MX2023003066A - Metodos para la reduccion de los niveles de proteina alfa-1 antitripsina z (z-aat). - Google Patents

Metodos para la reduccion de los niveles de proteina alfa-1 antitripsina z (z-aat).

Info

Publication number
MX2023003066A
MX2023003066A MX2023003066A MX2023003066A MX2023003066A MX 2023003066 A MX2023003066 A MX 2023003066A MX 2023003066 A MX2023003066 A MX 2023003066A MX 2023003066 A MX2023003066 A MX 2023003066A MX 2023003066 A MX2023003066 A MX 2023003066A
Authority
MX
Mexico
Prior art keywords
aat
protein levels
reduction
methods
aat protein
Prior art date
Application number
MX2023003066A
Other languages
English (en)
Spanish (es)
Inventor
James C Hamilton
Bruce Given
Dawn Christianson
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of MX2023003066A publication Critical patent/MX2023003066A/es

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

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)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (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)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
MX2023003066A 2020-09-15 2021-09-14 Metodos para la reduccion de los niveles de proteina alfa-1 antitripsina z (z-aat). MX2023003066A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063078658P 2020-09-15 2020-09-15
US202163180487P 2021-04-27 2021-04-27
PCT/US2021/050247 WO2022060721A2 (en) 2020-09-15 2021-09-14 Methods for the reduction of z-aat protein levels

Publications (1)

Publication Number Publication Date
MX2023003066A true MX2023003066A (es) 2023-04-10

Family

ID=78080560

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023003066A MX2023003066A (es) 2020-09-15 2021-09-14 Metodos para la reduccion de los niveles de proteina alfa-1 antitripsina z (z-aat).

Country Status (11)

Country Link
US (1) US20230348905A1 (https=)
EP (1) EP4214316A2 (https=)
JP (1) JP7846095B2 (https=)
KR (1) KR20230067648A (https=)
AU (1) AU2021345026A1 (https=)
BR (1) BR112023004756A2 (https=)
CA (1) CA3192372A1 (https=)
CO (1) CO2023003958A2 (https=)
MX (1) MX2023003066A (https=)
TW (1) TW202220675A (https=)
WO (1) WO2022060721A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
WO2008016391A2 (en) * 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
EP3366312A1 (en) * 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
DK3444350T3 (da) * 2013-07-03 2022-02-07 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna
KR102515329B1 (ko) 2016-03-07 2023-03-29 애로우헤드 파마슈티컬스 인코포레이티드 치료용 화합물을 위한 표적화 리간드
MX2018009853A (es) 2016-09-02 2018-11-09 Arrowhead Pharmaceuticals Inc Ligandos de direccion.
BR112019014282A2 (pt) 2017-01-10 2020-03-03 Arrowhead Pharmaceuticals, Inc. Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso
MX2021015003A (es) * 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).

Also Published As

Publication number Publication date
WO2022060721A3 (en) 2022-04-28
JP2023541170A (ja) 2023-09-28
TW202220675A (zh) 2022-06-01
WO2022060721A2 (en) 2022-03-24
CA3192372A1 (en) 2022-03-24
EP4214316A2 (en) 2023-07-26
CO2023003958A2 (es) 2023-04-17
AU2021345026A1 (en) 2023-05-11
AU2021345026A9 (en) 2024-06-13
US20230348905A1 (en) 2023-11-02
BR112023004756A2 (pt) 2024-02-06
JP7846095B2 (ja) 2026-04-14
KR20230067648A (ko) 2023-05-16

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