Patent · US Active

Compositions and methods for inhibiting expression of a mutant gene

US8273870B2 · kind B2 · utility

5Cited by

0References

33Claims

0Family size

Assignee

Alnylam Pharmaceuticals, Inc. · US

Inventors

Roland Kreutzer · Weidenberg, DE
Stefan Limmer · Neudrossenfeld, DE
Matthias John · Hallstadt, DE

Key dates

Filing date	Jul 6, 2011
Grant date	Sep 25, 2012
Priority date	—
Expiry date	Jul 6, 2031

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12N2320/50
WIPO fieldBiotechnology
WIPO sectorChemistry

Abstract

The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.