US2019276823A1PendingUtilityA1
Selective antisense compounds and uses thereof
Est. expiryOct 12, 2032(~6.2 yrs left)· nominal 20-yr term from priority
C12N 2310/345C07H 21/02C12N 2310/346A61P 25/14A61P 25/28C12N 2310/3125C12N 2310/335C12N 2310/3231C12N 2310/3341C12N 2320/34C12N 2310/11C12N 2310/313C12N 2310/341C12N 2310/315C12N 15/113
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Claims
Abstract
The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.
Claims
exact text as granted — not AI-modified1 .- 40 . (canceled)
41 . A compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the linked nucleosides comprise at least 8 contiguous nucleobases of a nucleobase sequence recited in SEQ ID NO: 5, 6, 27, 28, 29, 30, 38, 39, or 46.
42 . The compound of claim 41 , wherein the nucleobase sequence of the modified oligonucleotide is 100% complementary to SEQ ID NO. 1.
43 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 5.
44 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 6.
45 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 27.
46 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 28.
47 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 29.
48 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 30.
49 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 38.
50 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 39.
51 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 46.
52 . The compound of claim 43 , wherein the oligonucleotide has a ekek-d9-keke sugar motif.
53 . The compound of claim 44 , wherein the oligonucleotide has a kekk-d8-keke sugar motif.
54 . The compound of claim 45 , wherein the oligonucleotide has a ekkk-d8-kke sugar motif.
55 . The compound of claim 46 , wherein the oligonucleotide has a ekk-d8-kkke sugar motif.
56 . The compound of claim 47 , wherein the oligonucleotide has a ekekk-d8-keke sugar motif.
57 . The compound of claim 48 , wherein the oligonucleotide has a ekek-d8-kkeke sugar motif.
58 . The compound of claim 49 , wherein the oligonucleotide has a eekk-d8-kkeee sugar motif.
59 . The compound of claim 50 , wherein the oligonucleotide has a eekk-d8-kkeee sugar motif.
60 . The compound of claim 51 , wherein the oligonucleotide has a eeekk-d8-kkee sugar motif.
61 . The compound of claim 41 , comprising at least one phosphorothioate internucleoside linkage.
62 . A method of ameliorating a symptom of Huntington's disease, comprising administering an oligomeric compound of claim 41 to an animal in need thereof.Join the waitlist — get patent alerts
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