Title:
MODIFIED NUCLEOSIDE AND SYNTHETIC METHOD THEREFOR
Document Type and Number:
WIPO Patent Application WO/2020/168952
Kind Code:
A1
Abstract:
Disclosed are a modified cytidine compound, i.e. a new derivative cytidine generated by adding a guanidyl at position 4 of a cytidine, and a nucleic acid containing the modified compound, for example, RNA. The nucleic acid containing the modified cytidine, especially mRNA, can significantly increase the expression quantity of the mRNA in vivo.
Inventors:
CAI YUNSONG (CN)
LI HANGWEN (CN)
LIU LIANXIAO (CN)
LI NING (CN)
LI HANGWEN (CN)
LIU LIANXIAO (CN)
LI NING (CN)
Application Number:
PCT/CN2020/074825
Publication Date:
August 27, 2020
Filing Date:
February 12, 2020
Export Citation:
Assignee:
STEMIRNA THERAPEUTICS CO LTD (CN)
International Classes:
C07H19/06
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RAZVAZHNAYA, O.V. ET AL.: "Study of the Influence of Derivatization Conditions on the Structure and Stability of Nucleoside Derivatives Using Gas Chromatography-Mass Spectrometry", JOURNAL OF MEDICINAL CHEMISTRY, vol. 69, no. 14, 30 December 2014 (2014-12-30), XP035415107, ISSN: 1061-9348, DOI: 20200319162252X
DORONINA, S.O. ET AL.: "Synthesis of 4-Guanidinopyrimidine Nucleosides for Triple Helix-mediated Guanine and Cytosine Recognition", TETRAHEDRON LETTERS, vol. 39, no. 7, 12 February 1998 (1998-02-12), XP004106734, ISSN: 0040-4039, DOI: 20200319155111X
CESNEK, M. ET AL.: "6-Guanidinopurine Nucleosides and Their Analogues", TETRAHEDRON, vol. 58, no. 15, 8 April 2002 (2002-04-08), XP004347793, ISSN: 0040-4020, DOI: 20200324095024A
ROZENSKI, JCRAIN, PMCCLOSKEY, J: "The RNA Modification Database: 1999 update", NUCL ACIDS RES, vol. 27, 1999, pages 196 - 197
BUNDGARD, H.: "Design of Prodrugs", 1985, ELSEVIER, pages: 7 - 9
A. R. GENNARO: "Remington's The Science and Practice of Pharmacy", 2006, LIPPINCOTT, WILLIAMS & WILKINS
HARRISON ET AL.: "Compendium of Synthetic Organic Methods", vol. 1-8, 1971, JOHN WILEY & SONS
CAUDY A A ET AL., GENES & DEVEL, vol. 16, pages 2491 - 96
NEILSEN P E, CURR OPIN STRUCT BIOL, vol. 9, pages 353 - 57
RAZ N K ET AL., BIOCHEM BIOPHYS RES COMMUN, vol. 297, pages 1075 - 84
"Molecular Cloning", 2001
PURCHIOG. C. FAREED: "Methods in Enzymology: Methods for molecular cloning in eukaryotic cells", 2003
GREENE ET AL.: "Protective Groups in Organic Synthesis", 1991, WILEY & SONS
OGATA ET AL., JOURNAL OF ORGANIC CHEMISTRY, vol. 74, 2009, pages 2585 - 2588
PURMAL ET AL., NUCLEIC ACIDS RESEARCH, vol. 22, no. 1, 1994, pages 72 - 78
FUKUHARA ET AL., BIOCHEMISTRY, vol. 1, no. 4, 1962, pages 563 - 568
XU ET AL., TETRAHEDRON, vol. 48, no. 9, 1992, pages 1729 - 1740
"Oligonucleotide synthesis: a practical approach, Oxford [Oxfordshire", 1984, IRL PRESS
CLIFTON, N.J.: "Oligonucleotide synthesis: methods and applications, Methods in Molecular Biology", vol. 288, 2005, HUMANA PRESS
ASHIQUL HAQUE ET AL.: "Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis", SCIENTIFIC REPORTS, vol. 8, 2018, pages 16776
KORE ET AL.: "Recent Developments in 5'-Terminal Cap Analogs: Synthesis and Biological Ramifications", MINI-REVIEWS IN ORGANIC CHEMISTRY, vol. 5, 2008, pages 179 - 192
LANGER, SCIENCE, vol. 249, 1990, pages 1527 - 1533
TREAT ET AL.: "Liposomes in the Therapy of Infectious Disease and Cancer", 1989, pages: 353 - 365
HUM. GENE THER, vol. 19, no. 9, September 2008 (2008-09-01), pages 887 - 95
See also references of EP 3929204A4
GIRI, N. ET AL.: "Fast, Quantitative Nucleoside Protection under Solvent-free Conditions", GREEN CHEMISTRY, vol. 10, no. 6, 30 April 2008 (2008-04-30), XP055728583, ISSN: 1463-9270, DOI: 20200319160720X
FURUTA, H. ET AL.: "Enhanced Transport of Nucleosides and Nucleoside Analogues with Complementary Base-Pairing Agents", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 113, no. 12, 1 June 1991 (1991-06-01), XP055728581, ISSN: 0002-7863, DOI: 20200319160720X
RAZVAZHNAYA, O.V. ET AL.: "Study of the Influence of Derivatization Conditions on the Structure and Stability of Nucleoside Derivatives Using Gas Chromatography-Mass Spectrometry", JOURNAL OF MEDICINAL CHEMISTRY, vol. 69, no. 14, 30 December 2014 (2014-12-30), XP035415107, ISSN: 1061-9348, DOI: 20200319162252X
DORONINA, S.O. ET AL.: "Synthesis of 4-Guanidinopyrimidine Nucleosides for Triple Helix-mediated Guanine and Cytosine Recognition", TETRAHEDRON LETTERS, vol. 39, no. 7, 12 February 1998 (1998-02-12), XP004106734, ISSN: 0040-4039, DOI: 20200319155111X
CESNEK, M. ET AL.: "6-Guanidinopurine Nucleosides and Their Analogues", TETRAHEDRON, vol. 58, no. 15, 8 April 2002 (2002-04-08), XP004347793, ISSN: 0040-4020, DOI: 20200324095024A
ROZENSKI, JCRAIN, PMCCLOSKEY, J: "The RNA Modification Database: 1999 update", NUCL ACIDS RES, vol. 27, 1999, pages 196 - 197
BUNDGARD, H.: "Design of Prodrugs", 1985, ELSEVIER, pages: 7 - 9
A. R. GENNARO: "Remington's The Science and Practice of Pharmacy", 2006, LIPPINCOTT, WILLIAMS & WILKINS
HARRISON ET AL.: "Compendium of Synthetic Organic Methods", vol. 1-8, 1971, JOHN WILEY & SONS
CAUDY A A ET AL., GENES & DEVEL, vol. 16, pages 2491 - 96
NEILSEN P E, CURR OPIN STRUCT BIOL, vol. 9, pages 353 - 57
RAZ N K ET AL., BIOCHEM BIOPHYS RES COMMUN, vol. 297, pages 1075 - 84
"Molecular Cloning", 2001
PURCHIOG. C. FAREED: "Methods in Enzymology: Methods for molecular cloning in eukaryotic cells", 2003
GREENE ET AL.: "Protective Groups in Organic Synthesis", 1991, WILEY & SONS
OGATA ET AL., JOURNAL OF ORGANIC CHEMISTRY, vol. 74, 2009, pages 2585 - 2588
PURMAL ET AL., NUCLEIC ACIDS RESEARCH, vol. 22, no. 1, 1994, pages 72 - 78
FUKUHARA ET AL., BIOCHEMISTRY, vol. 1, no. 4, 1962, pages 563 - 568
XU ET AL., TETRAHEDRON, vol. 48, no. 9, 1992, pages 1729 - 1740
"Oligonucleotide synthesis: a practical approach, Oxford [Oxfordshire", 1984, IRL PRESS
CLIFTON, N.J.: "Oligonucleotide synthesis: methods and applications, Methods in Molecular Biology", vol. 288, 2005, HUMANA PRESS
ASHIQUL HAQUE ET AL.: "Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis", SCIENTIFIC REPORTS, vol. 8, 2018, pages 16776
KORE ET AL.: "Recent Developments in 5'-Terminal Cap Analogs: Synthesis and Biological Ramifications", MINI-REVIEWS IN ORGANIC CHEMISTRY, vol. 5, 2008, pages 179 - 192
LANGER, SCIENCE, vol. 249, 1990, pages 1527 - 1533
TREAT ET AL.: "Liposomes in the Therapy of Infectious Disease and Cancer", 1989, pages: 353 - 365
HUM. GENE THER, vol. 19, no. 9, September 2008 (2008-09-01), pages 887 - 95
See also references of EP 3929204A4
Attorney, Agent or Firm:
HANGZHOU TIANHAO PATENT AGENCY CO. LTD (CN)
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