SYSTEMS AND METHODS FOR TREATING HYPER-IGM SYNDROME

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EP 3 701 028 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:
	23.10.2024 Bulletin 2024/43

(45)	Mention of the grant of the patent:
	11.09.2024 Bulletin 2024/37

(21)	Application number: 18815370.4

(22)	Date of filing: 24.10.2018

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International Patent Classification (IPC):

C12N 15/113^(2010.01)
C12N 9/22^(2006.01)

C12N 15/90^(2006.01)
A61P 37/02^(2006.01)

(52)	Cooperative Patent Classification (CPC):
	A61K 48/005; C12N 9/22; C12N 15/113; C12N 15/907; C12N 2750/14143; C12N 2310/20; A61P 37/02; A61K 39/461; A61K 39/4611; A61K 39/464411

(86)	International application number:
	PCT/US2018/057354

(87)	International publication number:
	WO 2019/084168 (02.05.2019 Gazette 2019/18)

(54)	SYSTEMS AND METHODS FOR TREATING HYPER-IGM SYNDROME SYSTEME UND VERFAHREN ZUR BEHANDLUNG VON HYPER-IGM-SYNDROM SYSTÈMES ET MÉTHODES DE TRAITEMENT DU SYNDROME D'HYPER-IGM

(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

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Priority:

24.10.2017 US 201762576277 P
30.04.2018 US 201862664800 P
17.05.2018 US 201862673039 P
26.06.2018 US 201862690284 P
17.10.2018 US 201862746900 P

(43)	Date of publication of application:
	02.09.2020 Bulletin 2020/36

(73)	Proprietors:
	Editas Medicine, Inc. Cambridge, MA 02141 (US) Fondazione Telethon ETS 00185 Roma (IT) Ospedale San Raffaele S.r.l. 20132 Milano (IT)

(72)	Inventors:
	COTTA-RAMUSINO, Cecillia Cambridge,MA 02139 (US) MARGULIES, Carrie, M. Waban,MA 02468 (US) NALDINI, Luigi 20132 Milan (IT) GENOVESE, Pietro 20132 Milan (IT)

(74)	Representative: D Young & Co LLP
	3 Noble Street London EC2V 7BQ London EC2V 7BQ (GB)

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References cited: :

WO-A1-2016/183345

TAHARA MINORU ET AL: "Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency", NATURE MEDICINE, vol. 10, no. 8, 25 July 2004 (2004-07-25), New York, pages 835 - 841, XP055805708, ISSN: 1078-8956, Retrieved from the Internet <URL:https://www.nature.com/articles/nm1086.pdf> DOI: 10.1038/nm1086
TAHARA MINORU ET AL: "Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency - Supplementary Methods", NATURE MEDICINE, 25 July 2004 (2004-07-25), XP055805730, Retrieved from the Internet <URL:https://static-content.springer.com/esm/art:10.1038/nm1086/MediaObjects/41591_2004_BFnm1086_MOESM4_ESM.pdf> [retrieved on 20210519]
CAROLINE KUO: "Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome", CDI SYMPOSIUM 2016, May 2016 (2016-05-01), XP055554527, Retrieved from the Internet <URL:https://www.uclahealth.org/mattel/cdi/workfiles/Symposium/2016/CKuo-CDI-Symposium-2016.pdf> [retrieved on 20190211]
HUBBARD NICHOLAS ET AL: "Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome", BLOOD, vol. 127, no. 21, 22 February 2016 (2016-02-22), US, pages 2513 - 2522, XP055397792, ISSN: 0006-4971, DOI: 10.1182/blood-2015-11-683235
NICHOLAS HUBBARD ET AL: "Supplemental Methods", BLOOD, 22 February 2016 (2016-02-22), XP055554515, Retrieved from the Internet <URL:http://www.bloodjournal.org/content/bloodjournal/suppl/2016/02/22/blood-2015-11-683235.DC1/blood-2015-11-683235-1.pdf> [retrieved on 20190211]
DAISUKE TOMIZAWA ET AL: "Allogeneic hematopoietic stem cell transplantation for seven children with X-linked hyper-IgM syndrome: A single center experience", AMERICAN JOURNAL OF HEMATOLOGY, vol. 76, no. 1, 20 May 2004 (2004-05-20), US, pages 33 - 39, XP055554737, ISSN: 0361-8609, DOI: 10.1002/ajh.20044
CAROLINE Y. KUO ET AL: "121. Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 23, May 2015 (2015-05-01), US, pages S50, XP055554579, ISSN: 1525-0016, DOI: 10.1016/S1525-0016(16)33726-1
CAROLINE Y. KUO ET AL: "Site Specific Gene Correction of Defects in CD40 Ligand Using the Crispr/Cas9 Genome Editing Platform", JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, vol. 135, no. 2, 2 February 2015 (2015-02-02), AMSTERDAM, NL, pages AB17, XP055554584, ISSN: 0091-6749, DOI: 10.1016/j.jaci.2014.12.987
CAROLINE Y. KUO ET AL: "Patient Specific Targeted Gene Therapy In The Treatment Of X-Linked Hyper-IgM Syndrome", JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, vol. 133, no. 2, 23 January 2014 (2014-01-23), AMSTERDAM, NL, pages AB162, XP055554585, ISSN: 0091-6749, DOI: 10.1016/j.jaci.2013.12.588
MATTHEW H. PORTEUS: "Knock-in editing: it functionally corrects!", BLOOD, vol. 127, no. 21, 26 May 2016 (2016-05-26), US, pages 2507 - 2509, XP055554520, ISSN: 0006-4971, DOI: 10.1182/blood-2016-03-703181
A. JAIN ET AL: "Partial immune reconstitution of X-linked hyper IgM syndrome with recombinant CD40 ligand", BLOOD, vol. 118, no. 14, 12 August 2011 (2011-08-12), US, pages 3811 - 3817, XP055554490, ISSN: 0006-4971, DOI: 10.1182/blood-2011-04-351254
JULIETTE M. K. M. DELHOVE ET AL: "Genome-Edited T Cell Therapies", CURRENT STEM CELL REPORTS, vol. 3, no. 2, 18 April 2017 (2017-04-18), pages 124 - 136, XP055554337, DOI: 10.1007/s40778-017-0077-5
CAROLINE Y. KUO ET AL: "Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome", CELL REPORTS, vol. 23, no. 9, 29 May 2018 (2018-05-29), US, pages 2606 - 2616, XP055554129, ISSN: 2211-1247, DOI: 10.1016/j.celrep.2018.04.103

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