AU2019292134B2 - Aptamer preparation - Google Patents

Aptamer preparation Download PDF

Info

Publication number
AU2019292134B2
AU2019292134B2 AU2019292134A AU2019292134A AU2019292134B2 AU 2019292134 B2 AU2019292134 B2 AU 2019292134B2 AU 2019292134 A AU2019292134 A AU 2019292134A AU 2019292134 A AU2019292134 A AU 2019292134A AU 2019292134 B2 AU2019292134 B2 AU 2019292134B2
Authority
AU
Australia
Prior art keywords
aptamer
aqueous
group
atom
fgf2
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.)
Active
Application number
AU2019292134A
Other languages
English (en)
Other versions
AU2019292134A1 (en
Inventor
Kazumasa AKITA
Yusuf Ali
Yoshikazu Nakamura
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ribomic Inc
Original Assignee
Ribomic Inc
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 Ribomic Inc filed Critical Ribomic Inc
Publication of AU2019292134A1 publication Critical patent/AU2019292134A1/en
Application granted granted Critical
Publication of AU2019292134B2 publication Critical patent/AU2019292134B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • 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
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • Rheumatology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Ophthalmology & Optometry (AREA)
AU2019292134A 2018-06-29 2019-06-28 Aptamer preparation Active AU2019292134B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2018124390 2018-06-29
JP2018-124390 2018-06-29
PCT/JP2019/025766 WO2020004607A1 (ja) 2018-06-29 2019-06-28 アプタマー製剤

Publications (2)

Publication Number Publication Date
AU2019292134A1 AU2019292134A1 (en) 2021-02-11
AU2019292134B2 true AU2019292134B2 (en) 2025-07-10

Family

ID=68986754

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019292134A Active AU2019292134B2 (en) 2018-06-29 2019-06-28 Aptamer preparation

Country Status (14)

Country Link
US (1) US20210269802A1 (enExample)
EP (1) EP3815715B1 (enExample)
JP (1) JP7340264B2 (enExample)
KR (1) KR102841342B1 (enExample)
CN (1) CN112384246B (enExample)
AU (1) AU2019292134B2 (enExample)
BR (1) BR112020026634A2 (enExample)
CA (1) CA3105002A1 (enExample)
ES (1) ES2992308T3 (enExample)
IL (1) IL279595B1 (enExample)
MX (1) MX2020014124A (enExample)
SG (1) SG11202012933QA (enExample)
TW (1) TWI845521B (enExample)
WO (1) WO2020004607A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021157681A1 (ja) 2020-02-06 2021-08-12 株式会社リボミック 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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3124611A1 (en) * 2014-03-24 2017-02-01 Ribomic Inc. Aptamer for fgf2 and use thereof

Family Cites Families (11)

* 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
US5847086A (en) * 1991-06-20 1998-12-08 Centeon L.L.C. Therapeutic fragments of von Willebrand factor
MY147374A (en) * 2006-11-14 2012-11-30 Ribomic Inc Aptamer against midkine and use thereof
CN101317820B (zh) * 2007-04-19 2012-02-29 中国人民解放军军事医学科学院毒物药物研究所 生物亲和性配体分子介导的靶向脂质体,其制 备及其应用
US20090203766A1 (en) * 2007-06-01 2009-08-13 Archemix Corp. vWF aptamer formulations and methods for use
WO2009118108A1 (en) * 2008-03-26 2009-10-01 Merck Patent Gmbh Method of long term storage of substrate-coupled beads
US9052323B2 (en) * 2009-08-27 2015-06-09 The University Of Kansas Osmolyte mixture for protein stabilization
JP5899550B2 (ja) 2010-02-12 2016-04-06 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
WO2014116789A1 (en) * 2013-01-25 2014-07-31 Thymon, Llc Immunogenic and prophylactic compositions, methods of making same, and method for treating and preventing tnf-mediated disease and hiv-1 infection
WO2015200539A1 (en) * 2014-06-25 2015-12-30 Monsanto Technology Llc Methods and compositions for delivering nucleic acids to plant cells and regulating gene expression
JP2018124390A (ja) 2017-01-31 2018-08-09 株式会社沖データ 画像形成装置及び定着制御方法

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3124611A1 (en) * 2014-03-24 2017-02-01 Ribomic Inc. Aptamer for fgf2 and use thereof

Also Published As

Publication number Publication date
TWI845521B (zh) 2024-06-21
EP3815715B1 (en) 2024-09-11
ES2992308T3 (es) 2024-12-11
CA3105002A1 (en) 2020-01-02
AU2019292134A1 (en) 2021-02-11
KR20210025083A (ko) 2021-03-08
EP3815715A4 (en) 2022-03-16
MX2020014124A (es) 2021-05-31
BR112020026634A2 (pt) 2021-04-06
IL279595A (en) 2021-03-01
IL279595B1 (en) 2025-11-01
EP3815715A1 (en) 2021-05-05
SG11202012933QA (en) 2021-01-28
JPWO2020004607A1 (ja) 2021-07-15
TW202012625A (zh) 2020-04-01
KR102841342B1 (ko) 2025-07-31
CN112384246B (zh) 2025-02-21
JP7340264B2 (ja) 2023-09-07
EP3815715C0 (en) 2024-09-11
US20210269802A1 (en) 2021-09-02
CN112384246A (zh) 2021-02-19
WO2020004607A1 (ja) 2020-01-02

Similar Documents

Publication Publication Date Title
TWI823866B (zh) 用於抑制類血管生成素蛋白-3(ANGPTL3)表現的RNAi藥劑及組合物及使用方法
JP7488254B2 (ja) 17β-HSD13型(HSD17B13)の発現を阻害するためのRNAi剤、その組成物、および使用方法
US20230078200A1 (en) RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
US20220049252A1 (en) CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF
JP2023537943A (ja) MARC1発現を阻害するためのRNAiコンストラクト及び方法
CN114222820A (zh) 用于治疗α-1抗胰蛋白酶缺乏症(AATD)的方法
JP2023519246A (ja) PNPLA3の発現を阻害するためのRNAi薬、その医薬組成物、及び使用方法
AU2019292134B2 (en) Aptamer preparation
US20250283089A1 (en) Treatment of a non-alcoholic fatty liver disease
US20140309287A1 (en) Oligonucleotide formulation
EP4267191A1 (en) Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
HK40046627A (en) Aptamer preparation
KR20250054795A (ko) Angptl3-관련 질환 및 장애의 치료 방법
KR20230067648A (ko) Z-aat 단백질 수준을 감소시키기 위한 방법
RU2847618C2 (ru) Способы лечения дефицита альфа-1-антитрипсина (aatd)
RU2851259C1 (ru) Способы снижения уровней белка z-aat
EP4122471A1 (en) Therapeutic agent for subretinal hyperreflective material or retinal disorders accompanying subretinal hyperreflective material
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
CN116670278A (zh) 用于降低z-aat蛋白水平的方法

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)