CL2021002854A1 - Método para la predicción de eficacia del tratamiento de hemoglobinopatía - Google Patents

Método para la predicción de eficacia del tratamiento de hemoglobinopatía

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Publication number
CL2021002854A1
CL2021002854A1 CL2021002854A CL2021002854A CL2021002854A1 CL 2021002854 A1 CL2021002854 A1 CL 2021002854A1 CL 2021002854 A CL2021002854 A CL 2021002854A CL 2021002854 A CL2021002854 A CL 2021002854A CL 2021002854 A1 CL2021002854 A1 CL 2021002854A1
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CL
Chile
Prior art keywords
modified
individual
treatment
hemoglobinopathy
population
Prior art date
Application number
CL2021002854A
Other languages
English (en)
Spanish (es)
Inventor
Riguo Fang
Lingling Yu
Huihui Yang
Original Assignee
Edigene 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 Inc filed Critical Edigene Inc
Publication of CL2021002854A1 publication Critical patent/CL2021002854A1/es

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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • C12N15/113—Non-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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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/66—Phosphorus compounds
    • A61K31/675—Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
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    • A61K31/706—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
    • 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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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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)
    • A—HUMAN NECESSITIES
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Plant Pathology (AREA)
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CL2021002854A 2019-04-30 2021-10-29 Método para la predicción de eficacia del tratamiento de hemoglobinopatía CL2021002854A1 (es)

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CN2019085116 2019-04-30

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CL2021002854A1 true CL2021002854A1 (es) 2022-08-12

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CL2021002854A CL2021002854A1 (es) 2019-04-30 2021-10-29 Método para la predicción de eficacia del tratamiento de hemoglobinopatía

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US (1) US20220193142A1 (enExample)
EP (1) EP3964579A4 (enExample)
JP (1) JP2022531269A (enExample)
KR (1) KR20220002565A (enExample)
CN (1) CN113785059A (enExample)
AU (1) AU2020265060A1 (enExample)
BR (1) BR112021021744A8 (enExample)
CA (1) CA3138029A1 (enExample)
CL (1) CL2021002854A1 (enExample)
CO (1) CO2021016058A2 (enExample)
CR (1) CR20210591A (enExample)
EA (1) EA202192931A1 (enExample)
IL (1) IL287672A (enExample)
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MX (1) MX2021013363A (enExample)
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Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3136735A1 (en) 2019-04-15 2020-10-22 Edigene Inc. Methods and compositions for editing rnas
AU2020313143B2 (en) 2019-07-12 2024-04-04 Peking University Targeted RNA editing by leveraging endogenous adar using engineered RNAs
EP4314308A4 (en) * 2021-04-02 2026-05-20 Univ Shanghai Technology GENE THERAPY FOR THE TREATMENT OF BETA-HEMOGLOBINOPATHIES

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2836226B1 (en) * 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
CN116622704A (zh) 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
EP4397759A3 (en) 2012-10-23 2024-10-09 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
ES2708948T3 (es) * 2012-11-27 2019-04-12 Childrens Medical Ct Corp Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2016135558A2 (en) * 2015-02-23 2016-09-01 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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EA202192931A1 (ru) 2022-02-22
EP3964579A4 (en) 2023-07-26
US20220193142A1 (en) 2022-06-23
SG11202112072QA (en) 2021-12-30
EP3964579A1 (en) 2022-03-09
BR112021021744A8 (pt) 2023-03-21
IL287672A (en) 2021-12-01
MX2021013363A (es) 2021-11-18
WO2020221291A1 (zh) 2020-11-05
CO2021016058A2 (es) 2021-12-10
CA3138029A1 (en) 2020-11-05
BR112021021744A2 (enExample) 2021-12-28
PH12021552732A1 (en) 2023-01-16
JP2022531269A (ja) 2022-07-06
MA55817A (fr) 2022-03-09
CN113785059A (zh) 2021-12-10
PE20220003A1 (es) 2022-01-05
KR20220002565A (ko) 2022-01-06
CR20210591A (es) 2022-03-11
AU2020265060A1 (en) 2021-12-16

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