US2017327824A1PendingUtilityA1
Selective antisense compounds and uses thereof
Est. expiryOct 12, 2032(~6.2 yrs left)· nominal 20-yr term from priority
A61P 25/28C12N 2310/345C07H 21/02C12N 2310/3125C12N 2310/335C12N 2310/3231A61P 25/14C12N 2310/346C12N 2310/3341C12N 2310/341C12N 2310/11C12N 2310/315C12N 2320/34C12N 2310/313C12N 15/113
62
PatentIndex Score
0
Cited by
0
References
0
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: 24, 3, 67, 70, 71, 73, 77, 80, 91, or 577.
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: 24.
44 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 3.
45 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 67.
46 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 70.
47 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 71.
48 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 73.
49 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 77.
50 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 80.
51 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 91.
52 . The compound of claim 42 , wherein the modified oligonucleotide has the nucleobase sequence recited in SEQ ID NO: 577.
53 . The compound of claim 43 , wherein the oligonucleotide has a eeeekk-d7-kke sugar motif.
54 . The compound of claim 44 , wherein the oligonucleotide has a sugar motif selected from among: ekk-d-f-kke, ekk-d7-kkeee, ekk-d2-h-d6-kke, ekk-d3-z-d5-kke, ekk-d6-z-d2-kke, ekk-d8-z-kke, or ekk-d9-kke.
55 . The compound of claim 45 , wherein the oligonucleotide has a ekek-d9-keee sugar motif.
56 . The compound of claim 46 , wherein the oligonucleotide has a eeeeek-d7-kke sugar motif.
57 . The compound of claim 47 , wherein the oligonucleotide has a eeeekk-d7-kee sugar motif.
58 . The compound of claim 48 , wherein the oligonucleotide has a eeeeek-d7-kee sugar motif.
59 . The compound of claim 49 , wherein the oligonucleotide has a eeeeek-d7-eeeeee sugar motif.
60 . The compound of claim 50 , wherein the oligonucleotide has a eek-d7-eeeeeeeee sugar motif.
61 . The compound of claim 51 , wherein the oligonucleotide has a ekek-d9-keke sugar motif.
62 . The compound of claim 52 , wherein the oligonucleotide has a ekk-d2-z-d6-kke sugar motif.
63 . 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
Track US2017327824A1 — get alerts on status changes and closely related new filings.
We store only your email — no account needed. See our privacy policy.