HUE059843T2 - Exonátugró oligomerkonjugátumok izomdisztrófiára - Google Patents

Exonátugró oligomerkonjugátumok izomdisztrófiára

Info

Publication number
HUE059843T2
HUE059843T2 HUE17832401A HUE17832401A HUE059843T2 HU E059843 T2 HUE059843 T2 HU E059843T2 HU E17832401 A HUE17832401 A HU E17832401A HU E17832401 A HUE17832401 A HU E17832401A HU E059843 T2 HUE059843 T2 HU E059843T2
Authority
HU
Hungary
Prior art keywords
muscular dystrophy
exon skipping
oligomer conjugates
skipping oligomer
conjugates
Prior art date
Application number
HUE17832401A
Other languages
English (en)
Hungarian (hu)
Inventor
Marco A Passini
Gunnar J Hanson
Original Assignee
Sarepta Therapeutics 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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of HUE059843T2 publication Critical patent/HUE059843T2/hu

Links

Classifications

    • 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it

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)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
HUE17832401A 2016-12-19 2017-12-13 Exonátugró oligomerkonjugátumok izomdisztrófiára HUE059843T2 (hu)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662436182P 2016-12-19 2016-12-19
US201762443476P 2017-01-06 2017-01-06
US201762479173P 2017-03-30 2017-03-30
US201762562080P 2017-09-22 2017-09-22

Publications (1)

Publication Number Publication Date
HUE059843T2 true HUE059843T2 (hu) 2023-01-28

Family

ID=61006315

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE17832401A HUE059843T2 (hu) 2016-12-19 2017-12-13 Exonátugró oligomerkonjugátumok izomdisztrófiára

Country Status (27)

Country Link
US (4) US10888578B2 (enExample)
EP (1) EP3554553B1 (enExample)
JP (3) JP7118063B2 (enExample)
KR (2) KR102552428B1 (enExample)
CN (1) CN110290812B (enExample)
AU (1) AU2017382723B2 (enExample)
CA (1) CA3046793A1 (enExample)
CO (1) CO2019007397A2 (enExample)
DK (1) DK3554553T3 (enExample)
ES (1) ES2927079T3 (enExample)
HR (1) HRP20220943T1 (enExample)
HU (1) HUE059843T2 (enExample)
IL (2) IL267244B (enExample)
LT (1) LT3554553T (enExample)
MD (1) MD3554553T2 (enExample)
MX (1) MX2019006882A (enExample)
MY (1) MY195801A (enExample)
NZ (1) NZ755416A (enExample)
PL (1) PL3554553T3 (enExample)
PT (1) PT3554553T (enExample)
RS (1) RS63610B1 (enExample)
SA (1) SA519402154B1 (enExample)
SG (1) SG10202012839TA (enExample)
SM (1) SMT202200366T1 (enExample)
TW (1) TWI760402B (enExample)
WO (1) WO2018118599A1 (enExample)
ZA (1) ZA201904092B (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA49775A (fr) 2016-05-24 2020-06-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
EP3464306B1 (en) 2016-05-24 2024-03-27 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
KR102552428B1 (ko) * 2016-12-19 2023-07-06 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
EP3554552B1 (en) * 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
SMT202200440T1 (it) 2016-12-19 2023-01-13 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
KR20190104381A (ko) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
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
WO2020257489A1 (en) * 2019-06-19 2020-12-24 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
TWI874546B (zh) 2019-12-26 2025-03-01 日商日本新藥股份有限公司 誘導外顯子50的跳讀的反義核酸
CA3173049A1 (en) 2020-02-28 2021-09-02 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
BR112023021849A2 (pt) 2021-04-30 2023-12-19 Sarepta Therapeutics Inc Métodos de tratamento para distrofia muscular
IL309626A (en) 2021-06-23 2024-02-01 Nippon Shinyaku Co Ltd A combination of antisense oligomers
EP4368176A4 (en) 2021-07-08 2025-06-25 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
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
TW202346589A (zh) 2022-03-10 2023-12-01 日商日本新藥股份有限公司 抗病毒反義寡聚物
CN119013401A (zh) 2022-03-17 2024-11-22 萨勒普塔医疗公司 二氨基磷酸酯吗啉代寡聚物缀合物

Family Cites Families (177)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion 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
US5087617A (en) 1989-02-15 1992-02-11 Board Of Regents, The University Of Texas System Methods and compositions for treatment of cancer using oligonucleotides
US5670617A (en) 1989-12-21 1997-09-23 Biogen Inc Nucleic acid conjugates of tat-derived transport polypeptides
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
GB9209032D0 (en) 1992-04-25 1992-06-10 Ciba Geigy Ag New peptide derivatives
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent animal diseases
CA2135642C (en) 1992-08-21 1999-12-14 James G. Barsoum Tat-derived transport polypeptides
JPH08505397A (ja) 1993-01-07 1996-06-11 トーマス ジェファーソン ユニバーシティ 平滑筋細胞の増殖を調節するためのc‐mycのアンチセンス阻害
ES2290951T3 (es) 1993-05-11 2008-02-16 The University Of North Carolina At Chapel Hill Oligonucleotidos antisentido que combaten el corte y empalme aberrante y metodos de uso de los mismos.
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
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
BR9809138A (pt) 1997-05-21 2001-08-28 Trustees For The Leland Stanfo Conjugado e método para aumentar o transporte de um composto selecionado atravessando uma membrana biológica
US6329501B1 (en) 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
CA2340884A1 (en) 1998-08-25 2000-03-02 Human Genome Sciences, Inc. 49 human secreted proteins
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
JP2000125448A (ja) 1998-10-14 2000-04-28 Yazaki Corp 電気接続箱
CA2360997A1 (en) 1999-01-29 2000-08-03 Avi Biopharma, Inc. Non-invasive method for detecting target rna
JP2000256547A (ja) 1999-03-10 2000-09-19 Sumitomo Dow Ltd 耐熱性プラスチックカード用樹脂組成物
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
IL145586A0 (en) 1999-04-08 2002-06-30 Oasis Biosciences Inc Antisense oligonucleotides comprising universal and/or degenerate bases
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
ATE402257T1 (de) 1999-05-24 2008-08-15 Avi Biopharma Inc Antisense gegen c-myc zur behandlung von polyzystischer nierenkrankheit
US6303573B1 (en) 1999-06-07 2001-10-16 The Burnham Institute Heart homing peptides and methods of using same
EP2269654A3 (en) 1999-08-24 2011-04-13 Cellgate Inc. Enhancing drug delivery across and into epithelial tissues using oligo arginine moieties
US7229961B2 (en) 1999-08-24 2007-06-12 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into ocular tissues
US6669951B2 (en) 1999-08-24 2003-12-30 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into epithelial tissues
US20030104622A1 (en) 1999-09-01 2003-06-05 Robbins Paul D. Identification of peptides that facilitate uptake and cytoplasmic and/or nuclear transport of proteins, DNA and viruses
US7163695B2 (en) 1999-12-29 2007-01-16 Mixson A James Histidine copolymer and methods for using same
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
US20020009491A1 (en) 2000-02-14 2002-01-24 Rothbard Jonathan B. Compositions and methods for enhancing drug delivery across biological membranes and tissues
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
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
WO2002038764A2 (en) 2000-11-10 2002-05-16 The Regents Of The University Of California Il-17 receptor-like protein, uses thereof, and modulation of catabolic activity of il-17 cytokines on bone and cartilage
EP1364201A4 (en) 2001-02-06 2005-01-05 Univ Auburn LIGAND SENSOR DEVICES AND USES THEREOF
AU2002250034A1 (en) 2001-02-08 2002-08-19 Sequitur, Inc. Methods of light activated release 0f ligands from endosomes
US7585834B2 (en) 2001-02-16 2009-09-08 The Board Of Trustees Of The Leland Stanford Junior University Transporters comprising spaced arginine moieties
US7456146B2 (en) 2001-05-09 2008-11-25 Ghc Research Development Corporation Lytic peptide prodrugs
KR20040004629A (ko) 2001-05-17 2004-01-13 에이브이아이 바이오파마 인코포레이티드 c-myc 안티센스 올리고머를 사용한 암치료를 위한조합 접근법
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
US20090075377A1 (en) 2001-08-03 2009-03-19 Arbor Vita Corporation Molecular interactions in cells
WO2003020739A2 (en) 2001-09-04 2003-03-13 Exiqon A/S Novel lna compositions and uses thereof
CA2469336C (en) 2001-12-11 2013-06-11 The Board Of Trustees Of The Leland Stanford Junior University Guanidinium transport reagents and conjugates
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Pna 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Pna 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003295388A1 (en) 2002-11-05 2004-06-03 Isis Pharmaceuticals, Inc. 2'-substituted oligomeric compounds and compositions for use in gene modulations
EP1568769A4 (en) 2002-11-25 2008-08-06 Masafumi Matsuo ENA-NUCLEIC ACID MEDICAMENTS THAT CHANGE SPLICING IN THE MRNA PRESETTER
US7482016B2 (en) 2003-03-19 2009-01-27 The J. David Gladstone Institutes Immunogenic compositions comprising HIV-1 acetylated Tat polypeptides
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
US7468418B2 (en) 2003-04-29 2008-12-23 Avi Biopharma., Inc. Compositions for enhancing transport of molecules into cells
NZ573837A (en) 2003-07-08 2010-07-30 Genentech Inc IL-17 A/F heterologous polypeptides and therapeutic uses thereof
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
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
US7402574B2 (en) 2004-03-12 2008-07-22 Avi Biopharma, Inc. Antisense composition and method for treating cancer
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
US20060014712A1 (en) 2004-05-30 2006-01-19 Cemines, Inc. Controlled delivery of therapeutic compounds
EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006088490A2 (en) 2004-06-30 2006-08-24 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a non-phosphate backbone linkage
US7947645B2 (en) 2004-09-02 2011-05-24 Cognosci, Inc. APO E analogs and methods for their use
US7429481B2 (en) 2004-09-14 2008-09-30 University Of Pittsburgh Targeting viruses using a modified sindbis glycoprotein
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
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
PE20061391A1 (es) 2005-02-14 2007-01-09 Wyeth Corp Anticuerpos para interleucina-17f y otros antagonistas de la senalizacion de il-17f
KR20080031164A (ko) 2005-04-22 2008-04-08 아카데미슈 지켄후이스 라이덴 SR 단백질의 결합의 방해에 의해 그리고 이차 RNA구조의 방해에 의한 pre-mRNA에서 엑손 인식의조절
WO2006114478A1 (en) 2005-04-26 2006-11-02 Karyon-Ctt Ltd Diagnostic and therapeutic agents
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US7790694B2 (en) 2005-07-13 2010-09-07 Avi Biopharma Inc. Antisense antibacterial method and compound
AU2006267051B2 (en) 2005-07-13 2013-03-07 Sarepta Therapeutics, Inc. Antisense antibacterial method and compound
CA2621964A1 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8524676B2 (en) 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
US8137670B2 (en) 2005-09-29 2012-03-20 Medimmune, Llc Method of identifying membrane IgE specific antibodies and use thereof for targeting IgE producing precursor cells
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
AU2006311586A1 (en) 2005-11-08 2007-05-18 Avi Biopharma, Inc. Immunosuppression compound and treatment method
WO2007058894A2 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
WO2007103529A2 (en) 2006-03-07 2007-09-13 Avi Biopharma, Inc. Antisense antiviral compound and method for treating arenavirus infection
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
JP2010505741A (ja) 2006-05-10 2010-02-25 エイブイアイ バイオファーマ, インコーポレイテッド カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
JP3151554U (ja) 2006-05-17 2009-07-02 スヴェトラナ アナトレフナ ソコロヴァ 輸送手段
CA2558160C (en) 2006-06-30 2017-01-03 Oligos Etc., Inc. Antisense oligonucleotide targeting myostatin or fox01 for treatment of muscle wasting
NZ574807A (en) 2006-08-11 2011-01-28 Prosensa Technologies Bv Methods and means for treating dna repeat instability associated genetic disorders
US20080199961A1 (en) 2006-08-25 2008-08-21 Avi Biopharma, Inc. ANTISENSE COMPOSITION AND METHOD FOR INHIBITION OF miRNA BIOGENESIS
US20080267978A1 (en) 2006-08-28 2008-10-30 Mary Zutter Anti-angiogenic targets for cancer therapy
US20100190689A1 (en) 2006-09-21 2010-07-29 University Of Rochester Compositions and methods related to protein displacement therapy for myotonic distrophy
FR2908999B1 (fr) 2006-11-29 2012-04-27 Biomerieux Sa Nouveau medicament destine a l'inhibition, la prevention ou le traitement de la polyarthrite rhumatoide.
EP1938802A1 (en) 2006-12-22 2008-07-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Interfering RNAs targeting pro-inflammatory cytokines
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
CN101790385A (zh) 2007-07-12 2010-07-28 普罗森那技术公司 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子
US20110300154A1 (en) 2007-08-21 2011-12-08 Children's Medical Center Corporation Treatment of airway hyperreactivity
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
HRP20140646T1 (hr) 2007-11-15 2014-09-26 Sarepta Therapeutics, Inc. Postupak sinteze morfolino oligomera
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
CA2710013A1 (en) 2007-12-28 2009-07-09 Avi Biopharma, Inc. Immunomodulatory agents and methods of use
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
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
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
DK3133160T3 (en) 2008-10-24 2019-04-01 Sarepta Therapeutics Inc EXON SKIP COMPOSITIONS FOR DMD
DK2344637T4 (en) 2008-10-27 2018-04-23 Biomarin Tech Bv Methods and means for efficiently skipping exon 45 in Duchenne muscular dystrophy pre-mRNA
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
WO2010080554A1 (en) 2008-12-17 2010-07-15 Avi Biopharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
PL2417257T3 (pl) 2009-04-10 2016-11-30 Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
ES2586683T3 (es) 2009-09-16 2016-10-18 Wave Life Sciences Japan, Inc. Grupo protector novedoso para sintetizar ARN y derivados del mismo
BR112012011195B1 (pt) 2009-11-12 2024-02-15 The University Of Western Australia Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica
EP2569431B1 (en) 2010-05-13 2015-09-23 Sarepta Therapeutics, Inc. Methods for identifying compounds which modulate interleukins 17 and 23 signaling activity
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102183273B1 (ko) * 2011-05-05 2020-11-27 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9607308B2 (en) 2011-06-29 2017-03-28 American Express Travel Related Services Company, Inc. Spend based digital ad targeting and measurement
DK2581448T3 (en) 2011-10-13 2015-04-27 Ass Inst De Myologie Tricyclo-DNA phosphorothioate
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
ES2748868T3 (es) 2011-12-28 2020-03-18 Nippon Shinyaku Co Ltd Acido nucleico antisentido
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド
DE102012101676A1 (de) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage
CN110257379B (zh) * 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
SG10201912895PA (en) 2012-07-13 2020-02-27 Wave Life Sciences Ltd Chiral control
CN104684893B (zh) 2012-07-13 2016-10-26 日本波涛生命科学公司 不对称辅助基团
EP3885439A1 (en) 2012-12-20 2021-09-29 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
EP3633035A1 (en) 2013-03-14 2020-04-08 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
KR20220061280A (ko) 2013-03-14 2022-05-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
BR112015022998A2 (pt) * 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
IL282239B2 (en) * 2013-09-05 2023-10-01 Sarepta Therapeutics Inc Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
US10149905B2 (en) 2014-01-15 2018-12-11 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
RU2016133035A (ru) 2014-01-16 2018-02-21 Уэйв Лайф Сайенсес Лтд. Хиральный дизайн
DK3118311T3 (en) 2014-03-12 2019-03-11 Nippon Shinyaku Co Ltd Antisense nucleic acid
EP4596047A3 (en) 2014-06-17 2025-10-22 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for use in treating duchenne muscular dystrophy
AU2015338923B2 (en) 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
EA201890908A1 (ru) * 2015-10-09 2018-10-31 Сарепта Терапьютикс, Инк. Композиции и способы для лечения мышечной дистрофии дюшенна и сходных нарушений
TW201722439A (zh) 2015-10-09 2017-07-01 波濤生命科學有限公司 寡核苷酸組合物及其方法
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
KR102552428B1 (ko) * 2016-12-19 2023-07-06 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체

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