CA2951816A1 - Systemic in vivo delivery of oligonucleotides - Google Patents

Systemic in vivo delivery of oligonucleotides Download PDF

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Publication number
CA2951816A1
CA2951816A1 CA2951816A CA2951816A CA2951816A1 CA 2951816 A1 CA2951816 A1 CA 2951816A1 CA 2951816 A CA2951816 A CA 2951816A CA 2951816 A CA2951816 A CA 2951816A CA 2951816 A1 CA2951816 A1 CA 2951816A1
Authority
CA
Canada
Prior art keywords
oligonucleotide
hes
nucleic acid
disease
mirna
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.)
Abandoned
Application number
CA2951816A
Other languages
English (en)
French (fr)
Inventor
Beverly Packard
Akira Komoriya
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.)
Oncoimmunin Inc
Original Assignee
Oncoimmunin 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 Oncoimmunin Inc filed Critical Oncoimmunin Inc
Publication of CA2951816A1 publication Critical patent/CA2951816A1/en
Abandoned legal-status Critical Current

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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Communicable Diseases (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Dermatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Rheumatology (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Cardiology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
CA2951816A 2013-06-12 2014-06-12 Systemic in vivo delivery of oligonucleotides Abandoned CA2951816A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361834383P 2013-06-12 2013-06-12
US61/834,383 2013-06-12
PCT/US2014/042202 WO2014201306A1 (en) 2013-06-12 2014-06-12 Systemic in vivo delivery of oligonucleotides

Publications (1)

Publication Number Publication Date
CA2951816A1 true CA2951816A1 (en) 2014-12-18

Family

ID=52022784

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2951816A Abandoned CA2951816A1 (en) 2013-06-12 2014-06-12 Systemic in vivo delivery of oligonucleotides

Country Status (5)

Country Link
US (3) US20160367587A1 (https=)
EP (1) EP3007705A4 (https=)
JP (2) JP7011389B2 (https=)
CA (1) CA2951816A1 (https=)
WO (1) WO2014201306A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
EP3827838B1 (en) 2015-12-16 2023-06-07 The Walter and Eliza Hall Institute of Medical Research Inhibition of cytokine-induced sh2 protein in nk cells
PH12018501207B1 (en) 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
EP3806850A2 (en) * 2018-06-15 2021-04-21 Ideaya Biosciences, Inc. Methods of inhibiting proliferative cells
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US11820985B2 (en) 2019-03-26 2023-11-21 University Of Massachusetts Modified oligonucleotides with increased stability
EP3947680A2 (en) * 2019-04-03 2022-02-09 Bristol-Myers Squibb Company Angptl2 antisense oligonucleotides and uses thereof
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021132648A1 (ja) * 2019-12-27 2021-07-01 ルクサナバイオテク株式会社 Ｒｅｃｑｌの発現を阻害するアンチセンスオリゴヌクレオチド及びその用途
CA3167444A1 (en) * 2020-01-11 2021-07-15 Oncoimmunin, Inc. Targeted and localized in vivo delivery of oligonucleotides
CN115666636A (zh) * 2020-03-10 2023-01-31 中佛罗里达大学研究基金会有限公司 递送基因表达调节剂用于治疗癌症和病毒感染
JP7837560B2 (ja) 2020-03-26 2026-03-31 ユニバーシティー オブ マサチューセッツ 安定性の向上を有する修飾オリゴヌクレオチドの合成
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
US20230065782A1 (en) * 2021-07-14 2023-03-02 Oncoimmunin, Inc. Targeted and localized in vivo delivery of oligonucleotides
WO2025171411A1 (en) 2024-02-09 2025-08-14 Herophilus, Inc. Compositions and methods related to modulating macrophage migration inhibitory factor (mif)-cd74 signaling and related treatments for neuroinflammatory conditions
WO2025227100A2 (en) * 2024-04-26 2025-10-30 Purdue Research Foundation Targeted delivery of therapeutic agent to retinal pigment epithelium in treatment of ocular diseases

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6893868B2 (en) * 1997-02-20 2005-05-17 Onco Immunin, Inc. Homo-doubly labeled compositions for the detection of enzyme activity in biological samples
US6133032A (en) * 1999-09-09 2000-10-17 Isis Pharmaceutical Inc. Antisense modulation of PI3 kinase p110 beta expression
WO2002059137A1 (en) * 2000-11-03 2002-08-01 Isis Pharmaceuticals, Inc. Nuclease-based method for detecting and quantitating oligonucleotides
CA2459347C (en) * 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
AU2003225495B2 (en) * 2002-04-05 2009-01-15 Roche Innovation Center Copenhagen A/S Oligomeric compounds for the modulation HIF-1alpha expression
GB2406169B (en) * 2002-06-12 2006-11-01 Ambion Inc Methods and compositions relating to labeled rna molecules that reduce gene expression
US20050142581A1 (en) * 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
US20080199960A1 (en) * 2004-05-13 2008-08-21 Juliano Rudolph L Methods for the Delivery of Oligomeric Compounds
WO2008134593A1 (en) * 2007-04-25 2008-11-06 President And Fellows Of Harvard College Molecular circuits
EP2167660A1 (en) * 2007-06-22 2010-03-31 Keygene N.V. Targeted nucleotide exchange with improved modified oligonucleotides
US20100280098A1 (en) * 2007-10-05 2010-11-04 Juliano Rudolph L Receptor targeted oligonucleotides
US8911948B2 (en) * 2008-04-30 2014-12-16 Integrated Dna Technologies, Inc. RNase H-based assays utilizing modified RNA monomers
WO2010071852A2 (en) * 2008-12-19 2010-06-24 University Of Florida Research Foundation, Inc. Oligonucleotide micelles
WO2011072082A2 (en) * 2009-12-09 2011-06-16 Nitto Denko Corporation Modulation of hsp47 expression
JP2015502365A (ja) * 2011-12-12 2015-01-22 オンコイミューニン，インコーポレイティド オリゴヌクレオチドのイン−ビボ送達

Also Published As

Publication number Publication date
US20160367587A1 (en) 2016-12-22
EP3007705A4 (en) 2017-02-15
US20220033815A1 (en) 2022-02-03
JP7011389B2 (ja) 2022-01-26
EP3007705A1 (en) 2016-04-20
JP2019135259A (ja) 2019-08-15
US20190183918A1 (en) 2019-06-20
WO2014201306A1 (en) 2014-12-18
JP2016521753A (ja) 2016-07-25

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FZDE Discontinued

Effective date: 20200831