PH12021552732A1 - Method for predicting effectiveness of treatment of hemoglobinopathy - Google Patents

Method for predicting effectiveness of treatment of hemoglobinopathy

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Publication number
PH12021552732A1
PH12021552732A1 PH1/2021/552732A PH12021552732A PH12021552732A1 PH 12021552732 A1 PH12021552732 A1 PH 12021552732A1 PH 12021552732 A PH12021552732 A PH 12021552732A PH 12021552732 A1 PH12021552732 A1 PH 12021552732A1
Authority
PH
Philippines
Prior art keywords
modified
individual
hemoglobinopathy
population
treatment
Prior art date
Application number
PH1/2021/552732A
Other languages
English (en)
Inventor
Riguo Fang
Huihui Yang
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 PH12021552732A1 publication Critical patent/PH12021552732A1/en

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
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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
    • A—HUMAN NECESSITIES
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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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  • Plant Pathology (AREA)
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PH1/2021/552732A 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy PH12021552732A1 (en)

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

Publications (1)

Publication Number Publication Date
PH12021552732A1 true PH12021552732A1 (en) 2023-01-16

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Application Number Title Priority Date Filing Date
PH1/2021/552732A PH12021552732A1 (en) 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy

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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)
MA (1) MA55817A (enExample)
MX (1) MX2021013363A (enExample)
PE (1) PE20220003A1 (enExample)
PH (1) PH12021552732A1 (enExample)
SG (1) SG11202112072QA (enExample)
WO (1) WO2020221291A1 (enExample)

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
CL2021002854A1 (es) 2022-08-12
CO2021016058A2 (es) 2021-12-10
CA3138029A1 (en) 2020-11-05
BR112021021744A2 (enExample) 2021-12-28
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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