IP Library › Granted Patent US 9,101,643
Granted Patent B2
US 9,101,643 · App. 13/503,843 · Granted Aug 11, 2015

Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)

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Quick Facts
Patent No.
US 9,101,643
App. No.
13/503,843
Granted
Aug 11, 2015
Kind
B2
Abstract

The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.

Claims (4)

1. A method for reducing a TTR amyloid deposit by >90% in a subject having the TTR amyloid deposit, comprising administering to the subject an effective amount of a composition comprising a TTR dsRNA and determining TTR amyloid deposition in the subject, wherein: the TTR dsRNA comprises a sense strand and an antisense strand, the antisense strand consisting of SEQ ID NO:1010 and the sense strand consisting of SEQ ID NO:1009; the effective amount is 0.01-30 mg/kg TTR dsRNA; and the composition comprises a lipid formulation comprising a cationic lipid.

2. The method of claim 1 , wherein the TTR amyloid deposit comprises V30M mutant TTR.

3. The method of claim 1 , wherein the subject is a human subject.

4. The method of claim 1 , wherein the TTR amyloid deposit comprises V30M mutant TTR and the subject is a human subject.

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 Jul 5, 2012
From: SAH, DINAH WEN-YEE
To: ALNYLAM PHARMACEUTICALS, INC.
Reel/Frame 028492/0275 →