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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (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)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Dermatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Virology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 (enExample)
EP (1) EP3007705A4 (enExample)
JP (2) JP7011389B2 (enExample)
CA (1) CA2951816A1 (enExample)
WO (1) WO2014201306A1 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
AU2016374497B2 (en) 2015-12-16 2022-03-10 The Walter And Eliza Hall Institute Of Medical Research Inhibition of cytokine-induced SH2 protein in NK cells
KR20250112919A (ko) 2015-12-21 2025-07-24 노파르티스 아게 타우 발현을 감소시키기 위한 조성물 및 방법
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
EP3806850A2 (en) * 2018-06-15 2021-04-21 Ideaya Biosciences, Inc. Methods of inhibiting proliferative cells
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 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
EP3946369A4 (en) * 2019-03-26 2023-10-18 University Of Massachusetts MODIFIED OLIGONUCLEOTIDES WITH INCREASED STABILITY
JP7725370B2 (ja) * 2019-04-03 2025-08-19 ブリストル－マイヤーズ スクイブ カンパニー Ａｎｇｐｔｌ２アンチセンスオリゴヌクレオチドおよびその使用
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
EP4087587A4 (en) * 2020-01-11 2024-08-07 Oncoimmunin, Inc. TARGETED AND LOCALIZED IN VIVO DELIVERY OF OLIGONUCLEOTIDES
JP2023526719A (ja) * 2020-03-10 2023-06-23 ユニバーシティ オブ セントラル フロリダ リサーチ ファウンデーション，インコーポレイテッド がん及びウイルス感染症治療のための遺伝子発現調節剤の送達
AU2021244600A1 (en) 2020-03-26 2022-11-03 University Of Massachusetts Synthesis of modified oligonucleotides with increased stability
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova 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
US7060809B2 (en) * 2001-09-04 2006-06-13 Exiqon A/S LNA compositions and uses thereof
ES2649817T3 (es) * 2002-04-05 2018-01-15 Roche Innovation Center Copenhagen A/S Compuestos oligom¿¿ricos para la modulaci¿®n de la expresi¿®n de HIF-1¿Á
WO2003106630A2 (en) * 2002-06-12 2003-12-24 Ambion, Inc. Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference
US20050142581A1 (en) * 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005113571A2 (en) * 2004-05-13 2005-12-01 The University Of North Carolina At Chapel Hill Methods for the delivery of oligomeric compounds
WO2008134593A1 (en) * 2007-04-25 2008-11-06 President And Fellows Of Harvard College Molecular circuits
CA2690510A1 (en) * 2007-06-22 2008-12-31 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
CA2781896C (en) * 2009-12-09 2021-03-30 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
EP3007705A4 (en) 2017-02-15
JP2019135259A (ja) 2019-08-15
JP2016521753A (ja) 2016-07-25
US20190183918A1 (en) 2019-06-20
US20160367587A1 (en) 2016-12-22
EP3007705A1 (en) 2016-04-20
JP7011389B2 (ja) 2022-01-26
WO2014201306A1 (en) 2014-12-18
US20220033815A1 (en) 2022-02-03

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Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20200831