US2021180063A1PendingUtilityA1
Anti-arid3a treatments for inflammatory disorders
Est. expiryJul 15, 2036(~10 yrs left)· nominal 20-yr term from priority
A61P 37/02A61P 31/12C12N 15/113C12N 2310/3233C12N 2310/314C12N 2310/11C12N 2310/351
49
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Claims
Abstract
Antisense compounds for suppressing expression of ARID3a are disclosed, as well as pharmaceutical compositions containing same and methods of producing and using same. The antisense compounds can be used to treat inflammatory disorders and conditions related to interferon-alpha production.
Claims
exact text as granted — not AI-modifiedWhat is claimed is:
1 . An antisense compound, comprising:
an oligonucleotide consisting of 18-50 linked nucleosides, the oligonucleotide comprising a targeting sequence that is complementary and specifically hybridizable to a target sequence of an ARID3a nucleic acid selected from the group consisting of SEQ ID NOS:1-28, the target sequence comprising at least 18 contiguous nucleobases, and wherein the oligonucleotide is non-natural.
2 . The antisense compound of claim 1 , wherein the oligonucleotide comprises at least one non-natural internucleoside linkage.
3 . The antisense compound of claim 2 , wherein the at least one non-natural internucleoside linkage of the oligonucleotide is selected from the group consisting of phosphoramidite, phosphorodiamidate, phosphorothioate, phosphorodithioate, phosphotriester, aminoalkylphosphotriester, alkyl phosphonate, chiral phosphonate, phosphinate, aminoalkylphosphoramidate, thiono-phosphoramidate, thionoalkylphosphonate, thionoalkylphospho-triester, selenophosphate, boranophosphate, and peptide linkages.
4 . The antisense compound of claim 1 , wherein the oligonucleotide comprises at least one nucleoside having a modified sugar.
5 . The antisense compound of claim 1 , wherein the oligonucleotide comprises a phosphoroamidate backbone.
6 . The antisense compound of claim 1 , wherein the oligonucleotide comprises a phosphorodiamidate morpholino backbone.
7 . The antisense compound of claim 1 , wherein the oligonucleotide comprises at least one non-natural nucleobase.
8 . The antisense compound of claim 1 , wherein the targeting sequence is complementary and specifically hybridizable to a target sequence of a nucleic acid selected from the group consisting of SEQ ID NOS:1, 14, 17, and 27.
9 . The antisense compound of claim 8 , wherein the target sequence is SEQ ID NO:1.
10 . The antisense compound of claim 8 , wherein the target sequence is SEQ ID NO:14.
11 . The antisense compound of claim 8 , wherein the target sequence is SEQ ID NO:17.
12 . The antisense compound of claim 8 , wherein the target sequence is SEQ ID NO:27.
13 . The antisense compound of claim 1 , wherein the oligonucleotide hybridizes to at least one of an ARID3a precursor mRNA and an ARID3a mature mRNA.
14 . The antisense compound of claim 1 , wherein the antisense compound further comprises a cell penetrating moiety linked to the oligonucleotide.
15 . The antisense compound of claim 1 , wherein the oligonucleotide consists of 18-40 nucleobases.
16 . The antisense compound of claim 1 , wherein the oligonucleotide consists of 20-30 nucleobases.
17 . The antisense compound of claim 1 , wherein the oligonucleotide consists of 22-28 nucleobases.
18 . The antisense compound of claim 1 , wherein the targeting sequence is at least 84% complementary to the target sequence.
19 . The antisense compound of claim 1 , wherein the targeting sequence is at least 90% complementary to the target sequence.
20 . A pharmaceutical composition, comprising the antisense compound of claim 1 disposed in a pharmaceutically-acceptable diluent, vehicle, or carrier.Join the waitlist — get patent alerts
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