(19)
(11) EP 3 371 306 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:
22.02.2023 Bulletin 2023/08

(45) Mention of the grant of the patent:
04.01.2023 Bulletin 2023/01

(21) Application number: 16829306.6

(22) Date of filing: 04.11.2016
(51) International Patent Classification (IPC): 
C12N 9/22(2006.01)
C12N 15/10(2006.01)
C12N 15/113(2010.01)
C07K 14/805(2006.01)
C12N 15/63(2006.01)
(52) Cooperative Patent Classification (CPC):
C12N 15/102; C12N 15/113; C12N 15/63; C12N 2310/20; C07K 14/805; A61P 7/00
(86) International application number:
PCT/IB2016/001750
(87) International publication number:
WO 2017/077394 (11.05.2017 Gazette 2017/19)

(54)

MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES

MATERIALIEN UND VERFAHREN ZUR BEHANDLUNG VON HÄMOGLOBINOPATHIEN

MATÉRIELS ET MÉTHODES POUR LE TRAITEMENT D'HÉMOGLOBINOPATHIES


(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: 04.11.2015 US 201562250823 P
27.04.2016 US 201662328203 P

(43) Date of publication of application:
12.09.2018 Bulletin 2018/37

(73) Proprietor: Vertex Pharmaceuticals Incorporated
Boston, MA 02210 (US)

(72) Inventors:
  • PORTEUS, Matthew, Hebden
    Cambridge, MA 02139-3526 (US)
  • ALLEN, Melanie, Ruth
    Cambridge, MA 02139 (US)
  • COWAN, Chad, Albert
    Cambridge, MA 02139 (US)
  • LUNDBERG, Ante, Sven
    Cambridge, MA 02139 (US)
  • LIN, Michelle, I-ching
    Cambridge, MA 02139 (US)
  • LI, Jeffrey
    Cambdridge, MA 02139 (US)
  • NGUYEN, Thao, Thi
    Cambridge, MA 02139 (US)

(74) Representative: Michalski Hüttermann & Partner Patentanwälte mbB 
Kaistraße 16A
40221 Düsseldorf
40221 Düsseldorf (DE)


(56) References cited: : 
WO-A1-2013/126794
WO-A1-2015/113063
WO-A2-2014/186585
WO-A2-2015/148863
WO-A2-2016/135557
WO-A2-2016/135559
WO-A1-2015/113063
WO-A2-2014/036219
WO-A2-2015/006498
WO-A2-2015/148863
WO-A2-2016/135558
   
  • VITTORIO SEBASTIANO ET AL: "In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases", STEM CELLS., vol. 29, no. 11, 25 October 2011 (2011-10-25), pages 1717-1726, XP055260055, US ISSN: 1066-5099, DOI: 10.1002/stem.718
  • XIAOSONG HUANG ET AL: "Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation", STEM CELLS., vol. 33, no. 5, 20 February 2015 (2015-02-20), pages 1470-1479, XP055281582, US ISSN: 1066-5099, DOI: 10.1002/stem.1969
  • G Saglio ET AL: "Italian type of deletional hereditary persistence of fetal hemoglobin", Blood, 1 September 1986 (1986-09-01), page 646, XP055283183, UNITED STATES Retrieved from the Internet: URL:http://www.bloodjournal.org/content/bl oodjournal/68/3/646.full.pdf
  • HENTHORN P S ET AL: "(A gamma delta beta)0-Thalassaemia in Blacks is due to a deletion of 34 kbp of DNA.", BRITISH JOURNAL OF HAEMATOLOGY FEB 1985, vol. 59, no. 2, February 1985 (1985-02), pages 343-356, XP009193694, ISSN: 0007-1048
  • C Camaschella ET AL: "A new hereditary persistence of fetal hemoglobin deletion has the breakpoint within the 3' beta-globin gene enhancer", Blood, 15 February 1990 (1990-02-15), page 1000, XP055283589, UNITED STATES Retrieved from the Internet: URL:http://www.bloodjournal.org/content/bl oodjournal/75/4/1000.full.pdf
  • T. J. CRADICK ET AL: "CRISPR/Cas9 systems targeting -globin and CCR5 genes have substantial off-target activity", NUCLEIC ACIDS RESEARCH, vol. 41, no. 20, 1 November 2013 (2013-11-01), pages 9584-9592, XP055186069, ISSN: 0305-1048, DOI: 10.1093/nar/gkt714
  • Anonymous: "Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation - publication history", , 17 June 2016 (2016-06-17), XP055281580, Retrieved from the Internet: URL:http://onlinelibrary.wiley.com/doi/10. 1002/stem.1969/abstract [retrieved on 2016-06-17]
  • JOLY P ET AL: "Identification and molecular characterization of four new large deletions in the beta-globin gene cluster", BLOOD CELLS, MOLECULES AND DISEASES, LAJOLLA, US, vol. 43, no. 1, 1 July 2009 (2009-07-01), pages 53-57, XP026153334, ISSN: 1079-9796, DOI: 10.1016/J.BCMD.2009.01.017 [retrieved on 2009-03-09]
   
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