SG11202112072QA - Method for predicting effectiveness of treatment of hemoglobinopathy - Google Patents

Method for predicting effectiveness of treatment of hemoglobinopathy

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Publication number
SG11202112072QA
SG11202112072QA SG11202112072QA SG11202112072QA SG11202112072QA SG 11202112072Q A SG11202112072Q A SG 11202112072QA SG 11202112072Q A SG11202112072Q A SG 11202112072QA SG 11202112072Q A SG11202112072Q A SG 11202112072QA SG 11202112072Q A SG11202112072Q A SG 11202112072QA
Authority
SG
Singapore
Prior art keywords
hemoglobinopathy
treatment
predicting effectiveness
predicting
effectiveness
Prior art date
Application number
SG11202112072QA
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 SG11202112072QA publication Critical patent/SG11202112072QA/en

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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
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/095—Sulfur, selenium, or tellurium compounds, e.g. thiols
    • A61K31/10—Sulfides; Sulfoxides; Sulfones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P7/06—Antianaemics
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/14—Hydrolases (3)
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SG11202112072QA 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy SG11202112072QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2019085116 2019-04-30
PCT/CN2020/087766 WO2020221291A1 (en) 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy

Publications (1)

Publication Number Publication Date
SG11202112072QA true SG11202112072QA (en) 2021-12-30

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SG11202112072QA SG11202112072QA (en) 2019-04-30 2020-04-29 Method for predicting effectiveness of treatment of hemoglobinopathy

Country Status (18)

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220307020A1 (en) 2019-04-15 2022-09-29 Edigene Inc. Methods and compositions for editing rnas
CN117778385A (en) 2019-07-12 2024-03-29 北京大学 Targeted RNA editing with engineered RNA using endogenous ADAR
CN117120622A (en) * 2021-04-02 2023-11-24 上海科技大学 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
KR101833589B1 (en) * 2012-02-24 2018-03-02 프레드 헛친슨 켄서 리서치 센터 Compositions and methods for the treatment of hemoglobinopathies
DE202013012242U1 (en) 2012-05-25 2016-02-02 Emmanuelle Charpentier Compositions for RNA-directed modification of a target DNA and for RNA-driven modulation of transcription
CN105188767A (en) 2012-07-25 2015-12-23 布罗德研究所有限公司 Inducible DNA binding proteins and genome perturbation tools and applications thereof
DE202013012597U1 (en) 2012-10-23 2017-11-21 Toolgen, Inc. A composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and a Cas protein-encoding nucleic acid or Cas protein, and their use
CN109554350B (en) * 2012-11-27 2022-09-23 儿童医疗中心有限公司 Targeting BCL11A distal regulatory elements for fetal hemoglobin re-induction
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2016135557A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
BR112018013065A2 (en) * 2015-12-28 2018-12-11 Intellia Therapeutics Inc compositions and methods for the treatment of hemoglobinopathies
TW201839136A (en) * 2017-02-06 2018-11-01 瑞士商諾華公司 Compositions and methods for the treatment of hemoglobinopathies
WO2018218135A1 (en) * 2017-05-25 2018-11-29 The Children's Medical Center Corporation Bcl11a guide delivery

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EA202192931A1 (en) 2022-02-22
KR20220002565A (en) 2022-01-06
WO2020221291A1 (en) 2020-11-05
BR112021021744A2 (en) 2021-12-28
CN113785059A (en) 2021-12-10
PE20220003A1 (en) 2022-01-05
MX2021013363A (en) 2021-11-18
AU2020265060A1 (en) 2021-12-16
JP2022531269A (en) 2022-07-06
CO2021016058A2 (en) 2021-12-10
CR20210591A (en) 2022-03-11
CL2021002854A1 (en) 2022-08-12
EP3964579A4 (en) 2023-07-26
IL287672A (en) 2021-12-01
CA3138029A1 (en) 2020-11-05
BR112021021744A8 (en) 2023-03-21
US20220193142A1 (en) 2022-06-23
MA55817A (en) 2022-03-09
EP3964579A1 (en) 2022-03-09

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