Compositions and methods for inhibiting expression of a target gene

US7829693B2 · kind B2 · utility

Assignee

• Alnylam Pharmaceuticals, Inc. · US

Inventors

• Roland Kreutzer · Weidenberg, DE
• Stefan Limmer · Neudrossenfeld, DE
• Sylvia Limmer · Kulmbach, DE
• Philipp Hadwiger · Kulmbach, DE

Key dates

• Filing date: Mar 7, 2003
• Grant date: Nov 9, 2010
• Expiry date: Jan 31, 2028

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/50
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention relates to a double-stranded ribonucleic acid (dsRNA) having a nucleotide sequence which is substantially identical to at least a part of a target gene and which is no more than 49, preferably less than 25, nucleotides in length, and which comprises a complementary (antisense) RNA strand having a 1 to 4 nucleotide overhang at the 3′-end and a blunt 5′-end. 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 gene, as well as for treating diseases caused by expression of the target gene, at low dosages (i.e., less than 5 milligrams, preferably less than 25 micrograms, per kg body weight per day). The invention also relates to methods for inhibiting the expression of a target gene, as well as methods for treating diseases caused by the expression of the gene.