Title:
MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
Document Type and Number:
WIPO Patent Application WO/2016/135558
Kind Code:
A3
Abstract:
The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.
Inventors:
PORTEUS MATTHEW HEBDEN (US)
CRADICK THOMAS JAMES (US)
BAO GANG (US)
LEE CIARAN M (US)
CRADICK THOMAS JAMES (US)
BAO GANG (US)
LEE CIARAN M (US)
Application Number:
PCT/IB2016/000282
Publication Date:
October 20, 2016
Filing Date:
February 23, 2016
Export Citation:
Assignee:
CRISPR THERAPEUTICS AG (CH)
GEORGIA TECH RES INST (US)
UNIV RICE WILLIAM M (US)
GEORGIA TECH RES INST (US)
UNIV RICE WILLIAM M (US)
International Classes:
C12N15/113; C12N15/09; C12N15/63
Domestic Patent References:
| WO2013126794A1 | 2013-08-29 | |||
| WO2014036219A2 | 2014-03-06 | |||
| WO2015006498A2 | 2015-01-15 | |||
| WO2014186585A2 | 2014-11-20 |
Other References:
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), US, pages 1717 - 1726, XP055260055, ISSN: 1066-5099, DOI: 10.1002/stem.718
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, 11 August 2013 (2013-08-11), pages 9584 - 9592, XP055186069, ISSN: 0305-1048, DOI: 10.1093/nar/gkt714
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), US, pages 1470 - 1479, XP055281582, 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), UNITED STATES, pages 646, XP055283183, Retrieved from the Internet [retrieved on 20160623]
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), UNITED STATES, pages 1000, XP055283589, Retrieved from the Internet [retrieved on 20160624]
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, 9 March 2009 (2009-03-09), pages 53 - 57, XP026153334, ISSN: 1079-9796, [retrieved on 20090309], DOI: 10.1016/J.BCMD.2009.01.017
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, 11 August 2013 (2013-08-11), pages 9584 - 9592, XP055186069, ISSN: 0305-1048, DOI: 10.1093/nar/gkt714
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), US, pages 1470 - 1479, XP055281582, 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), UNITED STATES, pages 646, XP055283183, Retrieved from the Internet
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), UNITED STATES, pages 1000, XP055283589, Retrieved from the Internet
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, 9 March 2009 (2009-03-09), pages 53 - 57, XP026153334, ISSN: 1079-9796, [retrieved on 20090309], DOI: 10.1016/J.BCMD.2009.01.017
Attorney, Agent or Firm:
PILKINGTON, Stephanie, J. (The Belgrave CentreTalbot Street, Nottingham NG1 5GG, GB)
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