Title:
フェニルケトン尿症のためのｍＲＮＡ療法
Document Type and Number:
Japanese Patent JP6506749
Kind Code:
B2
Abstract:
The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids
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Inventors:
De rosa, frank
Hearthrain, michael
Diaz, Anusha
Hearthrain, michael
Diaz, Anusha
Application Number:
JP2016523331A
Publication Date:
April 24, 2019
Filing Date:
October 22, 2014
Export Citation:
Assignee:
Shire Human Genetic Therapy Inc.
International Classes:
A61K31/7105; A61K9/127; A61K47/14; A61K47/22; A61K47/24; A61K47/28; A61K48/00; A61P3/00; A61P25/00
Foreign References:
| WO2013063468A1 |
Other References:
Mol Ther,2013年 5月,Vol.21, Sup.1,S136, Abstract 354
Eur J Pharm Biopharm,2009年,Vol.71,p.484-489
Eur J Pharm Biopharm,2009年,Vol.71,p.484-489
Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto