy
compositions
facioscapulohumeral muscular
treating facioscapulohumeral
Prior art date
Application number
MX2024003266A
Other languages
English (en)
Inventor
Rob Burke
Beatrice Diana Darimont
Barbora Malecova
Cano David Sala
Original Assignee
Avidity Biosciences 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 Avidity Biosciences Inc filed Critical Avidity Biosciences Inc
Publication of MX2024003266A publication Critical patent/MX2024003266A/es

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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
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    • 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
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
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  • Immunology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)

Abstract

En la presente se describen moléculas de ácido polinucléico, composiciones farmacéuticas y métodos para tratar la distrofia muscular facioescapulohumeral (FSHD).
MX2024003266A 2021-09-16 2022-09-15 Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral. MX2024003266A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163245123P 2021-09-16 2021-09-16
PCT/US2022/043705 WO2023043953A1 (en) 2021-09-16 2022-09-15 Compositions and methods of treating facioscapulohumeral muscular dystrophy

Publications (1)

Publication Number Publication Date
MX2024003266A true MX2024003266A (es) 2024-04-03

Family

ID=85571689

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024003266A MX2024003266A (es) 2021-09-16 2022-09-15 Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral.

Country Status (10)

Country Link
US (4) US11912779B2 (es)
EP (1) EP4401792A1 (es)
JP (1) JP2024535797A (es)
KR (1) KR20240055874A (es)
CN (1) CN118265544A (es)
AU (1) AU2022345098A1 (es)
CA (1) CA3231330A1 (es)
IL (1) IL311452A (es)
MX (1) MX2024003266A (es)
WO (1) WO2023043953A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
MX2022011499A (es) 2020-03-19 2022-10-07 Avidity Biosciences Inc Composiciones y metodos para tratar la distrofia muscular facioescapulohumeral.
EP4401792A1 (en) 2021-09-16 2024-07-24 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy

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