AR127607A1 - DOUBLE CHAIN OLIGONUCLEOTIDE CONJUGATES OF RNAi MODULATORS OF THE EXPRESSION OF TARGET GENES - Google Patents
DOUBLE CHAIN OLIGONUCLEOTIDE CONJUGATES OF RNAi MODULATORS OF THE EXPRESSION OF TARGET GENESInfo
- Publication number
- AR127607A1 AR127607A1 ARP220103060A ARP220103060A AR127607A1 AR 127607 A1 AR127607 A1 AR 127607A1 AR P220103060 A ARP220103060 A AR P220103060A AR P220103060 A ARP220103060 A AR P220103060A AR 127607 A1 AR127607 A1 AR 127607A1
- Authority
- AR
- Argentina
- Prior art keywords
- expression
- antisense
- strand
- nucleotide
- target genes
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 3
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 108091030071 RNAI Proteins 0.000 title abstract 2
- 230000009368 gene silencing by RNA Effects 0.000 title abstract 2
- 230000000692 anti-sense effect Effects 0.000 abstract 5
- 239000002773 nucleotide Substances 0.000 abstract 5
- 125000003729 nucleotide group Chemical group 0.000 abstract 5
- 108091081021 Sense strand Proteins 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 125000003473 lipid group Chemical group 0.000 abstract 1
- 150000002632 lipids Chemical class 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
Landscapes
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
En el presente documento se proporcionan oligonucleótidos conjugados con lípidos que inhiben o reducen la expresión de genes diana. También se proporcionan composiciones que incluyen las mismas y usos de las mismas, en particular usos relacionados con el tratamiento de enfermedades, trastornos y/o condiciones asociadas con una disminución inducida por un desencadenante de ARNi en la expresión del gen diana. Reivindicación 1: Un oligonucleótido de doble cadena que comprende una cadena antisentido de aproximadamente 20 - 22 nucleótidos de longitud y una cadena con sentido de aproximadamente 8 - 20 nucleótidos de longitud, en donde las cadenas antisentido y sentido forman una región dúplex de aproximadamente 8 - 20 pares de bases, en donde la cadena antisentido comprende una orientación de 5 a 3, en donde la cadena antisentido comprende un saliente 5 de al menos un nucleótido y un saliente 3 de al menos un nucleótido, en donde la cadena antisentido comprende una región de complementariedad con una secuencia diana de ARNm, y en donde la cadena con sentido comprende al menos un resto lipídico conjugado con un nucleótido de la cadena con sentido.Provided herein are lipid-conjugated oligonucleotides that inhibit or reduce the expression of target genes. Also provided are compositions including the same and uses thereof, in particular uses related to the treatment of diseases, disorders and/or conditions associated with an RNAi trigger-induced decrease in the expression of the target gene. Claim 1: A double-stranded oligonucleotide comprising an antisense strand of approximately 20 - 22 nucleotides in length and a sense strand of approximately 8 - 20 nucleotides in length, wherein the antisense and sense strands form a duplex region of approximately 8 - 20 base pairs, wherein the antisense strand comprises a 5 to 3 orientation, wherein the antisense strand comprises a 5 overhang of at least one nucleotide and a 3 overhang of at least one nucleotide, wherein the strand antisense comprises a region of complementarity with a target mRNA sequence, and wherein the sense strand comprises at least one lipid residue conjugated to a nucleotide of the sense strand.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063340291P | 2020-05-10 | 2020-05-10 | |
| US202163277097P | 2021-11-08 | 2021-11-08 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR127607A1 true AR127607A1 (en) | 2024-02-14 |
Family
ID=90220687
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220103060A AR127607A1 (en) | 2020-05-10 | 2022-11-08 | DOUBLE CHAIN OLIGONUCLEOTIDE CONJUGATES OF RNAi MODULATORS OF THE EXPRESSION OF TARGET GENES |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR127607A1 (en) |
-
2022
- 2022-11-08 AR ARP220103060A patent/AR127607A1/en unknown
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| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |