The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Mar. 11, 2025
Filed:
May. 16, 2019
Alnylam Pharmaceuticals, Inc., Cambridge, MA (US);
Shigeo Matsuda, Cambridge, MA (US);
Mark K. Schlegel, Cambridge, MA (US);
Maja Janas, Cambridge, MA (US);
Vasant R. Jadhav, Cambridge, MA (US);
Martin Maier, Cambridge, MA (US);
Klaus Charisse, Cambridge, MA (US);
Muthiah Manoharan, Cambridge, MA (US);
Kallanthottathil Rajeev, Cambridge, MA (US);
Jayaprakash K. Nair, Cambridge, MA (US);
Abstract
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2'-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.