Short interfering nucleic acid (siNA) compositions
US-11530407-B2 · Dec 20, 2022 · US
Short interfering nucleic acid (siNA) compositions
US12486503B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12486503-B2 |
| Application number | US-202217984486-A |
| Country | US |
| Kind code | B2 |
| Filing date | Nov 10, 2022 |
| Priority date | May 2, 2012 |
| Publication date | Dec 2, 2025 |
| Grant date | Dec 2, 2025 |
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Abstract
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The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
First claim
Opening claim text (preview).
The invention claimed is: 1 . A composition comprising: a double-stranded short interfering nucleic acid (siNA) molecule that modulates expression of a target RNA sequence via RNA interference, wherein the molecule has a passenger strand and a guide strand and comprises structure selected from the structure represented by formula (A): 5′- B 1 -N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′- B 3 -( N s ) y N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (A); formula (B): 5′-B 1 -N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (B); formula (C): 5′-B 1 -N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (C); formula (D): 5′-B 1 -N N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (D); formula (E): 5′-B 1 -N N N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (E); formula (F): 5′-B 1 -N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (F); formula (G): 5′-B 1 -N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (G); formula (H): 5′-B 1 -N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (H); formula (I): 5′-B 1 -N N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (I); formula (J): 5′-B 1 -N N N N N N N N N N N N N N N N N N N N N N ( s N) x -B 2 3′-B 3 -(N s ) y N N N N N N N N N N N N N N N N N N N s 3 N s 2 N s 1 N (J); wherein, (a) the upper strand is the passenger strand and the lower strand is the guide strand of the double-stranded nucleic acid molecule; the guide strand is complementary to the target sequence and the passenger strand is complementary to the guide strand, wherein one or more mismatches between the guide strand and passenger strand and/or between the guide strand and the target sequence are tolerated so long as RNAi activity is maintained; (b) each N is independently a nucleotide wherein one or more Ns
Assignees
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Classifications
2'-O-R Modification · CPC title
modulating the chemical stability, e.g. nuclease-resistance · CPC title
Special delivery means, e.g. tissue-specific · CPC title
Conjugate · CPC title
having a combination of backbone and sugar modifications · CPC title
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Frequently asked questions
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- What does patent US12486503B2 cover?
- The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interferin…
- Who is the assignee on this patent?
- Sirna Therapeutics Inc
- What technology area does this patent fall under?
- Primary CPC classification C12N15/113. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Dec 02 2025 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 9 related publications on this page (citations in our corpus or others sharing the same primary CPC).