MX2021015003A - Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd). - Google Patents

Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).

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Publication number
MX2021015003A
MX2021015003A MX2021015003A MX2021015003A MX2021015003A MX 2021015003 A MX2021015003 A MX 2021015003A MX 2021015003 A MX2021015003 A MX 2021015003A MX 2021015003 A MX2021015003 A MX 2021015003A MX 2021015003 A MX2021015003 A MX 2021015003A
Authority
MX
Mexico
Prior art keywords
aatd
treatment
methods
alpha
antitrypsin deficiency
Prior art date
Application number
MX2021015003A
Other languages
English (en)
Inventor
Tao Pei
Zhen Li
Rui Zhu
James Hamilton
Bruce Given
Dawn Christianson
Christine Wooddell
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 MX2021015003A publication Critical patent/MX2021015003A/es

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    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
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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
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    • 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
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    • 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)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pulmonology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se describen métodos para tratar la deficiencia de alfa-1 antitripsina (AATD) en un paciente humano que necesita tratamiento, mediante el uso de composiciones farmacéuticas que incluyen agentes de iARN de AAT. Las composiciones farmacéuticas descritas en la presente que incluyen agentes de iARN de AAT, cuando se administran a un paciente humano que lo necesita, tratan enfermedades hepáticas asociadas a la deficiencia de AAT tales como la hepatitis crónica, la cirrosis, el aumento del riesgo de carcinoma hepatocelular, la transaminitis, la colestasis, la fibrosis, la insuficiencia hepática fulminante y otras enfermedades relacionadas con el hígado.
MX2021015003A 2019-06-06 2020-06-05 Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd). MX2021015003A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962858059P 2019-06-06 2019-06-06
PCT/US2020/036359 WO2020247774A1 (en) 2019-06-06 2020-06-05 Methods for the treatment of alpha-1 antitrypsin deficiency (aatd)

Publications (1)

Publication Number Publication Date
MX2021015003A true MX2021015003A (es) 2022-01-24

Family

ID=73652297

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021015003A MX2021015003A (es) 2019-06-06 2020-06-05 Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).

Country Status (12)

Country Link
US (1) US20220305046A1 (es)
EP (1) EP3980540A4 (es)
JP (1) JP2022535127A (es)
KR (1) KR20220019255A (es)
CN (1) CN114222820A (es)
AU (1) AU2020286462A1 (es)
BR (1) BR112021024534A2 (es)
CA (1) CA3141762A1 (es)
CO (1) CO2022000016A2 (es)
MX (1) MX2021015003A (es)
TW (1) TW202113081A (es)
WO (1) WO2020247774A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190098748A (ko) 2017-01-10 2019-08-22 애로우헤드 파마슈티컬스 인코포레이티드 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법
MX2023003066A (es) * 2020-09-15 2023-04-10 Arrowhead Pharmaceuticals Inc Metodos para la reduccion de los niveles de proteina alfa-1 antitripsina z (z-aat).
US20230107967A1 (en) * 2021-08-25 2023-04-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting alpha-1 antitrypsin expression
WO2024044542A2 (en) * 2022-08-22 2024-02-29 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of angptl3-related diseases and disorders

Family Cites Families (10)

* 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.
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
US8765700B2 (en) * 2004-10-20 2014-07-01 Antisense Therapeutics Ltd. Topical administrations of antisense compounds to VLA-4 for the treatment of respiratory conditions
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
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
UY37146A (es) 2016-03-07 2017-09-29 Arrowhead Pharmaceuticals Inc Ligandos de direccionamiento para compuestos terapéuticos
JP2019518028A (ja) * 2016-06-10 2019-06-27 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 補体成分Ｃ５ｉＲＮＡ組成物及び発作性夜間血色素尿症（ＰＮＨ）を処置するためのその使用方法
KR20230115344A (ko) 2016-09-02 2023-08-02 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
KR20190098748A (ko) * 2017-01-10 2019-08-22 애로우헤드 파마슈티컬스 인코포레이티드 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법
SE1750774A1 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy

Also Published As

Publication number Publication date
CA3141762A1 (en) 2020-12-10
JP2022535127A (ja) 2022-08-04
KR20220019255A (ko) 2022-02-16
TW202113081A (zh) 2021-04-01
CO2022000016A2 (es) 2022-04-08
WO2020247774A1 (en) 2020-12-10
US20220305046A1 (en) 2022-09-29
BR112021024534A2 (pt) 2022-02-08
CN114222820A (zh) 2022-03-22
AU2020286462A1 (en) 2022-01-27
EP3980540A4 (en) 2023-11-01
EP3980540A1 (en) 2022-04-13

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