Title:
ORGANIC COMPOUNDS TO TREAT HEPATITIS B VIRUS
Document Type and Number:
WIPO Patent Application WO/2015/050871
Kind Code:
A3
Abstract:
The disclosure relates to compositions comprising a HBV RNAi agent. In some embodiments, the HBV RNAi agent comprises a sense and an anti-sense strand, each strand being an 18-mer and the strands together forming a blunt-ended duplex, wherein the 3' end of at least one strand terminates in a phosphate or modified internucleoside linker and further comprises, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. In some embodiments, the 3' end of both the sense and anti-sense strand further comprise, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. The two strands can have the same or different spacers, phosphates or modified internucleoside linkers, and/or 3' end caps. The strands can be ribonucleotides, or, optionally, one or more nucleotide can be modified or substituted. Optionally, at least one nucleotide comprises a modified internucleoside linker. Optionally, the RNAi agent can be modified on one or both 5' end. Optionally, the sense strand can comprise a 5' end cap which reduces the amount of the RNA interference mediated by this strand. Optionally, the RNAi agent is attached to a ligand. This format can be used to devise RNAi agents to a variety of different targets and sequences. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference. The disclosure also pertains to methods of treating, ameliorating and preventing HBV in a patient involving the step of administering to the patient a therapeutic amount of a HBV RNAi agent.
Inventors:
BARYZA JEREMY LEE (US)
BLOMMERS MARCEL (CH)
FERNANDEZ CESAR (CH)
GENO ERIN (US)
GOSSERT ALVAR (CH)
GREENIDGE PAULETTE (CH)
HUESKEN DIETER (CH)
HUNZIKER JUERG (CH)
NATT FRANCOIS JEAN-CHARLES (CH)
PATNAIK ANUP (US)
PATTERSON ANDREW (US)
RONDEAU JEAN-MICHEL RENE (CH)
WEILER JAN (US)
ZHU MEICHENG (US)
HOLDORF MEGHAN (US)
BLOMMERS MARCEL (CH)
FERNANDEZ CESAR (CH)
GENO ERIN (US)
GOSSERT ALVAR (CH)
GREENIDGE PAULETTE (CH)
HUESKEN DIETER (CH)
HUNZIKER JUERG (CH)
NATT FRANCOIS JEAN-CHARLES (CH)
PATNAIK ANUP (US)
PATTERSON ANDREW (US)
RONDEAU JEAN-MICHEL RENE (CH)
WEILER JAN (US)
ZHU MEICHENG (US)
HOLDORF MEGHAN (US)
Application Number:
PCT/US2014/058314
Publication Date:
July 02, 2015
Filing Date:
September 30, 2014
Export Citation:
Assignee:
NOVARTIS AG (CH)
BARYZA JEREMY LEE (US)
BLOMMERS MARCEL (CH)
FERNANDEZ CESAR (CH)
GENO ERIN (US)
GOSSERT ALVAR (CH)
GREENIDGE PAULETTE (CH)
HUESKEN DIETER (CH)
HUNZIKER JUERG (CH)
NATT FRANCOIS JEAN-CHARLES (CH)
PATNAIK ANUP (US)
PATTERSON ANDREW (US)
RONDEAU JEAN-MICHEL RENE (CH)
WEILER JAN (US)
ZHU MEICHENG (US)
HOLDORF MEGHAN (US)
BARYZA JEREMY LEE (US)
BLOMMERS MARCEL (CH)
FERNANDEZ CESAR (CH)
GENO ERIN (US)
GOSSERT ALVAR (CH)
GREENIDGE PAULETTE (CH)
HUESKEN DIETER (CH)
HUNZIKER JUERG (CH)
NATT FRANCOIS JEAN-CHARLES (CH)
PATNAIK ANUP (US)
PATTERSON ANDREW (US)
RONDEAU JEAN-MICHEL RENE (CH)
WEILER JAN (US)
ZHU MEICHENG (US)
HOLDORF MEGHAN (US)
International Classes:
A61K31/713; C12N15/113
Domestic Patent References:
| WO2013003520A1 | 2013-01-03 | |||
| WO2003100059A2 | 2003-12-04 | |||
| WO2006078414A2 | 2006-07-27 | |||
| WO2007128477A2 | 2007-11-15 |
Other References:
CZAUDERNA F ET AL: "Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 31, no. 11, 1 June 2003 (2003-06-01), pages 2705 - 2716, XP002607732, ISSN: 0305-1048
Attorney, Agent or Firm:
NOVARTIS AG (4056 Basel, CH)
Download PDF:
Previous Patent: HAND AND FOOT WRAPS
Next Patent: MODULE WITH COLLAPSIBLE FLUID CHAMBER AND ONBOARD FLUID CHAMBER COMPRESSION ELEMENT
Next Patent: MODULE WITH COLLAPSIBLE FLUID CHAMBER AND ONBOARD FLUID CHAMBER COMPRESSION ELEMENT