Chemically Modified RNAi Constructs and Their Uses
Chemically modified RNAi constructs with specific nucleotide patterns improve in vivo efficacy and stability, addressing the limitations of existing RNAi constructs by achieving potent and long-lasting gene silencing.
KR102999190B1Active Publication Date: 2026-08-05AMGEN INC
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Patent Information
- Application Number
- KR1020217020966
- Authority / Receiving Office
- KR · KR
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2018-12-10
- Filing Date
- 2019-12-09
- Publication Date
- 2026-08-05
- Estimated Expiration
- 2039-12-09
AI Technical Summary
Technical Problem
Existing RNAi constructs face challenges with reduced efficacy and stability in vivo, limiting their therapeutic potential for gene silencing applications.
Method used
Chemically modified RNAi constructs with specific patterns of nucleotide modifications, including 2′-fluoro and other modified nucleotides, enhance the efficacy and stability of RNAi constructs for in vivo gene silencing.
Benefits of technology
The modified RNAi constructs achieve potent and long-lasting knockdown of target gene expression, improving therapeutic outcomes by enhancing stability and duration of gene silencing activity.
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Abstract
The present invention relates to a chemically modified RNAi construct for reducing the expression of a target gene. In particular, the present invention relates to a specific pattern of modified nucleotides incorporated into the RNAi construct to improve in vivo stability and efficacy. Additionally, a pharmaceutical composition comprising a chemically modified RNAi construct and a method for inhibiting target gene expression in vivo by administering the chemically modified RNAi construct, for example, to treat or improve various disease conditions are described.
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