Title:
LIPID FORMULATED COMPOSITION AND METHOD FOR INHIBITING EXPRESSION OF TRANSTHYRETIN (TTR)
Document Type and Number:
Japanese Patent JP2017071616
Kind Code:
A
Abstract:
PROBLEM TO BE SOLVED: To provide lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.SOLUTION: The present invention provides a method for reducing TTR amyloid deposition in a subject, comprising administration of a composition in an effective dose, the composition comprises TTR dsRNA that inhibits expression of TTR in the subject, and the TTR dsRNA has a sense strand and an antisense strand and a duplex structure between 15 and 30 base pairs in length, and the antisense strand has 15 or more contiguous nucleotides of a specific sequence.SELECTED DRAWING: None
Inventors:
DINAH WEN-YEE SAH
Application Number:
JP2016220184A
Publication Date:
April 13, 2017
Filing Date:
November 11, 2016
Export Citation:
Assignee:
ALNYLAM PHARMACEUTICALS INC
International Classes:
A61K31/713; A61K47/44; A61K47/50; A61K48/00; A61P9/00; A61P25/00; A61P25/02; A61P43/00; C12N15/113
Domestic Patent References:
| JP5791505B2 | 2015-10-07 | |||
| JP2012530059A | 2012-11-29 | |||
| JP2012506254A | 2012-03-15 |
Foreign References:
| US20090082300A1 | 2009-03-26 |
Other References:
NATURE BIOTECHNOLOGY, vol. 23(8), JPN6013051636, 2005, pages 1002 - 1007, ISSN: 0003629735
JOURNAL OF CONTROLLED RELEASE, vol. 107, JPN6013051637, 2005, pages 276 - 287, ISSN: 0003629736
JOURNAL OF CONTROLLED RELEASE, vol. 107, JPN6013051637, 2005, pages 276 - 287, ISSN: 0003629736
Attorney, Agent or Firm:
Mitsuo Tanaka
Hiroshi Yamazaki
Kenji Tomita
Fumio Inai
Manami Sasakura
Hiroshi Yamazaki
Kenji Tomita
Fumio Inai
Manami Sasakura