AU2020265060A1 - Method for predicting effectiveness of treatment of hemoglobinopathy - Google Patents

Method for predicting effectiveness of treatment of hemoglobinopathy Download PDF

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Publication number
AU2020265060A1
AU2020265060A1 AU2020265060A AU2020265060A AU2020265060A1 AU 2020265060 A1 AU2020265060 A1 AU 2020265060A1 AU 2020265060 A AU2020265060 A AU 2020265060A AU 2020265060 A AU2020265060 A AU 2020265060A AU 2020265060 A1 AU2020265060 A1 AU 2020265060A1
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modified
cells
individual
population
positive
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AU2020265060A
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Riguo FANG
Huihui YANG
Lingling Yu
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Edigene Guangzhou Inc
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Edigene Guangzhou Inc
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Assigned to EDIGENE (GUANGZHOU) INC. reassignment EDIGENE (GUANGZHOU) INC. Request for Assignment Assignors: EDIGENE INC.
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    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
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    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774—Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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AU2020265060A 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy Abandoned AU2020265060A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2019085116 2019-04-30
CNPCT/CN2019/085116 2019-04-30
PCT/CN2020/087766 WO2020221291A1 (zh) 2019-04-30 2020-04-29 一种血红蛋白病治疗有效性预测方法

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AU2020265060A1 true AU2020265060A1 (en) 2021-12-16

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AU2020265060A Abandoned AU2020265060A1 (en) 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy

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US (1) US20220193142A1 (https=)
EP (1) EP3964579A4 (https=)
JP (1) JP2022531269A (https=)
KR (1) KR20220002565A (https=)
CN (1) CN113785059A (https=)
AU (1) AU2020265060A1 (https=)
BR (1) BR112021021744A8 (https=)
CA (1) CA3138029A1 (https=)
CL (1) CL2021002854A1 (https=)
CO (1) CO2021016058A2 (https=)
CR (1) CR20210591A (https=)
EA (1) EA202192931A1 (https=)
IL (1) IL287672A (https=)
MA (1) MA55817A (https=)
MX (1) MX2021013363A (https=)
PE (1) PE20220003A1 (https=)
PH (1) PH12021552732A1 (https=)
SG (1) SG11202112072QA (https=)
WO (1) WO2020221291A1 (https=)

Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
EP3956449B1 (en) 2019-04-15 2026-02-25 EdiGene Therapeutics (Beijing) Inc. Methods and compositions for editing rnas
EP3997229A4 (en) 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
WO2022206986A1 (en) * 2021-04-02 2022-10-06 Shanghaitech University Gene therapy for treating beta-hemoglobinopathies

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US20150166969A1 (en) * 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
AU2013335451C1 (en) 2012-10-23 2024-07-04 Toolgen Incorporated Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof
CN109554350B (zh) * 2012-11-27 2022-09-23 儿童医疗中心有限公司 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
BR112017017812A2 (en) * 2015-02-23 2018-04-10 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
MX2018007987A (es) * 2015-12-28 2019-01-10 Novartis Ag Composiciones y metodos para el tratamiento de hemoglobinopatias.
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US11788087B2 (en) * 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery

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US20220193142A1 (en) 2022-06-23
CO2021016058A2 (es) 2021-12-10
EA202192931A1 (ru) 2022-02-22
CL2021002854A1 (es) 2022-08-12
SG11202112072QA (en) 2021-12-30
BR112021021744A2 (https=) 2021-12-28
CN113785059A (zh) 2021-12-10
JP2022531269A (ja) 2022-07-06
BR112021021744A8 (pt) 2023-03-21
CA3138029A1 (en) 2020-11-05
PH12021552732A1 (en) 2023-01-16
KR20220002565A (ko) 2022-01-06
EP3964579A1 (en) 2022-03-09
MA55817A (fr) 2022-03-09
PE20220003A1 (es) 2022-01-05
IL287672A (en) 2021-12-01
EP3964579A4 (en) 2023-07-26
MX2021013363A (es) 2021-11-18
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CR20210591A (es) 2022-03-11

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