Title:
COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION OF ALPHA-1 ANTITRYPSIN
Document Type and Number:
WIPO Patent Application WO/2015/195628
Kind Code:
A3
Abstract:
The invention relates to a RNA interference triggers for inhibiting the expression of an AAT gene through the mechanism of RNA interference. The invention also relates to a pharmaceutical composition comprising the AAT RNAi trigger together with an excipient capable of improving delivery of the RNAi trigger to a liver cell in vivo. Delivery of the AAT RNAi trigger to liver cells in vivo provides for inhibition of AAT gene expression and treatment of alpha 1-antitrypsin deficiency and associated diseases.
Inventors:
WOODDELL CHRISTINE I (US)
LEWIS DAVID L (US)
WAKEFIELD DARREN H (US)
ALMEIDA LAUREN (US)
KANNER STEVEN B (US)
LEWIS DAVID L (US)
WAKEFIELD DARREN H (US)
ALMEIDA LAUREN (US)
KANNER STEVEN B (US)
Application Number:
PCT/US2015/035976
Publication Date:
February 18, 2016
Filing Date:
June 16, 2015
Export Citation:
Assignee:
ARROWHEAD RES CORP (US)
International Classes:
A61K38/00; C12N15/113
Domestic Patent References:
| WO1999038987A1 | 1999-08-05 |
Foreign References:
| US20050137153A1 | 2005-06-23 | |||
| US20110028531A1 | 2011-02-03 | |||
| JP2013539967A | 2013-10-31 | |||
| US20100056768A1 | 2010-03-04 | |||
| US20130190484A1 | 2013-07-25 |
Other References:
See also references of EP 3157541A4
Attorney, Agent or Firm:
EKENA, Kirk (465 Science Drive Suite CMadison, WI, US)
Download PDF: