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

Rna aptamers against transferrin receptor (tfr) Download PDF

Info

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
Authority
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

Links

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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/005—Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
    • A61K49/0052—Small organic molecules
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/115—Aptamers, 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special 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
EP3953473A1 (en) * 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020245198A1 (en) 2019-06-04 2020-12-10 Apterna Limited APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
GB202019692D0 (en) 2020-12-14 2021-01-27 Apterna Ltd Aptamer-sirna fusions
KR20240004937A (ko) 2021-05-05 2024-01-11 이매틱스 바이오테크놀로지스 게엠베하 Bma031 항원 결합 폴리펩타이드
KR20260021596A (ko) 2023-04-05 2026-02-13 아이오니스 파마수티컬즈, 인코포레이티드 Pln 발현을 감소시키는 화합물 및 방법
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

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2006291005A1 (en) 2005-09-12 2007-03-22 Novimmune S.A. Anti-CD3 antibody formulations
WO2008119565A2 (en) 2007-04-03 2008-10-09 Micromet Ag Cross-species-specific binding domain
WO2008119567A2 (en) 2007-04-03 2008-10-09 Micromet Ag Cross-species-specific cd3-epsilon binding domain
PL2155788T3 (pl) 2007-04-03 2013-02-28 Amgen Res Munich Gmbh Swoiste międzygatunkowe bispecyficzne czynniki wiążące
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
WO2011071099A1 (ja) 2009-12-10 2011-06-16 独立行政法人産業技術総合研究所 抗癌性抗モータリンペプチド抗体
CA2817191A1 (en) * 2010-10-08 2012-04-12 Pal Saetrom Short rna molecules
AU2013246419A1 (en) 2012-04-10 2014-10-30 City Of Hope RNA aptamers for therapeutic and diagnostic delivery to pancreatic cancer cells
JP6636803B2 (ja) 2013-02-05 2020-01-29 エンクマフ エスアーエールエル Bcmaに対する抗体の選択のための方法
EP2976427A4 (en) 2013-03-18 2017-02-22 University Of The Witwatersrand, Johannesburg Cd7 receptor aptamers
EP2789630A1 (en) 2013-04-09 2014-10-15 EngMab AG Bispecific antibodies against CD3e and ROR1
HK1214605A1 (zh) 2013-04-19 2016-07-29 Covagen Ag 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
WO2016019270A1 (en) 2014-07-31 2016-02-04 Academia Sinica An 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
EP3206719B1 (en) * 2014-10-15 2020-04-08 City of Hope Rna aptamers against transferrin receptor (tfr)
WO2016120325A1 (en) 2015-01-27 2016-08-04 Klinikum Rechts Der Isar Der Technischen Universität München Quantitative assay for heat shock protein 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
WO2016161165A1 (en) 2015-03-31 2016-10-06 City Of Hope Anti-cancer rna aptamers
US11219635B2 (en) 2016-02-19 2022-01-11 City Of Hope Bi-specific aptamer
WO2019033050A1 (en) 2017-08-11 2019-02-14 City Of Hope BISPECIFIC MOLECULE FOR BINDING TO ANTIGEN

Also Published As

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

Similar Documents

Publication Publication Date Title
US11427826B2 (en) RNA aptamers against transferrin receptor (TfR)
US20220072029A1 (en) Bi specific aptamer
EP3206719B1 (en) Rna aptamers against transferrin receptor (tfr)
US20180243436A1 (en) Therapeutic cell internalizing conjugates
US11845938B2 (en) Aptamer compositions and the use thereof
US11261449B2 (en) Anti-cancer RNA aptamers
US20220396794A1 (en) APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
US20240352466A1 (en) APTAMER-siRNA FUSIONS
US11180761B2 (en) PDGFR RNA aptamers
WO2019014648A1 (en) PEPTIDES CONJUGATED TO A PHOSPHOROTHIOATE AND METHODS OF USE
WO2017173237A1 (en) Mortalin binding aptamers
EP3969591A1 (en) Aptamers against glioblastoma