DK2633052T3 - Behandling af interferonrelateret udviklingsregulator 1 (ifrd1)-relaterede sygdomme ved inhibering af naturligt antisense-transkript til ifrd1 - Google Patents

Behandling af interferonrelateret udviklingsregulator 1 (ifrd1)-relaterede sygdomme ved inhibering af naturligt antisense-transkript til ifrd1 Download PDF

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DK2633052T3
DK2633052T3 DK11836985.9T DK11836985T DK2633052T3 DK 2633052 T3 DK2633052 T3 DK 2633052T3 DK 11836985 T DK11836985 T DK 11836985T DK 2633052 T3 DK2633052 T3 DK 2633052T3
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oligonucleotide
ifrd1
disease
antisense
nucleic acid
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Joseph Collard
Sherman Olga Khorkova
Carlos Coito
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Curna Inc
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Claims (16)

1. Oligonukleotid, der har et interferonrelateret udviklingsregulator 1 (IFRD1)-naturligt antisense-transkript som mål til anvendelse som en terapeutisk forbindelse, hvor oligonukleotidet forøger ekspressionen af interferonrelateret udviklingsregulator 1 (IFRD1); hvor det naturlige antisense-transkript har nukleinsyresekvensen som angivet i én af sekvenserne SEQ ID NO: 2 til 6.
2. Oligonukleotid, der har et interferonrelateret udviklingsregulator 1 (IFRD1)-naturligt antisense-transkript som mål til anvendelse ved forebyggelse eller behandling afen interferonrelateret udviklingsregulator 1 (IFRDI)-forbundet sygdom eller lidelse, hvor oligonukleotidet forøger ekspressionen af interferonrelateret udviklingsregulator 1 (IFRD1); hvor det naturlige antisense-transkript har nukleinsyresekvensen som angivet i én af sekvenserne SEQ ID NO: 2 til 6.
3. Oligonukleotid til anvendelse ifølge krav 2, hvor sygdommen eller lidelsen er valgt fra gruppen bestående af en sygdom eller lidelse, der er forbundet med unormal funktion og/eller ekspression af "interferonrelateret udviklingsregulator 1"; cystisk fibrose; kronisk obstruktiv lungesygdom (KOL); en sygdom eller lidelse, der er forbundet med differentiering og regenerering af væv; en sygdom eller lidelse, der er forbundet med muskelregenerering; en sygdom eller lidelse, der er forbundet med skeletmuskelregenerering; en sygdom eller lidelse, der er forbundet med muskeldifferentiering; en sygdom eller lidelse, der er forbundet med myogen differentiering; oxidativt stress, en inflammatorisk sygdom eller lidelse; inflammation; en postkiurgisk tilstand, der er forbundet med intestinalt epitel; en immunsystemsygdom eller -lidelse; en lungesygdom eller -lidelse; en sygdom eller lidelse, der er forbundet med unormal metabolisk hastighed, adipositas og intestinal triglyceridabsorption; lungecancer; sensorisk/motorisk neuropati; en neuronskade, der er forbundet med cerebral iskæmi; en neuronskade og en neurologisk sygdom eller lidelse.
4. Oligonukleotid til anvendelse ifølge krav 3, hvor sygdommen eller lidelsen er cystisk fibrose.
5. Oligonukleotid til anvendelse ifølge krav 3, hvor sygdommen eller lidelsen er en sygdom eller lidelse, der er forbundet med muskelregenerering.
6. In w'/ro-fremgangsmåde til forøgelse af ekspressionen af interferonrelateret udviklingsregulator 1 (IFRD1) i patientceller eller -væv, hvilken fremgangsmåde omfatter, at: cellerne eller vævene bringes i kontakt med et oligonukleotid, der har et interferonrelateret udviklingsregulator 1 (IFRDI)-naturligt antisense-transkript som mål; hvorved ekspressionen af interferonrelateret udviklingsregulator 1 (IFRD1) forøges; hvor det naturlige antisense-transkript har nukleinsyresekvensen som angivet i én af sekvenserne SEQ ID NO: 2 til 6.
7. Oligonukleotid, der har et interferonrelateret udviklingsregulator 1 (IFRD1)-naturligt antisense-transkript som mål, hvor oligonukleotidet forøger ekspressionen af interferonrelateret udviklingsregulator 1 (IFRD1); hvor det naturlige antisense-transkript har nukleinsyresekvensen som angivet i én af sekvenserne SEQ ID NO: 2 til 6.
8. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5 eller fremgangsmåde ifølge krav 6 eller oligonukleotid ifølge krav 7, hvor oligonukleotidet er mellem 10 og 30 nukleotider langt.
9. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5 eller 8 eller fremgangsmåde ifølge krav 6 eller krav 8 eller oligonukleotid ifølge krav 7 eller krav 8, hvor oligonukleotidet har mindst 90 % sekvensidentitet med et komplement af et interferonrelateret udviklingsregulator 1 (IFRD1 )-naturligt antisense-transkript.
10. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5, 8 eller 9 eller fremgangsmåde ifølge et hvilket som helst af kravene 6, 8 eller 9 eller oligonukleotid ifølge et hvilket som helst af kravene 7 til 9, hvor oligonukleotidet er enkeltstrenget.
11. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5, 8 eller 9 eller fremgangsmåde ifølge et hvilket som helst af kravene 6, 8 eller 9 eller oligonukleotid ifølge et hvilket som helst af kravene 7 til 9, hvor oligonukleotidet er en siRNA-forbindelse.
12. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5 eller 8 til 11 eller fremgangsmåde ifølge et hvilket som helst af kravene 6 eller 8 til 11 eller oligonukleotid ifølge et hvilket som helst af kravene 7 til 11, hvor oligonukleotidet omfatter SEQ ID NO: 9.
13. Oligonukleotid til anvendelse ifølge krav 11 eller fremgangsmåde ifølge krav 11 eller oligonukleotid ifølge krav 11, hvor siRNA-forbindelsen omfatter et oligonukleotid angivet som SEQ ID NO: 9 og en revers komplement-sekvens for oligonukleotidet angivet som SEQ ID NO: 18.
14. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5 eller 8 til 13 eller fremgangsmåde ifølge et hvilket som helst af kravene 6 eller 8 til 13 eller oligonukleotid ifølge et hvilket som helst af kravene 7 til 13, hvor ekspressionen af interferonrelateret udviklingsregulator 1 (IFRD1) forøges med mindst 10 %.
15. Oligonukleotid til anvendelse ifølge et hvilket som helst af kravene 1 til 5 eller 8 til 14 eller fremgangsmåde ifølge et hvilket som helst af kravene 6 eller 8 til 14 eller oligonukleotid ifølge et hvilket som helst af kravene 7 til 14, hvor oligonukleotidet endvidere omfatter én eller flere modifikationer, som omfatter: a. mindst én modificeret internukleosidbinding, som er valgt blandt: et phosphorothioat, alkylphosphonat, phosphorodithioat, alkylphosphonothioat, phosphoramidat, carbamat, carbonat, phosphattriester, acetamidat, carboxymethylester og kombinationer deraf; b. mindst ét modificeret nukleotid, som er valgt blandt: en peptidnukleinsyre (PNA), en låst nukleinsyre (LNA), en arabino-nukleinsyre, en analog, et derivat og kombinationer deraf; eller c. mindst én modificeret sukkergruppe, som er valgt blandt: en 2'-0-methoxyethyl-modificeret sukkergruppe, en 2'-fluor-modificeret sukkergruppe, en 2'-methoxy-modificeret sukkergruppe, en 2'-0-alkyl-modificeret sukkergruppe, en bicyklisk sukkergruppe og kombinationer deraf.
16. Farmaceutisk sammensætning, der omfatter et oligonukleotid ifølge et hvilket som helst af kravene 7 til 15 og en farmaceutisk acceptabel excipiens.
DK11836985.9T 2010-10-27 2011-10-26 Behandling af interferonrelateret udviklingsregulator 1 (ifrd1)-relaterede sygdomme ved inhibering af naturligt antisense-transkript til ifrd1 DK2633052T3 (da)

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