Patent ID: 11932854
Assignee: SIRNA THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A composition comprising:
I) a double-stranded short interfering nucleic acid (siNA) molecule that inhibits the expression of a target gene via RNA interference, having a sense strand and an antisense strand and comprising formula (A):, wherein, the upper strand is the sense strand and the lower strand is the antisense strand of the double-stranded nucleic acid molecule; wherein the antisense strand comprises a sequence having at least 15 nucleotides that are complementary to a target RNA sequence encoded by the target gene and the sense strand comprises a sequence that is complementarity to the antisense strand; each N is independently a nucleotide which is unmodified or chemically modified or is optionally a non-nucleotide; each B is independently a terminal cap that is present or absent; (N) represents overhanging nucleotides, each of which is independently unmodified or chemically modified; [N] represents nucleotides at the 5′-terminus of the antisense strand; X1 and X2 are independently integers from 0 to 4; X3 is an integer from 15 to 30; X4 is an integer from 12 to 27; and X5 is an integer from 1-6, provided that the sum of X4 and X5 is an integer from 15-30; and wherein
(a) five or more pyrimidine nucleotides in NX4 positions are 2′-O-alkyl nucleotides;
(b) five or more purine nucleotides in NX4 positions are 2′-halo nucleotides;
(c) five or more pyrimidine nucleotides in NX3 positions are 2′-O-alkyl nucleotides;
(d) five or more purine nucleotides in Nx3 positions are 2′-halo nucleotides; and
(e) [N] position nucleotide(s) are any combination of ribonucleotides, deoxyribonucleotides, 2′-O-alkyl nucleotides, or 2′-halo nucleotides; and

II) a cationic lipid comprising 13Z,16Z)-N,N-dimethyl-3-nonyldocosa-13 16-dien-1-amine.