CN113891940B - 视网膜色素变性的新治疗方法 - Google Patents

视网膜色素变性的新治疗方法 Download PDF

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CN113891940B
CN113891940B CN202080040006.5A CN202080040006A CN113891940B CN 113891940 B CN113891940 B CN 113891940B CN 202080040006 A CN202080040006 A CN 202080040006A CN 113891940 B CN113891940 B CN 113891940B
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antisense
antisense oligomer
cnot3
oligomer
rna
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CN113891940A (zh
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S.弗莱彻
I.皮图特
J.格赖诺克
S.D.威尔顿
F.K.陈
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Vision Pharmaceutical Pte Ltd
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Vision Pharmaceutical Pte Ltd
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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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/35—Nature of the modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
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CN202080040006.5A 2019-05-27 2020-05-25 视网膜色素变性的新治疗方法 Active CN113891940B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
AU2019901804A AU2019901804A0 (en) 2019-05-27 Novel Retinitis Pigmentosa Treatment
AU2019901804 2019-05-27
AU2019903812A AU2019903812A0 (en) 2019-10-10 Novel Retinitis Pigmentosa Treatment
AU2019903812 2019-10-10
PCT/AU2020/050516 WO2020237294A1 (en) 2019-05-27 2020-05-25 Novel retinitis pigmentosa treatment

Publications (2)

Publication Number Publication Date
CN113891940A CN113891940A (zh) 2022-01-04
CN113891940B true CN113891940B (zh) 2024-12-20

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US (2) US12559747B2 (enExample)
EP (1) EP3976789A4 (enExample)
JP (2) JP7652713B2 (enExample)
KR (1) KR20220012276A (enExample)
CN (1) CN113891940B (enExample)
AU (1) AU2020285470B2 (enExample)
BR (1) BR112021023936A2 (enExample)
CA (1) CA3141633A1 (enExample)
IL (1) IL288442A (enExample)
SG (1) SG11202112946VA (enExample)
WO (1) WO2020237294A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
WO2025043278A1 (en) * 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions

Citations (3)

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CA3147970A1 (en) * 2019-08-19 2021-02-25 Isabel AZNAREZ Compositions and methods for modulating splicing and protein expression
CN113584038A (zh) * 2021-09-09 2021-11-02 安吉康尔（深圳）科技有限公司 一种治疗视网膜疾病的反义寡核苷酸组合及其应用
WO2021247540A1 (en) * 2020-06-01 2021-12-09 Dana-Farber Cancer Institute, Inc. Methods for modulating mhc-i expression and immunotherapy uses thereof

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US20020123476A1 (en) 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
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CA3147970A1 (en) * 2019-08-19 2021-02-25 Isabel AZNAREZ Compositions and methods for modulating splicing and protein expression
WO2021247540A1 (en) * 2020-06-01 2021-12-09 Dana-Farber Cancer Institute, Inc. Methods for modulating mhc-i expression and immunotherapy uses thereof
CN113584038A (zh) * 2021-09-09 2021-11-02 安吉康尔（深圳）科技有限公司 一种治疗视网膜疾病的反义寡核苷酸组合及其应用

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Splicing-correcting therapeutic approaches for retinal dystrophies:where endogenous gene regulation and specificity matter;Niccolò Bacchi 等;Inverstigative Ophthalmology &Visual Science;第55卷(第5期);摘要，第3287页右栏第2段-第3289页左栏第1段 *
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US12559747B2 (en) 2026-02-24
JP7652713B2 (ja) 2025-03-27
AU2020285470A1 (en) 2021-12-16
SG11202112946VA (en) 2021-12-30
AU2020285470B2 (en) 2025-08-28
JP2025085741A (ja) 2025-06-05
JP2022534086A (ja) 2022-07-27
US20250066782A1 (en) 2025-02-27
WO2020237294A1 (en) 2020-12-03
CN113891940A (zh) 2022-01-04
CA3141633A1 (en) 2020-12-03
BR112021023936A2 (pt) 2022-01-25
US20220298506A1 (en) 2022-09-22
IL288442A (en) 2022-01-01
EP3976789A4 (en) 2023-08-09
EP3976789A1 (en) 2022-04-06
KR20220012276A (ko) 2022-02-03

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