KR20220137699A - 망막하 고반사 병소 또는 망막하 고반사 병소를 수반하는 망막 질환의 치료제 - Google Patents

망막하 고반사 병소 또는 망막하 고반사 병소를 수반하는 망막 질환의 치료제 Download PDF

Info

Publication number
KR20220137699A
KR20220137699A KR1020227030369A KR20227030369A KR20220137699A KR 20220137699 A KR20220137699 A KR 20220137699A KR 1020227030369 A KR1020227030369 A KR 1020227030369A KR 20227030369 A KR20227030369 A KR 20227030369A KR 20220137699 A KR20220137699 A KR 20220137699A
Authority
KR
South Korea
Prior art keywords
aptamer
subretinal
hyperreflex
nucleotide
therapeutic agent
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
KR1020227030369A
Other languages
English (en)
Korean (ko)
Inventor
요시카즈 나카무라
마사토시 후지와라
유서프 알리
다니엘 페레이라
Original Assignee
가부시키가이샤 리보믹
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 가부시키가이샤 리보믹 filed Critical 가부시키가이샤 리보믹
Publication of KR20220137699A publication Critical patent/KR20220137699A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020227030369A 2020-02-06 2021-02-05 망막하 고반사 병소 또는 망막하 고반사 병소를 수반하는 망막 질환의 치료제 Pending KR20220137699A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062970842P 2020-02-06 2020-02-06
US62/970,842 2020-02-06
PCT/JP2021/004215 WO2021157681A1 (ja) 2020-02-06 2021-02-05 網膜下高反射病巣または網膜下高反射病巣を伴う網膜疾患の治療剤

Publications (1)

Publication Number Publication Date
KR20220137699A true KR20220137699A (ko) 2022-10-12

Family

ID=77199348

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227030369A Pending KR20220137699A (ko) 2020-02-06 2021-02-05 망막하 고반사 병소 또는 망막하 고반사 병소를 수반하는 망막 질환의 치료제

Country Status (7)

Country Link
US (1) US20230065106A1 (https=)
EP (1) EP4122471A4 (https=)
JP (1) JPWO2021157681A1 (https=)
KR (1) KR20220137699A (https=)
CN (1) CN115175684A (https=)
TW (1) TWI868308B (https=)
WO (1) WO2021157681A1 (https=)

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5660985A (en) 1990-06-11 1997-08-26 Nexstar Pharmaceuticals, Inc. High affinity nucleic acid ligands containing modified nucleotides
US20050043220A1 (en) * 2002-11-08 2005-02-24 Guyer David R. Methods and compositions for treating macular degeneration
NZ591443A (en) * 2005-09-22 2013-04-26 Intermune Inc Granule formation of pirfenidone and pharmaceutically acceptable excipients
EP2535410A4 (en) * 2010-02-12 2014-08-13 Ribomic Inc APTAMER FOR FGF2 AND USE THEREOF
WO2013186857A1 (ja) * 2012-06-12 2013-12-19 株式会社リボミック Fgf2に対するアプタマー及びその使用
CA2943772C (en) * 2014-03-24 2022-07-19 Ribomic Inc. Aptamer for fgf2 and use thereof
US11123411B2 (en) * 2016-12-08 2021-09-21 Gary E. Borodic Method of treating macular degeneration using botulinum toxin-based pharmaceuticals
ES2992308T3 (es) 2018-06-29 2024-12-11 Ribomic Inc Preparación de aptámero

Also Published As

Publication number Publication date
TWI868308B (zh) 2025-01-01
EP4122471A4 (en) 2025-02-19
EP4122471A1 (en) 2023-01-25
TW202142240A (zh) 2021-11-16
US20230065106A1 (en) 2023-03-02
WO2021157681A1 (ja) 2021-08-12
JPWO2021157681A1 (https=) 2021-08-12
CN115175684A (zh) 2022-10-11

Similar Documents

Publication Publication Date Title
JP7691462B2 (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2024113187A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
ES2819213T3 (es) Composiciones y métodos para modular la expresión de apolipoproteína (a)
JP2022528725A (ja) 筋ジストロフィーを治療するための組成物
US20190211330A1 (en) Compositions and methods for treatment of spinal muscular atrophy
KR20200057029A (ko) 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
TW201828995A (zh) 用於肌肉萎縮症之外顯子跳躍寡聚物結合物
JP2021526796A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマー
JP2021526017A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
CN115397436A (zh) 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
JP2021521794A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート
KR20220019255A (ko) 알파-1 항트립신 결핍증 (aatd)의 치료 방법
JP2025038098A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマー
EP4267191A1 (en) Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
JP2022511055A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
WO2026026776A1 (zh) 抑制APP基因表达的siRNA及其缀合物和应用
KR20220137699A (ko) 망막하 고반사 병소 또는 망막하 고반사 병소를 수반하는 망막 질환의 치료제
JP2021526807A (ja) 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート
JP7340264B2 (ja) アプタマー製剤
TW202308663A (zh) 人類染色體9開讀框72(C9ORF72)iRNA劑組成物及其使用方法
EP3978608A1 (en) Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
RU2847618C2 (ru) Способы лечения дефицита альфа-1-антитрипсина (aatd)
WO2025111497A1 (en) Compounds and methods for modulating acute activation
TW202430635A (zh) 用於抑制補體成分C3(C3)表現之RNAi藥劑、其醫藥組合物及使用方法
WO2025151250A2 (en) Complement factor b-modulating compositions and methods of use thereof

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902