US2018179537A1PendingUtilityA1

Modulation of transthyretin expression

Assignee: IONIS PHARMACEUTICALS INCPriority: Apr 5, 2004Filed: Feb 15, 2018Published: Jun 28, 2018
Est. expiryApr 5, 2024(expired)· nominal 20-yr term from priority
C12N 2310/315C12N 2310/11Y10T436/143333C12N 2310/321C12N 2310/341C12N 2310/3525C12N 15/1136C12Q 2600/158C12N 15/113C12N 2310/3341C12Q 1/6883C12Q 2600/136C12N 2310/346
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Claims

Abstract

Compounds, compositions and methods are provided for modulating the expression of transthyretin. The compositions comprise oligonucleotides, targeted to nucleic acid encoding transthyretin. Methods of using these compounds for modulation of transthyretin expression and for diagnosis and treatment of diseases and conditions associated with expression of transthyretin are provided.

Claims

exact text as granted — not AI-modified
1 .- 18 . (canceled) 
     
     
         19 . A formulation comprising a liposome and a modulator of transthyretin expression, wherein the liposome comprises a cationic lipid. 
     
     
         20 . The formulation of  claim 19 , wherein the modulator of transthyretin expression is an antisense compound, an antisense oligonucleotide, or a ribozyme capable of hybridizing to at least a portion of SEQ ID NO: 4. 
     
     
         21 . The formulation of  claim 20 , wherein the antisense compound is single-stranded. 
     
     
         22 . The formulation of  claim 20 , wherein the antisense compound comprises a modified oligonucleotide 20 to 25 nucleobases in length having at least one modified sugar moiety. 
     
     
         23 . The formulation of  claim 22 , wherein the modified oligonucleotide is at least 95% complementary to SEQ ID NO: 4. 
     
     
         24 . The formulation of  claim 19 , wherein the modulator is complexed to the cationic liposome. 
     
     
         25 . The formulation of  claim 19 , wherein the cationic lipid comprises dioleoyltetramethylaminopropyl or dioleoylphosphatidyl ethanolamine. 
     
     
         26 . The formulation of  claim 19 , wherein the modulator is encapsulated within the liposome. 
     
     
         27 . The formulation of  claim 20 , wherein the modulator is encapsulated within the liposome. 
     
     
         28 . The formulation of  claim 21 , wherein the modulator is encapsulated within the liposome. 
     
     
         29 . The formulation of  claim 22 , wherein the modulator is encapsulated within the liposome. 
     
     
         30 . The formulation of  claim 23 , wherein the modulator is encapsulated within the liposome. 
     
     
         31 . The formulation of  claim 25 , wherein the modulator is encapsulated within the liposome.

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