IP Library › Patent Application 16790557
Patent Application
App. No. 16/790,557

COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION

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Quick Facts
Patent No.
US None
App. No.
16/790,557
Abstract

Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.

Claims (7)

1 - 33 . (canceled)

34 . A method of treating transthyretin amyloidosis in a subject comprising administering a modified oligonucleotide, wherein the anion form of the modified oligonucleotide has the following chemical structure:

wherein the modified oligonucleotide is a salt, wherein the cation of the salt is sodium, and wherein the transthyretin amyloidosis is senile systemic amyloidosis (SSA), familial amyloid polyneuropathy (FAP), or familial amyloid cardiopathy (FAC).

35 - 39 . (canceled)

40 . The method of claim 34 , wherein the transthyretin amyloidosis is senile systemic amyloidosis (SSA).

41 . The method of claim 34 , wherein the transthyretin amyloidosis is familial amyloid polyneuropathy (FAP).

42 . The method of claim 34 , wherein the transthyretin amyloidosis is familial amyloid cardiopathy (FAC).

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 13, 2022
From: PRAKASH, THAZHA P.; SETH, PUNIT P.; SWAYZE, ERIC E.
To: IONIS PHARMACEUTICALS, INC.
Reel/Frame 059585/0524 →