RNAi modulation of SCAP and therapeutic uses thereof

US8383805B2 · kind B2 · utility

Assignees

• Alnylam Pharmaceuticals, Inc. · US
• The Board of Regents of the University of Texas System · US

Inventors

• Juergen Soutschek · Kasendorf, DE
• Pamela Tan · Kulmbach, DE
• Jay D. Horton · Plano, US
• Michael S. Brown · Dallas, US
• Joseph L. Goldstein · Dallas, US
• Young-Ah Moon · Irving, US

Key dates

• Filing date: Apr 5, 2011
• Grant date: Feb 26, 2013
• Expiry date: Apr 5, 2031

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/3515
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a SCAP gene (Human SCAP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of a SCAP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Human SCAP expression and the expression of a SCAP gene using the pharmaceutical composition; and methods for inhibiting the expression of a SCAP gene in a cell.