COMBINED THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY

(19)

(11)

EP 3 307 891 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:
	13.11.2019 Bulletin 2019/46

(45)	Mention of the grant of the patent:
	02.10.2019 Bulletin 2019/40

(21)	Application number: 16734566.9

(22)	Date of filing: 10.06.2016

(51)

International Patent Classification (IPC):

C12N 15/113^(2010.01)
A61K 31/7105^(2006.01)

C12N 15/11^(2006.01)
A61K 31/712^(2006.01)

(86)	International application number:
	PCT/EP2016/063404

(87)	International publication number:
	WO 2016/198676 (15.12.2016 Gazette 2016/50)

(54)	COMBINED THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY KOMBINATIONSTHERAPIE DER DUCHENNE-MUSKELDYSTROPHIE THÉRAPIE COMBINÉE POUR LA DYSTROPHIE MUSCULAIRE DE DUCHENNE

(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:

10.06.2015 EP 15305890

(43)	Date of publication of application:
	18.04.2018 Bulletin 2018/16

(73)	Proprietors:
	Association Institut de Myologie 75013 Paris (FR) Sorbonne Université 75006 Paris (FR) INSERM - Institut National de la Santé et de la Recherche Médicale 75013 Paris (FR) Centre National de la Recherche Scientifique 75016 Paris (FR)

(72)	Inventors:
	LORAIN, Stéphanie 94300 Vincennes (FR) VOIT, Thomas London E149RU (GB) WOOD, Matthew Oxford OX2 6HY (GB) MCCLOREY, Graham Oxford OX4 4HS (GB)

(74)	Representative: Cabinet Becker et Associés
	25, rue Louis le Grand 75002 Paris 75002 Paris (FR)

(56)

References cited: :

MAËVA LE HIR ET AL: "AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy", MOLECULAR THERAPY, vol. 21, no. 8, 1 August 2013 (2013-08-01) , pages 1551-1558, XP055229819, GB ISSN: 1525-0016, DOI: 10.1038/mt.2013.121
WILLEM M.H. HOOGAARS ET AL: "Combined Effect of AAV-U7-Induced Dystrophin Exon Skipping and Soluble Activin Type IIB Receptor in mdx Mice", HUMAN GENE THERAPY, vol. 23, no. 12, 1 December 2012 (2012-12-01), pages 1269-1279, XP055229829, US ISSN: 1043-0342, DOI: 10.1089/hum.2012.056
CAROLINE LE GUINER ET AL: "Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients", MOLECULAR THERAPY, vol. 22, no. 11, 4 August 2014 (2014-08-04), pages 1923-1935, XP055230114, GB ISSN: 1525-0016, DOI: 10.1038/mt.2014.151
JEAN-BAPTISTE DUPONT ET AL: "Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 2, 8 April 2015 (2015-04-08), page 15010, XP055229821, DOI: 10.1038/mtm.2015.10
CORINNE A. BETTS ET AL: "Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice", SCIENTIFIC REPORTS, vol. 5, 11 March 2015 (2015-03-11), page 8986, XP055229827, DOI: 10.1038/srep08986

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).