EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

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EP 2 970 964 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:
	06.03.2019 Bulletin 2019/10

(45)	Mention of the grant of the patent:
	26.12.2018 Bulletin 2018/52

(21)	Application number: 14723583.2

(22)	Date of filing: 14.03.2014

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International Patent Classification (IPC):

C12N 15/11^(2006.01)
A61K 31/7088^(2006.01)

C12N 15/113^(2010.01)
A61P 21/00^(2006.01)

(86)	International application number:
	PCT/US2014/029766

(87)	International publication number:
	WO 2014/153240 (25.09.2014 Gazette 2014/39)

(54)	EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY VERBESSERTE EXON-SKIPPING-ZUSAMMENSETZUNGEN ZUR BEHANDLUNG VON MUSKELDYSTROPHIE COMPOSITIONS DE SAUTS D'EXON POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE

(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
	Designated Extension States:
	BA ME

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Priority:

14.03.2013 US 201361782706 P

(43)	Date of publication of application:
	20.01.2016 Bulletin 2016/03

(60)	Divisional application:
	18215621.6

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

(72)	Inventors:
	BESTWICK, Richard, K. Bend, OR 97707 (US) FRANK, Diane, Elizabeth Seattle, WA 98115 (US)

(74)	Representative: D Young & Co LLP
	120 Holborn London EC1N 2DY London EC1N 2DY (GB)

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References cited: :

WO-A1-2010/048586
US-A1- 2010 168 212

WO-A1-2012/029986

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 2010-01-15]
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, DOI: 10.1089/HUM.2012.099 [retrieved on 2012-05-30]
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), page 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, DOI: 10.1016/J.BBAMEM.2010.02.012 [retrieved on 2010-02-17]

Note: Within nine months from the publication of the mention of the grant of the European patent, any person may give notice to the European Patent Office of opposition to the European patent granted. Notice of opposition shall be filed in a written reasoned statement. It shall not be deemed to have been filed until the opposition fee has been paid. (Art. 99(1) European Patent Convention).