Title:
ANTISENSE NUCLEIC ACID
Document Type and Number:
WIPO Patent Application WO/2015/137409
Kind Code:
A1
Abstract:
Provided is a drug that allows highly-efficient skipping of exon 51 in the human dystrophin gene. The present invention provides an antisense oligomer which enables exon 51 exon in the human dystrophin gene to be skipped.
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Inventors:
WAKAYAMA TATSUSHI (JP)
SEO HARUNA (JP)
SATOU YOUHEI (JP)
TAKEDA SHIN ICHI (JP)
NAGATA TETSUYA (JP)
SEO HARUNA (JP)
SATOU YOUHEI (JP)
TAKEDA SHIN ICHI (JP)
NAGATA TETSUYA (JP)
Application Number:
PCT/JP2015/057180
Publication Date:
September 17, 2015
Filing Date:
March 11, 2015
Export Citation:
Assignee:
NIPPON SHINYAKU CO LTD (JP)
NAT CT NEUROLOGY & PSYCHIATRY (JP)
NAT CT NEUROLOGY & PSYCHIATRY (JP)
International Classes:
C12N15/09; A61K31/711; A61K31/712; A61K31/7125; A61P21/00; C12N15/113
Domestic Patent References:
| WO2012029986A1 | 2012-03-08 | |||
| WO2004048570A1 | 2004-06-10 | |||
| WO2012150960A1 | 2012-11-08 |
Foreign References:
| JP2012506703A | 2012-03-22 | |||
| JP2004509622A | 2004-04-02 | |||
| JP2012506697A | 2012-03-22 | |||
| US20100168212A1 | 2010-07-01 | |||
| JP2013530154A | 2013-07-25 |
Other References:
TAKASHI SAITO ET AL.: "Development of Exon Skipping Therapy for Duchenne Muscular Dystrophy Using Patient-Derived Cells", JAPANESE JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS, vol. 43, no. 2, 31 March 2012 (2012-03-31), pages 91 - 92, XP008174733
INCITTI T ET AL.: "Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping", MOL. THER., vol. 18, no. 9, 2010, pages 1675 - 1682, XP008131506
CIRAK S ET AL.: "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study", LANCET, vol. 378, no. 9791, 2011, pages 595 - 605, XP009160164
ARECHAVALA-GOMEZA V ET AL.: "Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle", HUM. GENE THER., vol. 18, no. 9, 2007, pages 798 - 810, XP055038387
AOKI Y ET AL.: "In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse", MOL. THER., vol. 18, no. 11, 2010, pages 1995 - 2005, XP055224044
NAKANO S ET AL.: "Exon-skipping events in candidates for clinical trials of morpholino", PEDIATR. INT., vol. 53, no. 4, 2011, pages 524 - 529, XP055224048
See also references of EP 3118311A4
INCITTI T ET AL.: "Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping", MOL. THER., vol. 18, no. 9, 2010, pages 1675 - 1682, XP008131506
CIRAK S ET AL.: "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study", LANCET, vol. 378, no. 9791, 2011, pages 595 - 605, XP009160164
ARECHAVALA-GOMEZA V ET AL.: "Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle", HUM. GENE THER., vol. 18, no. 9, 2007, pages 798 - 810, XP055038387
AOKI Y ET AL.: "In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse", MOL. THER., vol. 18, no. 11, 2010, pages 1995 - 2005, XP055224044
NAKANO S ET AL.: "Exon-skipping events in candidates for clinical trials of morpholino", PEDIATR. INT., vol. 53, no. 4, 2011, pages 524 - 529, XP055224048
See also references of EP 3118311A4
Attorney, Agent or Firm:
KOBAYASHI Hiroshi et al. (JP)
Kobayashi 浩 (JP)
Kobayashi 浩 (JP)
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