CN115867657A - 用于治疗疾患和疾病的opa1反义寡聚物 - Google Patents

用于治疗疾患和疾病的opa1反义寡聚物 Download PDF

Info

Publication number
CN115867657A
CN115867657A CN202180049789.8A CN202180049789A CN115867657A CN 115867657 A CN115867657 A CN 115867657A CN 202180049789 A CN202180049789 A CN 202180049789A CN 115867657 A CN115867657 A CN 115867657A
Authority
CN
China
Prior art keywords
fold
nucleotides
mrna
exon
target portion
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.)
Pending
Application number
CN202180049789.8A
Other languages
English (en)
Chinese (zh)
Inventor
伊莎贝尔·阿兹纳雷兹
阿迪蒂亚·文卡塔斯
吉恩·里奥
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.)
Stoke Therapeutics Inc
Original Assignee
Stoke 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 Stoke Therapeutics Inc filed Critical Stoke Therapeutics Inc
Publication of CN115867657A publication Critical patent/CN115867657A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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
    • A61K31/7125—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1137—Non-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 against enzymes
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05005—Dynamin GTPase (3.6.5.5)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202180049789.8A 2020-05-11 2021-04-30 用于治疗疾患和疾病的opa1反义寡聚物 Pending CN115867657A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063023013P 2020-05-11 2020-05-11
US63/023,013 2020-05-11
US202063112458P 2020-11-11 2020-11-11
US63/112,458 2020-11-11
PCT/US2021/030254 WO2021231107A1 (en) 2020-05-11 2021-04-30 Opa1 antisense oligomers for treatment of conditions and diseases

Publications (1)

Publication Number Publication Date
CN115867657A true CN115867657A (zh) 2023-03-28

Family

ID=78526029

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180049789.8A Pending CN115867657A (zh) 2020-05-11 2021-04-30 用于治疗疾患和疾病的opa1反义寡聚物

Country Status (11)

Country Link
US (3) US12338437B2 (enExample)
EP (1) EP4150092A4 (enExample)
JP (1) JP2023525799A (enExample)
KR (1) KR20230022409A (enExample)
CN (1) CN115867657A (enExample)
BR (1) BR112022022889A2 (enExample)
CA (1) CA3173647A1 (enExample)
IL (1) IL298063A (enExample)
MX (1) MX2022014151A (enExample)
TW (1) TWI901675B (enExample)
WO (1) WO2021231107A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
AU2021354152A1 (en) * 2020-10-02 2023-06-01 PYC Therapeutics Limited Treatment of optic atrophy
AU2023212479A1 (en) * 2022-01-31 2024-08-01 PYC Therapeutics Limited Method of treatment for optic atrophy
KR20240165951A (ko) * 2022-03-23 2024-11-25 피와이씨 테라퓨틱스 리미티드 녹내장 치료 방법
EP4594505A1 (en) * 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
EP4612295A1 (en) * 2022-10-31 2025-09-10 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
WO2024221049A1 (en) * 2023-04-28 2024-10-31 PYC Therapeutics Limited Agents and method of treatment for optic conditions
EP4720297A2 (en) * 2023-05-31 2026-04-08 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases

Family Cites Families (324)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3933961A (en) 1974-12-13 1976-01-20 Pennwalt Corporation Tabletting spherical dental amalgam alloy
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US4476116A (en) 1982-12-10 1984-10-09 Syntex (U.S.A.) Inc. Polypeptides/chelating agent nasal compositions having enhanced peptide absorption
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
US4866042A (en) 1987-11-18 1989-09-12 Neuwelt Edward A Method for the delivery of genetic material across the blood brain barrier
US6294520B1 (en) 1989-03-27 2001-09-25 Albert T. Naito Material for passage through the blood-brain barrier
WO1992003568A1 (en) 1990-08-13 1992-03-05 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
ATE299509T1 (de) 1992-07-23 2005-07-15 Isis Pharmaceuticals Inc Neue 2'-o-alkyl-nukleoside und -phosphoramidite, verfahren zu ihrer herstellung und ihre verwendungen
ATE404683T1 (de) 1992-09-25 2008-08-15 Aventis Pharma Sa Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
AU1322795A (en) 1993-12-24 1995-07-17 Erasmus University Rotterdam Tuberous sclerosis 2 gene and uses thereof
US5656612A (en) 1994-05-31 1997-08-12 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
FR2727867B1 (fr) 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
NZ331217A (en) 1996-02-14 2000-02-28 Novartis Ag Sugar-modified gapped oligonucleotides for eliciting RNase H activity for strand cleavage in an opposing strand
GB9605808D0 (en) 1996-03-20 1996-05-22 Isis Innovation CFTR gene regulator
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5955279A (en) 1996-06-13 1999-09-21 Gatti; Richard A. Ataxia-telangiectasia: mutations in the ATM gene
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
US7011973B1 (en) 1997-06-03 2006-03-14 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. Regulatory sequences capable of conferring expression of a heterologous DNA sequence in endothelial cells in vivo and uses thereof
US6963589B1 (en) 1997-07-03 2005-11-08 Canon Kabushiki Kaisha Information processing apparatus for and method of transmitting and/or receiving broadcast signal
US6391452B1 (en) 1997-07-18 2002-05-21 Bayer Corporation Compositions for nasal drug delivery, methods of making same, and methods of removing residual solvent from pharmaceutical preparations
GB9716231D0 (en) 1997-07-31 1997-10-08 Amersham Int Ltd Base analogues
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
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
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
WO2000010608A1 (en) 1998-08-24 2000-03-02 Meiji Milk Products Co., Ltd. Preventives/remedies for arteriosclerosis and post-ptca reangiostenosis
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US7071324B2 (en) 1998-10-13 2006-07-04 Brown University Research Foundation Systems and methods for sequencing by hybridization
US20030224514A1 (en) 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of PPAR-delta expression
US6436657B1 (en) 1998-12-18 2002-08-20 E. I. Du Pont De Nemours And Company Polynucleotides encoding aminomethyltransferases
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
KR20020013513A (ko) 1999-03-18 2002-02-20 추후제출 Ｘｙｌｏ-ｌｎａ 유사체
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
DE60029314T2 (de) 1999-03-24 2007-07-12 Exiqon A/S Verbesserte Synthese für -2.2.1. I Bicyclo-Nukleoside
US6383752B1 (en) 1999-03-31 2002-05-07 Hybridon, Inc. Pseudo-cyclic oligonucleobases
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6083482A (en) 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
US6531591B1 (en) 1999-07-07 2003-03-11 Exiqon A/S Synthesis of stable quinone and photoreactive ketone phosphoramidite reagents for solid phase synthesis of photoreactive-oligomer conjugates
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
US6458536B1 (en) 1999-07-23 2002-10-01 The Regents Of The University Of California Modified SSCP method using sequential electrophoresis of multiple nucleic acid segments
US6147200A (en) 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6677445B1 (en) 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
US6187586B1 (en) 1999-12-29 2001-02-13 Isis Pharmaceuticals, Inc. Antisense modulation of AKT-3 expression
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
US6809194B1 (en) 2000-05-10 2004-10-26 Chiron Corporation Akt3 inhibitors
CA2395781C (en) 2000-07-13 2010-04-13 The Johns Hopkins University School Of Medicine Detection and treatment of polycystic kidney disease
US7053199B2 (en) 2000-08-29 2006-05-30 Takeshi Imanishi Nucleoside analogs and oligonucleotide derivatives containing these analogs
AU2001295531A1 (en) 2000-09-02 2002-03-13 Grünenthal GmbH Antisense oligonucleotides against vanilloid receptor 1
JP4413493B2 (ja) 2000-10-04 2010-02-10 サンタリス ファーマ アー／エス プリンｌｎａ類似体の改善された合成方法
JP2002345489A (ja) 2000-11-03 2002-12-03 Astrazeneca Ab 化学物質
NZ530101A (en) 2001-05-17 2007-01-26 Avi Biopharma Inc Methods for treating cancer by administering an oligomer antisense to c-myc together with a cancer chemotherapeutic agent
ATE287413T1 (de) 2001-07-12 2005-02-15 Santaris Pharma As Verfahren zur herstellung des lna phosphoramidite
US20040078837A1 (en) 2001-08-02 2004-04-22 Shannon Mark E. Four human zinc-finger-containing proteins: MDZ3, MDZ4, MDZ7 and MDZ12
AU2002336367A1 (en) 2001-08-16 2003-03-03 The Regents Of The University Of Michigan Adamts13 genes and proteins and variants, and uses thereof
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
EP1481983A4 (en) 2002-02-13 2008-04-02 Takeshi Imanishi NUCLEOSIDE ANALOGUE AND OLIGONUCLEOTIDE DERIVATIVES CONTAINING A NUCLEOTIDE ANALOGON THEREOF
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003222743B2 (en) 2002-05-08 2008-12-11 Roche Innovation Center Copenhagen A/S Synthesis of locked nucleic acid derivatives
US20040102401A1 (en) 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of jagged 1 expression
AU2003247610A1 (en) 2002-06-21 2004-01-06 Ptc Therapeutics, Inc. METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
US20060058253A1 (en) 2002-08-12 2006-03-16 Benoit Chabot Methods to reprogram splice site selection in pre-messenger rnas
AU2003259803B2 (en) 2002-08-12 2007-08-02 The Regents Of The University Of Michigan Diagnosis and treatment of tuberous sclerosis
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
ATE443765T1 (de) 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
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
NZ542687A (en) 2003-04-13 2010-02-26 Enzon Pharmaceuticals Inc Polymeric oligonucleotide prodrugs
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
GB0326578D0 (en) 2003-11-14 2003-12-17 Univ Belfast Cancer diagnosis and therapy
US20050244851A1 (en) * 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US20050221354A1 (en) 2004-02-18 2005-10-06 Wyeth Nucleic acid arrays for monitoring expression profiles of drug target genes
JP2007534772A (ja) 2004-04-27 2007-11-29 イルミジェン バイオサイエンシーズ， インコーポレイテッド ヒト肝細胞癌細胞を特異的に標的化するための方法および組成物
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
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US20070087376A1 (en) 2004-08-30 2007-04-19 Potashkin Judith A Splice variants of pre-mRNA transcripts as biomarkers in idiopathic neurodegenerative diseases
US20060134670A1 (en) 2004-11-19 2006-06-22 Fabrice Piu Enabling tools to identify ligands for hormone nuclear receptors
WO2006081311A2 (en) 2005-01-26 2006-08-03 Medical College Of Georgia Research Institute Compositions and methods for the intracellular disruption of vegf and vegfr-2 by intraceptors
US7718625B2 (en) 2005-01-27 2010-05-18 University Of South Florida Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof
US20100150839A1 (en) 2005-02-04 2010-06-17 Massachusetts Institute Of Technology Compositions and Methods for Modulating Cognitive Function
ES2732071T3 (es) 2005-04-01 2019-11-20 Univ Florida Biomarcadores de lesiones hepáticas
EP1904630A4 (en) 2005-06-16 2009-10-21 Bionomics Ltd METHODS OF TREATING AND DIAGNOSING EPILEPSY BY DETECTING MUTATIONS OF THE SCN1A GENE
ATE544866T1 (de) 2005-06-17 2012-02-15 Baxter Int Adamts13-haltige zusammensetzungen mit thrombolytischer wirkung
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
US8501703B2 (en) 2005-08-30 2013-08-06 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
WO2007048629A2 (en) 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation of rna silencing efficiency by argonaute proteins
US20080280848A1 (en) 2005-10-28 2008-11-13 Volker Patzel Structures of Active Guide Rna Molecules and Method of Selection
US20100022495A1 (en) 2005-11-01 2010-01-28 President And Fellows Of Harvard College Modulating endoplasmic reticulum stress in the treatment of tuberous sclerosis
US20090291437A1 (en) 2005-11-02 2009-11-26 O'brien Sean Methods for targeting quadruplex sequences
US7785834B2 (en) 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
EP1792981A1 (en) 2005-12-02 2007-06-06 Humboldt-Universität zu Berlin Microginin producing proteins and nucleic acids encoding a microginin gene cluster as well as methods for creating microginins
EP1991677A2 (en) 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
AU2007211082B2 (en) 2006-01-27 2012-09-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
US8007790B2 (en) 2006-04-03 2011-08-30 Stowers Institute For Medical Research Methods for treating polycystic kidney disease (PKD) or other cyst forming diseases
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｐｃｓｋ９の発現を調節するための化合物および方法
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US8927704B2 (en) 2006-06-08 2015-01-06 Amino Up Chemical Co., Ltd Sense oligonucleotide capable of controlling the expression of iNOS and composition comprising the same
JPWO2008001778A1 (ja) 2006-06-27 2009-11-26 武田薬品工業株式会社 遺伝子改変動物およびその用途
WO2008094194A2 (en) 2006-07-24 2008-08-07 Athena Diagnostics, Inc. Pkd mutations and evaluation of same
ES2526295T5 (es) 2006-10-18 2021-05-04 Ionis Pharmaceuticals Inc Compuestos antisentido
US20090264353A1 (en) 2007-10-19 2009-10-22 Santaris Pharma A/S Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
DK2099461T3 (da) 2006-11-13 2012-07-02 Santaris Pharma As LNA Nukleoside Phosphoramidates
US8293684B2 (en) 2006-11-29 2012-10-23 Exiqon Locked nucleic acid reagents for labelling nucleic acids
WO2008086807A2 (en) 2007-01-19 2008-07-24 Exiqon A/S Mediated cellular delivery of lna oligonucleotides
EP2114981B1 (en) 2007-01-29 2013-05-08 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
AR065648A1 (es) 2007-03-09 2009-06-24 Pioneer Hi Bred Int Identificacion de transportadores de amonio (amts) para la regulacion del metabolismo de nitrogeno en plantas
EP2130835B1 (en) 2007-03-09 2012-05-23 Riken Compound having structure derived from mononucleoside or mononucleotide, nucleic acid, labeling substance, and method and kit for detection of nucleic acid
WO2008111908A1 (en) 2007-03-15 2008-09-18 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2',4'- carbocyclic ribo-thymidines for the treatment of infections and cancer
EP2149605B1 (en) 2007-03-22 2013-07-03 Santaris Pharma A/S Short RNA antagonist compounds for the modulation of target mRNA
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
EP2170374A2 (en) 2007-07-03 2010-04-07 Andreas Reichert Method for treating diseases related to mitochondrial dysfunction
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
KR101654007B1 (ko) 2007-08-15 2016-09-05 아이오니스 파마수티컬즈, 인코포레이티드 테트라하이드로피란 핵산 유사체
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
WO2009064920A2 (en) 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US8846386B2 (en) 2007-12-18 2014-09-30 University Of Kentucky Research Foundation sVEGFR-2 and its role in lymphangiogenesis modulation
JP5608863B2 (ja) 2007-12-28 2014-10-15 公立大学法人横浜市立大学 新生児期〜乳児期発症の難治性てんかんの検出方法
WO2009099942A2 (en) 2008-01-31 2009-08-13 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2009151691A2 (en) 2008-03-13 2009-12-17 Celera Corporation Genetic polymorphisms associated wiith venous thrombosis, methods of detection and uses thereof
EP2274423A2 (en) 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
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
WO2009149921A2 (en) 2008-06-11 2009-12-17 Bionucleon S.R.L. Inhibition of hrp-3 using modified oligonucleotides
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
KR20110081336A (ko) 2008-11-04 2011-07-13 이데라 파마슈티칼즈, 인코포레이티드 안티센스 올리고누클레오티드에 의한 ｔｏｌｌ―유사 수용체 2 발현의 조절
EP2364325A4 (en) 2008-11-07 2012-07-25 Ct Hospitalier Universitaire Sainte Justine SYNGAP1 DYSFUNCTIONS AND THEIR USE IN DIAGNOSTIC AND THERAPEUTIC APPLICATIONS FOR INTELLECTUAL SENTENCING
ES2600781T3 (es) 2008-12-04 2017-02-10 Curna, Inc. Tratamiento para enfermedades relacionadas con el factor de crecimiento del endotelio vascular (vegf) mediante la inhibición de transcritos antisentido naturales de vegf
US9006194B2 (en) 2008-12-19 2015-04-14 Drexel University Compositions and methods for diminishing viral infection and inflammation associated with viral infection
US20100166784A1 (en) 2008-12-30 2010-07-01 The Washington University Method and compositions for modulating th17 cell development
CA2750379A1 (en) 2009-01-14 2010-07-22 Gordon J. Lutz Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
JP5931720B2 (ja) 2009-05-08 2016-06-08 クルナ・インコーポレーテッド Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
US20140154783A1 (en) 2009-07-06 2014-06-05 Alnylam Pharmaceuticals, Inc. Bioprocessing
EP2275545A1 (en) 2009-07-16 2011-01-19 Julius-Maximilians-Universität Würzburg Use of microRNA-24 and/or its targets for the treatment and prevention of ischemia and induction of angiogenesis
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
MX2012002189A (es) * 2009-08-21 2012-03-16 Beeologics Inc Prevencion y cura de las enfermedades de los insectos beneficiosos a traves de moleculas.
CN102918163B (zh) 2009-09-08 2016-10-05 美国控股实验室公司 用于诊断自闭症谱系障碍的组合物和方法
WO2011032034A2 (en) 2009-09-10 2011-03-17 University Of Idaho Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids
CA2778171C (en) 2009-10-29 2017-07-25 Osaka University Bridged artificial nucleoside and nucleotide
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
US9334495B2 (en) 2009-11-25 2016-05-10 Elitechgroup B.V. Minor groove binder (MGB)-oligonucleotide miRNA antagonists
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
US8957040B2 (en) 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
JP2013529181A (ja) 2010-03-25 2013-07-18 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 神経障害を治療するための組成物および方法
WO2011127210A1 (en) 2010-04-06 2011-10-13 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
US20110269735A1 (en) 2010-04-19 2011-11-03 Celera Corporation Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof
CA2796722C (en) 2010-04-19 2021-06-01 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
US9156873B2 (en) 2010-04-28 2015-10-13 Isis Pharmaceuticals, Inc. Modified 5′ diphosphate nucleosides and oligomeric compounds prepared therefrom
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
US20130184223A1 (en) 2010-05-20 2013-07-18 University Of Rochester Methods and compositions related to modulating autophagy
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US9518259B2 (en) 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
CN109112126B (zh) 2010-06-23 2024-06-14 库尔纳公司 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病
KR101865433B1 (ko) 2010-10-22 2018-07-13 큐알엔에이, 인크. 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US20130309246A1 (en) 2011-02-02 2013-11-21 The Trustees Of Princeton University Jagged1 as a marker and therapeutic target for breast cancer bone metastasis
WO2012138487A2 (en) 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9644035B2 (en) 2011-04-08 2017-05-09 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
EP3495497B1 (en) 2011-04-28 2021-03-24 Life Technologies Corporation Methods and compositions for multiplex pcr
WO2012168435A1 (en) 2011-06-10 2012-12-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of leber congenital amaurosis
US20140357558A1 (en) 2011-06-24 2014-12-04 Cold Spring Harbor Laboratory Compositions and methods for treatment of spinal muscular atrophy
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
US8592156B2 (en) 2011-08-08 2013-11-26 Roche Molecular Systems, Inc. Predicting response to anti-CD20 therapy in DLBCL patients
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2753694B1 (en) 2011-09-05 2017-08-23 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
MX365525B (es) 2011-09-06 2019-06-06 Curna Inc Compuestos que regulan la expresión de subunidades alfa de canales de sodio regulados por voltaje en enfermedades relacionadas con epilepsia mioclónica severa de la infancia.
WO2013043878A2 (en) 2011-09-20 2013-03-28 The George Washington University Alternative splicing variants of genes associated with prostate cancer risk and survival
WO2013068441A1 (en) 2011-11-11 2013-05-16 Santaris Pharma A/S Compounds for the modulation of smn2 splicing
WO2013074814A2 (en) 2011-11-15 2013-05-23 University Of Utah Research Fourdation Morpholinos, morpholino upregulating, and associated methods
EP2966180B1 (en) 2011-11-29 2017-08-16 Life Technologies Corporation Methods and compositions for multiplex pcr
EP2802674B1 (en) 2012-01-11 2020-12-16 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of ikbkap splicing
WO2013120003A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
DK2812004T3 (en) 2012-02-10 2018-10-15 Ptc Therapeutics Inc COMPOUNDS FOR TREATMENT OF SPINAL MUSCLE DROPHY
US8846885B2 (en) 2012-02-17 2014-09-30 Ajinomoto Co., Inc. Oligonucleotide with protected base
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US20140378526A1 (en) 2012-05-11 2014-12-25 City Of Hope Design of nucleic acid binding molecules with non-watson crick and non-canonical pairing based on artificial mutation consensus sequences to counter escape mutations
US9296778B2 (en) 2012-05-22 2016-03-29 Idenix Pharmaceuticals, Inc. 3′,5′-cyclic phosphate prodrugs for HCV infection
WO2013177188A1 (en) 2012-05-22 2013-11-28 Idenix Pharmaceuticals, Inc. 3',5'-cyclic phosphoramidate prodrugs for hcv infection
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
CN104781271B (zh) 2012-08-20 2018-07-06 加利福尼亚大学董事会 具有生物可逆的基团的多核苷酸
WO2014045283A1 (en) 2012-09-24 2014-03-27 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Restoration of the cftr function by splicing modulation
WO2014049536A2 (en) 2012-09-25 2014-04-03 Universidade De Lisboa Drug targets for cystic fibrosis and other conditions
WO2014052638A1 (en) 2012-09-27 2014-04-03 Idenix Pharmaceuticals, Inc. Esters and malonates of sate prodrugs
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2014066915A2 (en) 2012-10-26 2014-05-01 Smith Larry J Methods and compositions to produce ss-rnai activity with enhanced potency
HRP20190303T1 (hr) 2012-10-31 2019-05-17 Ionis Pharmaceuticals, Inc. Liječenje raka
US9909128B2 (en) 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
EP2935304A1 (en) 2012-12-19 2015-10-28 IDENIX Pharmaceuticals, Inc. 4'-fluoro nucleosides for the treatment of hcv
CN103667438B (zh) 2013-01-07 2015-04-01 赵晨 一种筛查HRDs致病突变的方法及涉及的基因芯片杂交探针设计方法
CA2897522A1 (en) 2013-01-18 2014-07-24 Anna Mary Rose Therapeutics and diagnostics based on minisatellite repeat element 1 (msr1)
WO2014121287A2 (en) 2013-02-04 2014-08-07 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2014126229A1 (ja) 2013-02-18 2014-08-21 塩野義製薬株式会社 含窒素複素環構造を有するヌクレオシド及びヌクレオチド
WO2014137930A1 (en) 2013-03-04 2014-09-12 Idenix Pharmaceuticals, Inc. Thiophosphate nucleosides for the treatment of hcv
WO2014137926A1 (en) 2013-03-04 2014-09-12 Idenix Pharmaceuticals, Inc. 3'-deoxy nucleosides for the treatment of hcv
EP2981542B1 (en) 2013-04-01 2021-09-15 Idenix Pharmaceuticals LLC 2',4'-fluoro nucleosides for the treatment of hcv
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
WO2014198890A1 (en) 2013-06-13 2014-12-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of erythropoietic protoporphyria
WO2014201413A1 (en) 2013-06-14 2014-12-18 Isis Pharmaceuticals, Inc. Compounds and methods for modulating non-coding rna
WO2014209841A2 (en) 2013-06-25 2014-12-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
US20160201064A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
WO2015023938A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Epigenetic regulators of frataxin
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
CA2921556A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
EP3041958B1 (en) 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
US11236338B2 (en) 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3044315B1 (en) 2013-09-11 2019-02-20 Synthena AG Nucleic acids and methods for the treatment of pompe disease
AU2014352773B2 (en) 2013-11-21 2021-04-29 Memorial Sloan-Kettering Cancer Center Specification of functional cranial placode derivatives from human pluripotent stem cells
US10119168B2 (en) 2014-03-12 2018-11-06 The Brigham And Women's Hospital, Inc. Methods for the treatment of kidney fibrosis
US20170044540A1 (en) 2014-04-22 2017-02-16 Mina Therapeutics Limited Sarna compositions and methods of use
KR102524543B1 (ko) 2014-06-10 2023-04-20 에라스무스 유니버시티 메디컬 센터 로테르담 폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
GB201411468D0 (en) 2014-06-27 2014-08-13 Univ Edinburgh Novel treatment for endometriosis
US20180085391A1 (en) 2014-08-08 2018-03-29 Modernatx, Inc. Compositions and methods for the treatment of ophthalmic diseases and conditions
CA2958524A1 (en) 2014-08-20 2016-02-25 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
WO2016027168A2 (en) 2014-08-20 2016-02-25 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3207048A4 (en) 2014-10-17 2018-05-30 The Broad Institute, Inc. Compositions and methods of treating muscular dystrophy
CA2964979A1 (en) 2014-11-14 2016-05-19 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins
US20180346981A1 (en) 2014-11-20 2018-12-06 Massachusetts Eye And Ear Infirmary Panel-based Genetic Diagnostic Testing for Inherited Eye Diseases
GB201421379D0 (en) 2014-12-02 2015-01-14 Isis Innovation Ltd And Medical Res Council Molecule
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
WO2016128343A1 (en) 2015-02-09 2016-08-18 F. Hoffmann-La Roche Ag Compounds for the treatment of cancer
EP3259356B1 (en) 2015-02-20 2021-12-01 Rosalind Franklin University of Medicine and Science Antisense compounds targeting genes associated with cystic fibrosis
EP3262056A4 (en) 2015-02-27 2018-09-19 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
MX2017012426A (es) 2015-04-03 2018-01-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de tmprss6.
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
WO2017024111A1 (en) 2015-08-04 2017-02-09 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
US20180265911A1 (en) 2015-09-24 2018-09-20 The Trustees Of The University Of Pennsylvania Triptycene derivatives for nucleic acid junction stabilization
GB2591194B (en) 2015-10-09 2021-10-13 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
JP2018531975A (ja) 2015-10-30 2018-11-01 ピーティーシー セラピューティクス，インコーポレーテッド てんかんの治療方法
JP7036723B2 (ja) 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
EP3390666A4 (en) 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
JP7049249B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 中枢神経系疾患の処置のための組成物および方法
JP2018538287A (ja) 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
JP2019500345A (ja) 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
ES2903394T3 (es) 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
EP3390635A4 (en) 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
CN109843914B (zh) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 用于治疗疼痛相关病症的材料和方法
US20190218255A1 (en) 2016-09-16 2019-07-18 Olipass Corporation Scn9a antisense oligonucleotides
EP3548039B1 (en) * 2016-12-04 2023-07-19 Alavi Khorassani Moghadam, Marcel Victor Ribavirin for use in the treatment of a mitochondrial disease
MX2019011772A (es) 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.
CA3059775A1 (en) 2017-04-13 2018-10-18 Ovid Therapeutics Inc. Methods of treating developmental encephalopathies
JP2020519594A (ja) 2017-05-09 2020-07-02 ゾゲニクス インターナショナル リミテッド フェンフルラミンを用いるドゥーゼ症候群の治療方法
US20200085838A1 (en) 2017-05-16 2020-03-19 Praxis Precision Medicines, Inc. Methods of treating epilepsy and neurodevelopmental disorders
WO2019018898A1 (en) 2017-07-28 2019-01-31 Phylogica Limited CELL PENETRATING PEPTIDES AND RELATED COMPOSITIONS AND METHODS
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
HUE070436T2 (hu) 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére
EP3717505A4 (en) 2017-12-01 2021-12-01 Encoded Therapeutics, Inc. GENENICALLY MODIFIED DNA-BINDING PROTEINS
WO2019191341A1 (en) 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
CA3096407A1 (en) 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019224864A1 (ja) 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法とそれによるドラベ症候群の治療法
EP3806834A4 (en) 2018-05-25 2022-07-27 The Florey Institute of Neuroscience and Mental Health DYNAMIC CLAMPS AND METHODS OF USE THEREOF
US20210228531A1 (en) 2018-06-05 2021-07-29 Tufts Medical Center, Inc. Targeted treatment of autism spectrum disorder and other neurological or psychiatric disorders
EP3810776A1 (en) 2018-06-22 2021-04-28 F. Hoffmann-La Roche AG Oligonucleotides for modulating scn9a expression
CN112399849A (zh) 2018-06-26 2021-02-23 日本新药株式会社 含有反义寡核苷酸的组合物及其在迪谢内肌营养不良症的治疗中的用途
BR112021003224A2 (pt) 2018-08-20 2021-07-20 Rogcon, Inc. oligonucleotídeos antissentido direcionados a scn2a para o tratamento de encefalopatias por scn1a
US10905778B2 (en) 2018-09-26 2021-02-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
CN111518898B (zh) 2019-02-01 2022-07-26 中国科学院广州生物医药与健康研究院 Opa1异形体蛋白在调控肝癌细胞增殖能力中的用途
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
JP2022535717A (ja) 2019-05-24 2022-08-10 エンピリコ インク． アンジオポエチン様７（ａｎｇｐｔｌ７）関連疾患の処置
JP7652713B2 (ja) 2019-05-27 2025-03-27 ビジョン ファーマ ピーティーワイ リミテッド 新規の網膜色素変性処置
BR112022010882A2 (pt) 2019-12-06 2022-10-04 Stoke Therapeutics Inc Oligômeros antissenso para tratamento de condições e doenças
WO2021119756A1 (en) 2019-12-20 2021-06-24 PYC Therapeutics Limited Novel cellular delivery methods
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
AU2021354152A1 (en) 2020-10-02 2023-06-01 PYC Therapeutics Limited Treatment of optic atrophy
CN117980479A (zh) 2021-06-21 2024-05-03 斯托克制药公司 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体
JP2024534157A (ja) 2021-08-27 2024-09-18 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｓｃｎ１ａ発現を調節するための化合物及び方法
EP4429714A2 (en) 2021-11-09 2024-09-18 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases
AU2023212479A1 (en) 2022-01-31 2024-08-01 PYC Therapeutics Limited Method of treatment for optic atrophy
KR20240165951A (ko) 2022-03-23 2024-11-25 피와이씨 테라퓨틱스 리미티드 녹내장 치료 방법
KR20250087733A (ko) 2022-07-29 2025-06-16 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 화합물
CN121194802A (zh) 2023-02-14 2025-12-23 斯托克制药公司 反义寡聚物调配物
EP4720297A2 (en) 2023-05-31 2026-04-08 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases

Also Published As

Publication number Publication date
JP2023525799A (ja) 2023-06-19
IL298063A (en) 2023-01-01
TWI901675B (zh) 2025-10-21
KR20230022409A (ko) 2023-02-15
TW202208627A (zh) 2022-03-01
BR112022022889A2 (pt) 2023-04-04
US11814622B2 (en) 2023-11-14
US20230250429A1 (en) 2023-08-10
US20240102011A1 (en) 2024-03-28
US20230287410A1 (en) 2023-09-14
US12338437B2 (en) 2025-06-24
WO2021231107A1 (en) 2021-11-18
EP4150092A1 (en) 2023-03-22
EP4150092A4 (en) 2024-11-06
CA3173647A1 (en) 2021-11-18
MX2022014151A (es) 2022-11-30
AU2021270720A1 (en) 2022-12-08

Similar Documents

Publication Publication Date Title
US20240033378A1 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
US11814622B2 (en) OPA1 antisense oligomers for treatment of conditions and diseases
JP7420679B2 (ja) 状態および疾患の処置のためのアンチセンスオリゴマー
US20250059535A1 (en) Opa1 antisense oligomers for treatment of conditions and diseases
JP2019500347A (ja) 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法
CN117980479A (zh) 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体
WO2024249949A2 (en) Opa1 antisense oligomers for treatment of conditions and diseases
AU2021270720B2 (en) OPA1 antisense oligomers for treatment of conditions and diseases
CN118510551A (zh) 用于治疗病状和疾病的opa1反义寡聚体
HK40122411A (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination