Title:
THERAPEUTIC AGENT FOR X-LINKED SIDEROBLASTIC ANEMIA
Document Type and Number:
WIPO Patent Application WO/2022/145144
Kind Code:
A1
Abstract:
The present invention provides: a pharmaceutical composition for treating X-linked sideroblastic anemia, the composition comprising a demethylating agent, in particular, 5−azacitidine or a derivative thereof; and normal erythroid cells that are differentiated and induced from iPS cells derived from a patient with X-linked sideroblastic anemia in the presence of a demethylating agent.
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Inventors:
YOSHIDA YOSHINORI (JP)
MORIMOTO YUKI (JP)
CHONABAYASHI KAZUHISA (JP)
TAKAORI AKIFUMI (JP)
KAWABATA HIROSHI (JP)
MORIMOTO YUKI (JP)
CHONABAYASHI KAZUHISA (JP)
TAKAORI AKIFUMI (JP)
KAWABATA HIROSHI (JP)
Application Number:
PCT/JP2021/042347
Publication Date:
July 07, 2022
Filing Date:
November 11, 2021
Export Citation:
Assignee:
UNIV KYOTO (JP)
International Classes:
A61K45/00; A61K31/706; A61P7/06; A61P43/00; C12N5/078; C12N5/10
Domestic Patent References:
| WO2018230505A1 | 2018-12-20 |
Other References:
CAZZOLA MARIO, MAY ALISON, BERGAMASCHI GAETANO, CERANI PAOLA, ROSTI VITTORIO, BISHOP DAVID F.: "Familial-skewed X-chromosome inactivation as a predisposing factor for late-onset X-linked sideroblastic anemia in carrier females", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 96, no. 13, 15 December 2000 (2000-12-15), US , pages 4363 - 4365, XP055947457, ISSN: 0006-4971, DOI: 10.1182/blood.V96.13.4363.h8004363_4363_4365
INOKURA KYOKO; FUJIWARA TOHRU; SAITO KEI; IINO TATSUYA; HATTA SHUNSUKE; OKITSU YOKO; FUKUHARA NORIKO; ONISHI YASUSHI; ISHIZAWA KEN: "Impact of TET2 deficiency on iron metabolism in erythroblasts", EXPERIMENTAL HEMATALOGY, ELSEVIER INC., US, vol. 49, 5 February 2017 (2017-02-05), US , pages 56, XP029980229, ISSN: 0301-472X, DOI: 10.1016/j.exphem.2017.01.002
MORITA YASUYOSHI, MAEDA YASUHIRO, YAMAGUCHI TERUFUMI, URASE FUMIAKI, KAWATA SHUHEI, HANAMOTO HITOSHI, TSUBAKI KAZUO, ISHIKAWA JUN,: "Five-day regimen of azacitidine for lower-risk myelodysplastic syndromes (refractory anemia or refractory anemia with ringed sideroblasts): A prospective single-arm phase 2 trial", CANCER SCIENCE, JAPANESE CANCER ASSOCIATION, TOKYO, JP, vol. 109, no. 10, 1 October 2018 (2018-10-01), JP , pages 3209 - 3215, XP055947458, ISSN: 1347-9032, DOI: 10.1111/cas.13739
INOKURA KYOKO; FUJIWARA TOHRU; SAITO KEI; IINO TATSUYA; HATTA SHUNSUKE; OKITSU YOKO; FUKUHARA NORIKO; ONISHI YASUSHI; ISHIZAWA KEN: "Impact of TET2 deficiency on iron metabolism in erythroblasts", EXPERIMENTAL HEMATALOGY, ELSEVIER INC., US, vol. 49, 5 February 2017 (2017-02-05), US , pages 56, XP029980229, ISSN: 0301-472X, DOI: 10.1016/j.exphem.2017.01.002
MORITA YASUYOSHI, MAEDA YASUHIRO, YAMAGUCHI TERUFUMI, URASE FUMIAKI, KAWATA SHUHEI, HANAMOTO HITOSHI, TSUBAKI KAZUO, ISHIKAWA JUN,: "Five-day regimen of azacitidine for lower-risk myelodysplastic syndromes (refractory anemia or refractory anemia with ringed sideroblasts): A prospective single-arm phase 2 trial", CANCER SCIENCE, JAPANESE CANCER ASSOCIATION, TOKYO, JP, vol. 109, no. 10, 1 October 2018 (2018-10-01), JP , pages 3209 - 3215, XP055947458, ISSN: 1347-9032, DOI: 10.1111/cas.13739
Attorney, Agent or Firm:
YAMAO, Norihito et al. (JP)
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