Title:
METHOD FOR PREDICTING EFFECTIVENESS OF TREATMENT OF HEMOGLOBINOPATHY
Document Type and Number:
WIPO Patent Application WO/2020/221291
Kind Code:
A1
Abstract:
The present invention relates to a method for treating hemoglobinopathy in an individual, comprising: (a) an evaluation step: the evaluation step comprises evaluating the ability of a first population of modified CD34-positive hematopoietic stem cells/progenitor cells to produce a desired level of γ-globin or fetal hemoglobin after differentiation, the modified CD34-positive HSPCs of the first population being derived from the individual and being modified to reduce BCL11A function; and (b) a treatment step: the treatment step comprises administering to the individual a second population of modified CD34-positive HSPCs, the modified CD34-positive HSPCs being derived from the individual and being modified to reduce BCL11A function. At the same time, the invention also relates to a method for treating hemoglobinopathy in individuals, a method for selecting individuals suffering from hemoglobinopathy for treatment using the modified CD34-positive HSPCs of the second population, and a method for determining whether an individual suffering from hemoglobinopathy is suitable or unsuitable for treatment using the second population of modified CD34-positive HSPCs derived from the individual and modified to reduce the function of BCL11A.
Inventors:
FANG RIGUO (CN)
YU LINGLING (CN)
YANG HUIHUI (CN)
YU LINGLING (CN)
YANG HUIHUI (CN)
Application Number:
PCT/CN2020/087766
Publication Date:
November 05, 2020
Filing Date:
April 29, 2020
Export Citation:
Assignee:
EDIGENE INC (CN)
International Classes:
C12N15/11; A61P7/00
Domestic Patent References:
| WO2018142364A1 | 2018-08-09 | |||
| WO2016135558A2 | 2016-09-01 | |||
| WO2013126794A1 | 2013-08-29 | |||
| WO2013176772A1 | 2013-11-28 | |||
| WO2014065596A1 | 2014-05-01 | |||
| WO2014018423A2 | 2014-01-30 |
Foreign References:
| CN108779462A | 2018-11-09 | |||
| CN104955943A | 2015-09-30 | |||
| US8697359B1 | 2014-04-15 | |||
| CN2018112068W | 2018-10-26 | |||
| CN2018112027W | 2018-10-26 |
Other References:
CHANG K.H. ET AL.: "Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing inBone-Marrow-Derived CD 34+ Hematopoietic Stem and Progenitor Cells.", MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT., vol. 4, 31 March 2017 (2017-03-31), pages 137 - 148, XP055552856, DOI: 10.1016/j.omtm.2016.12.009
D. RUND ET AL., NEW ENGLAND JOURNAL OF MEDICINE, 2005, pages 718 - 739
MARINA ET AL., MOLECULAR THERAPY, 2017
MEGAN D ET AL., BLOOD, 2015
VINJAMUR DS ET AL., BR. J. HAEMATOL., 2018
U. MANUELA ET AL., PNAS, 2008, pages 11869 - 11874
P. LIU ET AL., NATURE IMMUNOLOGY, 2003
BAUER ET AL., SCIENCE, vol. 342, 2013, pages 253 - 257
V.G. SANKARAN ET AL., SCIENCE, 2008
MATTHEW C. ET AL., NATURE, 2015
DELEAVEY GF1DAMHA MJ: "Designing chemically modified oligonucleotides for targeted gene silencing", CHEM BIOL, vol. 19, no. 8, 24 August 2012 (2012-08-24), pages 937 - 54, XP055107150, DOI: 10.1016/j.chembiol.2012.07.011
HENDEL ET AL.: "Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells", NAT BIOTECHNOL., vol. 33, no. 9, September 2015 (2015-09-01), pages 985 - 989, XP055548372, DOI: 10.1038/nbt.3290
PAIKARI A ET AL., BR J HAEMATOL., 2018
LETTRE G ET AL., LANCET, 2016
See also references of EP 3964579A4
D. RUND ET AL., NEW ENGLAND JOURNAL OF MEDICINE, 2005, pages 718 - 739
MARINA ET AL., MOLECULAR THERAPY, 2017
MEGAN D ET AL., BLOOD, 2015
VINJAMUR DS ET AL., BR. J. HAEMATOL., 2018
U. MANUELA ET AL., PNAS, 2008, pages 11869 - 11874
P. LIU ET AL., NATURE IMMUNOLOGY, 2003
BAUER ET AL., SCIENCE, vol. 342, 2013, pages 253 - 257
V.G. SANKARAN ET AL., SCIENCE, 2008
MATTHEW C. ET AL., NATURE, 2015
DELEAVEY GF1DAMHA MJ: "Designing chemically modified oligonucleotides for targeted gene silencing", CHEM BIOL, vol. 19, no. 8, 24 August 2012 (2012-08-24), pages 937 - 54, XP055107150, DOI: 10.1016/j.chembiol.2012.07.011
HENDEL ET AL.: "Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells", NAT BIOTECHNOL., vol. 33, no. 9, September 2015 (2015-09-01), pages 985 - 989, XP055548372, DOI: 10.1038/nbt.3290
PAIKARI A ET AL., BR J HAEMATOL., 2018
LETTRE G ET AL., LANCET, 2016
See also references of EP 3964579A4
Attorney, Agent or Firm:
BEIJING CAI HE LAW FIRM (CN)
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