MX2021013363A - Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia. - Google Patents

Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia.

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Publication number
MX2021013363A
MX2021013363A MX2021013363A MX2021013363A MX2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A MX 2021013363 A MX2021013363 A MX 2021013363A
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MX
Mexico
Prior art keywords
modified
individual
hemoglobinopathy
population
treatment
Prior art date
Application number
MX2021013363A
Other languages
English (en)
Spanish (es)
Inventor
Huihui Yang
Riguo Fang
Lingling Yu
Original Assignee
Edigene Guangzhou Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Edigene Guangzhou Inc filed Critical Edigene Guangzhou Inc
Publication of MX2021013363A publication Critical patent/MX2021013363A/es

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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K31/10Sulfides; Sulfoxides; Sulfones
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    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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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    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
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MX2021013363A 2019-04-30 2020-04-29 Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia. MX2021013363A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2019085116 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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MX2021013363A true MX2021013363A (es) 2021-11-18

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MX2021013363A MX2021013363A (es) 2019-04-30 2020-04-29 Metodo para la prediccion de eficacia del tratamiento de hemoglobinopatia.

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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)

* Cited by examiner, † Cited by third party
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2020265060A1 (en) 2021-12-16
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
WO2020221291A1 (zh) 2020-11-05
CR20210591A (es) 2022-03-11

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