Title:
NUCLEIC ACID MOLECULE CONTAINING 5'-CYCLOPROPYLENE MODIFICATION
Document Type and Number:
WIPO Patent Application WO/2024/071099
Kind Code:
A1
Abstract:
The present invention addresses the problem of providing a novel nucleic acid molecule capable of simultaneously achieving both a high gene regulatory effect and toxicity reduction. Provided is a nucleic acid molecule that contains a base sequence that is complementary to at least a portion of a target gene or a transcription product thereof and that has an antisense effect on the target gene or a transcription product thereof, the nucleic acid molecule: containing [1] a center region containing at least three continuous deoxyribonucleosides, [2] a 5' wing region that is disposed on a 5'-end side of the center region and that contains a nonnatural nucleoside, and [3] a 3' wing region that is disposed on a 3'-end side of the center region and that contains a nonnatural nucleoside; and containing a 5'-modified nucleoside represented by formula (I) at the first, third, and/or ninth base positions from the 5' side of the center region.
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Inventors:
YOKOTA TAKANORI (JP)
YOSHIOKA KOTARO (JP)
KURODA TAKAYUKI (JP)
OBIKA SATOSHI (JP)
YAMAGUCHI TAKAO (JP)
YOSHIOKA KOTARO (JP)
KURODA TAKAYUKI (JP)
OBIKA SATOSHI (JP)
YAMAGUCHI TAKAO (JP)
Application Number:
PCT/JP2023/034892
Publication Date:
April 04, 2024
Filing Date:
September 26, 2023
Export Citation:
Assignee:
UNIV NAT CORP TOKYO MEDICAL & DENTAL (JP)
UNIV OSAKA (JP)
UNIV OSAKA (JP)
International Classes:
C12N15/113; A61K31/712; A61K48/00; A61P25/00
Domestic Patent References:
| WO2022097727A1 | 2022-05-12 | |||
| WO2020158910A1 | 2020-08-06 | |||
| WO2021054370A1 | 2021-03-25 | |||
| WO2022234855A1 | 2022-11-10 | |||
| WO2022211095A1 | 2022-10-06 |
Foreign References:
| JP2015231978A | 2015-12-24 |
Other References:
YAMAGUCHI, TAKAO: "Decrease in hepatotoxicity by an incorporation of novel artificial nucleic acid into antisense oligonucleotide", LECTURE ABSTRACTS OF THE 7TH ANNUAL MEETING OF THE NUCLEIC ACIDS THERAPEUTICS SOCIETY OF JAPAN, OS1-06, 20 July 2022 (2022-07-20), pages 119
KAWANOBE, TAKAAKI ET AL.: "The effect of 5’-cyclopropylene DNA modification in gapmer antisense oligonucleotides", LECTURE ABSTRACTS OF THE 7TH ANNUAL MEETING OF THE NUCLEIC ACIDS THERAPEUTICS SOCIETY OF JAPAN, O2-02, 20 July 2022 (2022-07-20), pages 71
KAWANOBE, TAKAAKI ET AL.: "The effect of 5’-cyclopropylene DNA modification in gapmer antisense oligonucleotides", LECTURE ABSTRACTS OF THE 7TH ANNUAL MEETING OF THE NUCLEIC ACIDS THERAPEUTICS SOCIETY OF JAPAN, O2-02, 20 July 2022 (2022-07-20), pages 71
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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