Title:
THERAPEUTIC AGENT FOR CARDIAC DISEASE
Document Type and Number:
WIPO Patent Application WO/2022/250072
Kind Code:
A1
Abstract:
The present invention provides a therapeutic agent for cardiac disease that includes an oligonucleotide having one or more nucleotides represented by formula I or formula II below: (in formulas I and II: Base represents a 2-oxo-1, 2-dihydropyrimidine-1-yl group or purine-9-yl group that may have one or more arbitrary substituents selected from an α group; the α group comprises hydroxyl groups, hydroxyl groups protected by a nucleic-acid-synthesis protective group, C1 to C6 straight-chain alkyl groups, C1 to C6 straight-chain alkoxy groups, mercapto groups, mercapto groups protected by a nucleic-acid-synthesis protective group, C1 to C6 straight-chain alkylthio groups, amino groups, C1 to C6 straight-chain alkylamino groups, amino groups protected by a nucleic-acid-synthesis protective group, and halogen atoms; R1, R12, and R13 each independently represent a hydrogen atom, a C1 to C7 alkyl group that may form a branch or ring, or a protective group for an amino group; and R14 represents a hydrogen atom).
Inventors:
MIYASAKA YUI (JP)
SHRESTHA AJAYA RAM (JP)
CHIBA KOSUKE (JP)
KAWANOBE TAKAAKI (JP)
SHRESTHA AJAYA RAM (JP)
CHIBA KOSUKE (JP)
KAWANOBE TAKAAKI (JP)
Application Number:
PCT/JP2022/021330
Publication Date:
December 01, 2022
Filing Date:
May 25, 2022
Export Citation:
Assignee:
LUXNA BIOTECH CO LTD (JP)
International Classes:
A61K31/7088; A61K48/00; A61P9/04; A61P43/00; C07K14/47; C12N15/11; C12N15/13
Domestic Patent References:
| WO2014046212A1 | 2014-03-27 | |||
| WO2017047816A1 | 2017-03-23 |
Other References:
KUMAGAI SHINJI, SAWAMOTO HIROAKI, TAKEGAWA-ARAKI TOMO, ARAI YUUKI, YAMAKOSHI SHUHEI, YAMADA KATSUYA, OHTA TETSUYA, KAWANISHI EIJI,: "Synthesis and properties of GuNA purine/pyrimidine nucleosides and oligonucleotides", ORGANIC & BIOMOLECULAR CHEMISTRY, ROYAL SOCIETY OF CHEMISTRY, vol. 18, no. 46, 7 December 2020 (2020-12-07), pages 9461 - 9472, XP093008825, ISSN: 1477-0520, DOI: 10.1039/D0OB01970D
AJAYA R. SHRESTHA, YUTARO KOTOBUKI, YOSHIYUKI HARI, SATOSHI OBIKA: "Guanidine bridged nucleic acid (GuNA): an effect of a cationic bridged nucleic acid on DNA binding affinity", CHEMICAL COMMUNICATIONS, ROYAL SOCIETY OF CHEMISTRY, UK, vol. 50, no. 5, 1 January 2014 (2014-01-01), UK , pages 575 - 577, XP055246151, ISSN: 1359-7345, DOI: 10.1039/C3CC46017G
NAOHIRO HORIE, SHINJI KUMAGAI, YUTARO KOTOBUKI, TAKAO YAMAGUCHI, SATOSHI OBIKA: "Facile synthesis and fundamental properties of an N -methylguanidine-bridged nucleic acid (GuNA[NMe])", ORGANIC & BIOMOLECULAR CHEMISTRY, ROYAL SOCIETY OF CHEMISTRY, vol. 16, no. 35, 1 January 2018 (2018-01-01), pages 6531 - 6536, XP055754022, ISSN: 1477-0520, DOI: 10.1039/C8OB01307A
MASAKI YAMAGAMI, TAKURO KOBORI, TAKAO SUZUKI, KAZUO SEKIGUCHI, HIDEAKI SATO, NAOHIRO HORIE, RYUSUKE HATANAKA, TAKAO YAMAGUCHI, SAT: "Synthesis and Properties of Antisense Oligonucleotides Containing AmNA and GuNA", THE 46TH INTERNATIONAL SYMPOSIUM ON NUCLEIC ACIDS CHEMISTRY 2019. THE 3RD ANNUAL MEETING OF JAPAN SOCIETY OF NUCLEIC ACIDS CHEMISTRY. ISNAC 2019; OCTOBER 29-31, 2019, JAPAN SOCIETY OF NUCLEIC ACIDS CHEMISTRY, JP, vol. 46, 1 January 2019 (2019-01-01) - 31 October 2019 (2019-10-31), JP, pages 184 - 185, XP009541587
YOKO HIRAKAWA, FUMIKO YAMAIRI, TAKASHI KURITA, KARIN MURAHASHI, TAKASHI TATEOKA, TETSUYA OTA, TAKASHI SASAKI, JUN KODERA, SATOSHI : "P-069 Evaluation of biological activity and safety of guanidine-bridged artificial nucleic acid (GuNA)-loaded Malat1 antisense oligonucleotides", ABSTRACTS OF THE 5TH ANNUAL MEETING OF THE NUCLEIC ACID MEDICINE SOCIETY OF JAPAN; JULY 10-12, 2019, NUCLEIC ACID MEDICINE SOCIETY OF JAPAN, JP, 1 January 2019 (2019-01-01) - 12 July 2019 (2019-07-12), JP, pages 149, XP009541527
MIYASAKA, YUI ET AL.: "2P-23 The knockdown activity of GuNA[t-Bu]-gapmer antisense oligonucleotides in heart", ABSTRACTS OF THE 6TH ANNUAL MEETING OF THE JAPANESE SOCIETY OF NUCLEIC ACID MEDICINE; JUNE 27 - JUNE 29, 2020, JAPANESE SOCIETY OF NUCLEIC ACID MEDICINE, JP, 18 June 2021 (2021-06-18) - 29 June 2020 (2020-06-29), JP, pages 155, XP009541528
AJAYA R. SHRESTHA, YUTARO KOTOBUKI, YOSHIYUKI HARI, SATOSHI OBIKA: "Guanidine bridged nucleic acid (GuNA): an effect of a cationic bridged nucleic acid on DNA binding affinity", CHEMICAL COMMUNICATIONS, ROYAL SOCIETY OF CHEMISTRY, UK, vol. 50, no. 5, 1 January 2014 (2014-01-01), UK , pages 575 - 577, XP055246151, ISSN: 1359-7345, DOI: 10.1039/C3CC46017G
NAOHIRO HORIE, SHINJI KUMAGAI, YUTARO KOTOBUKI, TAKAO YAMAGUCHI, SATOSHI OBIKA: "Facile synthesis and fundamental properties of an N -methylguanidine-bridged nucleic acid (GuNA[NMe])", ORGANIC & BIOMOLECULAR CHEMISTRY, ROYAL SOCIETY OF CHEMISTRY, vol. 16, no. 35, 1 January 2018 (2018-01-01), pages 6531 - 6536, XP055754022, ISSN: 1477-0520, DOI: 10.1039/C8OB01307A
MASAKI YAMAGAMI, TAKURO KOBORI, TAKAO SUZUKI, KAZUO SEKIGUCHI, HIDEAKI SATO, NAOHIRO HORIE, RYUSUKE HATANAKA, TAKAO YAMAGUCHI, SAT: "Synthesis and Properties of Antisense Oligonucleotides Containing AmNA and GuNA", THE 46TH INTERNATIONAL SYMPOSIUM ON NUCLEIC ACIDS CHEMISTRY 2019. THE 3RD ANNUAL MEETING OF JAPAN SOCIETY OF NUCLEIC ACIDS CHEMISTRY. ISNAC 2019; OCTOBER 29-31, 2019, JAPAN SOCIETY OF NUCLEIC ACIDS CHEMISTRY, JP, vol. 46, 1 January 2019 (2019-01-01) - 31 October 2019 (2019-10-31), JP, pages 184 - 185, XP009541587
YOKO HIRAKAWA, FUMIKO YAMAIRI, TAKASHI KURITA, KARIN MURAHASHI, TAKASHI TATEOKA, TETSUYA OTA, TAKASHI SASAKI, JUN KODERA, SATOSHI : "P-069 Evaluation of biological activity and safety of guanidine-bridged artificial nucleic acid (GuNA)-loaded Malat1 antisense oligonucleotides", ABSTRACTS OF THE 5TH ANNUAL MEETING OF THE NUCLEIC ACID MEDICINE SOCIETY OF JAPAN; JULY 10-12, 2019, NUCLEIC ACID MEDICINE SOCIETY OF JAPAN, JP, 1 January 2019 (2019-01-01) - 12 July 2019 (2019-07-12), JP, pages 149, XP009541527
MIYASAKA, YUI ET AL.: "2P-23 The knockdown activity of GuNA[t-Bu]-gapmer antisense oligonucleotides in heart", ABSTRACTS OF THE 6TH ANNUAL MEETING OF THE JAPANESE SOCIETY OF NUCLEIC ACID MEDICINE; JUNE 27 - JUNE 29, 2020, JAPANESE SOCIETY OF NUCLEIC ACID MEDICINE, JP, 18 June 2021 (2021-06-18) - 29 June 2020 (2020-06-29), JP, pages 155, XP009541528
Attorney, Agent or Firm:
MURAOKA, Koichiro et al. (JP)
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