Methods for treatment of polycystic kidney disease
US-2024141350-A1 · May 2, 2024 · US
US12559750B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12559750-B2 |
| Application number | US-202519052052-A |
| Country | US |
| Kind code | B2 |
| Filing date | Feb 12, 2025 |
| Priority date | Oct 8, 2021 |
| Publication date | Feb 24, 2026 |
| Grant date | Feb 24, 2026 |
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Provided herein are methods for the treatment of polycystic kidney disease, including autosomal dominant polycystic kidney disease, using modified oligonucleotides targeted to miR-17.
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What is claimed is: 1 . A modified oligonucleotide having the structure: or a pharmaceutically acceptable salt thereof. 2 . The modified oligonucleotide of claim 1 , wherein the pharmaceutically acceptable salt is a sodium salt. 3 . A modified oligonucleotide having the structure: 4 . A pharmaceutical composition comprising the modified oligonucleotide of claim 1 and a pharmaceutically acceptable diluent. 5 . The pharmaceutical composition of claim 4 , wherein the pharmaceutically acceptable diluent is an aqueous solution. 6 . The pharmaceutical composition of claim 5 , wherein the aqueous solution is a saline solution. 7 . A pharmaceutical composition comprising a modified oligonucleotide of claim 2 and a pharmaceutically acceptable diluent. 8 . The pharmaceutical composition of claim 7 , wherein the pharmaceutically acceptable diluent is an aqueous solution. 9 . The pharmaceutical composition of claim 8 , wherein the aqueous solution is a saline solution. 10 . A pharmaceutical composition comprising a modified oligonucleotide of claim 3 and a pharmaceutically acceptable diluent. 11 . The pharmaceutical composition of claim 10 , wherein the pharmaceutically acceptable diluent is an aqueous solution. 12 . The pharmaceutical composition of claim 11 , wherein the aqueous solution is a saline solution. 13 . A pharmaceutical composition comprising a modified oligonucleotide of claim 1 , which is a lyophilized composition. 14 . A pharmaceutical composition comprising a modified oligonucleotide of claim 2 , which is a lyophilized composition. 15 . A pharmaceutical composition comprising a modified oligonucleotide of claim 3 , which is a lyophilized composition.
5-Methylcytosine · CPC title
2'-R Modification · CPC title
2'-O-R Modification · CPC title
Phosphorothioates · CPC title
targeting other non-coding nucleic acids, e.g. antagomirs · CPC title
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