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Title:
HETERONUCLEIC ACID INCLUDING 2'-MODIFIED NUCLEOSIDE
Document Type and Number:
WIPO Patent Application WO/2023/042876
Kind Code:
A1
Abstract:
A problem addressed is to provide a double-stranded nucleic acid complex with reduced central nervous system toxicity. Provided is a double-stranded nucleic acid complex including a first nucleic acid strand and a second nucleic acid strand, wherein the first nucleic acid strand can hybridize with at least a part of a target gene or a transcript thereof and has an antisense effect on the target gene or transcript thereof, and the second nucleic acid strand includes a base sequence complementary to that of the first nucleic acid strand and includes one or more 2'-modified nucleosides.

Inventors:
YOKOTA TAKANORI (JP)
YOSHIOKA KOTARO (JP)
Application Number:
PCT/JP2022/034531
Publication Date:
March 23, 2023
Filing Date:
September 15, 2022
Export Citation:
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Assignee:
UNIV NAT CORP TOKYO MEDICAL & DENTAL (JP)
International Classes:
A61K48/00; A61K31/713; A61K47/54; A61K47/64; A61P25/00; A61P43/00; C12N15/113
Foreign References:
JP2015502134A2015-01-22
Other References:
TAKAO INOUE, KIYOMI SASAKI, TOKUYUKI YOSHIDA: "Current status of development of oligonucleotide therapeutics", DRUG DELIVERY SYSTEM., NIHON D D S GAKKAI, JAPAN, vol. 34, no. 2, 25 March 2019 (2019-03-25), Japan , pages 86 - 98, XP009539425, ISSN: 0913-5006, DOI: 10.2745/dds.34.86
ASAMI YUTARO, NAGATA TETSUYA, YOSHIOKA KOTARO, KUNIEDA TAIKI, YOSHIDA-TANAKA KIE, BENNETT C. FRANK, SETH PUNIT P., YOKOTA TAKANORI: "Efficient Gene Suppression by DNA/DNA Double-Stranded Oligonucleotide In Vivo", MOLECULAR THERAPY, ELSEVIER INC., US, vol. 29, no. 2, 1 February 2021 (2021-02-01), US , pages 838 - 847, XP055834158, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2020.10.017
NISHIKAWA MAKIYA, YUKITAKE YOSHIOKA, MAKOTO NAGAOKA, KOSUKE KUSAMORI: "Development of Oligonucleotide Therapeutics: Tissue Distribution and Drug Delivery System", DDS. DRUG DELIVERY SYSTEM, vol. 36, no. 1, 25 January 2021 (2021-01-25), pages 40 - 50, XP093006747, DOI: 10.2745/dds.36.40
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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