JP7034951B2 - 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する - Google Patents

操作されたウイルスベクターは炎症および免疫応答の誘導を低減する Download PDF

Info

Publication number
JP7034951B2
JP7034951B2 JP2018563666A JP2018563666A JP7034951B2 JP 7034951 B2 JP7034951 B2 JP 7034951B2 JP 2018563666 A JP2018563666 A JP 2018563666A JP 2018563666 A JP2018563666 A JP 2018563666A JP 7034951 B2 JP7034951 B2 JP 7034951B2
Authority
JP
Japan
Prior art keywords
seq
aav
nucleic acid
egfp
genome
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
JP2018563666A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019517266A (ja
JP2019517266A5 (https=
Inventor
カイ チャン,イン
エム. チャーチ,ジョージ
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.)
Harvard University
Original Assignee
Harvard University
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 Harvard University filed Critical Harvard University
Publication of JP2019517266A publication Critical patent/JP2019517266A/ja
Publication of JP2019517266A5 publication Critical patent/JP2019517266A5/ja
Priority to JP2022031422A priority Critical patent/JP7394898B2/ja
Application granted granted Critical
Publication of JP7034951B2 publication Critical patent/JP7034951B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • 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
    • 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
    • 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]
    • 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
    • C12N15/117Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • 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
    • C12N2310/17Immunomodulatory nucleic acids
    • 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
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/00032Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/00041Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2018563666A 2016-06-08 2017-06-08 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する Active JP7034951B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022031422A JP7394898B2 (ja) 2016-06-08 2022-03-02 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662347302P 2016-06-08 2016-06-08
US62/347,302 2016-06-08
PCT/US2017/036525 WO2017214378A1 (en) 2016-06-08 2017-06-08 Engineered viral vector reduces induction of inflammatory and immune responses

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022031422A Division JP7394898B2 (ja) 2016-06-08 2022-03-02 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する

Publications (3)

Publication Number Publication Date
JP2019517266A JP2019517266A (ja) 2019-06-24
JP2019517266A5 JP2019517266A5 (https=) 2020-07-16
JP7034951B2 true JP7034951B2 (ja) 2022-03-14

Family

ID=60578967

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018563666A Active JP7034951B2 (ja) 2016-06-08 2017-06-08 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する
JP2022031422A Active JP7394898B2 (ja) 2016-06-08 2022-03-02 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022031422A Active JP7394898B2 (ja) 2016-06-08 2022-03-02 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する

Country Status (7)

Country Link
US (3) US11339396B2 (https=)
EP (1) EP3468605A4 (https=)
JP (2) JP7034951B2 (https=)
KR (1) KR20190017872A (https=)
CN (2) CN114807152A (https=)
AU (2) AU2017277647B2 (https=)
WO (1) WO2017214378A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7034951B2 (ja) 2016-06-08 2022-03-14 プレジデント アンド フェローズ オブ ハーバード カレッジ 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する
JP2021502381A (ja) 2017-11-08 2021-01-28 プレジデント アンド フェローズ オブ ハーバード カレッジ ウイルスベクター誘発性炎症反応を阻害するための組成物および方法
US10736957B2 (en) 2017-12-19 2020-08-11 President And Fellows Of Harvard College Enhanced immunogenicity of mRNA with co-encoded adjuvant sequences
EP3833745A1 (en) 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production
JP2022518504A (ja) * 2019-01-24 2022-03-15 ジェネレーション バイオ カンパニー 閉端dna(cedna)ならびに遺伝子療法または核酸療法に関連する免疫応答を低減させる方法における使用
CA3130108A1 (en) 2019-02-22 2020-08-27 Life Technologies Corporation Suspension system for adeno associated virus production
US20220259599A1 (en) * 2019-05-15 2022-08-18 President And Fellows Of Harvard College Compositions and methods for inhibiting nucleic acid vector-induced inflammatory responses
CA3174553A1 (en) * 2020-03-05 2021-09-10 Flagship Pioneering Innovations Vi, Llc Host defense suppressing methods and compositions for modulating a genome
CN115698332A (zh) * 2020-06-12 2023-02-03 新加坡科技研究局 在单细胞水平上评估载体转导效率和/或特异性的方法
WO2022187679A1 (en) * 2021-03-04 2022-09-09 Kriya Therapeutics, Inc. Viral vector constructs incorporating dna for inhibiting toll like receptors and methods of using the same
WO2025087987A1 (en) * 2023-10-23 2025-05-01 Sartorius Stedim Cellca Gmbh Plasmids for improved aav titers and empty full ratios

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002505580A (ja) 1997-06-06 2002-02-19 ザ リージェンツ オブ ザ ユニバーシティー オブ カリフォルニア Dna免疫刺激配列活性の阻害剤
JP2013544816A (ja) 2010-11-19 2013-12-19 イデラ ファーマシューティカルズ インコーポレイテッド トール様受容体に基づく免疫反応を調節するための免疫調節オリゴヌクレオチド(iro)化合物
WO2014105870A1 (en) 2012-12-27 2014-07-03 Sierra Sciences, Inc. Enhancing health in mammals using telomerase reverse transcriptase gene therapy
JP2015532095A (ja) 2012-09-28 2015-11-09 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター
JP2015536659A (ja) 2012-11-29 2015-12-24 Sbiバイオテック株式会社 阻害性オリゴヌクレオチド及びその使用

Family Cites Families (79)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL104314A0 (en) 1992-01-07 1993-05-13 Novo Nordisk As Human kunitz-type protease inhibitor and variants thereof,their production and pharmaceutical compositions containing them
US5489508A (en) 1992-05-13 1996-02-06 University Of Texas System Board Of Regents Therapy and diagnosis of conditions related to telomere length and/or telomerase activity
US5643890A (en) 1995-01-31 1997-07-01 The Board Of Regents Of The University Of Nebraska Synthetic oligonucleotides which mimic telomeric sequences for use in treatment of cancer and other diseases
US6551795B1 (en) 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
JP2002541264A (ja) 1999-04-08 2002-12-03 ユーエイビー・リサーチ・ファウンデーション 冨gオリゴヌクレオチドの抗増殖活性およびヌクレオリンに結合するためのその使用方法
NZ517929A (en) 1999-09-25 2004-02-27 Univ Iowa Res Found Immunostimulatory nucleic acids
MXPA00011713A (es) * 2000-11-28 2002-05-31 Tgt Lab S A De C V Vectores recombinantes virales y no virales conteniendo el gen humano del activador de plasminogeno derivado de urocinasa y su utilidad en el tratamiento de diversos tipos de fibrosis hepatica, renal, pulmonar, pancreatica, cardiaca y cicatrices hipe
US7060690B2 (en) 2001-01-22 2006-06-13 Genta Incorporated Methods and compositions for treating a cell-proliferative disorder using CRE decoy oligomers, BCL-2 antisense oligomers, and hybrid oligomers thereof
WO2003027313A2 (en) 2001-09-24 2003-04-03 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services SUPPRESSORS OF CpG OLIGONUCLEOTIDES AND METHODS OF USE
EP3006043B1 (en) 2002-04-04 2019-05-29 Zoetis Belgium S.A. Immunostimulatory g,u-containing oligoribonucleotides
US7357928B2 (en) 2002-04-08 2008-04-15 University Of Louisville Research Foundation, Inc. Method for the diagnosis and prognosis of malignant diseases
US20040009949A1 (en) 2002-06-05 2004-01-15 Coley Pharmaceutical Group, Inc. Method for treating autoimmune or inflammatory diseases with combinations of inhibitory oligonucleotides and small molecule antagonists of immunostimulatory CpG nucleic acids
EP1575504A4 (en) 2002-08-01 2009-11-04 Us Gov Health & Human Serv METHOD FOR THE TREATMENT OF INFLAMMATORY ARTHROPATHIES WITH SUPPRESSORS OF THE CPG OLIGONUCLEOTIDES
US8043622B2 (en) 2002-10-08 2011-10-25 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Method of treating inflammatory lung disease with suppressors of CpG oligonucleotides
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
US20050013812A1 (en) 2003-07-14 2005-01-20 Dow Steven W. Vaccines using pattern recognition receptor-ligand:lipid complexes
US20050239733A1 (en) 2003-10-31 2005-10-27 Coley Pharmaceutical Gmbh Sequence requirements for inhibitory oligonucleotides
WO2005072097A2 (en) 2003-12-16 2005-08-11 University Of Massachusetts Toll-like receptor 9 modulators
CN1989439B (zh) 2004-05-06 2010-12-29 美国政府健康及人类服务部 治疗葡萄膜炎的方法以及组合物
JP5697297B2 (ja) 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロnasおよびその使用
WO2005116250A2 (en) 2004-05-26 2005-12-08 Rosetta Genomics Ltd. Viral and viral associated mirnas and uses thereof
JP2008514187A (ja) 2004-09-01 2008-05-08 ダイナバックス テクノロジーズ コーポレイション 先天性免疫応答及び自己免疫疾患の阻害法及び阻害用組成物
JP2008000001A (ja) 2004-09-30 2008-01-10 Osaka Univ 免疫刺激オリゴヌクレオチドおよびその医薬用途
CA2591582A1 (en) 2004-12-17 2006-06-22 Dynavax Technologies Corporation Methods and compositions for induction or promotion of immune tolerance
US20070077231A1 (en) * 2005-09-30 2007-04-05 Contag Christopher H Immune effector cells pre-infected with oncolytic virus
EP2982679A1 (en) 2005-10-12 2016-02-10 Idera Pharmaceuticals, Inc. Immune regulatory oligonucleotide (iro) compounds to modulate toll-like receptor based immune response
EP2441846A3 (en) 2006-01-09 2012-07-25 The Regents Of the University of California Immunostimulatory combinations of TNFRSF, TLR, NLR, RHR, purinergic receptor, and cytokine receptor agoinsts for vaccines and tumor immunotherapy
WO2007127428A2 (en) * 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
AU2007260779A1 (en) 2006-06-13 2007-12-21 Bayhill Therapeutics, Inc. Polynucleotide therapy
US8377898B2 (en) 2006-10-12 2013-02-19 Idera Pharmaceuticals, Inc. Immune regulatory oligonucleotide (IRO) compounds to modulate toll-like receptor based immune response
WO2008076981A2 (en) 2006-12-15 2008-06-26 Board Of Regents, The University Of Texas System Inhibitors of ll-37- mediated immune reactivity to self nucleic acids
JP2010519899A (ja) 2007-03-01 2010-06-10 ロゼッタ ゲノミックス エルティーディー. 肺扁平上皮癌とその他の非小細胞肺癌とを見分けるための方法
WO2009006141A2 (en) 2007-07-05 2009-01-08 Bausch & Lomb Incorporated Authority to read as follows: compositions and methods for treating or controlling infections of the eye a sequelae thereof
US7879812B2 (en) 2007-08-06 2011-02-01 University Of Iowa Research Foundation Immunomodulatory oligonucleotides and methods of use therefor
JP2010536787A (ja) 2007-08-15 2010-12-02 イデラ ファーマシューティカルズ インコーポレイテッド Toll様受容体モジュレータ
EP2209896B1 (en) 2007-10-26 2017-03-01 Dynavax Technologies Corporation Methods and compositions for inhibition of immune responses and autoimmunity
WO2009089401A2 (en) 2008-01-10 2009-07-16 Bausch & Lomb Incorporated Compositions comprising toll-like receptor or coreceptor antagonists and methods for treating or controlling ocular allergy using same
WO2009089399A2 (en) 2008-01-10 2009-07-16 Bausch & Lomb Incorporated Compositions comprising toll-like receptor or coreceptor antagonists and methods for ocular neuroprotection
WO2009143292A2 (en) 2008-05-21 2009-11-26 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Method of treating pneumoconiosis with oligodeoxynucleotides
JP5415724B2 (ja) * 2008-08-05 2014-02-12 長春華普生物技術有限公司 一種のオリゴヌクレオチド及びその応用
US8030289B2 (en) 2008-08-06 2011-10-04 Changchun Huapu Biotechnology Co., Ltd. Oligonucleotide and use thereof
EP2334300B1 (en) 2008-09-24 2016-02-17 The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin Composition and method for treatment of preterm labor
GB0906130D0 (en) 2008-10-03 2009-05-20 Procrata Biosystems Ltd Transcription factor decoys
WO2010042543A2 (en) 2008-10-06 2010-04-15 Idera Pharmaceuticals, Inc. Use of inhibitors of toll-like receptors in the prevention and treatment of hypercholesterolemia and hyperlipidemia and diseases related thereto
US8053422B2 (en) 2008-12-04 2011-11-08 The United States Of America As Represented By The Department Of Health And Human Services Anti-cancer oligodeoxynucleotides
WO2010108035A1 (en) * 2009-03-18 2010-09-23 Isis Pharmaceuticals, Inc. Compounds and methods for modulating toxic and proinflammatory effects
WO2010127166A2 (en) 2009-04-30 2010-11-04 The Regents Of The University Of California Combination anti-hiv vectors, targeting vectors, and methods of use
ES2624767T3 (es) 2009-06-01 2017-07-17 Idera Pharmaceuticals, Inc Potenciación de tratamientos de enfermedades autoinmunitarias e inflamatorias mediante oligonucleótidos inmunorreguladores (IRO) antagonistas de TLR7 y TLR9
US20110070241A1 (en) 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
EP2451974A2 (en) 2009-07-08 2012-05-16 Idera Pharmaceuticals, Inc. Oligonucleotide-based compounds as inhibitors of toll-like receptors
CN101712957B (zh) 2009-11-25 2011-11-23 中国人民解放军第三军医大学第一附属医院 Toll样受体9阻断剂及应用
US8771669B1 (en) 2010-02-09 2014-07-08 David Gordon Bermudes Immunization and/or treatment of parasites and infectious agents by live bacteria
EP3444346B1 (en) 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
JP5921535B2 (ja) 2010-06-16 2016-05-24 ディナバックス テクノロジーズ コーポレイション Tlr7および/またはtlr9阻害剤を用いる治療の方法
EP2582398B1 (en) 2010-06-16 2016-04-13 Dynavax Technologies Corporation Methods of treatment using tlr7 and/or tlr9 inhibitors
GB201014026D0 (en) 2010-08-20 2010-10-06 Ucl Business Plc Treatment
WO2012051491A1 (en) * 2010-10-14 2012-04-19 The United States Of America, As Represented By The Secretary National Institutes Of Health Compositions and methods for controlling neurotropic viral pathogenesis by micro-rna targeting
US20140378531A1 (en) 2012-02-01 2014-12-25 New York University Inhibition of pattern recognition receptors in pancreatic cancer treatment using tlr inhibitors
AR091569A1 (es) 2012-06-28 2015-02-11 Intervet Int Bv Receptores de tipo toll
CN102925485B (zh) 2012-11-09 2014-07-09 中国农业科学院兰州兽医研究所 双表达的aav重组病毒的制备方法
JP6385957B2 (ja) 2013-01-08 2018-09-05 イデラ ファーマシューティカルズ インコーポレイテッドIdera Pharmaceuticals, Inc. トール様受容体に基づく免疫応答を調節するための免疫調節オリゴヌクレオチド(iro)化合物
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US20170035864A1 (en) 2013-12-09 2017-02-09 Bullet Biotechnology, Inc. SPECIFIC VIRUS-LIKE PARTICLE-CpG OLIGONUCLEOTIDE VACCINES AND USES THEREOF
WO2015109391A1 (en) 2014-01-24 2015-07-30 Children's Hospital Of Eastern Ontario Research Institute Inc. Smc combination therapy for the treatment of cancer
US12139542B2 (en) 2014-05-27 2024-11-12 Trustees Of Boston University Inhibitors of aortic carboxypeptidase-like protein (ACLP)
EP2982756A1 (en) 2014-08-04 2016-02-10 Berlin Cures Holding AG Aptamers for use against autoantibody-associated diseases
AU2015339063A1 (en) 2014-10-31 2017-06-29 H. Lee Moffitt Cancer Center And Research Institute, Inc. TLR9 targeted cytotoxic agents
WO2016130832A1 (en) 2015-02-13 2016-08-18 Idera Pharmaceuticals, Inc. Toll-like receptor 9 antagonist and methods of use thereof
US20180110777A1 (en) 2015-03-19 2018-04-26 The Johns Hopkins University Method for regulation of telomere length
WO2016168656A1 (en) 2015-04-15 2016-10-20 Ji Hoon Lee Aptasensor and method of detecting target material
WO2016183370A1 (en) 2015-05-13 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Synthetic immunosuppressive oligodeoxynucleotides reduce ischemic tissue damage
ES2781459T3 (es) 2015-07-29 2020-09-02 Ifom Fondazione St Firc Di Oncologia Molecolare Oligonucleótidos terapéuticos
WO2017085115A1 (en) 2015-11-18 2017-05-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for treating rhabdomyolysis
US10806749B2 (en) 2015-12-25 2020-10-20 Sbi Biotech Co., Ltd. TLR inhibitory oligonucleotides and their use
CN105597079A (zh) * 2016-01-11 2016-05-25 中国人民解放军第三军医大学第一附属医院 用于治疗银屑病的药物
US10124062B2 (en) 2016-02-26 2018-11-13 The United States Of America As Represented By The Department Of Veterans Affairs Methods of treating, inhibiting and/or preventing an auditory impairment
JP7034951B2 (ja) 2016-06-08 2022-03-14 プレジデント アンド フェローズ オブ ハーバード カレッジ 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する
CN117653654A (zh) 2016-11-23 2024-03-08 柏林制药股份有限公司 用于抑制和/或压制tlr9激活的适体
JP2021502381A (ja) 2017-11-08 2021-01-28 プレジデント アンド フェローズ オブ ハーバード カレッジ ウイルスベクター誘発性炎症反応を阻害するための組成物および方法

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002505580A (ja) 1997-06-06 2002-02-19 ザ リージェンツ オブ ザ ユニバーシティー オブ カリフォルニア Dna免疫刺激配列活性の阻害剤
JP2013544816A (ja) 2010-11-19 2013-12-19 イデラ ファーマシューティカルズ インコーポレイテッド トール様受容体に基づく免疫反応を調節するための免疫調節オリゴヌクレオチド(iro)化合物
JP2015532095A (ja) 2012-09-28 2015-11-09 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター
JP2015536659A (ja) 2012-11-29 2015-12-24 Sbiバイオテック株式会社 阻害性オリゴヌクレオチド及びその使用
WO2014105870A1 (en) 2012-12-27 2014-07-03 Sierra Sciences, Inc. Enhancing health in mammals using telomerase reverse transcriptase gene therapy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Gursel I. et al.,The Journal of Immunology,2003年,Vol. 171,p. 1393-1400
Li Y. et al.,Vaccine,2011年,Vol. 29,p. 2193-2198
Martino A. T. et al.,Blood,2011年,Vol. 117, No. 24,p. 6459-6468

Also Published As

Publication number Publication date
CN109451729A (zh) 2019-03-08
JP7394898B2 (ja) 2023-12-08
AU2017277647A1 (en) 2018-12-13
US20190316151A1 (en) 2019-10-17
JP2019517266A (ja) 2019-06-24
EP3468605A4 (en) 2020-01-08
US20190177731A1 (en) 2019-06-13
AU2017277647B2 (en) 2023-07-27
KR20190017872A (ko) 2019-02-20
US20240043849A1 (en) 2024-02-08
AU2023251424A1 (en) 2023-11-09
JP2022066336A (ja) 2022-04-28
CN109451729B (zh) 2022-03-22
EP3468605A1 (en) 2019-04-17
WO2017214378A1 (en) 2017-12-14
US11339396B2 (en) 2022-05-24
CN114807152A (zh) 2022-07-29

Similar Documents

Publication Publication Date Title
JP7394898B2 (ja) 操作されたウイルスベクターは炎症および免疫応答の誘導を低減する
JP7754872B2 (ja) 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途
US20260049334A1 (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
US9725485B2 (en) AAV vectors with high transduction efficiency and uses thereof for gene therapy
JP2022093425A (ja) 高効率ゲノム編集のためのアデノ随伴ウイルスベクターバリアント及びその方法
CN105408352B (zh) 通过双重aav载体有效递送大基因
US9611302B2 (en) High-transduction-efficiency RAAV vectors, compositions, and methods of use
US11999965B2 (en) Bocaparvovirus small noncoding RNA and uses thereof
TW201741458A (zh) 治療a型血友病之基因治療
KR20160026841A (ko) 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법
KR20160033217A (ko) 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
CN117858891A (zh) 用于基因疗法的经修饰aav衣壳及其方法
TW201629225A (zh) 第九因子基因療法
JP2011516049A (ja) 遺伝子障害を治療する方法
JP2023548746A (ja) サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用
EP4509606A1 (en) Nucleic acid construct for treating hereditary coagulation factor deficiency and use thereof
JP2023543361A (ja) Neurod1及びdlx2ベクター
Cho et al. Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics
JP2023507174A (ja) Dmd変異の修正のための方法及び組成物
CN117867025A (zh) ScAAV-DJ/8装载的精炼凝血因子IX载体的制备及用途
Luo et al. Identification of AAV Capsids with Enhanced Intravitreal Transduction and Favorable Safety Profile in Non-Human Primates
HK40096161B (zh) 一种用於遗传性凝血因子缺乏病治疗的核酸构建体及其用途
HK40096161A (zh) 一种用於遗传性凝血因子缺乏病治疗的核酸构建体及其用途
US20160237141A1 (en) Methods of treating alzheimer's disease with apo a-1 milano
EA048431B1 (ru) Вектор на основе аденоассоциированного вируса (aav) клады f и его применения

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200608

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200608

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210519

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210806

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210903

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211203

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220201

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220302

R150 Certificate of patent or registration of utility model

Ref document number: 7034951

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250