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MX2023010958A - Agentes de arn modificados con efecto reducido fuera de la diana. - Google Patents

Agentes de arn modificados con efecto reducido fuera de la diana.

Info

Publication number
MX2023010958A
MX2023010958A MX2023010958A MX2023010958A MX2023010958A MX 2023010958 A MX2023010958 A MX 2023010958A MX 2023010958 A MX2023010958 A MX 2023010958A MX 2023010958 A MX2023010958 A MX 2023010958A MX 2023010958 A MX2023010958 A MX 2023010958A
Authority
MX
Mexico
Prior art keywords
dsrna
ligand
reduced
modified rna
target effect
Prior art date
Application number
MX2023010958A
Other languages
English (en)
Inventor
Stuart Milstein
Klaus Charisse
Alfica Sehgal
Donald Foster
Muthiah Manoharan
Christopher Theile
Maja Janas
Vasant R Jadhav
Muthusamy Jayaraman
Alexander V Kel'in
Shigeo Matsuda
Jayaprakash K Nair
Martin A Maier
Kallanthottathil G Rajeev
Christopher Brown
Mark K Schlegel
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=60888585&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX2023010958(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of MX2023010958A publication Critical patent/MX2023010958A/es

Links

Classifications

    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Un aspecto de la presente invención se refiere a un agente de ARN de doble cadena (ARNdc) capaz de inhibir la expresión de un gen diana. La cadena no codificante de la molécula de ARNdc comprende al menos un nucleótido térmicamente desestabilizante que se encuentra en una región semilla; el ARNdc comprende al menos cuatro modificaciones de 2'-fluoro, y la cadena codificante de la molécula de ARNdc comprende un ligando, en donde el ligando es un ligando de ASGPR. Otros aspectos de la invención se refieren a composiciones farmacéuticas que comprenden estas moléculas de ARNdc adecuadas para su uso terapéutico, y a métodos para inhibir la expresión de un gen diana al administrar estas moléculas de ARNdc, por ejemplo, para el tratamiento de varias afecciones patológicas.
MX2023010958A 2016-11-23 2019-05-17 Agentes de arn modificados con efecto reducido fuera de la diana. MX2023010958A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662425907P 2016-11-23 2016-11-23
US201762548589P 2017-08-22 2017-08-22
US201762561514P 2017-09-21 2017-09-21

Publications (1)

Publication Number Publication Date
MX2023010958A true MX2023010958A (es) 2023-12-07

Family

ID=60888585

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019005816A MX2019005816A (es) 2016-11-23 2017-11-22 Agentes de arn modificados con efecto reducido fuera de la diana.
MX2023010958A MX2023010958A (es) 2016-11-23 2019-05-17 Agentes de arn modificados con efecto reducido fuera de la diana.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2019005816A MX2019005816A (es) 2016-11-23 2017-11-22 Agentes de arn modificados con efecto reducido fuera de la diana.

Country Status (11)

Country Link
US (3) US11504391B1 (es)
EP (1) EP3544617A4 (es)
JP (3) JP7288852B2 (es)
KR (2) KR102645243B1 (es)
CN (2) CN110582283B (es)
AU (3) AU2017363892B2 (es)
CA (1) CA3044598A1 (es)
IL (2) IL266780B1 (es)
MX (2) MX2019005816A (es)
SG (1) SG10201913786SA (es)
WO (1) WO2018098328A1 (es)

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CN117757791A (zh) 2024-03-26
MX2019005816A (es) 2019-10-07
JP2023053963A (ja) 2023-04-13
US20250205272A1 (en) 2025-06-26
KR20240035907A (ko) 2024-03-18
IL266780B1 (en) 2025-11-01
AU2023226646A1 (en) 2023-11-16
IL266780A (en) 2019-08-29
EP3544617A1 (en) 2019-10-02
JP2019535288A (ja) 2019-12-12
JP7288852B2 (ja) 2023-06-08
AU2017363892A1 (en) 2019-06-13
WO2018098328A1 (en) 2018-05-31
JP2025168400A (ja) 2025-11-07
AU2025202185A1 (en) 2025-04-17
KR102645243B1 (ko) 2024-03-11
KR20190086001A (ko) 2019-07-19
SG10201913786SA (en) 2020-03-30
CN110582283B (zh) 2024-01-02
US11504391B1 (en) 2022-11-22
NZ753920A (en) 2024-10-25
EP3544617A4 (en) 2020-08-05
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