Title:
EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
Document Type and Number:
WIPO Patent Application WO/2014/153240
Kind Code:
A3
Abstract:
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
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Inventors:
BESTWICK RICHARD K (US)
FRANK DIANE ELIZABETH (US)
FRANK DIANE ELIZABETH (US)
Application Number:
PCT/US2014/029766
Publication Date:
December 31, 2014
Filing Date:
March 14, 2014
Export Citation:
Assignee:
SAREPTA THERAPEUTICS INC (US)
International Classes:
C12N15/11; A61K31/7088; A61P21/00; C12N15/113
Domestic Patent References:
| WO2010048586A1 | 2010-04-29 | |||
| WO2012029986A1 | 2012-03-08 |
Foreign References:
| US20100168212A1 | 2010-07-01 |
Other References:
POPPLEWELL L J ET AL: "Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials", NEUROMUSCULAR DISORDERS, PERGAMON PRESS, GB, vol. 20, no. 2, 15 January 2010 (2010-01-15), pages 102 - 110, XP026878306, ISSN: 0960-8966, [retrieved on 20100115]
FOSTER H ET AL: "Genetic therapeutic approaches for Duchenne muscular dystrophy", HUMAN GENE THERAPY, MARY ANN LIEBERT, NEW YORK ,NY, US, vol. 23, no. 7, 30 May 2012 (2012-05-30), pages 676 - 687, XP002688790, ISSN: 1043-0342, [retrieved on 20120530], DOI: 10.1089/HUM.2012.099
VAN VLIET LAURA ET AL: "Assessment of the feasibility of exon 45-55 multiexon skipping for duchenne muscular dystrophy", BMC MEDICAL GENETICS, BIOMED CENTRAL, LONDON, GB, vol. 9, no. 1, 1 December 2008 (2008-12-01), pages 105, XP021042784, ISSN: 1471-2350, DOI: 10.1186/1471-2350-9-105
JERRY R. MENDELL ET AL: "Gene therapy for muscular dystrophy: Lessons learned and path forward", NEUROSCIENCE LETTERS, vol. 527, no. 2, 17 May 2012 (2012-05-17), pages 90 - 99, XP055128775, ISSN: 0304-3940, DOI: 10.1016/j.neulet.2012.04.078
MOULTON H M ET AL: "Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - BIOMEMBRANES, ELSEVIER, AMSTERDAM, NL, vol. 1798, no. 12, 17 February 2010 (2010-02-17), pages 2296 - 2303, XP027430152, ISSN: 0005-2736, [retrieved on 20100217], DOI: 10.1016/J.BBAMEM.2010.02.012
FOSTER H ET AL: "Genetic therapeutic approaches for Duchenne muscular dystrophy", HUMAN GENE THERAPY, MARY ANN LIEBERT, NEW YORK ,NY, US, vol. 23, no. 7, 30 May 2012 (2012-05-30), pages 676 - 687, XP002688790, ISSN: 1043-0342, [retrieved on 20120530], DOI: 10.1089/HUM.2012.099
VAN VLIET LAURA ET AL: "Assessment of the feasibility of exon 45-55 multiexon skipping for duchenne muscular dystrophy", BMC MEDICAL GENETICS, BIOMED CENTRAL, LONDON, GB, vol. 9, no. 1, 1 December 2008 (2008-12-01), pages 105, XP021042784, ISSN: 1471-2350, DOI: 10.1186/1471-2350-9-105
JERRY R. MENDELL ET AL: "Gene therapy for muscular dystrophy: Lessons learned and path forward", NEUROSCIENCE LETTERS, vol. 527, no. 2, 17 May 2012 (2012-05-17), pages 90 - 99, XP055128775, ISSN: 0304-3940, DOI: 10.1016/j.neulet.2012.04.078
MOULTON H M ET AL: "Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - BIOMEMBRANES, ELSEVIER, AMSTERDAM, NL, vol. 1798, no. 12, 17 February 2010 (2010-02-17), pages 2296 - 2303, XP027430152, ISSN: 0005-2736, [retrieved on 20100217], DOI: 10.1016/J.BBAMEM.2010.02.012
Attorney, Agent or Firm:
MANDRAGOURAS, Amy, E. et al. (One Post Office SquareBoston, MA, US)
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