TW202134438A - 著色性乾皮症ｆ群組治療藥 - Google Patents

著色性乾皮症ｆ群組治療藥 Download PDF

Info

Publication number
TW202134438A
TW202134438A TW109142335A TW109142335A TW202134438A TW 202134438 A TW202134438 A TW 202134438A TW 109142335 A TW109142335 A TW 109142335A TW 109142335 A TW109142335 A TW 109142335A TW 202134438 A TW202134438 A TW 202134438A
Authority
TW
Taiwan
Prior art keywords
sequence
antisense oligonucleotide
pharmaceutically acceptable
acceptable salt
base sequence
Prior art date
Application number
TW109142335A
Other languages
English (en)
Chinese (zh)
Inventor
荻朋男
中澤由華
小泉誠
小路貴生
Original Assignee
日商第一三共股份有限公司
國立大學法人東海國立大學機構
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 日商第一三共股份有限公司, 國立大學法人東海國立大學機構 filed Critical 日商第一三共股份有限公司
Publication of TW202134438A publication Critical patent/TW202134438A/zh

Links

Images

Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW109142335A 2019-12-02 2020-12-02 著色性乾皮症ｆ群組治療藥 TW202134438A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2019218038 2019-12-02
JP2019-218038 2019-12-02

Publications (1)

Publication Number Publication Date
TW202134438A true TW202134438A (zh) 2021-09-16

Family

ID=76221697

Family Applications (1)

Application Number Title Priority Date Filing Date
TW109142335A TW202134438A (zh) 2019-12-02 2020-12-02 著色性乾皮症ｆ群組治療藥

Country Status (3)

Country Link
JP (2) JPWO2021112106A1 (https=)
TW (1) TW202134438A (https=)
WO (1) WO2021112106A1 (https=)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69435005T2 (de) * 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation

Also Published As

Publication number Publication date
JP2025114590A (ja) 2025-08-05
WO2021112106A1 (ja) 2021-06-10
JPWO2021112106A1 (https=) 2021-06-10

Similar Documents

Publication Publication Date Title
JP6865871B2 (ja) 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含
JP6188686B2 (ja) フラタキシン（ｆｘｎ）への天然アンチセンス転写物の阻害によるｆｘｎ関連疾患の治療
RU2618688C2 (ru) Лечение связанных с геном-супрессором опухолей заболеваний посредством ингибирования природного транскрипта в антисмысловой ориентации относительно этого гена
JP5973996B2 (ja) Ａｔｏｈ１に対する天然アンチセンス転写物の阻害による無調ホモログ１（ａｔｏｈ１）関連疾患の治療
CA3169252A1 (en) Oligonucleotide compositions and methods thereof
TWI600759B (zh) 藉由抑制群落刺激因子３（ｃｓｆ３）之天然反義轉錄物以治療ｃｓｆ３相關疾病
KR101838308B1 (ko) 피롤린-5-카르복실레이트 환원효소 1(ｐｙｃｒ1)에 대한 천연 안티센스 전사체의 억제에 의한 ｐｙｃｒ1과 관련된 질환의 치료
KR20210121199A (ko) 올리고뉴클레오티드 조성물 및 이의 방법
JP2020189874A (ja) 状態および疾患の処置のためのアンチセンスオリゴマー
JP5978203B2 (ja) Ｆｇｆ２１に対する天然アンチセンス転写物の阻害による線維芽細胞増殖因子（ｆｇｆ２１）線維芽細胞増殖因子ｆｇｆ２１）関連疾患の治療
EP3390642B1 (en) Compositions for treatment of retinitis pigmentosa 13
CN121271865A (zh) 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸
TWI575068B (zh) 藉由抑制ｍｓｒａ天然反股轉錄本治療甲硫胺亞磺酸還原酶ａ（ｍｓｒａ）相關疾病
TWI531370B (zh) 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
JP2018082703A (ja) ナトリウムチャネル、電位依存性、αサブユニット（ＳＣＮＡ）に対する天然アンチセンス転写物の阻害によるＳＣＮＡ関連疾患の治療
CN107532162A (zh) 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
JP7060525B2 (ja) Ｈｔｒａ１の発現を調節するためのアンチセンスオリゴヌクレオチド
KR20190040098A (ko) 안티센스 핵산
TW201201819A (en) Treatment of BCL2-like 11 (BCL2L11) related diseases by inhibition of natural antisense transcript to BCL2L11
US10731160B2 (en) Allele-specific therapy for Huntington disease haplotypes
TW202134438A (zh) 著色性乾皮症ｆ群組治療藥
CN121057820A (zh) 使用环状前药核酸递送rna治疗药物
JPWO2019009299A1 (ja) α−シヌクレイン発現抑制するＥＮＡアンチセンスオリゴヌクレオチド
EP4069255B1 (en) Identifying non-productive splice sites
JP2025520199A (ja) 発現を調節するための薬剤