JP5864100B2 - 組織特異的ペプチドコンジュゲートおよび方法 - Google Patents

組織特異的ペプチドコンジュゲートおよび方法 Download PDF

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JP5864100B2
JP5864100B2 JP2010514854A JP2010514854A JP5864100B2 JP 5864100 B2 JP5864100 B2 JP 5864100B2 JP 2010514854 A JP2010514854 A JP 2010514854A JP 2010514854 A JP2010514854 A JP 2010514854A JP 5864100 B2 JP5864100 B2 JP 5864100B2
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peptide
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antisense
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JP2010532168A (ja
JP2010532168A5 (enExample
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ホン エム． モールトン
ホン エム． モールトン
パトリック エル． アイバーセン
パトリック エル． アイバーセン
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サレプタ セラピューティクス インコーポレイテッド
サレプタ セラピューティクス インコーポレイテッド
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    • 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
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    • A—HUMAN NECESSITIES
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    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P31/12—Antivirals
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    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
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    • 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
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
    • 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
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

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JP2010514854A 2007-06-29 2008-06-30 組織特異的ペプチドコンジュゲートおよび方法 Active JP5864100B2 (ja)

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US93772507P 2007-06-29 2007-06-29
US60/937,725 2007-06-29
PCT/US2008/008168 WO2009005793A2 (en) 2007-06-29 2008-06-30 Tissue specific peptide conjugates and methods

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JP2010532168A JP2010532168A (ja) 2010-10-07
JP2010532168A5 JP2010532168A5 (enExample) 2011-08-11
JP5864100B2 true JP5864100B2 (ja) 2016-02-17

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JP2015182739A Active JP6584888B2 (ja) 2007-06-29 2015-09-16 組織特異的ペプチドコンジュゲートおよび方法
JP2018121949A Pending JP2018171066A (ja) 2007-06-29 2018-06-27 組織特異的ペプチドコンジュゲートおよび方法

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US (1) US20090099066A1 (enExample)
EP (2) EP2170363B1 (enExample)
JP (3) JP5864100B2 (enExample)
AU (1) AU2008271050B2 (enExample)
CA (1) CA2691673A1 (enExample)
ES (1) ES2694726T3 (enExample)
WO (1) WO2009005793A2 (enExample)

Families Citing this family (117)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003225410A1 (en) * 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
ES2351976T3 (es) 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
EP1713332A4 (en) * 2004-01-23 2010-08-18 Avi Biopharma Inc ANTISENSE OLIGOMERS AND METHOD FOR INDUCTION OF IMMUNOTHERAPY AND IMMUNOSUPPRESSION
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
ES2852549T3 (es) * 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
CA2638823A1 (en) 2006-02-13 2007-08-23 Alethia Biotherapeutics Inc. Polynucleotides and polypeptide sequences involved in the process of bone remodeling
US8168181B2 (en) 2006-02-13 2012-05-01 Alethia Biotherapeutics, Inc. Methods of impairing osteoclast differentiation using antibodies that bind siglec-15
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
CA2710013A1 (en) * 2007-12-28 2009-07-09 Avi Biopharma, Inc. Immunomodulatory agents and methods of use
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8231895B2 (en) 2008-05-22 2012-07-31 Universidade De Coimbra Targeted delivery to human diseases and disorders
US20110130346A1 (en) * 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
US8575305B2 (en) 2008-06-04 2013-11-05 Medical Research Council Cell penetrating peptides
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2762567B1 (en) 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
US8592386B2 (en) * 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
CA2779830C (en) 2009-11-13 2020-07-21 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
KR20120125455A (ko) * 2009-12-11 2012-11-15 광주과학기술원 세포내 타겟 결합용 바이포달 펩타이드 바인더
US9006415B2 (en) * 2010-04-06 2015-04-14 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
US9050373B2 (en) * 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
US20130225659A1 (en) * 2010-07-19 2013-08-29 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
WO2013030569A2 (en) 2011-08-30 2013-03-07 Michael John Gait Peptides
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
ES2535654T3 (es) * 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
PT2788487T (pt) 2011-12-08 2018-07-03 Sarepta Therapeutics Inc Análogos de oligonucleótido visando lmna humano
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
SG11201407483YA (en) * 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
CA2873769A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating hemoglobin gene family expression
BR112014028634A2 (pt) * 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
BR112014028644A2 (pt) 2012-05-16 2017-08-15 Rana Therapeutics Inc Composições e métodos para modulação da expressão de atp2a2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
KR101329411B1 (ko) * 2012-05-31 2013-11-14 주식회사 엘지생활건강 피부투과성 펩타이드
ES2723885T3 (es) 2012-07-19 2019-09-03 Daiichi Sankyo Co Ltd Anticuerpos anti-Siglec-15
CA2882292C (en) 2012-08-13 2023-10-17 The Rockefeller University Treatment and diagnosis of melanoma
MX369519B (es) * 2012-09-25 2019-11-11 Genzyme Corp Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.
JP6452614B2 (ja) 2012-11-15 2019-01-16 ロシュ イノベーション センター コペンハーゲン エーエス オリゴヌクレオチドコンジュゲート
CN103981147B (zh) 2013-02-08 2017-11-10 中国科学院上海生命科学研究院 一种新的制备肝实质细胞的方法
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
US10010562B2 (en) 2013-11-06 2018-07-03 Merck Sharp & Dohme Corp. Dual molecular delivery of oligonucleotides and peptide containing conjugates
WO2015175977A2 (en) 2014-05-16 2015-11-19 Geller Bruce L Antisense antibacterial compounds and methods
EP3569252B1 (en) 2014-05-19 2021-12-15 Oregon State University Antisense antibacterial compounds and methods
US20170182189A1 (en) * 2014-05-23 2017-06-29 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
JP6825914B2 (ja) 2014-05-23 2021-02-03 ジェンザイム・コーポレーション ペプチド担体上の多重オリゴヌクレオチド部分
AU2015372560B2 (en) * 2014-12-31 2021-12-02 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
MA41795A (fr) * 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016196897A1 (en) 2015-06-04 2016-12-08 Sarepta Therapeutics, Inc. Methods and compounds for treatment of lymphocyte-related diseases and conditions
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
AU2016379399B2 (en) 2015-12-23 2022-12-08 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
AU2016379402B2 (en) * 2015-12-23 2023-01-12 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
NZ747685A (en) 2016-04-29 2023-05-26 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
US10800817B2 (en) 2016-12-19 2020-10-13 Morehouse School Of Medicine Compositions and methods for treating diseases by inhibiting exosome release
HRP20240705T1 (hr) * 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US20180207293A1 (en) * 2017-01-25 2018-07-26 2C Tech Corp. Nanoparticles for sustained ophthalmic drug delivery and methods of use
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP2020536060A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
TW202423453A (zh) 2017-10-17 2024-06-16 美商薩羅塔治療公司 用於反義遞送之細胞穿透肽
EP3713575A4 (en) 2017-11-21 2021-08-25 Rgenix, Inc. POLYMORPHS AND THEIR USES
JP7013626B2 (ja) * 2018-01-05 2022-02-01 国立医薬品食品衛生研究所長 細胞膜透過ペプチド、構築物、及び、カーゴ分子を細胞内に輸送する方法
US11987647B2 (en) 2018-05-09 2024-05-21 Ohio State Innovation Foundation Cyclic cell-penetrating peptides with one or more hydrophobic residues
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
TW202449155A (zh) 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US20210308272A1 (en) * 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
EP3890783A1 (en) 2018-12-07 2021-10-13 Oxford University Innovation Limited Linkers
SG11202104960PA (en) 2018-12-13 2021-06-29 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
JP2022528725A (ja) 2019-04-18 2022-06-15 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを治療するための組成物
GB201911403D0 (en) * 2019-08-09 2019-09-25 Univ Oxford Innovation Ltd Conjugate and uses thereof
KR102270700B1 (ko) * 2019-11-01 2021-06-30 한국과학기술연구원 폐 섬유증의 예방 또는 치료를 위한 올리고뉴클레오티드 중합체와 생체 적합성 양이온성 펩타이드로 이루어지는 폐 특이적 약물 전달체 및 이의 이용
JP7689964B2 (ja) 2019-12-13 2025-06-09 インスピルナ，インコーポレーテッド 金属塩及びその使用
AU2020411964A1 (en) 2019-12-26 2022-06-16 National Center Of Neurology And Psychiatry Antisense nucleic acid that induces skipping of exon 50
PE20230237A1 (es) 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
US11987795B2 (en) 2020-11-24 2024-05-21 The Broad Institute, Inc. Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11
US20240327831A1 (en) 2020-12-23 2024-10-03 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
AU2022298028A1 (en) 2021-06-23 2023-12-21 National Center Of Neurology And Psychiatry Combination of antisense oligomers
JPWO2023282344A1 (enExample) 2021-07-08 2023-01-12
IL310003A (en) 2021-07-08 2024-03-01 Nippon Shinyaku Co Ltd Nephrotoxicity reducing agent
WO2023282346A1 (ja) 2021-07-08 2023-01-12 日本新薬株式会社 析出抑制剤
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US20250011777A1 (en) * 2021-09-03 2025-01-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
JP2025509438A (ja) 2022-03-17 2025-04-11 サレプタ セラピューティクス， インコーポレイテッド ホスホロジアミデートモルホリノオリゴマーコンジュゲート
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
WO2023205451A1 (en) 2022-04-22 2023-10-26 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
WO2024091824A1 (en) 2022-10-26 2024-05-02 Ada Forsyth Institute, Inc. Differentiation and reprogramming of chondrocyte
JP2025537104A (ja) 2022-11-02 2025-11-14 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマーコンジュゲートの製剤化
WO2024129459A1 (en) 2022-12-16 2024-06-20 University Of Rochester Repairmen! of barrier dysfunction in esophagus
WO2025085868A2 (en) 2023-10-20 2025-04-24 Radiation Control Technologies, Inc. Cd47-targeting morpholinos

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US946496A (en) 1909-08-21 1910-01-11 Henry J Cox Fulcrum-block.
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5525465A (en) * 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
US5670617A (en) * 1989-12-21 1997-09-23 Biogen Inc Nucleic acid conjugates of tat-derived transport polypeptides
CN1119830A (zh) * 1993-01-07 1996-04-03 托马斯·杰弗逊大学 反义抑制C-myc以调节平滑肌细胞的增殖
US6686338B1 (en) 1996-02-23 2004-02-03 The Board Of Regents Of The University Of Nebraska Enzyme inhibitors for metabolic redirection
US5849727A (en) * 1996-06-28 1998-12-15 Board Of Regents Of The University Of Nebraska Compositions and methods for altering the biodistribution of biological agents
IL132941A0 (en) * 1997-05-21 2001-03-19 Univ Leland Stanford Junior Composition and method for enhancing transport across biological membranes
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
AUPP398498A0 (en) 1998-06-09 1998-07-02 Silverbrook Research Pty Ltd A method of manufacture of an image creation apparatus (ijm44)
AU743695B2 (en) 1998-07-13 2002-01-31 Board Of Regents Of The University Of Nebraska, The Targeted site specific drug delivery compositions and method of use
KR20010102992A (ko) 1999-01-29 2001-11-17 추후보정 표적 ｒｎａ를 검출하는 비-침입적 방법
US7094765B1 (en) 1999-01-29 2006-08-22 Avi Biopharma, Inc. Antisense restenosis composition and method
JP2002541825A (ja) * 1999-04-08 2002-12-10 オアシス バイオサイエンシーズ インコーポレイティッド ユニバーサル及び／又は同義性塩基を含むアンチセンスオリゴヌクレオチド
US6303573B1 (en) * 1999-06-07 2001-10-16 The Burnham Institute Heart homing peptides and methods of using same
US6669951B2 (en) * 1999-08-24 2003-12-30 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into epithelial tissues
DE60143085D1 (de) 2000-05-17 2010-10-28 Univ Nebraska Morpholino antisense oligomere als enzyminhibitoren zur verbesserung der pharmacokinetik eines wirkstoffs
US20040170955A1 (en) 2000-09-08 2004-09-02 Wadih Arap Human and mouse targeting peptides identified by phage display
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
EP1364201A4 (en) * 2001-02-06 2005-01-05 Univ Auburn LIGAND SENSOR DEVICES AND USES THEREOF
AU2002306500C1 (en) * 2001-02-16 2006-09-28 Cellgate, Inc. Transporters comprising spaced arginine moieties
CN1509292A (zh) * 2001-05-17 2004-06-30 AVI������ҩ��˾ 使用c-myc反义寡聚物治疗癌症的结合的方法
JP3735292B2 (ja) * 2001-07-26 2006-01-18 三菱重工業株式会社 ダイエット効果のある健康食品および製剤
US6645974B2 (en) * 2001-07-31 2003-11-11 Merck & Co., Inc. Androgen receptor modulators and methods for use thereof
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
EP1461084A2 (en) * 2001-12-11 2004-09-29 The Board Of Trustees Of The Leland Stanford Junior University Guanidinium transport reagents and conjugates
US20030207907A1 (en) 2002-05-03 2003-11-06 Iversen Patrick L. Delivery of microparticle-conjugated drugs for inhibition of stenosis
ES2351976T3 (es) * 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
US20050203041A1 (en) 2003-09-23 2005-09-15 Mourich Dan V. Antisense compound and method for selectively killing activated T cells
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US7786151B2 (en) * 2004-01-09 2010-08-31 Kinopharma, Inc. Therapeutic composition of treating abnormal splicing caused by the excessive kinase induction
EP1713332A4 (en) * 2004-01-23 2010-08-18 Avi Biopharma Inc ANTISENSE OLIGOMERS AND METHOD FOR INDUCTION OF IMMUNOTHERAPY AND IMMUNOSUPPRESSION
WO2005077333A2 (en) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
WO2005117928A1 (en) * 2004-05-30 2005-12-15 Cemines, Inc. Compositions and methods for the treatment of skin cancer
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
US7489981B2 (en) * 2004-09-01 2009-02-10 Honda Motor Co., Ltd. Assembly line control system and immobilizer data protocol and communication process flow
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8357664B2 (en) * 2004-10-26 2013-01-22 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
NZ538097A (en) * 2005-02-07 2006-07-28 Ovita Ltd Method and compositions for improving wound healing
ES2852549T3 (es) * 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
US7790694B2 (en) * 2005-07-13 2010-09-07 Avi Biopharma Inc. Antisense antibacterial method and compound
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
CA2621964A1 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
CA2628093A1 (en) * 2005-11-08 2007-05-18 Avi Biopharma, Inc. Antisense oligonucleotide analog compounds targeting proprocessed ctla-4 mrna
US8501704B2 (en) * 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
EP2002018B1 (en) * 2006-03-07 2010-05-12 AVI BioPharm, Inc. Antisense antiviral compound and method for treating arenavirus infection
ES2657400T3 (es) 2006-05-10 2018-03-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
CA2710013A1 (en) * 2007-12-28 2009-07-09 Avi Biopharma, Inc. Immunomodulatory agents and methods of use
EP2762567B1 (en) * 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
US8592386B2 (en) * 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis

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