Title:
COMPOUND, METHOD AND PHARMACEUTICAL COMPOSITION FOR REGULATING EXPRESSION OF ATAXIN 3
Document Type and Number:
WIPO Patent Application WO/2021/230286
Kind Code:
A1
Abstract:
A modified oligonucleotide that has an activity of inhibiting the expression of Ataxin 3 and has any of the following nucleic acid base sequences or a nucleic acid base sequence having 17 consecutive bases contained in the nucleic acid base sequences. 1) TCGGGTAAGTAGATTTTC (SEQ ID NO. 239), 2) GAAGTATCTGTAGGCCTA (SEQ ID NO. 240), 3) GGACTGTATAGGAGATTA (SEQ ID NO. 241), 4) GGTTATAGGATGCAGGTA (SEQ ID NO.242), 5) AGGTTATAGGATGCAGGT (SEQ ID NO.243), 6) GAAGCTAAGTAGGTGACT (SEQ ID NO. 244), 7) TGAAGCTAAGTAGGTGAC (SEQ ID NO. 245), 8) CCTAGTCACTTTGATAGA (SEQ ID NO. 246), 9) GGAACATCTTGAGTAGGT (SEQ ID NO. 247), 10) GGTGTTCAGGGTAGATGT (SEQ ID NO. 248), 11) GGATACTCTGCCCTGTTC (SEQ ID NO. 249), 12) GGTGTCAAACGTGTGGTT (SEQ ID NO. 250), 13) CCGTGTGCTAGTATTTGT (SEQ ID NO. 251), 14) TAGTAGAGTTTTGCTTGG (SEQ ID NO.252), 15) GATGTAGTAGAGTTTTGC (SEQ ID NO. 253), 16) TGATGTAGTAGAGTTTTG (SEQ ID NO. 254), 17) CTGATGTAGTAGAGTTTT (SEQ ID NO. 255), 19)GCAAGTTGGTTTGTGGTA (SEQ ID NO. 256), 20) TCTAGGCAATTGTGGTGG (SEQ ID NO. 257), 21) GTAACTCTGCACTTCCCA (SEQ ID NO. 258), 22) GTCATCCCTATGTCTTAT (SEQ ID NO. 259), 23) GTCATATGGTCAGGGTAT (SEQ ID NO. 260), 24) TGTCATATGGTCAGGGTA (SEQ ID NO. 261), 25) ATGTCATATGGTCAGGGT (SEQ ID NO. 262) and 26) TATGTCATATGGTCAGGG (SEQ ID NO. 263).
Inventors:
SAWAMOTO HIROAKI (JP)
HIGO TAKUYA (JP)
MURATA SHUMPEI (JP)
ARAKI YUU (JP)
HIGO TAKUYA (JP)
MURATA SHUMPEI (JP)
ARAKI YUU (JP)
Application Number:
PCT/JP2021/018040
Publication Date:
November 18, 2021
Filing Date:
May 12, 2021
Export Citation:
Assignee:
MITSUBISHI TANABE PHARMA CORP (JP)
International Classes:
A61K48/00; A61K31/7088; A61K31/7115; A61K31/712; A61K31/7125; A61P25/28; A61P43/00; C12N15/113
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TAKIYAMA ET AL., NATURE GENETICS, vol. 4, 1993, pages 300 - 304
KAWAGUCHI ET AL., NATURE GENETICS, vol. 8, 1994, pages 221 - 228
TAKIYAMA ET AL., NEUROLOGY, vol. 49, 1997, pages 604 - 606
M. M. EVERS ET AL., MOLECULAR NEUROBIOLOGY, vol. 49, 2014, pages 1513 - 1531
AARTSMA-RUS, A., NUCLEIC ACID THER., vol. 27, 2017, pages 67
HAYLEY S. MCLOUGHLIN ET AL., ANNALS OF NEUROLOGY, vol. 84, 2018, pages 64 - 77
ALTSCHUL ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403 - 410
ZHANGMADDEN, GENOME RES., vol. 7, 1997, pages 649 - 656
SMITHWATERMAN, ADV. APPL. MATH., vol. 2, 1981, pages 482 - 489
CHATTOPADHYAYA ET AL., J. ORG. CHEM., vol. 74, 2009, pages 118 - 134
SRIVASTAVA ET AL., J.AM.CHEM.SOC., vol. 129, no. 26, 2007, pages 8362 - 8379
FRIEDEN ET AL., NUCLEIC ACIDS RESEARCH, vol. 21, 2003, pages 6365 - 6372
ELAYADI ET AL., CURR.OPINION INVENS.DRUGS, vol. 2, 2001, pages 558 - 561
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
NAUWELAERTS ET AL., J. AM. CHEM. SOC., vol. 129, no. 30, 2007, pages 9340 - 9348
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, STRUCTURAL BIOLOGY AND CRYSTALLIZATION COMMUNICATIONS, vol. F61, 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, CHEM, vol. 122, 2000, pages 8595 - 8602
"Antisense Research and Applications", 1993, CRC PRESS, pages: 276 - 278
Attorney, Agent or Firm:
IP FIRM SHUWA (JP)
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