Patent ID: 11884920
Assignee: ARROWHEAD PHARMACEUTICALS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A  G | IPC A  C  G

Claim 0:
1. An RNAi agent for inhibiting the expression of an alpha-1 antitrypsin (AAT) gene comprising a sense strand and an antisense strand,
wherein nucleotides 2-18 of the antisense strand comprise nucleotides 2-18 of, (SEQ ID NO: 83)

 
NGUUAAACAUGCCUAAACN,

wherein the sense strand is at least substantially complementary to the antisense strand; and
wherein the antisense strand comprises (i) 2 or 3 phosphorothioate linkages at its 5′ end,
(ii) at least 8 nucleotides selected from 2′-fluoro adenosine, 2′-fluoro cytidine, 2′-fluoro guanosine, and 2′-fluoro uridine, (iii) at least 11 nucleotides selected from 2′-O-methyl adenosine, 2′-O-methyl cytidine, 2′-O-methyl guanosine, and 2′-O-methyl uridine, or (iv) any combination thereof.