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.

Date of Patent:
Aug. 12, 2025

Filed:

Aug. 23, 2024
Applicant:

Alnylam Pharmaceuticals, Inc., Cambridge, MA (US);

Inventors:

Martin Maier, Cambridge, MA (US);

Don Foster, Cambridge, MA (US);

Stuart Milstein, Cambridge, MA (US);

Satya Kuchimanchi, Cambridge, MA (US);

Vasant Jadhav, Cambridge, MA (US);

Kallanthottathil Rajeev, Cambridge, MA (US);

Muthiah Manoharan, Cambridge, MA (US);

Rubina Parmar, Cambridge, MA (US);

Assignee:

ALNYLAM PHARMACEUTICALS, INC., Cambridge, MA (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/02 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); C12N 15/111 (2013.01); C12N 2310/14 (2013.01); C12N 2310/313 (2013.01); C12N 2310/32 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/332 (2013.01); C12N 2310/335 (2013.01); C12N 2310/344 (2013.01); C12N 2310/346 (2013.01); C12N 2310/351 (2013.01); C12N 2310/533 (2013.01); C12N 2320/51 (2013.01); C12N 2320/52 (2013.01);
Abstract

One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2'-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.


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