Title:
LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY
Document Type and Number:
Japanese Patent JP2019048900
Kind Code:
A
Abstract:
To provide compositions and methods for modulating protein production in a target cell.SOLUTION: The compositions and methods disclosed herein can ameliorate diseases related to protein or enzyme deficiencies. The invention provides a composition comprising: (a) at least one mRNA molecule at least part of which encodes a functional secretion polypeptide; and (b) a transfer vehicle comprising lipid nanoparticles. The invention also provides a method for treating a subject deficient in a functional polypeptide the method comprising administering a composition comprising: (a) at least one mRNA molecule at least part of which encodes a functional secretion polypeptide; and (b) a transfer vehicle comprising lipid nanoparticles, where after the administration of the composition, the mRNA is expressed in a target cell to produce the functional secretion polypeptide.SELECTED DRAWING: None
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Inventors:
BRAYDON CHARLES GUILD
FRANK DEROSA
MICHAEL HEARTLEIN
FRANK DEROSA
MICHAEL HEARTLEIN
Application Number:
JP2019000252A
Publication Date:
March 28, 2019
Filing Date:
January 04, 2019
Export Citation:
Assignee:
SHIRE HUMAN GENETIC THERAPIES
International Classes:
A61K48/00; A61K9/16; A61K47/22; A61K47/24; A61K47/28; A61K47/34; A61P3/00; A61P43/00; C12N15/56; C12N15/88
Foreign References:
| WO2010053572A2 | 2010-05-14 | |||
| US20090286852A1 | 2009-11-19 |
Other References:
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Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto