Title:
ANTISENSE NUCLEIC ACID FOR TREATING AMYOTROPHY
Document Type and Number:
WIPO Patent Application WO/2017/047741
Kind Code:
A1
Abstract:
A novel antisense nucleic acid and the like inhibiting myostatin at an mRNA level have been required. Provided is a definite antisense oligomer that enables skipping of the second exon of myostatin gene and induces degradation of mRNA of myostatin gene.
Inventors:
NAKAGAWA SHINICHIRO (JP)
NAGATA SEIGO (JP)
SUNADA YOSHIHIDE (JP)
OHSAWA YUTAKA (JP)
NISHIMATSU SHIN-ICHIRO (JP)
NAGATA SEIGO (JP)
SUNADA YOSHIHIDE (JP)
OHSAWA YUTAKA (JP)
NISHIMATSU SHIN-ICHIRO (JP)
Application Number:
PCT/JP2016/077416
Publication Date:
March 23, 2017
Filing Date:
September 16, 2016
Export Citation:
Assignee:
NIPPON SHINYAKU CO LTD (JP)
KAWASAKI GAKUEN EDUCATIONAL FOUND (JP)
KAWASAKI GAKUEN EDUCATIONAL FOUND (JP)
International Classes:
C12N15/113; A61K31/7088; A61P21/02; A61P21/04
Domestic Patent References:
| WO2006086667A2 | 2006-08-17 | |||
| WO2012029986A1 | 2012-03-08 | |||
| WO2015194520A1 | 2015-12-23 |
Foreign References:
| JP2007104971A | 2007-04-26 | |||
| US20130085139A1 | 2013-04-04 |
Other References:
MALERBA A. ET AL.: "Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy", MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 1, no. 12, December 2012 (2012-12-01), pages 1 - 8, XP055128896, ISSN: 2162-2531, Retrieved from the Internet
LU -NGUYEN N. ET AL.: "Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.", MOLECULAR THERAPY, vol. 23, no. 8, 23 August 2015 (2015-08-23), pages 1341 - 1348, XP055370966, ISSN: 1525-0016
KEMALADEWI D. ET AL.: "Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy.", BMC MEDICAL GENOMICS, vol. 4, no. 36, 2011, pages 1 - 10, XP021102061, ISSN: 1755-8794, Retrieved from the Internet [retrieved on 20161003]
BESTAS B. ET AL.: "Design and Application of Bispecific Splice-Switching Oligonucleotides.", NUCLEIC ACID THERAPEUTICS, vol. 24, no. 1, 2014, pages 13 - 24, XP055256406, ISSN: 2159-3345
KANG J. ET AL.: "Antisense-induced Myostatin Exon Skipping Leads to Muscle Hypertrophy in Mice Following Octa-guanidine Morpholino Oligomer Treatment.", MOLECULAR THERAPY, vol. 19, no. 1, January 2011 (2011-01-01), pages 159 - 164, XP002657135, ISSN: 1525-0016
AOKI Y. ET AL.: "Feasibility and Effectiveness of Exon 51 skipping in Human-Like mdx Mutation.", MOLECULAR THERAPY, vol. 18, no. Supplement 1, May 2010 (2010-05-01), pages S218 - S219, XP055370970, ISSN: 1525-0016
PAO P. ET AL.: "Dual Masking of Specific Negative Splicing Regulatory Elements Resulted in Maximal Exon 7 Inclusion of SMN2 Gene .", MOLECULAR THERAPY, vol. 22, no. 4, 2014, pages 854 - 861, XP055237786, ISSN: 1525-0016
DATABASE GenBank 15 March 2015 (2015-03-15), "Homo sapiens myostatin (MSTN), mRNA", XP055370979, Database accession no. NM_005259.2
JANSON J.A.M. ET AL.: "Prospects for single antisense oligonucleotide-induced multiple exon skipping for rare non-hotspot mutations in Duchenne muscular dystrophy.", NEUROMUSCULAR DISORDERS, vol. 24, 2014, pages 820 - 821, XP055370986, ISSN: 0960-8966
See also references of EP 3351634A4
LU -NGUYEN N. ET AL.: "Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.", MOLECULAR THERAPY, vol. 23, no. 8, 23 August 2015 (2015-08-23), pages 1341 - 1348, XP055370966, ISSN: 1525-0016
KEMALADEWI D. ET AL.: "Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy.", BMC MEDICAL GENOMICS, vol. 4, no. 36, 2011, pages 1 - 10, XP021102061, ISSN: 1755-8794, Retrieved from the Internet
BESTAS B. ET AL.: "Design and Application of Bispecific Splice-Switching Oligonucleotides.", NUCLEIC ACID THERAPEUTICS, vol. 24, no. 1, 2014, pages 13 - 24, XP055256406, ISSN: 2159-3345
KANG J. ET AL.: "Antisense-induced Myostatin Exon Skipping Leads to Muscle Hypertrophy in Mice Following Octa-guanidine Morpholino Oligomer Treatment.", MOLECULAR THERAPY, vol. 19, no. 1, January 2011 (2011-01-01), pages 159 - 164, XP002657135, ISSN: 1525-0016
AOKI Y. ET AL.: "Feasibility and Effectiveness of Exon 51 skipping in Human-Like mdx Mutation.", MOLECULAR THERAPY, vol. 18, no. Supplement 1, May 2010 (2010-05-01), pages S218 - S219, XP055370970, ISSN: 1525-0016
PAO P. ET AL.: "Dual Masking of Specific Negative Splicing Regulatory Elements Resulted in Maximal Exon 7 Inclusion of SMN2 Gene .", MOLECULAR THERAPY, vol. 22, no. 4, 2014, pages 854 - 861, XP055237786, ISSN: 1525-0016
DATABASE GenBank 15 March 2015 (2015-03-15), "Homo sapiens myostatin (MSTN), mRNA", XP055370979, Database accession no. NM_005259.2
JANSON J.A.M. ET AL.: "Prospects for single antisense oligonucleotide-induced multiple exon skipping for rare non-hotspot mutations in Duchenne muscular dystrophy.", NEUROMUSCULAR DISORDERS, vol. 24, 2014, pages 820 - 821, XP055370986, ISSN: 0960-8966
See also references of EP 3351634A4
Attorney, Agent or Firm:
KOBAYASHI Hiroshi et al. (JP)
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