AR110742A1 - IRNA A-1 ANTITRIPSIN AGENTS (AAT), COMPOSITIONS INCLUDING iRNA AAT AGENTS AND METHODS OF USE - Google Patents

IRNA A-1 ANTITRIPSIN AGENTS (AAT), COMPOSITIONS INCLUDING iRNA AAT AGENTS AND METHODS OF USE

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Publication number
AR110742A1
AR110742A1 ARP180100065A ARP180100065A AR110742A1 AR 110742 A1 AR110742 A1 AR 110742A1 AR P180100065 A ARP180100065 A AR P180100065A AR P180100065 A ARP180100065 A AR P180100065A AR 110742 A1 AR110742 A1 AR 110742A1
Authority
AR
Argentina
Prior art keywords
aat
irna
agents
methods
irna agents
Prior art date
Application number
ARP180100065A
Other languages
Spanish (es)
Inventor
Christine I Wooddell
Rui Zhu
Zhen Li
Tao Pei
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 AR110742A1 publication Critical patent/AR110742A1/en

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se describen agentes de iARN para inhibir la expresión del gen a-1 antitripsina (AAT), composiciones que incluyen agente de iARN AAT y métodos para su uso, También se describen composiciones farmacéuticas que comprenden uno o más agentes de iARN AAT junto con uno o más excipientes capaces de suministrar uno o más agentes de iARN AAT a una célula hepática in vivo. El suministro de uno o más agentes de iARN AAT a células hepáticas in vivo prevé la inhibición de la expresión del gen AAT y el tratamiento de enfermedades asociadas con la deficiencia de AAT, como hepatitis crónica, cirrosis, carcinoma hepatocelular, transaminitis, colectasia, fibrosis o insuficiencia hepática fulminante.IRNA agents are described to inhibit the expression of the a-1 antitrypsin (AAT) gene, compositions that include AAT iRNA agent and methods for its use. Pharmaceutical compositions comprising one or more AAT iRNA agents together with one or more are also described. more excipients capable of delivering one or more AAT iRNA agents to a liver cell in vivo. The provision of one or more AAT iRNA agents to liver cells in vivo provides for the inhibition of AAT gene expression and the treatment of diseases associated with AAT deficiency, such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholectasis, fibrosis. or fulminant liver failure.

ARP180100065A 2017-01-10 2018-01-10 IRNA A-1 ANTITRIPSIN AGENTS (AAT), COMPOSITIONS INCLUDING iRNA AAT AGENTS AND METHODS OF USE AR110742A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762444452P 2017-01-10 2017-01-10

Publications (1)

Publication Number Publication Date
AR110742A1 true AR110742A1 (en) 2019-05-02

Family

ID=66669155

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP180100065A AR110742A1 (en) 2017-01-10 2018-01-10 IRNA A-1 ANTITRIPSIN AGENTS (AAT), COMPOSITIONS INCLUDING iRNA AAT AGENTS AND METHODS OF USE

Country Status (1)

Country Link
AR (1) AR110742A1 (en)

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