Modified Double-Stranded RNA Agents Inhibit Gene Expression
Summary
USPTO published patent application US20260098263A1 for modified double-stranded RNA (dsRNA) agents capable of inhibiting gene expression. The application was published April 9, 2026, with inventors Martin MAIER, Don FOSTER, Stuart MILSTEIN, Satya KUCHIMANCHI, Vasant JADHAV, Kallanthottathil RAJEEV, Muthiah MANOHARAN, and Rubina PARMAR. The dsRNA agents contain thermally destabilizing nucleotides positioned opposite the seed region of the antisense strand and modified nucleotides with reduced steric bulk, with pharmaceutical compositions and therapeutic methods for treating disease conditions also disclosed.
What changed
USPTO published application US20260098263A1 covering modified double-stranded RNA (dsRNA) agents designed to inhibit gene expression. The dsRNA agents feature sense strands with thermally destabilizing nucleotides positioned opposite the antisense strand seed region (positions 2-8), and antisense strands containing modified nucleotides with steric bulk equal to or less than 2'-OMe modifications, separated by 11 nucleotides. The application also discloses pharmaceutical compositions suitable for therapeutic use and methods for treating disease conditions by administering these agents. This is a published application, not a granted patent—enforceability rights have not yet attached.
Stakeholders in the RNAi therapeutics space should monitor prosecution of this application for potential licensing opportunities or to assess freedom-to-operate implications. The scope of any eventual granted claims may significantly impact competitive dynamics in gene-silencing therapeutics.
What to do next
- Monitor for patent grant status
- Review claims for freedom-to-operate implications
Archived snapshot
Apr 13, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
MODIFIED DOUBLE-STRANDED RNA AGENTS
Application US20260098263A1 Kind: A1 Apr 09, 2026
Inventors
Martin MAIER, Don FOSTER, Stuart MILSTEIN, Satya KUCHIMANCHI, Vasant JADHAV, Kallanthottathil RAJEEV, Muthiah MANOHARAN, Rubina PARMAR
Abstract
One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises. at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′—OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.
CPC Classifications
C12N 15/113 C12N 15/111 C12N 2310/14 C12N 2310/313 C12N 2310/32 C12N 2310/3231 C12N 2310/332 C12N 2310/335 C12N 2310/344 C12N 2310/346 C12N 2310/351 C12N 2310/533 C12N 2320/51 C12N 2320/52
Filing Date
2025-03-13
Application No.
19079175
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