Title:
ANTISENSE NUCLEIC ACID THAT INDUCES SKIPPING OF EXON 50
Document Type and Number:
WIPO Patent Application WO/2021/132591
Kind Code:
A1
Abstract:
The present description provides a drug that causes a highly efficient skipping of the 50th exon of the human dystrophin gene. The present description also provides an antisense oligomer that induces skipping of the 50th exon of the human dystrophin gene.
Inventors:
ENYA YUKIKO (JP)
SUNADOI YUTA (JP)
WAKI REIKO (JP)
MUCHIMA KANAME (JP)
TAKEDA SHIN'ICHI (JP)
AOKI YOSHITSUGU (JP)
SUNADOI YUTA (JP)
WAKI REIKO (JP)
MUCHIMA KANAME (JP)
TAKEDA SHIN'ICHI (JP)
AOKI YOSHITSUGU (JP)
Application Number:
PCT/JP2020/048803
Publication Date:
July 01, 2021
Filing Date:
December 25, 2020
Export Citation:
Assignee:
NIPPON SHINYAKU CO LTD (JP)
NAT CENTER NEUROLOGY & PSYCHIATRY (JP)
NAT CENTER NEUROLOGY & PSYCHIATRY (JP)
International Classes:
C12N15/113; A61K31/7088; A61P21/04
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Other References:
WU, B. ET AL.: "Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development", PLOS ONE, vol. 6, no. 5, 2011, pages el9906, XP009171295, DOI: 10.1371/journal.pone.0019906
ANNEMIEKE AARTSMA-RUS ET AL., NEUROMUSCULAR DISORDERS, vol. 12, 2002, pages S71 - S77
ROBERTS, RG ET AL., GENOMICS, vol. 16, 1993, pages 536 - 538
KOENIG, M. ET AL., CELL, vol. 53, 1988, pages 219 - 228
SAMBROOKRUSSELL: "Molecular Cloning: A Laboratory Manual", vol. 3, 2001, COLD SPRING HARBOR, LABORATORY PRESS
AUSUBEL: "Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
SCIENCE, vol. 227, no. 4693, 1985, pages 1435 - 1441
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. NIELSENM. EGHOLMR. H. BERGO. BUCHARDT, SCIENCE, vol. 254, 1991, pages 1497
M. EGHOLMO. BUCHARDTP. E. NIELSENR. H. BERG, JACS, vol. 114, 1992, pages 1895
K. L. DUEHOLMM. EGHOLMC. BEHRENSL. CHRISTENSENH. F. HANSENT. VULPIUSK. H. PETERSENR. H. BERGP. E. NIELSENO. BUCHARDT, J. ORG. CHEM., vol. 59, 1994, pages 5767
L. CHRISTENSENR. FITZPATRICKB. GILDEAK. H. PETERSENH. F. HANSENT. KOCHM. EGHOLMO. BUCHARDTP. E. NIELSENJ. COULL, SCI, vol. 1, 1995, pages 175
T. KOCHH. F. HANSENP. ANDERSENT. LARSENH. G. BATZK. OTTESONH. ORUM, J. PEPT. RES., vol. 49, 1997, pages 80
J. D. RAMSEYN. H. FLYNN, PHARMACOLOGY & THERAPEUTICS, vol. 154, 2015, pages 78 - 86
M. K. TSOUMPRA ET AL., EBIOMEDICINE
"New RRC, Liposomes: A practical approach", 1990, IRL PRESS, pages: 33 - 104
See also references of EP 4083208A4
ANNEMIEKE AARTSMA-RUS ET AL., NEUROMUSCULAR DISORDERS, vol. 12, 2002, pages S71 - S77
ROBERTS, RG ET AL., GENOMICS, vol. 16, 1993, pages 536 - 538
KOENIG, M. ET AL., CELL, vol. 53, 1988, pages 219 - 228
SAMBROOKRUSSELL: "Molecular Cloning: A Laboratory Manual", vol. 3, 2001, COLD SPRING HARBOR, LABORATORY PRESS
AUSUBEL: "Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
SCIENCE, vol. 227, no. 4693, 1985, pages 1435 - 1441
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. NIELSENM. EGHOLMR. H. BERGO. BUCHARDT, SCIENCE, vol. 254, 1991, pages 1497
M. EGHOLMO. BUCHARDTP. E. NIELSENR. H. BERG, JACS, vol. 114, 1992, pages 1895
K. L. DUEHOLMM. EGHOLMC. BEHRENSL. CHRISTENSENH. F. HANSENT. VULPIUSK. H. PETERSENR. H. BERGP. E. NIELSENO. BUCHARDT, J. ORG. CHEM., vol. 59, 1994, pages 5767
L. CHRISTENSENR. FITZPATRICKB. GILDEAK. H. PETERSENH. F. HANSENT. KOCHM. EGHOLMO. BUCHARDTP. E. NIELSENJ. COULL, SCI, vol. 1, 1995, pages 175
T. KOCHH. F. HANSENP. ANDERSENT. LARSENH. G. BATZK. OTTESONH. ORUM, J. PEPT. RES., vol. 49, 1997, pages 80
J. D. RAMSEYN. H. FLYNN, PHARMACOLOGY & THERAPEUTICS, vol. 154, 2015, pages 78 - 86
M. K. TSOUMPRA ET AL., EBIOMEDICINE
"New RRC, Liposomes: A practical approach", 1990, IRL PRESS, pages: 33 - 104
See also references of EP 4083208A4
Attorney, Agent or Firm:
KOBAYASHI Hiroshi et al. (JP)
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