Title:
COMPOUND, METHOD AND PHARMACEUTICAL COMPOSITION FOR DUX4 EXPRESSION ADJUSTMENT
Document Type and Number:
WIPO Patent Application WO/2020/203880
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a compound, a method, and a pharmaceutical composition for normalizing DUX4 of an individual having abnormal expression of double homeobox 4 (DUX4) gene. Provided is a modified oligonucleotide comprising 12 to 30 residues and including at least an eight contiguous nucleobase sequence that is complementary to an equal length portion in, from the 5' end, positions 126-147, 232-248, 1306-1325 or 1472-1495 of the nucleic acid bases of mature mRNA of DUX4 of SEQ ID NO: 1. The nucleic acid bases sequence of the modified oligonucleotide is at least 90% complementary to the equal length portion of the nucleic acid base sequence of mature mRNA of DUX4 of SEQ ID NO: 1.
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Inventors:
KUMAGAI SHINJI (JP)
YASHIRO TAKASHI (JP)
ARAKI TOMO (JP)
KANAGAWA TAKAYUKI (JP)
OKAGAKI CHIEKO (JP)
FURUKAWA HIROYUKI (JP)
YASHIRO TAKASHI (JP)
ARAKI TOMO (JP)
KANAGAWA TAKAYUKI (JP)
OKAGAKI CHIEKO (JP)
FURUKAWA HIROYUKI (JP)
Application Number:
PCT/JP2020/014307
Publication Date:
October 08, 2020
Filing Date:
March 27, 2020
Export Citation:
Assignee:
MITSUBISHI TANABE PHARMA CORP (JP)
International Classes:
A61K48/00; A61K31/7088; A61P21/00; A61P43/00; C12N15/113
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JONES, L. J. ET AL., ANALYTICAL BIOCHEMISTRY, vol. 265, 1998, pages 368 - 374
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WALLACE, L M ET AL., MOL THER, vol. 20, no. 7, 2012, pages 1417
WALLACE, L M ET AL., ANN NEUROL, vol. 69, no. 3, 2011, pages 540
SNIDER ET AL., PLOS, vol. 6, no. 10, 2010
FERREBOEUF ET AL., HUMAN MOLECULAR GENETICS, vol. 23, no. 1, 2013, pages 171
SACCONI ET AL., BIOCHIM. BIOPHYS. ACTA, 2015, pages 607
"GenBank", Database accession no. NM 001363820.1
YASUDA ET AL., NATURE GENETICS, vol. 48, no. 5, 2016, pages 569
YOSHIMOTO ET AL., CANCER RESEARCH, vol. 77, 2017, pages 2927
SIRVENT ET AL., CANCER GENETICS AND CYTOGENETICS, vol. 195, 2009, pages 12
WOOLF ET AL., PROC. NATL. ACAD. SCI. USA, vol. 89, 1992, pages 7305 - 7309
GAUTSCHI ET AL., J. NATL. CANCER INST., vol. 93, March 2001 (2001-03-01), pages 463 - 471
ELAYADI ET AL., CURR. OPINION INVENS. DRUGS, vol. 2, 2001, pages 558 - 561
ALTSCHUL ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403 410
ZHANGMADDEN, GENOME RES., vol. 7, 1997, pages 649 656
ADV. APPL. MATH., vol. 2, 1981, pages 482 489
CHATTOPADHYAYA ET AL., J. ORG. CHEM., vol. 74, 2009, pages 118 - 134
NAUWELAERTS ET AL., J. AM. CHEM. SOC., vol. 129, no. 30, 2007, pages 9340 - 9348
FRIEDEN ET AL., NUCLEIC ACIDS RESEARCH, vol. 21, 2003, pages 6365 - 6372
BRAASCH ET AL., CHEM. BIOL., vol. 8, 2001, pages 1 - 7
ORUM ET AL., CURR. OPINION MOL. THER., vol. 3, 2001, pages 239 - 243
WAHLESTEDT ET AL., PROC. NATL. ACAD. SCI. U.S.A., vol. 97, 2000, pages 5633 - 5638
SINGH ET AL., CHEM. COMMUN., vol. 4, 1998, pages 455 - 456
KOSHKIN ET AL., TETRAHEDRON, vol. 54, 1998, pages 3607 - 3630
KUMAR ET AL., BIOORG. MED. CHEM. LETT., vol. 8, 1998, pages 2219 - 2222
SINGH ET AL., J. ORG. CHEM., vol. 63, 1998, pages 10035 - 10039
FRIER ET AL., NUCLEIC ACIDS RESEARCH, vol. 25, no. 22, 1997, pages 4429 - 4443
ALBAEK ET AL., J. ORG. CHEM., vol. 71, 2006, pages 7731 - 7740
LEUMANN, CHRISTIAN J., BIOORG. & MED. CHEM., vol. 10, 2002, pages 841 - 854
BRAASCH ET AL., BIOCHEMISTRY, vol. 41, 2002, pages 4503 - 4510
ROBEYNS ET AL., J. AM. CHEM. SOC., vol. 130, no. 6, 2008, pages 1979 - 1984
HORVATH ET AL., TETRAHEDRON LETTERS, vol. 48, 2007, pages 3621 - 3623
GU ET AL., NUCLEOSIDES, NUCLEOTIDES & NUCLEIC ACIDS, vol. 24, no. 5 - 7, 2005, pages 993 - 998
NAUWELAERTS ET AL., NUCLEIC ACIDS RESEARCH, vol. 33, no. 8, 2005, pages 2452 - 2463
ROBEYNS ET AL., ACTA CRYSTALLOGRAPHICA, SECTION F: STRUCTURAL BIOLOGY AND CRYSTALLIZATION COMMUNICATIONS, no. 6, 2005, pages 585 - 586
GU ET AL., TETRAHEDRON, vol. 60, no. 9, 2004, pages 2111 - 2123
GU ET AL., OLIGONUCLEOTIDES, vol. 13, no. 6, 2003, pages 479 - 489
WANG ET AL., J. ORG. CHEM., vol. 68, 2003, pages 4499 - 4505
VERBEURE ET AL., NUCLEIC ACIDS RESEARCH, vol. 29, no. 24, 2001, pages 4941 - 4947
WANG ET AL., J. ORG. CHEM., vol. 66, 2001, pages 8478 - 82
WANG ET AL., NUCLEOSIDES, NUCLEOTIDES & NUCLEIC ACIDS, vol. 20, no. 4 - 7, 2001, pages 785 - 788
WANG ET AL., J. AM. CHEM., vol. 122, 2000, pages 8595 - 8602
"Antisense Research and Applications", 1993, CRC PRESS, pages: 276 - 278
JONES, L. J. ET AL., ANALYTICAL BIOCHEMISTRY, vol. 265, 1998, pages 368 - 374
JONES, T. ET AL., PLOS ONE, vol. 13, no. 2, 2018
WALLACE, L M ET AL., MOL THER, vol. 20, no. 7, 2012, pages 1417
WALLACE, L M ET AL., ANN NEUROL, vol. 69, no. 3, 2011, pages 540
Attorney, Agent or Firm:
IP FIRM SHUWA (JP)
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