Title:
ANTISENSE NUCLEIC ACID
Document Type and Number:
WIPO Patent Application WO/2012/029986
Kind Code:
A1
Abstract:
Disclosed is a drug which efficiently allows skipping of the 53rd exon in the human dystrophin gene. Disclosed is an oligomer which makes possible skipping of the 53rd exon of the human dystrophin gene.
Inventors:
WATANABE NAOKI (JP)
SATOU YOUHEI (JP)
TAKEDA SHIN ICHI (JP)
NAGATA TETSUYA (JP)
SATOU YOUHEI (JP)
TAKEDA SHIN ICHI (JP)
NAGATA TETSUYA (JP)
Application Number:
PCT/JP2011/070318
Publication Date:
March 08, 2012
Filing Date:
August 31, 2011
Export Citation:
Assignee:
NIPPON SHINYAKU CO LTD (JP)
NAT CT NEUROLOGY & PSYCHIATRY (JP)
WATANABE NAOKI (JP)
SATOU YOUHEI (JP)
TAKEDA SHIN ICHI (JP)
NAGATA TETSUYA (JP)
NAT CT NEUROLOGY & PSYCHIATRY (JP)
WATANABE NAOKI (JP)
SATOU YOUHEI (JP)
TAKEDA SHIN ICHI (JP)
NAGATA TETSUYA (JP)
International Classes:
C12N15/113; A61K31/7125; A61P21/04; C07H21/04
Domestic Patent References:
| WO2010048586A1 | 2010-04-29 | |||
| WO2008036127A2 | 2008-03-27 | |||
| WO2011057350A1 | 2011-05-19 | |||
| WO2006000057A1 | 2006-01-05 | |||
| WO2004048570A1 | 2004-06-10 | |||
| WO2010048586A1 | 2010-04-29 | |||
| WO2006129594A1 | 2006-12-07 | |||
| WO2006038608A1 | 2006-04-13 | |||
| WO1991009033A1 | 1991-06-27 | |||
| WO2009064471A1 | 2009-05-22 | |||
| WO1991009033A1 | 1991-06-27 | |||
| WO2008096690A1 | 2008-08-14 |
Foreign References:
| US20100168212A1 | 2010-07-01 | |||
| JP2002010790A | 2002-01-15 | |||
| US20100168212A1 | 2010-07-01 | |||
| JP2924179B2 | 1999-07-26 |
Other References:
POPPLEWELL L.J. ET AL.: "Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene.", MOL. THER., vol. 17, no. 3, 2009, pages 554 - 561, XP002573471
POPPLEWELL L.J. ET AL.: "Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.", NEUROMUSCUL. DISORD., vol. 20, no. 2, February 2010 (2010-02-01), pages 102 - 110, XP026878306
AARTSMA-RUS A. ET AL.: "Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.", NEUROMUSCUL. DISORD., vol. 12, 2002, pages S71 - S77, XP002250906
WILTON S.D. ET AL.: "Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.", MOL. THER., vol. 15, no. 7, 2007, pages 1288 - 1296, XP002544728
MONACO A. P. ET AL., GENOMICS, vol. 2, 1988, pages 90 - 95
MATSUO M., BRAIN DEV, vol. 18, 1996, pages 167 - 172
WILTON S. D., MOLECULAR THERAPY, vol. 15, 2007, pages 1288 - 96
ANNEMIEKE AARTSMA-RUS ET AL., NEUROMUSCULAR DISORDERS, vol. 12, 2002, pages S71 - S77
LINDA J. POPPLEWELL ET AL., NEUROMUSCULAR DISORDERS, vol. 20, no. 2, 2010, pages 102 - 10
ROBERTS, RG. ET AL., GENOMICS, vol. 16, 1993, pages 536 - 538
SAMBROOK; RUSSELL: "Molecular Cloning: A Laboratory Manual", vol. 3, 2001, COLD SPRING HARBOR, LABORATORY PRESS
AUSUBEL: "Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
KARLIN; ALTSCHUL, PROC. NATL. ACAD. SCI. USA, 1990, pages 872264 - 2268
PROC. NATL. ACAD. SCI. USA, vol. 90, 1993, pages 5873
ALTSCHUL SF ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403
ENYA ET AL., BIOORGANIC & MEDICINAL CHEMISTRY, vol. 18, 2008, pages 9154 - 9160
ALUL ET AL., NUCLEIC ACIDS RESEARCH, vol. 19, 1991, pages 1527
WRIGHT ET AL., TETRAHEDRON LETTERS, vol. 34, 1993, pages 3373
P. E. NIELSEN; M. EGHOLM; R. H. BERG; O. BUCHARDT, SCIENCE, vol. 254, 1991, pages 1497
M. EGHOLM; O. BUCHARDT; P. E. NIELSEN; R. H. BERG, JACS, vol. 114, 1992, pages 1895
K. L. DUEHOLM; M. EGHOLM; C. BEHRENS; L. CHRISTENSEN; H. F. HANSEN; T. VULPIUS; K. H. PETERSEN; R. H. BERG; P. E. NIELSEN; O. BUCH, J. ORG. CHEM., vol. 59, 1994, pages 5767
L. CHRISTENSEN; R. FITZPATRICK; B. GILDEA; K. H. PETERSEN; H. F. HANSEN; T. KOCH; M. EGHOLM; O. BUCHARDT; P. E. NIELSEN; J. COULL, J. PEPT. SCI., vol. 1, 1995, pages 175
T. KOCH; H. F. HANSEN; P. ANDERSEN; T. LARSEN; H. G. BATZ; K. OTTESON; H. ORUM, J. PEPT. RES., vol. 49, 1997, pages 80
ORG. LETT., vol. 6, no. 15, 2004, pages 2555 - 2557
See also references of EP 2612917A4
POPPLEWELL L.J. ET AL.: "Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.", NEUROMUSCUL. DISORD., vol. 20, no. 2, February 2010 (2010-02-01), pages 102 - 110, XP026878306
AARTSMA-RUS A. ET AL.: "Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.", NEUROMUSCUL. DISORD., vol. 12, 2002, pages S71 - S77, XP002250906
WILTON S.D. ET AL.: "Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.", MOL. THER., vol. 15, no. 7, 2007, pages 1288 - 1296, XP002544728
MONACO A. P. ET AL., GENOMICS, vol. 2, 1988, pages 90 - 95
MATSUO M., BRAIN DEV, vol. 18, 1996, pages 167 - 172
WILTON S. D., MOLECULAR THERAPY, vol. 15, 2007, pages 1288 - 96
ANNEMIEKE AARTSMA-RUS ET AL., NEUROMUSCULAR DISORDERS, vol. 12, 2002, pages S71 - S77
LINDA J. POPPLEWELL ET AL., NEUROMUSCULAR DISORDERS, vol. 20, no. 2, 2010, pages 102 - 10
ROBERTS, RG. ET AL., GENOMICS, vol. 16, 1993, pages 536 - 538
SAMBROOK; RUSSELL: "Molecular Cloning: A Laboratory Manual", vol. 3, 2001, COLD SPRING HARBOR, LABORATORY PRESS
AUSUBEL: "Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
KARLIN; ALTSCHUL, PROC. NATL. ACAD. SCI. USA, 1990, pages 872264 - 2268
PROC. NATL. ACAD. SCI. USA, vol. 90, 1993, pages 5873
ALTSCHUL SF ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403
ENYA ET AL., BIOORGANIC & MEDICINAL CHEMISTRY, vol. 18, 2008, pages 9154 - 9160
ALUL ET AL., NUCLEIC ACIDS RESEARCH, vol. 19, 1991, pages 1527
WRIGHT ET AL., TETRAHEDRON LETTERS, vol. 34, 1993, pages 3373
P. E. NIELSEN; M. EGHOLM; R. H. BERG; O. BUCHARDT, SCIENCE, vol. 254, 1991, pages 1497
M. EGHOLM; O. BUCHARDT; P. E. NIELSEN; R. H. BERG, JACS, vol. 114, 1992, pages 1895
K. L. DUEHOLM; M. EGHOLM; C. BEHRENS; L. CHRISTENSEN; H. F. HANSEN; T. VULPIUS; K. H. PETERSEN; R. H. BERG; P. E. NIELSEN; O. BUCH, J. ORG. CHEM., vol. 59, 1994, pages 5767
L. CHRISTENSEN; R. FITZPATRICK; B. GILDEA; K. H. PETERSEN; H. F. HANSEN; T. KOCH; M. EGHOLM; O. BUCHARDT; P. E. NIELSEN; J. COULL, J. PEPT. SCI., vol. 1, 1995, pages 175
T. KOCH; H. F. HANSEN; P. ANDERSEN; T. LARSEN; H. G. BATZ; K. OTTESON; H. ORUM, J. PEPT. RES., vol. 49, 1997, pages 80
ORG. LETT., vol. 6, no. 15, 2004, pages 2555 - 2557
See also references of EP 2612917A4
Attorney, Agent or Firm:
KOBAYASHI, Hiroshi et al. (JP)
Hiroshi Kobayashi (JP)
Hiroshi Kobayashi (JP)
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Claims: