(19)
(11) EP 2 785 840 B8

(12) CORRECTED EUROPEAN PATENT SPECIFICATION
Note: Bibliography reflects the latest situation

(15) Correction information:
Corrected version no 1 (W1 B1)

(48) Corrigendum issued on:
22.05.2019 Bulletin 2019/21

(45) Mention of the grant of the patent:
20.03.2019 Bulletin 2019/12

(21) Application number: 12812432.8

(22) Date of filing: 30.11.2012
(51) International Patent Classification (IPC): 
C12N 15/113(2010.01)
A61P 25/00(2006.01)
 A61K 31/7125(2006.01)
(86) International application number:
PCT/US2012/067475
(87) International publication number:
WO 2013/082551 (06.06.2013 Gazette 2013/23)

(54)

INDUCED EXON INCLUSION IN SPINAL MUSCLE ATROPHY

INDUZIERTE EXON-INKLUSION IN SPINALER MUSKELATROPHIE

INCLUSION D'EXON INDUITE POUR L'AMYOTROPHIE SPINALE

(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

(30) Priority: 30.11.2011 US 201161565499 P

(43) Date of publication of application:
08.10.2014 Bulletin 2014/41

(73) Proprietor: Sarepta Therapeutics, Inc.
Cambridge, MA 02142 (US)

(72) Inventors:
  • LINSLEY, Peter
    Seattle, WA 98119 (US)
  • LEPPERT, Brian, James
    Kenmore, WA 98028 (US)

(74) Representative: D Young & Co LLP 
Briton House Briton Street
Southampton SO14 3EB
Southampton SO14 3EB (GB)


(56) References cited: : 
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US-A1- 2006 287 268
US-A1- 2011 269 820
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WO-A2-2011/150408
US-A1- 2008 194 463
   
  • A. H. M. BURGHES ET AL: 'Antisense oligonucleotides and spinal muscular atrophy: skipping along' GENES & DEVELOPMENT vol. 24, no. 15, 01 August 2010, pages 1574 - 1579, XP55044388 DOI: 10.1101/gad.1961710 ISSN: 0890-9369
  • CARTEGNI L ET AL: "Correction of disease-associated exon skipping by synthetic exon-specific activators", NATURE STRUCTURAL BIOLOGY, NATURE PUBLISHING GROUP, NEW YORK, NY, US, vol. 10, no. 2, 1 February 2003 (2003-02-01), pages 120-125, XP002262731, ISSN: 1072-8368, DOI: 10.1038/NSB887
  • N. N. SINGH ET AL: "An antisense microwalk reveals critical role of an intronic position linked to a unique long-distance interaction in pre-mRNA splicing", RNA, vol. 16, no. 6, 1 June 2010 (2010-06-01), pages 1167-1181, XP55056239, ISSN: 1355-8382, DOI: 10.1261/rna.2154310
  • HUA YIMIN ET AL: "Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon", PLOS BIOLOGY, PUBLIC LIBRARY OF SCIENCE, US, vol. 5, no. 4, 1 April 2007 (2007-04-01), page E73, XP002596113, ISSN: 1544-9173
  • LIM S R ET AL: "Modulation of Survival Motor Neuron Pre-MRNA Splicing by Inhibition of Alternative 3'Splice Site Pairing", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 276, no. 48, 30 November 2001 (2001-11-30), pages 45476-45483, XP008124558, ISSN: 0021-9258, DOI: 10.1074/JBC.M107632200 [retrieved on 2001-10-02]
  • D. L. SWENSON ET AL: "Chemical Modifications of Antisense Morpholino Oligomers Enhance Their Efficacy against Ebola Virus Infection", ANTIMICROBIAL AGENTS AND CHEMOTHERAPY, vol. 53, no. 5, 1 May 2009 (2009-05-01), pages 2089-2099, XP55056348, ISSN: 0066-4804, DOI: 10.1128/AAC.00936-08
  • SAZANI P ET AL: "Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 29, no. 19, 1 October 2001 (2001-10-01), pages 3965-3974, XP002978135, ISSN: 0305-1048
   
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