Title:
MYOCARDIAL DYSFUNCTION THERAPEUTIC AGENT
Document Type and Number:
WIPO Patent Application WO/2019/240164
Kind Code:
A1
Abstract:
Established is a method for treating myocardial dysfunctions. Provided are: an oligonucleotide having 15-30 bases has complementary nucleotide sequences in some parts of an intron 55 region of a dystrophin gene, the oligonucleotide comprising 5'-TGTCTTCCT-3' or 5'-CAGCTTGAACCGGGC-3' (SEQ ID NO: 64) sequence (where T in the sequence may be U); and a pharmacologically acceptable salt or a solvate thereof. Provided is a prophylactic or therapeutic agent for myocardial dysfunction comprising said oligonucleotide, and a pharmacologically acceptable salt or solvate thereof. Provided is a Dp116 expression inhibitor comprising said oligonucleotide, and a pharmacologically acceptable salt or solvate thereof.
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Inventors:
MATSUO MASAFUMI (JP)
KOIZUMI MAKOTO (JP)
ONISHI YOSHIYUKI (JP)
SHOJI TAKAO (JP)
KOIZUMI MAKOTO (JP)
ONISHI YOSHIYUKI (JP)
SHOJI TAKAO (JP)
Application Number:
PCT/JP2019/023265
Publication Date:
December 19, 2019
Filing Date:
June 12, 2019
Export Citation:
Assignee:
DAIICHI SANKYO CO LTD (JP)
KOBE GAKUIN EDUCATIONAL FOUND (JP)
KOBE GAKUIN EDUCATIONAL FOUND (JP)
International Classes:
C12N15/113; A61K31/7088; A61K48/00; A61P9/00; A61P43/00
Domestic Patent References:
| WO1999014226A2 | 1999-03-25 | |||
| WO2000047599A1 | 2000-08-17 | |||
| WO2014109384A1 | 2014-07-17 | |||
| WO1998054198A1 | 1998-12-03 |
Foreign References:
| JP2018112863A | 2018-07-19 | |||
| US6261840B1 | 2001-07-17 | |||
| JPH0787982A | 1995-04-04 | |||
| JP2000297097A | 2000-10-24 |
Other References:
YAMAMOTO, TETSUSHI ET AL.: "Cardiac Dysfunction in Duchenne Muscular Dystrophy Is Less Frequent in Patients With Mutations in the Dystrophin Dp116 Coding Region Than in Other Regions", CIRCULATION: GENOMIC AND PRECISION MEDICINE, vol. 11, no. 1, 11 January 2018 (2018-01-11), pages e001782, XP055663418
MATSUO, MASAFUMI ET AL.: "Dystrophin Dp116: a yet to be investigated product of the Duchenne muscular dystrophy gene", GENES, vol. 8, no. 10, 2 October 2017 (2017-10-02), pages 251/1 - 251/11, XP055663421
KAMDAR FGARRY DJ: "Dystrophin-deficient cardiomyopathy", J AM COLL CARDIOL, vol. 67, 2016, pages 2533 - 2546, XP029548666, DOI: 10.1016/j.jacc.2016.02.081
NIGRO GCOMI LIPOLITANO LBAIN RJ: "The incidence and evolutive of cardiomyopathy in Duchenne muscular dystrophy", INT J CARDIOL, vol. 26, 1990, pages 271 - 277
MARKHAM LWSPICER RLKHOURY PRWONG BLMATHEWS KDCRIPE LH: "Steroid therapy and cardiac function in Duchenne muscular dystrophy", PEDIATR CARDIOL, vol. 26, 2005, pages 768 - 771, XP019363802
YAMAMOTO TAWANO HZHAN ZENOMOTO-SAKUMA MKITAAKI SMATSUMOTO MNAGAI MSATO IIMANISHI THAYASHI N: "Cardiac dysfunction in Duchenne muscular dystrophy is less frequent in patients with mutations in the dystrophin Dp 116 coding region than in other regions", CIRS GENOM PRECIS MED, vol. 11, 2018, pages e001782, XP055663418, DOI: 10.1161/CIRCGEN.117.001782
NUCLEIC ACIDS RESEARCH, vol. 12, 1984, pages 4539
BLOMMERS ET AL., BIOCHEMISTRY, vol. 37, 1998, pages 17714 - 17725
LESNIK, E.A. ET AL., BIOCHEMISTRY, vol. 32, 1993, pages 7832 - 7838
MARTIN, P, HELV. CHIM. ACTA., vol. 78, 1995, pages 486 - 504
WANG, G ET AL., TETRAHEDRON, vol. 55, 1999, pages 7707 - 7724
SETH, P.P. ET AL., J. ORG. CHEM, vol. 75, 2010, pages 1569 - 1581
YAHARA, A ET AL., CHEMBIOCHEM, vol. 13, 2012, pages 2513 - 2516
TETRAHEDRON LETTERS, vol. 32, 1991, pages 3005
J. AM. CHEM. SOC., vol. 112, 1990, pages 1253
"Oligonucleotide Synthesis", 1984, OXFORD UNIVERSITY PRESS
See also references of EP 3816286A4
MATSUO, MASAFUMI ET AL.: "Dystrophin Dp116: a yet to be investigated product of the Duchenne muscular dystrophy gene", GENES, vol. 8, no. 10, 2 October 2017 (2017-10-02), pages 251/1 - 251/11, XP055663421
KAMDAR FGARRY DJ: "Dystrophin-deficient cardiomyopathy", J AM COLL CARDIOL, vol. 67, 2016, pages 2533 - 2546, XP029548666, DOI: 10.1016/j.jacc.2016.02.081
NIGRO GCOMI LIPOLITANO LBAIN RJ: "The incidence and evolutive of cardiomyopathy in Duchenne muscular dystrophy", INT J CARDIOL, vol. 26, 1990, pages 271 - 277
MARKHAM LWSPICER RLKHOURY PRWONG BLMATHEWS KDCRIPE LH: "Steroid therapy and cardiac function in Duchenne muscular dystrophy", PEDIATR CARDIOL, vol. 26, 2005, pages 768 - 771, XP019363802
YAMAMOTO TAWANO HZHAN ZENOMOTO-SAKUMA MKITAAKI SMATSUMOTO MNAGAI MSATO IIMANISHI THAYASHI N: "Cardiac dysfunction in Duchenne muscular dystrophy is less frequent in patients with mutations in the dystrophin Dp 116 coding region than in other regions", CIRS GENOM PRECIS MED, vol. 11, 2018, pages e001782, XP055663418, DOI: 10.1161/CIRCGEN.117.001782
NUCLEIC ACIDS RESEARCH, vol. 12, 1984, pages 4539
BLOMMERS ET AL., BIOCHEMISTRY, vol. 37, 1998, pages 17714 - 17725
LESNIK, E.A. ET AL., BIOCHEMISTRY, vol. 32, 1993, pages 7832 - 7838
MARTIN, P, HELV. CHIM. ACTA., vol. 78, 1995, pages 486 - 504
WANG, G ET AL., TETRAHEDRON, vol. 55, 1999, pages 7707 - 7724
SETH, P.P. ET AL., J. ORG. CHEM, vol. 75, 2010, pages 1569 - 1581
YAHARA, A ET AL., CHEMBIOCHEM, vol. 13, 2012, pages 2513 - 2516
TETRAHEDRON LETTERS, vol. 32, 1991, pages 3005
J. AM. CHEM. SOC., vol. 112, 1990, pages 1253
"Oligonucleotide Synthesis", 1984, OXFORD UNIVERSITY PRESS
See also references of EP 3816286A4
Attorney, Agent or Firm:
MAYAMA, Setsuko et al. (JP)
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