AR107824A1 - ADDRESSING LINKS FOR THERAPEUTIC COMPOUNDS - Google Patents
ADDRESSING LINKS FOR THERAPEUTIC COMPOUNDSInfo
- Publication number
- AR107824A1 AR107824A1 ARP170100566A ARP170100566A AR107824A1 AR 107824 A1 AR107824 A1 AR 107824A1 AR P170100566 A ARP170100566 A AR P170100566A AR P170100566 A ARP170100566 A AR P170100566A AR 107824 A1 AR107824 A1 AR 107824A1
- Authority
- AR
- Argentina
- Prior art keywords
- galactosamine
- expression
- targeting ligands
- butanoylgalactosamine
- integer
- Prior art date
Links
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Los ligandos de direccionamiento divulgados en la presente pueden servir para dirigir los compuestos oligoméricos que inhiben la expresión, como agentes de ARNi, a células hepáticas para modular la expresión génica. Los ligandos de direccionamiento descritos en la presente, al conjugarse a un compuesto oligomérico que inhibe la expresión, se pueden usar en una variedad de aplicaciones, incluyendo el uso en aplicaciones terapéuticas, de diagnóstico, validación de diana y descubrimiento genómico. Las composiciones que incluyen los ligandos de direccionamiento divulgados en la presente, al unirse a compuestos oligoméricos que inhiben la expresión, son capaces de mediar la expresión de secuencias de ácido nucleico diana en células hepáticas, como hepatocitos, que pueden ser útiles en el tratamiento de enfermedades o afecciones que responden a la inhibición de la expresión génica o la actividad en una célula, tejido u organismo. Reivindicación 1: Un ligando de direccionamiento que comprende la estructura de fórmula (1), donde n es un entero de 1 a 20; X es O, S, o NH y el resto de direccionamiento se selecciona del grupo que consiste en: N-acetil-galactosamina, galactosa, galactosamina, N-formil-galactosamina, N-propionil-galactosamina, N-n-butanoilgalactosamina y N-iso-butanoilgalactosamina. Reivindicación 18: Un compuesto que tiene la estructura de fórmula (2), donde n es un entero de 1 a 20.The targeting ligands disclosed herein may serve to direct oligomeric compounds that inhibit expression, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands described herein, when conjugated to an oligomeric compound that inhibits expression, can be used in a variety of applications, including use in therapeutic, diagnostic, target validation and genomic discovery applications. Compositions that include the targeting ligands disclosed herein, by binding to oligomeric compounds that inhibit expression, are capable of mediating the expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to the inhibition of gene expression or activity in a cell, tissue or organism. Claim 1: An addressing ligand comprising the structure of formula (1), wherein n is an integer from 1 to 20; X is O, S, or NH and the targeting moiety is selected from the group consisting of: N-acetyl-galactosamine, galactose, galactosamine, N-formyl-galactosamine, N-propionyl-galactosamine, Nn-butanoylgalactosamine and N-iso -butanoylgalactosamine. Claim 18: A compound having the structure of formula (2), wherein n is an integer from 1 to 20.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662304652P | 2016-03-07 | 2016-03-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR107824A1 true AR107824A1 (en) | 2018-06-06 |
Family
ID=62683527
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP170100566A AR107824A1 (en) | 2016-03-07 | 2017-03-07 | ADDRESSING LINKS FOR THERAPEUTIC COMPOUNDS |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR107824A1 (en) |
-
2017
- 2017-03-07 AR ARP170100566A patent/AR107824A1/en unknown
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