US2022275365A9PendingUtilityA9
Compositions and methods for modulating ttr expression
Est. expiryMay 1, 2033(~6.7 yrs left)· nominal 20-yr term from priority
C12N 15/113C12N 2310/315C12N 2310/11A61K 31/713A61K 31/7088C07H 21/00C12N 2310/35C12N 2310/32A61K 48/00A61K 31/7125C12N 2310/34C12N 2310/3511C12N 2310/3515C07H 21/04A61K 47/59A61K 47/549C12N 2310/17C12N 2310/31C12N 2310/3231C12N 2310/3513C12N 2310/351C12N 2320/32A61P 43/00C12N 2310/346C12N 2310/321C12N 2310/353C12N 2310/3341C12Y 301/03048C12N 2310/113C12N 15/111C12N 2310/322C12N 2310/341A61P 25/00C12N 2310/3525C12N 2310/14A61K 31/7105C12N 15/1137
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Claims
Abstract
Provided herein are oligomeric compounds with conjugate groups. In certain embodiments, the oligomeric compounds are conjugated to N-Acetylgalactosamine.
Claims
exact text as granted — not AI-modified1 - 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).Join the waitlist — get patent alerts
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