CA3072575A1 - Rna aptamers against transferrin receptor (tfr) - Google Patents

Rna aptamers against transferrin receptor (tfr) Download PDF

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Publication number
CA3072575A1
CA3072575A1 CA3072575A CA3072575A CA3072575A1 CA 3072575 A1 CA3072575 A1 CA 3072575A1 CA 3072575 A CA3072575 A CA 3072575A CA 3072575 A CA3072575 A CA 3072575A CA 3072575 A1 CA3072575 A1 CA 3072575A1
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CA
Canada
Prior art keywords
cell
acid compound
nucleotides
moiety
ribonucleic acid
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
CA3072575A
Other languages
English (en)
French (fr)
Inventor
John J. Rossi
Sorah YOON
Nagy Habib
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.)
Apterna Ltd
City of Hope
Original Assignee
Apterna Ltd
City of Hope
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 Apterna Ltd, City of Hope filed Critical Apterna Ltd
Publication of CA3072575A1 publication Critical patent/CA3072575A1/en
Pending legal-status Critical Current

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    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/001Preparation for luminescence or biological staining
    • A61K49/0013Luminescence
    • A61K49/0017Fluorescence in vivo
    • A61K49/005Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
    • A61K49/0052Small organic molecules
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • C12N2310/10Type 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • 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
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
CA3072575A 2017-08-11 2018-08-10 Rna aptamers against transferrin receptor (tfr) Pending CA3072575A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762544220P 2017-08-11 2017-08-11
US62/544,220 2017-08-11
US201862671604P 2018-05-15 2018-05-15
US62/671,604 2018-05-15
PCT/US2018/046343 WO2019033051A1 (en) 2017-08-11 2018-08-10 RNA APTAMERS AGAINST THE TRANSFERRIN RECEPTOR (TFR)

Publications (1)

Publication Number Publication Date
CA3072575A1 true CA3072575A1 (en) 2019-02-14

Family

ID=65271776

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3072575A Pending CA3072575A1 (en) 2017-08-11 2018-08-10 Rna aptamers against transferrin receptor (tfr)

Country Status (8)

Country Link
US (1) US11427826B2 (enExample)
EP (1) EP3664814A4 (enExample)
JP (1) JP7382919B2 (enExample)
KR (1) KR20200068647A (enExample)
CN (2) CN111542326B (enExample)
AU (1) AU2018314236B2 (enExample)
CA (1) CA3072575A1 (enExample)
WO (1) WO2019033051A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020208361A1 (en) * 2019-04-12 2020-10-15 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220396794A1 (en) 2019-06-04 2022-12-15 Apterna Limited APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
GB202019692D0 (en) 2020-12-14 2021-01-27 Apterna Ltd Aptamer-sirna fusions
BR112023022975A2 (pt) 2021-05-05 2024-01-23 Immatics Biotechnologies Gmbh Polipeptídeos de ligação ao antígeno bma031 melhorados
UY40699A (es) 2023-04-05 2024-08-30 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de pln
WO2026011165A1 (en) * 2024-07-03 2026-01-08 Creyon Bio, Inc. Transferrin receptor (tfr) aptamers and use thereof
CN118879700B (zh) * 2024-07-18 2025-11-07 四川大学 具有sirt1基因靶向激活功能的框架核酸药物的制备及其用途

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US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US6344321B1 (en) * 1990-06-11 2002-02-05 Gilead Sciences, Inc. Nucleic acid ligands which bind to hepatocyte growth factor/scatter factor (HGF/SF) or its receptor c-met
US5830645A (en) 1994-12-09 1998-11-03 The Regents Of The University Of California Comparative fluorescence hybridization to nucleic acid arrays
US20020142000A1 (en) 1999-01-15 2002-10-03 Digan Mary Ellen Anti-CD3 immunotoxins and therapeutic uses therefor
AU2004242846A1 (en) 2003-05-31 2004-12-09 Micromet Ag Pharmaceutical compositions comprising bispecific anti-CD3, anti-CD19 antibody constructs for the treatment of B-cell related disorders
JP2006089471A (ja) 2004-08-26 2006-04-06 National Institute Of Advanced Industrial & Technology 癌の治療における抗モータリン2抗体と機能性核酸の使用
AR057807A1 (es) 2005-09-12 2007-12-19 Novimmune Sa Formulaciones de anticuerpo anti-cd3
US20100183615A1 (en) 2007-04-03 2010-07-22 Micromet Ag Cross-species-specific bispecific binders
SG10201808730VA (en) 2007-04-03 2018-11-29 Amgen Res Munich Gmbh Cross-species-specific binding domain
CN109456410B (zh) 2007-04-03 2022-01-28 安进研发(慕尼黑)股份有限公司 跨物种特异性CD3-ε结合结构域
JP5142265B2 (ja) 2007-05-28 2013-02-13 独立行政法人産業技術総合研究所 抗モータリン抗体のパラトープ及びエピトープ
EP2511371B1 (en) 2009-12-10 2015-02-25 National Institute of Advanced Industrial Science And Technology Anticancer anti-mortalin peptide antibodies
KR20130132795A (ko) * 2010-10-08 2013-12-05 미나 테라퓨틱스 리미티드 짧은 rna 분자
CA2870288A1 (en) 2012-04-10 2013-10-17 John J. Rossi Rna aptamers for therapeutic and diagnostic delivery to pancreatic cancer cells
US9963513B2 (en) 2013-02-05 2018-05-08 Engmab Sàrl Method for the selection of antibodies against BCMA
US9422563B2 (en) 2013-03-18 2016-08-23 University Of The Witwatersrand, Johannesburg CD7 receptor aptamers
EP2789630A1 (en) 2013-04-09 2014-10-15 EngMab AG Bispecific antibodies against CD3e and ROR1
CN105189544A (zh) 2013-04-19 2015-12-23 科瓦根股份公司 具有抗肿瘤活性的新颖的双特异性结合分子
US20160326249A1 (en) 2014-01-15 2016-11-10 Zymeworks Inc. Bi-specific cd3 and cd19 antigen-binding constructs
US9605266B2 (en) 2014-07-16 2017-03-28 City Of Hope Cell-specific internalizing RNA aptamers against human CCR5 and uses therefore
US10329570B2 (en) 2014-07-31 2019-06-25 Academia Sinica Antagonistic PD-1 aptamer and its applications in cancer therapy related applications
CN107106697B (zh) 2014-10-15 2021-06-18 希望之城 Pdgfr rna适体
CN107249644A (zh) 2014-10-15 2017-10-13 希望之城 针对转铁蛋白受体(TfR)的RNA适体
US11460472B2 (en) 2015-01-27 2022-10-04 Multimmune Gmbh Quantitative assay for heat shock proteins 70 (Hsp70) protein in body fluids
JP6978317B2 (ja) * 2015-02-11 2021-12-08 ディーキン・ユニバーシティー EpCAMアプタマー及びそのコンジュゲート
US10550394B2 (en) * 2015-03-31 2020-02-04 City Of Hope Anti-cancer RNA aptamers
US11219635B2 (en) 2016-02-19 2022-01-11 City Of Hope Bi-specific aptamer
US20200181264A1 (en) 2017-08-11 2020-06-11 City Of Hope Bispecific antigen-binding molecule

Also Published As

Publication number Publication date
AU2018314236A1 (en) 2020-03-19
AU2018314236B2 (en) 2025-02-06
JP2020532958A (ja) 2020-11-19
WO2019033051A1 (en) 2019-02-14
JP7382919B2 (ja) 2023-11-17
US11427826B2 (en) 2022-08-30
US20200172905A1 (en) 2020-06-04
EP3664814A1 (en) 2020-06-17
CN118931907A (zh) 2024-11-12
EP3664814A4 (en) 2021-05-05
CN111542326A (zh) 2020-08-14
KR20200068647A (ko) 2020-06-15
CN111542326B (zh) 2024-06-04

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