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MX2024001573A - Composiciones de acido ribonucleico de interferencia (arni) contra el factor xii (f12) y sus metodos de uso. - Google Patents

Composiciones de acido ribonucleico de interferencia (arni) contra el factor xii (f12) y sus metodos de uso.

Info

Publication number
MX2024001573A
MX2024001573A MX2024001573A MX2024001573A MX2024001573A MX 2024001573 A MX2024001573 A MX 2024001573A MX 2024001573 A MX2024001573 A MX 2024001573A MX 2024001573 A MX2024001573 A MX 2024001573A MX 2024001573 A MX2024001573 A MX 2024001573A
Authority
MX
Mexico
Prior art keywords
methods
disease
factor xii
gene
relates
Prior art date
Application number
MX2024001573A
Other languages
English (en)
Inventor
Mark K Schlegel
Adam Castoreno
Karyn Schmidt
Original Assignee
Alnylam Pharmaceuticals 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of MX2024001573A publication Critical patent/MX2024001573A/es

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • A61K47/54—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 organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21038—Coagulation factor XIIa (3.4.21.38)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/30—Chemical structure
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
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  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
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  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente invención se relaciona con agentes de ARNi, por ejemplo, agentes de ARN de hebra doble (ARNhd), dirigidos contra el gen del Factor XII (F12). La invención también se relaciona con métodos para usar los agentes de agente de ARNi para inhibir la expresión de un gen F12 y con métodos para prevenir y tratar un trastorno asociado con F12, por ejemplo, angioedema hereditario (HAE), deficiencia de precalicreína, hipertensión esencial maligna, hipertensión, enfermedad renal en estadio terminal, deficiencia del Factor de Fletcher, enfermedad tromboembólica, enfermedad inflamatoria o Enfermedad de Alzheimer.
MX2024001573A 2021-08-13 2022-08-12 Composiciones de acido ribonucleico de interferencia (arni) contra el factor xii (f12) y sus metodos de uso. MX2024001573A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163232840P 2021-08-13 2021-08-13
PCT/US2022/074910 WO2023019246A1 (en) 2021-08-13 2022-08-12 Factor xii (f12) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2024001573A true MX2024001573A (es) 2024-02-14

Family

ID=83192185

Family Applications (1)

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MX2024001573A MX2024001573A (es) 2021-08-13 2022-08-12 Composiciones de acido ribonucleico de interferencia (arni) contra el factor xii (f12) y sus metodos de uso.

Country Status (15)

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US (2) US12049630B2 (es)
EP (1) EP4384617A1 (es)
JP (1) JP2024534766A (es)
KR (1) KR20240045300A (es)
CN (1) CN118076737A (es)
AR (1) AR126771A1 (es)
AU (1) AU2022328347A1 (es)
BR (1) BR112024001923A2 (es)
CA (1) CA3228733A1 (es)
CL (1) CL2024000363A1 (es)
CO (1) CO2024001402A2 (es)
IL (1) IL310407A (es)
MX (1) MX2024001573A (es)
TW (1) TW202334413A (es)
WO (1) WO2023019246A1 (es)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2024001573A (es) 2021-08-13 2024-02-14 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) contra el factor xii (f12) y sus metodos de uso.

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