US2016090593A1PendingUtilityA1

Lipid Formulated Compositions And Methods For Inhibiting Expression Of Transthyretin (TTR)

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Assignee: ALNYLAM PHARMACEUTICALS INCPriority: Nov 3, 2009Filed: Jul 1, 2015Published: Mar 31, 2016
Est. expiryNov 3, 2029(~3.3 yrs left)· nominal 20-yr term from priority
Inventors:Dinah Wen-Yee Sah
A61P 9/00A61P 43/00A61P 9/04A61P 25/28A61P 27/02A61P 25/00A61P 25/02A61P 13/12A61P 1/12A61K 31/713C12N 15/1138C12N 2310/14C12N 15/113A61P 1/18C12N 2320/32A61P 1/16A61K 31/7088C12N 2310/3521C12N 2320/30A61P 1/04C12N 2310/321A61K 31/7115
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Claims

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

exact text as granted — not AI-modified
1 .- 24 . (canceled) 
     
     
         25 . A method for reducing a TTR amyloid deposit by >90% in a human subject having the TTR amyloid deposit, comprising administering to the human subject an effective amount of a composition comprising a TTR dsRNA and a lipid formulation comprising a cationic lipid, wherein the TTR dsRNA comprises a sense strand consisting of SEQ ID NO:1009 and an antisense strand consisting of SEQ ID NO:1010 and the effective amount is 0.01-30 mg/kg TTR dsRNA. 
     
     
         26 . The method of  claim 25 , further comprising determining TTR amyloid deposition in the subject.

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