CN115038789A - 治疗性编辑 - Google Patents

治疗性编辑 Download PDF

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Publication number
CN115038789A
CN115038789A CN202080095469.1A CN202080095469A CN115038789A CN 115038789 A CN115038789 A CN 115038789A CN 202080095469 A CN202080095469 A CN 202080095469A CN 115038789 A CN115038789 A CN 115038789A
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target rna
rna
polypeptide
sequence
composition
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CN202080095469.1A
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Chinese (zh)
Inventor
大卫·胡斯
普拉桑特·马里
阿努帕玛·拉克什马南
克里斯托弗·奈耶
扬尼斯·萨瓦
利亚纳·斯泰因
理查德·沙利文
拉斐尔·庞塞
苏珊·伯恩
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Shape Therapy Co
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Shape Therapy Co
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    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
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    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080095469.1A 2019-12-02 2020-12-01 治疗性编辑 Pending CN115038789A (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201962942667P 2019-12-02 2019-12-02
US201962942693P 2019-12-02 2019-12-02
US201962942683P 2019-12-02 2019-12-02
US62/942,693 2019-12-02
US62/942,683 2019-12-02
US62/942,667 2019-12-02
US202063022727P 2020-05-11 2020-05-11
US63/022,727 2020-05-11
US202063030165P 2020-05-26 2020-05-26
US63/030,165 2020-05-26
US202063112286P 2020-11-11 2020-11-11
US63/112,286 2020-11-11
PCT/US2020/062756 WO2021113270A1 (en) 2019-12-02 2020-12-01 Therapeutic editing

Publications (1)

Publication Number Publication Date
CN115038789A true CN115038789A (zh) 2022-09-09

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ID=74046152

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CN202080095469.1A Pending CN115038789A (zh) 2019-12-02 2020-12-01 治疗性编辑

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US (2) US11827880B2 (https=)
EP (1) EP4069842A1 (https=)
JP (1) JP2023504314A (https=)
CN (1) CN115038789A (https=)
AU (1) AU2020395113A1 (https=)
CA (1) CA3159944A1 (https=)
WO (1) WO2021113270A1 (https=)

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WO2024256620A1 (en) 2023-06-16 2024-12-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurodegenerative disease
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
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