BR112023026862A2 - Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos - Google Patents

Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos

Info

Publication number
BR112023026862A2
BR112023026862A2 BR112023026862A BR112023026862A BR112023026862A2 BR 112023026862 A2 BR112023026862 A2 BR 112023026862A2 BR 112023026862 A BR112023026862 A BR 112023026862A BR 112023026862 A BR112023026862 A BR 112023026862A BR 112023026862 A2 BR112023026862 A2 BR 112023026862A2
Authority
BR
Brazil
Prior art keywords
treatment
angiogenic disorders
flt1
eclampsia
oligonucleotide compounds
Prior art date
Application number
BR112023026862A
Other languages
English (en)
Inventor
Ananth Karumanchi
Anastasia Khvorova
Annabelle Biscans
Sarah Davis
Narayan Hariharan Vignesh
Original Assignee
Beth Israel Deaconess Medical Ct Inc
Univ Massachusetts
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 Beth Israel Deaconess Medical Ct Inc, Univ Massachusetts filed Critical Beth Israel Deaconess Medical Ct Inc
Publication of BR112023026862A2 publication Critical patent/BR112023026862A2/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1138Non-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 receptors or cell surface proteins
    • 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
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • 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
    • C12N2310/14Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de préeclâmpsia e outros distúrbios angiogênicos. a presente invenção refere-se a novos alvos para distúrbios angiogênicos. novos oligonucleotídeos também são fornecidos. métodos de uso dos novos oligonucleotídeos para o tratamento de distúrbios angiogênicos (por exemplo, pré-eclâmpsia) também são fornecidos.
BR112023026862A 2021-06-23 2022-06-22 Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos BR112023026862A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163214224P 2021-06-23 2021-06-23
PCT/US2022/034461 WO2022271786A1 (en) 2021-06-23 2022-06-22 Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders

Publications (1)

Publication Number Publication Date
BR112023026862A2 true BR112023026862A2 (pt) 2024-03-05

Family

ID=84544941

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023026862A BR112023026862A2 (pt) 2021-06-23 2022-06-22 Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos

Country Status (11)

Country Link
US (2) US11702659B2 (pt)
EP (1) EP4359539A1 (pt)
KR (1) KR20240040729A (pt)
CN (1) CN117677699A (pt)
AR (1) AR126207A1 (pt)
AU (1) AU2022299169A1 (pt)
BR (1) BR112023026862A2 (pt)
CA (1) CA3174095A1 (pt)
IL (1) IL309334A (pt)
TW (1) TW202315943A (pt)
WO (1) WO2022271786A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3334499A4 (en) * 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
WO2018031933A2 (en) * 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides

Family Cites Families (162)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5240848A (en) 1988-11-21 1993-08-31 Monsanto Company Dna sequences encoding human vascular permeability factor having 189 amino acids
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5332671A (en) 1989-05-12 1994-07-26 Genetech, Inc. Production of vascular endothelial cell growth factor and DNA encoding same
US5194596A (en) 1989-07-27 1993-03-16 California Biotechnology Inc. Production of vascular endothelial cell growth factor
US5219739A (en) 1989-07-27 1993-06-15 Scios Nova Inc. DNA sequences encoding bVEGF120 and hVEGF121 and methods for the production of bovine and human vascular endothelial cell growth factors, bVEGF120 and hVEGF121
AU6964191A (en) 1989-11-16 1991-06-13 Cornell Research Foundation Inc. Particle-mediated transformation of animal tissue cells
US5985558A (en) 1997-04-14 1999-11-16 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
TW404844B (en) 1993-04-08 2000-09-11 Oxford Biosciences Ltd Needleless syringe
ATE188479T1 (de) 1994-06-01 2000-01-15 Hybridon Inc Verzweigte oligonukleotide als pathogenhemmende mittel
US6383814B1 (en) 1994-12-09 2002-05-07 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
WO1996033761A1 (en) 1995-04-28 1996-10-31 Medtronic, Inc. Intraparenchymal infusion catheter system
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US5735814A (en) 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
EP1016726A1 (en) 1998-12-30 2000-07-05 Introgene B.V. Gene therapy to promote angiogenesis
US7608681B2 (en) 1999-12-24 2009-10-27 Genentech, Inc. Methods and compositions for prolonging elimination half-times of bioactive compounds
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
ES2728168T3 (es) 2000-12-01 2019-10-22 Max Planck Gesellschaft Moléculas pequeñas de ARN que median en la interferencia de ARN
EP2428569B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
DK2308507T3 (da) 2002-07-19 2015-04-20 Beth Israel Hospital Fremgangsmåder til behandling af præeklampsi
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US7923547B2 (en) 2002-09-05 2011-04-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2004044132A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Modified oligonucleotides for use in rna interference
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
US6913890B2 (en) 2002-12-18 2005-07-05 Palo Alto Research Center Incorporated Process for preparing albumin protein conjugated oligonucleotide probes
AU2003299864A1 (en) 2002-12-27 2004-07-29 P. Radhakrishnan Sirna compounds and methods for the downregulation of gene expression
US20040198640A1 (en) 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
EP1620544B1 (en) 2003-04-17 2018-09-19 Alnylam Pharmaceuticals Inc. MODIFIED iRNA AGENTS
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
WO2005001043A2 (en) 2003-06-02 2005-01-06 University Of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
PL1633767T3 (pl) 2003-06-02 2019-07-31 University Of Massachusetts Sposoby i kompozycje do kontrolowania wydajności wyciszania rna
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
CA2539181A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. Modulation of eif4e expression
MXPA06006892A (es) 2003-12-19 2008-02-13 Novartis Vaccines & Diagnostic Formulaciones para transfeccion celular de arn de interferencia corta, composiciones relacionadas y metodos de elaboracion y uso.
US20060078542A1 (en) 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
EP2899278A1 (en) 2004-03-12 2015-07-29 Alnylam Pharmaceuticals Inc. iRNA agents targeting VEGF
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
EP2145957B1 (en) 2004-04-20 2013-12-25 Marina Biotech, Inc. Compositions for enhancing delivery of double-stranded RNA to regulate gene expression in mammalian cells
WO2005116204A1 (ja) 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
JP2008501693A (ja) 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク. 遺伝子調節で使用するための個別に調節された鎖を有する二本鎖組成物
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
WO2007022470A2 (en) 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
WO2007027894A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
US7320965B2 (en) 2005-10-28 2008-01-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of Huntingtin gene
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
JP2009514877A (ja) 2005-11-04 2009-04-09 エムディーアールエヌエー,インコーポレイテッド siRNAの送達媒体としてのペプチド−ダイザ基質RNA抱合体
WO2007087451A2 (en) 2006-01-25 2007-08-02 University Of Massachusetts Compositions and methods for enhancing discriminatory rna interference
US9198981B2 (en) 2006-02-01 2015-12-01 The University Of Kentucky Modulation of angiogenesis
WO2007091269A2 (en) 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2007094218A1 (ja) 2006-02-16 2007-08-23 Gifu University 修飾オリゴヌクレオチド
EP1986697B1 (en) 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
WO2007112414A2 (en) 2006-03-27 2007-10-04 Isis Pharmaceuticals, Inc. Conjugated double strand compositions for use in gene modulation
US20080039415A1 (en) 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
AU2007299705B2 (en) 2006-09-22 2012-09-06 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
CA2666657A1 (en) 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof
US8906874B2 (en) 2006-11-09 2014-12-09 Gradalis, Inc. Bi-functional shRNA targeting Stathmin 1 and uses thereof
CA2664271A1 (en) 2006-11-27 2008-06-12 Enzon Pharmaceuticals, Inc. Polymeric short interfering rna conjugates
JP2010519908A (ja) 2007-03-02 2010-06-10 エムディーアールエヌエー,インコーポレイテッド Hif1a遺伝子の発現を抑制するための核酸化合物およびその使用
WO2008154482A2 (en) 2007-06-08 2008-12-18 Sirnaomics, Inc. Sirna compositions and methods of use in treatment of ocular diseases
WO2009002944A1 (en) 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
CA2735166C (en) 2007-08-27 2020-12-01 Boston Biomedical, Inc. Compositions of asymmetric interfering rna and uses thereof
CN101199858A (zh) 2007-10-18 2008-06-18 广州拓谱基因技术有限公司 治疗眼科疾病的多靶点小干扰rna鸡尾酒制剂及制备方法
TW200927177A (en) 2007-10-24 2009-07-01 Nat Inst Of Advanced Ind Scien Lipid-modified double-stranded RNA having potent RNA interference effect
US20090247608A1 (en) 2007-12-04 2009-10-01 Alnylam Pharmaceuticals, Inc. Targeting Lipids
US9029524B2 (en) 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
WO2009099991A2 (en) 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
EP3643782A1 (en) 2008-02-11 2020-04-29 Phio Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
WO2010011346A1 (en) 2008-07-24 2010-01-28 Rxi Pharmaceuticals Corporation Rnai constructs and uses therof
US20110223665A1 (en) 2008-07-25 2011-09-15 Alnylam Pharmaceuticals, Inc. ENHANCEMENT OF siRNA SILENCING ACTIVITY USING UNIVERSAL BASES OR MISMATCHES IN THE SENSE STRAND
US8664189B2 (en) 2008-09-22 2014-03-04 Rxi Pharmaceuticals Corporation RNA interference in skin indications
JP2012505644A (ja) 2008-10-15 2012-03-08 エフ.ホフマン−ラ ロシュ アーゲー オリゴヌクレオチド検出法
EP2358398A2 (en) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
CA2750561C (en) 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
WO2010118263A1 (en) 2009-04-08 2010-10-14 University Of Massachusetts Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
ES2646097T3 (es) 2009-08-27 2017-12-12 Idera Pharmaceuticals, Inc. Composición para inhibir la expresión de genes y sus usos
US20150025122A1 (en) 2009-10-12 2015-01-22 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
EP2513337A4 (en) 2009-12-17 2013-12-25 Merck Sharp & Dohme METHOD FOR QUICKLY ASSESSING THE PERFORMANCE OF SHORT INTERFERENCE RNA USING NEW CHEMICAL MODIFICATIONS
WO2011109698A1 (en) 2010-03-04 2011-09-09 Rxi Pharmaceuticals Corporation Formulations and methods for targeted delivery to phagocyte cells
CN105131067B (zh) 2010-03-24 2019-02-19 雷克西制药公司 皮肤与纤维化症候中的rna干扰
WO2011119852A1 (en) 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
WO2011119871A1 (en) 2010-03-24 2011-09-29 Rxi Phrmaceuticals Corporation Rna interference in ocular indications
KR101869570B1 (ko) 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
US8586301B2 (en) 2010-06-30 2013-11-19 Stratos Genomics, Inc. Multiplexed identification of nucleic acid sequences
JP2013541334A (ja) 2010-09-15 2013-11-14 アルニラム ファーマスーティカルズ インコーポレイテッド 修飾されたiRNA剤
US10378016B2 (en) 2010-12-06 2019-08-13 Children's Medical Center Corporation Composition and method for oligonucleotide delivery
WO2012083046A2 (en) 2010-12-17 2012-06-21 Arrowhead Research Corporation Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
CA2828544A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
WO2012177906A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
EP3453762B1 (en) 2012-05-02 2021-04-21 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
ES2716818T3 (es) 2012-05-22 2019-06-17 Olix Pharmaceuticals Inc Molécula de ácido nucleico inductora de interferencias de arn capaz de penetrar en las células y uso de la misma
CA2877985C (en) 2012-07-10 2020-10-27 Baseclick Gmbh Anandamide-modified nucleic acid molecules
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
WO2014047649A1 (en) 2012-09-24 2014-03-27 The Regents Of The University Of California Methods for arranging and packing nucleic acids for unusual resistance to nucleases and targeted delivery for gene therapy
AU2013346767B2 (en) 2012-11-15 2019-04-11 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
SI2929031T1 (en) 2012-12-05 2018-02-28 Alnylam Pharmaceuticals, Inc. PCSK9 IRNA assemblies and procedures for their use
US20140200124A1 (en) 2013-01-15 2014-07-17 Karen Michelle JAMES Physical Therapy Device and Methods for Use Thereof
NZ631552A (en) 2013-05-01 2017-02-24 Ionis Pharmaceuticals Inc Compositions and methods for modulating hbv expression
US10190117B2 (en) 2013-06-16 2019-01-29 National University Corporation Tokyo Medical And Dental University Double-stranded antisense nucleic acid with exon-skipping effect
US9862350B2 (en) 2013-08-12 2018-01-09 Tk Holdings Inc. Dual chambered passenger airbag
GB201315141D0 (en) 2013-08-23 2013-10-09 Logicor R & D Ltd Improvements to electric heating systems and method of use thereof
WO2015057847A1 (en) 2013-10-16 2015-04-23 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
US10752940B2 (en) 2013-11-08 2020-08-25 Ionis Pharmaceuticals, Inc. Compounds and methods for detecting oligonucleotides
US10513710B2 (en) 2014-04-18 2019-12-24 University Of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
MA39941A (fr) 2014-05-01 2017-03-08 Larry J Smith Procédés et modifications permettant de produire des composés arni simple brin à activité, potentiel et durée d'effet améliorés
JP6811094B2 (ja) 2014-05-22 2021-01-13 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. アンジオテンシノーゲン(AGT)iRNA組成物およびその使用
US10588980B2 (en) 2014-06-23 2020-03-17 Novartis Ag Fatty acids and their use in conjugation to biomolecules
EP3186377A1 (en) 2014-08-20 2017-07-05 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
US20170327524A1 (en) 2014-10-10 2017-11-16 Hoffmann-La Roche, Inc. Galnac phosphoramidites, nucleic acid conjugates thereof and their use
JP2018509913A (ja) 2015-03-17 2018-04-12 アローヘッド ファーマシューティカルズ インコーポレイテッド 第xii因子の遺伝子発現を阻害するための組成物と方法
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts 十分に安定化された非対称sirna
DK3277814T3 (da) 2015-04-03 2020-08-24 Univ Massachusetts Oligonukleotidforbindelser til målretning mod huntingtin-mrna
RS62672B1 (sr) 2015-04-03 2021-12-31 Univ Massachusetts Oligonukleotidna jedinjenja za tretman preeklampsije i drugih angiogenskih poremećaja
CA2982369A1 (en) 2015-04-10 2016-10-13 Hudsonalpha Institute For Biotechnology Method for blocking mirna
CA2988603A1 (en) 2015-06-15 2016-12-22 Mpeg La, Llc Defined multi-conjugate oligonucleotides
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JP2018525001A (ja) 2015-08-06 2018-09-06 シティ・オブ・ホープCity of Hope 治療用細胞内在化コンジュゲート
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
KR20180056766A (ko) 2015-10-09 2018-05-29 웨이브 라이프 사이언시스 리미티드 뉴클레오티드 조성물 및 이의 방법
CN105194689A (zh) 2015-10-10 2015-12-30 湖南中楚博生物科技有限公司 一种基于siRNA的双偶联复合物
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
EP3440222A1 (en) 2016-04-04 2019-02-13 H. Hoffnabb-La Roche Ag Nucleic acid sample preparation methods
WO2018031933A2 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
LT3607069T (lt) 2017-04-05 2023-01-10 Silence Therapeutics Gmbh Produktai ir kompozicijos
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
EP3444336B1 (en) 2017-08-18 2024-05-08 The Procter & Gamble Company Cleaning agent
EP3550021A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
MX2021001590A (es) 2018-08-10 2021-07-02 Univ Massachusetts Oligonucleótidos modificados dirigidos a snp.
JP2021533804A (ja) 2018-08-23 2021-12-09 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts O−メチルリッチ完全安定化オリゴヌクレオチド
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
JP2022528840A (ja) 2019-03-26 2022-06-16 ユニバーシティ・オブ・マサチューセッツ 安定性が増加した修飾オリゴヌクレオチド
EP4017504A4 (en) 2019-08-23 2024-01-24 Univ Massachusetts O-METHYL-RICH FULLY STABILIZED OLIGONUCLEOTIDES
GB201914296D0 (en) * 2019-10-03 2019-11-20 Univ Oxford Innovation Ltd Treatment
US20220010309A1 (en) 2020-03-26 2022-01-13 University Of Massachusetts Synthesis of modified oligonucleotides with increased stability
EP4157289A4 (en) 2020-05-26 2024-06-26 Univ Massachusetts SYNTHETIC OLIGONUCLEOTIDES WITH BLOCK AND CLUSTER MODIFICATION REGIONS
US20220228141A1 (en) 2020-11-23 2022-07-21 University Of Massachusetts Oligonucleotides for dgat2 modulation

Also Published As

Publication number Publication date
US11702659B2 (en) 2023-07-18
TW202315943A (zh) 2023-04-16
WO2022271786A1 (en) 2022-12-29
AU2022299169A1 (en) 2024-02-08
AR126207A1 (es) 2023-09-27
EP4359539A1 (en) 2024-05-01
US20230078622A1 (en) 2023-03-16
IL309334A (en) 2024-02-01
CA3174095A1 (en) 2022-12-29
CN117677699A (zh) 2024-03-08
US20240067967A1 (en) 2024-02-29
KR20240040729A (ko) 2024-03-28

Similar Documents

Publication Publication Date Title
BR112023026862A2 (pt) Compostos de oligonucleotídeos anti-flt1 otimizados para tratamento de pré-eclâmpsia e outros distúrbios angiogênicos
EP3995581A3 (en) Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
MX2022004101A (es) Composiciones de oligonucleotidos y metodos de uso de las mismas.
BR112022004451A2 (pt) Antagonistas de hpk1 e usos dos mesmos
EA201490993A1 (ru) МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи
BR112021017583A2 (pt) Compostos, composições e métodos para o tratamento de doença
BRPI0617165B1 (pt) Compostos inibidores mek, composições farmacêuticas que os contem e métodos de uso dos mesmos
EA201790420A1 (ru) Средства, представляющие собой модифицированную двухнитевую рнк
BRPI0606542A8 (pt) métodos para aumentar o tempo de progressão de uma doença (ttp)
BR112022000503A2 (pt) Compostos úteis para tratar infecções por vírus influenza
BR112019005548A2 (pt) tratamento da doença de huntington com aav
BRPI0511078A (pt) compostos de pirimidinona cìclicos, composição compreendendo os mesmos, método de inibição e uso
BRPI0507308A (pt) nanossuspensões de agentes anti-retrovirais para entrega ao sistema nervoso central aumentada
BR112022009938A2 (pt) Compostos macrocíclicos substituídos e métodos de tratamento relacionados
BRPI0410669A (pt) inibidores de caspase e usos dos mesmo
BR112023020781A2 (pt) Moduladores de cbl-b e usos dos mesmos
AR107579A1 (es) Composiciones y métodos para disminuir la expresión de tau
BRPI0508102A (pt) inibidores de caspase e usos dos mesmos
BR112022002691A2 (pt) Construtos de vesícula-aso extracelular visando stat6
BRPI0509653A (pt) inibidores cinesina mitótica
WO2016164463A1 (en) Methods for reactivating genes on the inactive x chromosome
BR112023015210A2 (pt) Inibidores de cdk2 e métodos de uso dos mesmos
WO2019217397A3 (en) Compositions and methods for improving strand biased
BR112022001365A2 (pt) Formas cristalinas de um inibidor de cd73
BR112022020147A2 (pt) Compostos para tratar a doença de huntington