TW202320808A - 用於治療多囊性腎病之方法及組合物 - Google Patents

用於治療多囊性腎病之方法及組合物 Download PDF

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TW202320808A
TW202320808A TW111138235A TW111138235A TW202320808A TW 202320808 A TW202320808 A TW 202320808A TW 111138235 A TW111138235 A TW 111138235A TW 111138235 A TW111138235 A TW 111138235A TW 202320808 A TW202320808 A TW 202320808A
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individual
nucleobase
compound
modified oligonucleotide
certain embodiments
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TW111138235A
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English (en)
Chinese (zh)
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丹尼斯 德利金
加斯 Ａ 金伯傑
震宇 李
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美商雷格勒斯治療公司
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Publication of TW202320808A publication Critical patent/TW202320808A/zh

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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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G

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  • Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW111138235A 2021-10-08 2022-10-07 用於治療多囊性腎病之方法及組合物 TW202320808A (zh)

Applications Claiming Priority (2)

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US202163253933P 2021-10-08 2021-10-08
US63/253,933 2021-10-08

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TW202320808A true TW202320808A (zh) 2023-06-01

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TW111138235A TW202320808A (zh) 2021-10-08 2022-10-07 用於治療多囊性腎病之方法及組合物

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US (2) US20240294913A1 (https=)
EP (1) EP4413132A1 (https=)
JP (1) JP2024537043A (https=)
KR (1) KR20240073028A (https=)
CN (1) CN118103510A (https=)
AR (1) AR127325A1 (https=)
AU (1) AU2022361062A1 (https=)
CA (1) CA3234547A1 (https=)
CL (1) CL2024000990A1 (https=)
CO (1) CO2024004227A2 (https=)
IL (1) IL311734A (https=)
MX (1) MX2024003529A (https=)
TW (1) TW202320808A (https=)
WO (1) WO2023060237A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023060237A1 (en) 2021-10-08 2023-04-13 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
KR20260005213A (ko) * 2023-04-12 2026-01-09 레굴루스 테라퓨틱스 인크 다낭성 신장 질환 치료를 위한 변형 올리고뉴클레오타이드
EP4680746A1 (en) * 2023-06-16 2026-01-21 Pyc Therapeutics Limited Compositions and methods for treatment of kidney disease
WO2025193584A1 (en) * 2024-03-11 2025-09-18 Regulus Therapeutics Inc. Compound for use in methods for treatment of polycystic kidney disease

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KR20260005213A (ko) 2023-04-12 2026-01-09 레굴루스 테라퓨틱스 인크 다낭성 신장 질환 치료를 위한 변형 올리고뉴클레오타이드

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US20240294913A1 (en) 2024-09-05
JP2024537043A (ja) 2024-10-10
WO2023060237A1 (en) 2023-04-13
IL311734A (en) 2024-05-01
CA3234547A1 (en) 2023-04-13
KR20240073028A (ko) 2024-05-24
CL2024000990A1 (es) 2024-09-27
AU2022361062A1 (en) 2024-03-21
US20250188466A1 (en) 2025-06-12
CO2024004227A2 (es) 2024-04-18
AR127325A1 (es) 2024-01-10
CN118103510A (zh) 2024-05-28
US12559750B2 (en) 2026-02-24
MX2024003529A (es) 2024-04-01
EP4413132A1 (en) 2024-08-14

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