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Patent Application
App. No. 18/921,732

COMPOSITIONS AND METHODS FOR IMPROVING STRAND BIASED

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Quick Facts
Patent No.
US None
App. No.
18/921,732
Abstract

One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. 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.

Claims (45)

1 . A compound of the formula,

wherein:

(i) R 3 is

 (t-butyldimethylsilyl), and B is

 wherein B is benzoyl; or

(ii) R 3 is H or

 and B is

 wherein Bz benzoyl.

2 . The compound of claim 1 , wherein the compound is selected from the group consisting of

wherein Bz benzoyl.

3 . The compound of claim 2 that is

4 . The compound of claim 2 that is

5 . The compound of claim 2 that is

wherein Bz benzoyl.

6 . The compound of claim 2 that is

7 . The compound of claim 1 , wherein the compound is selected from the group consisting of:

wherein Bz is benzoyl,

8 . The compound of claim 7 that is OH

9 . The compound of claim 7 that is

10 . The compound of claim 7 that is

wherein Bz is benzoyl.

11 . The compound of claim 7 that is

12 . The compound of claim 1 , wherein the compound is selected from the group consisting of:

13 . The compound of claim 12 that is

14 . The compound of claim 12 that is

15 . The compound of claim 12 that is

wherein Bz is benzoyl.

16 . The compound of claim 12 that is

17 . A Compound of structure:

wherein:

(i) R 3 is

 and B is

 or

(ii) R 3 is H or

 and B is

18 . The compound of claim 17 , wherein the compound is selected from the group consisting of:

19 . The compound of claim 18 that is

20 . The compound of claim 18 that is

21 . The compound of claim 18 that is

22 . The compound of claim 17 , wherein the compound is selected from the group consisting of:

23 . The compound of claim 22 that is

24 . The compound of claim 22 that is.

25 . The compound of claim 17 , wherein the compound is selected from the group consisting of:

26 . The compound of claim 25 that is

27 . The compound of claim 25 that is

Assignments (2)
SECURITY INTEREST Recorded Oct 1, 2025
From: ALNYLAM PHARMACEUTICALS, INC.; SIRNA THERAPEUTICS, INC.
To: BANK OF AMERICA, N.A.
Reel/Frame 072996/0337 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 2, 2025
From: MANOHARAN, MUTHIAH; PARMAR, RUBINA G.; KUMAR, PAWAN; AKABANE-NAKATA, MASAAKI; MATSUDA, SHIGEO
To: ALNYLAM PHARMACEUTICALS, INC.
Reel/Frame 070707/0655 →